clinical research therapeutic areas

A Comprehensive Guide to Therapeutic Areas in Clinical Research

Clinical research plays a crucial role in advancing medical knowledge and improving patient outcomes. The field encompasses a wide range of therapeutic areas, each with its own unique challenges and opportunities. In this comprehensive guide, we will explore different therapeutic areas in clinical research and shed light on the latest advancements and breakthroughs.

Understanding Different Therapeutic Areas in Clinical Research

When it comes to clinical research, it's essential to have a deep understanding of the various therapeutic areas. This knowledge helps researchers design robust and effective studies that can address the specific needs of each medical specialty.

Therapeutic areas in clinical research encompass a wide range of medical specialties, each focusing on a specific aspect of human health. From oncology and hematology to dermatology, immunology, neurology, gastroenterology, cardiology, ophthalmology, rare diseases, endocrinology, gene therapy, medical devices, and digital therapeutics, researchers are constantly exploring new frontiers to improve patient outcomes and advance medical knowledge.

Exploring the World of Oncology and Hematology Research

Oncology and hematology research focus on understanding and treating cancer and blood disorders, respectively. With advancements in genomics and targeted therapies, these fields have witnessed remarkable progress in recent years. Researchers are constantly striving to develop more effective treatments and improve the quality of life for patients battling these diseases.

In oncology research, scientists investigate the underlying mechanisms of cancer development, progression, and metastasis. They explore different types of cancer, such as breast, lung, prostate, and colorectal cancer, to gain insights into their unique characteristics and develop tailored treatment approaches. Hematology research, on the other hand, delves into blood disorders like leukemia, lymphoma, and anemia, aiming to understand the causes and develop innovative therapies.

Unveiling the Latest Advancements in Dermatology Research

Dermatology research aims to enhance our understanding of skin conditions and develop innovative therapies to treat them. From studying the mechanisms behind various dermatological disorders to conducting clinical trials for breakthrough treatments, researchers in this field are making significant strides towards improving skin health.

Researchers in dermatology explore conditions such as acne, psoriasis, eczema, and skin cancer. They investigate the role of genetics, environmental factors, and immune responses in the development of these conditions. By uncovering the underlying causes, researchers can develop targeted treatments that address the specific needs of patients with different skin conditions.

The Role of Immunology in Clinical Trials

Immunology plays a crucial role in clinical trials by investigating the complex interactions between the immune system and diseases. With the rise of immunotherapies and personalized medicine, researchers are harnessing the power of the immune system to target and eradicate diseases like never before.

Immunology research focuses on understanding how the immune system functions, how it responds to pathogens and cancer cells, and how it can be modulated to enhance therapeutic outcomes. Scientists study immune cells, antibodies, cytokines, and other components of the immune system to develop novel immunotherapies that can effectively treat conditions such as autoimmune diseases, infectious diseases, and cancer.

Navigating the Complexities of Neurology and Central Nervous System Research

Neurology and central nervous system research delve into the intricate workings of the brain and the nervous system. From studying the causes and mechanisms of neurological disorders to testing potential treatments, researchers in this field are dedicated to improving the lives of patients with conditions such as Alzheimer's, Parkinson's, and multiple sclerosis.

Neurological research encompasses a wide range of conditions, including neurodegenerative diseases, movement disorders, epilepsy, and stroke. Scientists investigate the underlying mechanisms of these disorders, explore potential biomarkers for early diagnosis, and develop innovative therapies to slow down or halt disease progression. They also focus on neurorehabilitation techniques to improve the quality of life for individuals living with neurological conditions.

Investigating Breakthroughs in Gastroenterology Research

Gastroenterology research aims to advance our understanding of the digestive system and develop new treatments for conditions such as inflammatory bowel disease, gastroesophageal reflux disease, and liver diseases. With emerging technologies and innovative therapies, researchers are pushing the boundaries of what is possible in gastrointestinal care.

Gastrointestinal research covers a broad spectrum of diseases and disorders affecting the digestive tract, including the esophagus, stomach, intestines, liver, and pancreas. Scientists investigate the causes of conditions like Crohn's disease, ulcerative colitis, and liver cirrhosis, aiming to develop targeted therapies that can alleviate symptoms and improve long-term outcomes for patients.

Advancing Cardiology Research for Better Heart Health

Cardiology research focuses on preventing, diagnosing, and treating cardiovascular diseases, which remain one of the leading causes of death worldwide. Through clinical trials and groundbreaking studies, researchers are constantly striving to develop more effective interventions and therapies to improve heart health.

Cardiovascular research encompasses a wide range of conditions, including coronary artery disease, heart failure, arrhythmias, and congenital heart defects. Scientists investigate the risk factors, genetic predispositions, and lifestyle interventions that can prevent the development of cardiovascular diseases. They also explore innovative treatments such as minimally invasive procedures, drug therapies, and regenerative medicine approaches to repair damaged heart tissue.

Shedding Light on Ophthalmology Research and Innovations

Ophthalmology research aims to improve our understanding of eye diseases and develop innovative treatments to preserve vision. From investigating the causes of conditions like cataracts and glaucoma to exploring the potential of gene therapies and regenerative medicine, researchers in this field are at the forefront of eye care innovation.

Researchers in ophthalmology study a wide range of eye conditions, including age-related macular degeneration, diabetic retinopathy, and retinal detachment. They investigate the underlying mechanisms of these diseases, develop diagnostic tools for early detection , and explore novel treatment approaches such as gene therapies, stem cell therapies, and artificial retinas.

Uncovering the Challenges and Progress in Rare Disease Research

Rare disease research focuses on understanding and addressing conditions that affect a small percentage of the population. These diseases often present unique challenges due to their complexity and limited treatment options. Researchers in this field work tirelessly to unravel the mysteries of rare diseases and develop targeted therapies to improve the lives of affected individuals.

There are thousands of rare diseases, each with its own set of challenges and complexities. Researchers investigate the genetic and molecular basis of these diseases, explore potential biomarkers for early diagnosis, and develop innovative therapies tailored to the specific needs of patients. They also collaborate with patient advocacy groups and healthcare professionals to raise awareness and improve access to care for individuals with rare diseases.

Delving into the Realm of Endocrinology Research

Endocrinology research explores the hormonal imbalances that contribute to conditions such as diabetes, thyroid disorders, and hormonal cancers. Through clinical trials and innovative treatments, researchers are committed to improving our understanding of endocrine disorders and developing personalized approaches to management.

Endocrine disorders encompass a wide range of conditions, including diabetes mellitus, hypothyroidism, hyperthyroidism, and adrenal disorders. Researchers investigate the underlying causes of these disorders, explore the role of genetics and environmental factors, and develop novel therapies such as hormone replacement therapies, targeted drug therapies, and lifestyle interventions.

Gene Therapy: Revolutionizing the Future of Medicine

Gene therapy has the potential to revolutionize the treatment of various diseases by targeting the underlying genetic causes. From inherited disorders to certain types of cancer, researchers are harnessing the power of gene editing and delivery techniques to develop transformative therapies that could change the landscape of medicine.

Gene therapy research focuses on understanding the genetic basis of diseases, identifying specific gene mutations or dysregulations, and developing techniques to modify or replace faulty genes. Scientists explore various delivery methods, such as viral vectors and nanoparticles, to safely and effectively deliver therapeutic genes to the target cells or tissues. Gene therapy holds promise for conditions like cystic fibrosis, muscular dystrophy, and certain types of cancer.

Exploring the Intersection of Medical Devices and Clinical Trials

Medical devices play a crucial role in healthcare, and their impact is not limited to diagnosis and treatment. Clinical trials involving medical devices are essential to ensure safety, efficacy, and usability. Researchers in this field focus on developing and testing innovative devices that can improve patient outcomes and enhance the delivery of healthcare services.

Medical device research encompasses a wide range of technologies, including implantable devices, diagnostic tools, and therapeutic equipment. Scientists work on improving the design and functionality of devices, conducting preclinical and clinical trials to assess their performance, and collaborating with healthcare professionals to integrate devices into patient care pathways. The development of advanced medical devices has the potential to revolutionize healthcare delivery and improve patient outcomes.

Harnessing the Power of Digital Therapeutics in Clinical Research

Digital therapeutics leverage technology to deliver evidence-based interventions for the prevention, management, and treatment of various conditions. From smartphone apps to virtual reality-based therapies, researchers are exploring the potential of digital therapeutics to empower patients and revolutionize healthcare delivery.

Researchers in digital therapeutics investigate the effectiveness of digital interventions in improving patient outcomes, enhancing medication adherence, and promoting behavior change. They develop and test smartphone applications, wearable devices, and virtual reality platforms that can deliver personalized interventions, track patient progress, and provide real-time feedback. Digital therapeutics have the potential to increase access to healthcare, reduce healthcare costs, and empower patients to take control of their health.

The Global Landscape of Clinical Trials

As the field of clinical research continues to expand, it's essential to understand the global landscape of clinical trials. These trials play a critical role in bringing new treatments to patients and advancing medical knowledge.

Analyzing the Growth and Trends in the Global Clinical Trials Market

The global clinical trials market is witnessing significant growth, driven by factors such as increasing disease burden, technological advancements, and growing interest from pharmaceutical companies. Analyzing the trends and dynamics of this market provides valuable insights into the future of clinical research and the potential impact on patient care.

A Closer Look at Clinical Trials Across Different Therapeutic Areas

Clinical trials span across various therapeutic areas, with each specialty presenting its unique challenges and considerations. Understanding the nuances of conducting clinical trials within different therapeutic areas is crucial for researchers and stakeholders involved in these studies. It helps ensure that trials are designed and executed effectively, leading to reliable and meaningful results.

In conclusion, this comprehensive guide has provided an overview of the therapeutic areas in clinical research. From oncology and hematology to dermatology, immunology, and beyond, each field presents exciting opportunities for researchers to make a significant impact on patients' lives. By continually pushing boundaries, embracing innovation, and collaborating across disciplines, the clinical research community can drive advancements that transform healthcare and improve outcomes for patients worldwide.

If you're inspired by the potential of clinical research across diverse therapeutic areas and are seeking a partner to navigate the complexities of clinical trials, look no further than Lindus Health. Our comprehensive suite of services provides an all-in-one solution for your clinical trial needs, from protocol writing to data delivery, including site services and an innovative eClinical platform. To discover how Lindus Health can support your clinical research journey and drive your study towards success, book a meeting with our team today.

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THERAPEUTIC AREAS OF PRECLINICAL AND CLINICAL RESEARCH

Our expertise in many therapeutic areas of preclinical and clinical research ensures that we move your molecule forward, whether through our preclinical, clinical, or bioanalytical service offerings. As a full development program or a stand-alone service, we apply our expertise in therapeutic areas to everything we do. We will help seamlessly transition your molecule through the phases of development in any of our therapeutic specialties, helping you get your drug to market faster.

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Our experience in general toxicology, safety pharmacology, and clinical pharmacology spans a wide range of therapeutic specialties, both small and large molecules. Across the therapeutic areas of both preclinical and clinical research, we analyze numerous biomarkers, and are always adding new validated methods to our panels that cover preclinical and clinical matrices. Finally, we have extensive experience in designing and conducting proof-of-concept studies in healthy participants or patients.

Move your cardiology compound from preclinical to early clinical efficiently, with a CRO that has worked on 30 different cardiology compounds across more than 70 studies. We can quickly de-risk your molecule with preclinical cardiovascular studies and with early cardiac safety assessment in early clinical studies.

Our preclinical and clinical experience spans a wide range of cardiology indications, including arrhythmias, hypertension, dyslipidemias, and congestive heart failure. Preclinical programs can be conducted across different species with robust safety pharmacology cardiovascular studies. Our preclinical facility has a specially designed room for ECG monitoring by telemetry. Our clinics are equipped with telemetry and staffed by a trained cardiac team.

CLINICAL STUDIES

DIFFERENT COMPOUNDS STUDIED

Dermatology

Working with a dermatology expert from preclinical to proof of concept can help to speed up your development program. As one of our therapeutic specialties, Altasciences conducts specific safety assessments for locally acting dermatology therapies and proof of concept in locally or centrally acting therapies, including both small and large molecules.

We assess toxicology of topical therapies, including edema/erythema measurement and Draize scoring, using mini pigs or NHPs. In the clinic, we collaborate closely with our consulting dermatologists who perform specialized assessments, such as biopsies or scoring lesion severity. If your locally acting product requires irritation and sensitization studies, our dedicated clinic areas can offer different climates, and can enroll up to 200 participants in one day.

Consult our  Topical Clinical Trials Fact Sheet and our Nonclinical Dermal Safety Assessment Fact Sheet .

Gastroenterology

We offer extensive preclinical and clinical expertise in the gastrointestinal therapeutic area, with over 100 Phase I/II PK or PD studies involving drugs acting systemically or locally in the GI tract. This includes general toxicology studies in a number of different species, as well as examining preclinical colitis and proctitis models. Our clinical pharmacology capabilities cover all study types, supported by a gastroenterologist Principal Investigator with experience in over 1,000 clinical studies.

Safety or efficacy monitoring is done via endoscopy, colonoscopy, intragastric pH monitoring, and X-ray imaging with barium. Additionally, we have experience with barostat model of colon pain and with PK sampling from ileostomy pouch.

Case Studies

• A Study to Evaluate the Effect of SYN-004 on the PK of IV Ceftriaxone in Adults With a Functioning  Ileostomy
• An Interaction Study Evaluating the Effect of Esomeprazole on SYN-004 Degradation of Ceftriaxone In Adults With an  Ileostomy
• A Study to Evaluate Safety, Tolerability and Pharmacokinetics of trimebutine 3-Thiocarbamoylbenzenesulfonate (GIC-1001) in a Randomized Phase I Integrated Design Study: Single and Multiple Ascending Doses and Effect of Food in Healthy  Volunteers
• A Phase Ib Clinical Study on the Analgesic Effect of NCE on Visceral Pain under Rectal Distension and Rectal Sensory Threshold using the Barostat Method in Male and Female Healthy  Volunteers

Consult our  Gastroenterology Trials Fact Sheet

PHASE I/II PK/PD GI

Support your early phase hematology program by partnering with a CRO that has relevant nonclinical and clinical expertise, aided by efficacy measures using flow cytometry or coagulation measures, including thromboelastography (TEG) and bioanalytical methods for a number of biomarkers.

Both our preclinical and clinical facilities perform flow cytometry, including when methods require samples to be analyzed within four hours of collection. We investigate coagulation directly or look for changes in different components of the coagulation cascade. If the focus is on the immune system, we have validated methods for a number of steps in the complement pathway.

Support the development of your immunology compound by working with a CRO that has the relevant therapeutic area expertise — and the flexible, collaborative approach — you need. With over 250,000 square feet of analytical laboratories spanning both our preclinical and clinical sites, Altasciences’ experience in analyzing immune endpoints is best positioned to meet your molecule’s development needs. Our biomarker panels, including the complement pathway and flow cytometry assessments, will allow you to seamlessly translate your preclinical findings to the clinical phase. Whether small or large molecules, we have the experience in conducting many types of programs, with scientific and technical expertise in the preclinical setting, and clinics that are set up and staffed with expert teams to ensure the safety in first-in-human studies for immune modulators.

With a comprehensive understanding of FDA regulations and requirements for vaccine approval, and access to a large database of participants, we are uniquely positioned to streamline the clinical development of new vaccines and delivery systems. We have conducted vaccine trials for the following conditions:

• Seasonal influenza
• Pandemic influenza
• Hepatitis B

In-house vaccine development supported by:

• Respiratory panel testing through the multiplex PCR BIOFIRE® FILMARRAY® system to evaluate the presence of an infection in the respiratory tract of immuno-compromised patients
• Ability to isolate peripheral blood mononuclear cells (PBMCs) through multiple collection methods
• Biosafety Level 2 (BSL-2) laboratories with limited badge access, biosafety cabinets, biohazard-trained staff, cleaning protocols, and all required containment procedures
• Long-term sample storage
• Dedicated   large molecule bioanalytical laboratory   with capabilities and state of the art equipment for flow cytometry , cell-based functional assays, immunogenticity expertise, etc. to support all clinical activities.

Infectious Diseases 

Maximize the efficiency of your infectious disease program by leveraging our significant preclinical, clinical, and bioanalytical therapeutic area expertise. We support the streamlined development of new vaccines, anti-viral therapies, delivery systems, and antimicrobial agents with our integrated, multi-disciplinary services. Partner with a leader that has a proven track record in infectious diseases and vaccine development.

Our preclinical site has the advantage of BSL-2 designation because of its NHP population. Our experts in infectious disease have conducted more than 220 clinical studies for acute or chronic infections, performed in healthy participants and patient populations.

Indications include:

• Bacterial infection
• Common cold
• Complicated skin infections
• Fungal infections
• Herpes zoster

We have performed clinical immune monitoring to establish proof-of-concept in diseases such as hepatitis C and influenza. Our experience in healthy participants includes antivirals, antifungals, antibiotics, and antiparasitics.

Infectious Diseases Case Studies

• A Phase Ib Study Evaluating GS-7340 in Treatment-Naive Adults with Chronic Hepatitis B (CHB) Infection
• A Phase Ib Study Evaluating GS-9620 in Virologically Suppressed Subjects with Chronic Hepatitis B Virus Infection
• A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple-Dose Ranging, Adaptive Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Antiviral Activity of GS 9620 in Treatment-Naive Subjects with Chronic Hepatitis B Virus Infection
• A Single and Multiple Dose Study of MK-3682 (IDX21437) in Healthy and Hepatitis C Virus (HCV)-Infected participants (MK-3682-001)

Consult our  Hepatitis Trials Fact Sheet Consult our  Infectious Diseases Trials Fact Sheet

STUDIES FOR ACUTE OR CHRONIC INFECTIONS

Metabolism and Endocrinology

Beyond the standard clinical chemistry and lipid panels to assess disorders in this therapeutic specialty, our preclinical services include specialized assays such as A1C, and specialized tests such as glucose tolerance to further evaluate molecules in this area. In the clinic, we have conducted over 75 early stage trials involving antidiabetic and hypoglycemic agents in patients who are diabetic (Type I and II), obese, or have metabolic syndrome or NASH. These include a large number of proof-of-concept studies investigating different measures of glucose tolerance, including clamping studies using state-of-the-art YSI-2900 that can analyze multiple glucose samples per run.

Consult our  Dyslipidemia Trials Fact Sheet Consult our  Metabolic Disorders Trials Fact Sheet Consult our issue of  The Altascientist   on Metabolic Disorders

Read the results of a Phase I study conducted by Altasciences combining obesity medications with different mechanisms of actions to provide more efficient treatment options for weight management. 

STUDIES INVOLVING ANTIDIABETIC AND HYPOGLYCEMIC AGENTS

Neurology is one of the key therapeutic areas of research conducted at Altasciences. Recognized globally as a CNS Center of Excellence, our scientific team has completed more than 200 preclinical and clinical neurology studies , in addition to providing formulation, manufacturing, and analytical testing services, as well as bioanalytical support. Whether large or small molecule, we can help move your compound through early development seamlessly and efficiently, from prototype formulation through commercialization.

Discover how working with a single partner for your end-to-end CNS drug development program can lead up to 40% in time savings .

Our preclinical oncology expertise can move your compound into patients, and we have clinical expertise with a number of non-cytotoxic oncology compounds. Drawing on a broad range of preclinical experience in oncology, we are accustomed to fast-tracking development so that you can proceed to patient studies as rapidly as possible. Our experience in oncology extends to the clinic, with experience testing nine different tyrosine kinase inhibitors in healthy participants.

DIFFERENT TYROSINE KINASE INHIBITORS STUDIED

Ophthalmology

As a leading partner in ocular therapy, our integrated CRO/CDMO solutions can support your ophthalmic drug development program from lead candidate selection to market. You will benefit from working with a single partner as your compound advances through each phase of drug development—from prototype formulation through preclinical testing, to early phase clinical trials, and manufacturing.

Discover our end-to-end ophthalmic drug development solutions that can lead up to 40% in time savings.

Orthopedics

Altasciences brings your orthopedics and musculoskeletal molecule through preclinical to proof of concept. Our experience with proof-of-concept studies in healthy participants and patient populations, such as those with osteoarthritis, includes models such as the 6-minute walk test or stair challenge, and imaging using X-ray, MRI, CT, or DEXA at our preclinical and clinical sites. Finally, our panels include a number of inflammatory biomarkers.

Consult our  Pain Models Fact Sheet

Altasciences has therapeutic specialty expertise in psychiatric research, conducting studies from preclinical to clinical proof of concept in patients. Our preclinical expertise covers the complete range of general toxicology and safety pharmacology studies, and includes 24-hour video monitoring and performing functional observation battery tests to assess behavioral changes.

Our clinical expertise is augmented by two Principal Investigators who are psychiatrists, and our experience covers substance abuse, epilepsy, anxiety disorder, panic disorder, depression, ADHD, and sleep disorders. As your clinical development proceeds, we can conduct the  human abuse potential  testing and  driving simulator studies  that will likely be required, two areas where we have extensive expertise.

Consult our  Central Nervous System Trials Fact Sheet

DECADES IN PSYCHIATRIC RESEARCH

Respirology

You can count on an efficient development of your compound to treat respiratory/pulmonary disorders, leveraging our preclinical and clinical experience with investigating both systemic and locally acting therapies. Our clinics have units with specialized air handling systems that quickly exchange air to minimize cross-contamination between dosed participants. Our staff consistently trains patients on using different pulmonary devices to ensure consistent dosing. We are adept at pulmonary function testing, and have pulmonologists and respiratory technicians on site to assist in study design or with assessments.

Consult our  Pulmonary Trials Fact Sheet

Rheumatology

Altasciences can help support you in the development of your rheumatology compound, delivering the preclinical, clinical, and bioanalytical expertise and experience you need. We have extensive preclinical and clinical experience testing immune modulators backed up by a battery of biomarkers, including components of the complement pathway and cytokines.

Substance Abuse

Altasciences offers leading preclinical and clinical sites for substance use disorders, one of our key therapeutic areas of clinical trials, including expertise across a wide variety of mechanisms of action. Our experience in general toxicology, safety pharmacology, and clinical pharmacology covers a wide range of compounds, including oral, IM, IV, and implanted products. We also analyze a number of biomarkers and efficacy outcomes using visual analogue scales (VAS) and questionnaires.

Our robust database of patients combined with our upscale facilities enable us to deliver rapid enrolment and excellent retention rates. Our sites have controlled substance licenses, including a DEA Schedule I license to conduct research with tetrahydrocannabinol and cannabidiol in Kansas City.  Cannabis study  start times at our Montreal site have been positively impacted by the legalization of cannabis in Canada.

YEARS COMBINED EXPERIENCE BY OUR PRINCIPAL INVESTIGATORS

Leverage our  preclinical  and  clinical  expertise to help you tackle the development needs of your urology compound. Having conducted almost 30 studies, we are skilled in general toxicology, safety pharmacology, and clinical pharmacology of a wide range of compounds. Our experience covers treatments for urinary incontinence, erectile dysfunction, benign prostate hyperplasia, and prostate cancer. We can also analyze a number of different biomarkers. With our extensive patient database and outreach, we can help you establish proof of concept by recruiting patients across a range of urological conditions.

STUDIES COMPLETED

Women’s Health Clinical Trials

With extensive expertise, experience, and a multitude of solutions for sponsors developing therapeutic products and diagnostics in the area of women’s health, Altasciences is ideally positioned to attain your drug development goals.

Altasciences has conducted preclinical and clinical studies with products for breast cancer, oral contraception, fertility treatment, hot flashes, and hypoactive sexual disorder. Our experience includes a wide range of compounds delivered orally, vaginally, or by implant, and we have validated methods for the majority of marketed contraceptives, fertility treatments, and hormone replacement therapies.

Consult our women’s health fact sheet for more information on the specific therapeutic areas we have experience in.

Our clinical pharmacology units are located in densely populated areas, allowing access to a wide variety of ages, ethnicities, lifestyles, and health conditions. We have a diverse participant database of over 100,000 healthy pre- and postmenopausal women who are actively interested in participating in our clinical trials, as well as access to a number of patient populations through our own database and partnerships with local fertility clinics. This enables us to tailor your study and obtain the data you need to move your project forward.

We work closely with a network of local gynecologists for specialized exams and biopsies, and offer imaging service like transvaginal ultrasounds and DEXA.

Some of the study types we offer include:

• Alcohol interaction
• Comparative BA/BE
• Drug-drug interaction
• Driving impairment
• Proof of concept, efficacy
• Transdermal adhesion/HRIPT
• FIH (SAD/MAD)
• Food effect
• Genotyping PK
• Renal impairment
• Safety, PK, tolerability

In addition, Altasciences’ large and small molecule bioanalytical expertise and comprehensive research support services ensure an integrated, efficient, and holistic solution for your women’s health studies.

YEARS AS A LEADING CRO IN WOMEN'S HEALTH

Therapeutic - FAQs

Is your participant database searchable by therapeutic area.

Our database of over 400,000 participants contains comprehensive, detailed individual medical profiles and screening histories, searchable by therapeutic area. We also develop recruitment strategies specific to the therapeutic areas of clinical research required by our sponsors.

DOES ALTASCIENCES HAVE EXPERTISE IN THERAPEUTIC SPECIALTIES WITH BOTH LARGE AND SMALL MOLECULES?

The clinical trials we conduct include both large and small molecules, in therapeutic specialties ranging from CNS to immunology, dermatology to cardiology, and many other therapeutic areas. We also have  manufacturing  for small molecules and  supporting services  for all our preclinical and clinical therapeutic specialties.

WHICH THERAPEUTIC AREAS OF CLINICAL RESEARCH REQUIRE PURPOSE-BUILT FACILITIES?

Altasciences is proud of our specialized facilities, including 10 on-site  driving simulators  for CNS or other studies, negative pressure  inhalation facilities  for inhaled products, and secured pharmacies and clinical pharmacology units for controlled substance studies. Of course we also have the facilities (400 beds in two North American locations) to conduct clinical trials in a wide range of therapeutic areas not requiring specialized facilities.

DO YOU HAVE ACCESS TO PATIENTS OR SPECIAL POPULATIONS AS NECESSARY FOR SPECIFIC CLINICAL TRIALS BY THERAPEUTIC AREA?

We have relationships with local hospitals and clinics to ensure that we can conduct clinical trials with patients, or in specific population or age groups, as necessary for your projects. We can accommodate long or short stays in our upscale, well-appointed, North-American clinical pharmacology units.

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Most common clinical trial therapy areas

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As of January 2024, there are 20,465 clinical trials recruiting patients in the U.S., according to the National Library of Medicine (NLM) . Worldwide, NLM reported 65,474 trials recruiting subjects.

While clinical trials are FDA-mandated for all new treatments—including drugs , medical devices , vaccines, and gene therapies —these studies also produce crucial data used in making other care decisions. In addition to evaluating the safety and efficacy of new treatment options, clinical trials can help both providers and manufacturers determine which medical approaches work best for certain illnesses or patient populations .

What are the different types of clinical trials?

Clinical studies are classified into two main groups based on the research protocol and whether or not participants receive medical interventions as part of their testing. These categories are defined as:

• Interventional studies (more commonly referred to as clinical trials) Investigators assess participant health outcomes based on specific interventions administered as part of the research protocol in order to test the safety and effectiveness of a candidate drug, therapy, or experimental treatment.
• Observational studies Investigators assess participant health outcomes as established in the research protocol without administering interventions or procedures.

Within this framework, clinical studies can be further segmented by subtype—including prevention, treatment, diagnostic , screening, genetic, epidemiological, or quality-of-life trials.

Definitive Healthcare tracks both interventional and observational trials across 14 different therapy areas . Data is compiled from The Clinical Trials Transformation Initiative (CTTI) and can be viewed in our HospitalView , PhysicianView , and PhysicianGroupView products. In this blog, we’ve compiled a list of the top 10 most common clinical trials by therapeutic area.

Cancer treatments have the highest clinical trial volume

With a projected global market value of $300 billion by 2026 , it’s no surprise that cancer drugs are among the most highly-tested therapies in clinical trials across the nation.

Top 10 clinical trial therapeutic areas

Fig 1 Data from the Definitive Healthcare Atlas Dataset and sourced from The Clinical Trials Transformation Initiative (CTTI) . The total number of clinical trials includes all historically reported. Accessed January 2024.

Treatments for cancer have the highest number of clinical trials by therapeutic area – 15.4% of all trials analyzed. With a projected global market value of $300 billion by 2026, it’s no surprise that cancer drugs are among the most highly-tested therapies in clinical trials across the nation. With an increasing number of new cancer drugs being developed each year, this clinical trial volume can be expected to grow.

Clinical trials for mental health and behavioral disorders represent 6.3% of the trials analyzed. From antidepressants and anti-anxiety medications to emerging psychedelic options , our mental health treatment choices continue to expand.

Next, trials for endocrinology and metabolic disease, including diabetes , and cardiovascular and circulatory diseases each represent more than five percent of the clinical trials on the list. The additional top therapeutic areas by clinical trial volume include:

• Nervous system diseases
• Musculoskeletal diseases
• Digestive diseases
• Infectious diseases

Where do clinical trials take place?

Below, we mapped the number of clinical trials by each state. The analysis includes 36,788 active clinical trials listed on ClinicalTrials.gov, which are those identified as “recruiting,” “not yet recruiting,” “active,” and “not recruiting.”

Clinical trial volume by state

Fig. 2 Data is sourced from the National Library of Medicine at clinicaltrials.gov and include trials with a status of recruiting, not yet recruiting, active, or not recruiting. Accessed January 2024.

Which states have the most clinical trials?

As expected, population density is a significant contributing factor in the geographic distribution of clinical trials across the U.S. Greater patient volumes, after all, increase the likelihood of finding eligible participants for clinical research. For this reason, we see a strong correlation between those states with the largest populations and the highest reported clinical trial volumes—including California , Texas , New York , and Florida .

Though it’s not explicitly represented on the above map, there also appears to be a strong correlation between high clinical trial volumes and facility types—where states with high-performing cancer centers , academic medical centers , or other research facilities report greater volumes of clinical trials than areas with fewer numbers of these facility types.

Massachusetts and Ohio , for instance, each have more than 5,500 active clinical trials despite their sizes. Both states are in the top five for most academic medical centers in the country.   

Top hospitals by clinical trial volume

Fig 3 Data from the Definitive Healthcare Atlas Dataset and sourced from The Clinical Trials Transformation Initiative (CTTI) . The total number of clinical trials includes all historically reported. Accessed January 2024.

Clinical studies can be sponsored by a number of different agencies, ranging from industry organizations like pharmaceutical companies to academic medical centers or federal agencies—including the National Institutes of Health, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs.

While clinical studies can be conducted in a variety of different locations, access to specific equipment, physician expertise, or sample patient populations quite often influence this decision. With greater access to these critical resources, places like cancer research facilities and university medical centers are frequently selected as locations for hosting clinical trial research.

In fact, all 10 of the top hospitals by historic clinical trial volume are cancer research centers, university hospitals, or another type of dedicated research facility, according to Definitive Healthcare data.

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Read our blogs to catch up on the latest clinical trial trends and learn more about recruiting the right patients for your studies. Or find out how the Atlas All-Payor Claims dataset and PhysicianView product can help you target the right physicians for your new therapies . Start a free trial today.

• Most Common Clinical Trial Therapy Areas

Therapeutic Areas

Focus and expertise.

Clinical trials present unique challenges for different therapeutic areas (TAs).

The complexity of trial design, the availability of patients, and the regulatory environment of each therapeutic area vary greatly and impact study success.

Our  eSource technology  combined with years of expertise in therapeutic areas—including neurology , immunology , infectious disease , oncology , Dermatology, Gastrointestinal, Rare Disease, Respiratory—ensure any TA-focused study achieves successful patient outcomes.

Patient-Centric Research, from the Ground Up

Patient-centric research is the Clinical ink commitment to focus on the needs and perspectives of patients throughout a clinical trial. We ensure research is relevant and meaningful, thus increasing engagement and participation of each patient.

Clinical ink delivers  eSource technology solutions that go beyond EDC, integrating Direct Data Capture (DDC) with eCOA  and ePRO modules, eConsent ,  sensors and wearables , and  digital biomarkers .

Our therapeutic experience and pioneering patient-centric research approach translate into trials no others can deliver. Learn more about our therapeutic area-specific expertise.

Solutions for Therapeutic Areas

Diabetes trials are intricate due to the complexity of this metabolic condition. Measuring and quantifying symptoms can be challenging.

Discover how Clinical ink Continuous Glucose Monitoring and eSource technology offer a better patient experience and improve trial outcomes for diabetes research.

Encompassing a wide range of conditions from autoimmune disorders to allergies, the mechanisms of action of immunotherapies — and how to target them effectively — are complex.

Leverage Clinical ink technology and expertise to simplify challenging and complex clinical trials.

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Therapeutic Areas in Drug Discovery & Clinical Research

COVID-19   Infectious Diseases/Vaccines   Immuno-Oncology (IO)/Cancer     Pain     Auto-Immunity     Heart Disease

The Carterra LSA is a real-time label-free biosensor unique from other technologies. The system is purpose built to characterize up to 384 samples with exquisite resolution. The capabilities of the LSA are well suited for long term infectious disease research objectives but with a throughput capability that allows the system to easily meet the limited timelines during rapidly evolving threats such as COVID-19. Additionally, the LSA’s patented microfluidics enable it to characterize epitope and affinity using sample quantities far below other technologies. Speed in conjunction with high resolution is vital towards making informed decisions on hundreds if not thousands of samples during viral pandemics.

• Publication: Jones et al, Science Magazine, The neutralizing antibody, LY-CoV555, protects against SARS-CoV-2 infection in non-human primates Supplementary Materials
• Publication: Schasfoort et al, Research Square 2020, Presence and strength of binding of IgM, IgG and IgA antibodies against SARS-CoV-2 during CoViD-19 infection
• White Paper: Addressing Viral Pandemics Such as COVID-19 Using the Carterra LSA
• Webinar: Antibody Discovery and Development for COVID-19 Diagnostic Testing
• Webinar: Rapid Discovery of Therapeutic Antibodies to Combat COVID-19 at Twist Bioscience
• Application Note: Discovery of Therapeutic Antibody Cocktails
• Press Release: Immunity Monitoring of COVID-19 Patients Using a SARS-CoV-2 Label-free Assay and Biosensor Chip
• Podcast: The Bamlanivimab story – how it reached the clinic in eight months

Infectious Diseases/Vaccines

Therapeutic cocktails comprise a collection of antibodies targeting several distinct epitopes to collectively achieve a broad neutralization of the virulence factor. Defeating an ever-mutating virus poses challenges in designing an effective medicine but targeting multiple epitopes at once can offset biological redundancy – when one pathway fails, another often compensates.

Carterra’s LSA facilitates epitope characterization on hundreds of antibodies in parallel, allowing you to quickly understand the epitope coverage of your antibody library and focus resources on studying the epitope clusters with the most biologically-relevant function. When epitope binning studies are performed on a large panel of antibodies, the competition picture gains resolution and nuance, allowing a deep appreciation of the epitope diversity contained in the antibody library.  Developing a potently neutralizing therapeutic antibody cocktail relies on the proper selection of antibodies with complementary epitopes. The Carterra LSA accelerates the epitope characterization of an antibody library, such as one that is recovered by B-cell cloning of an Ebola survivor, to understand the immune response and guide the discovery of therapeutic antibody cocktails.

• Application Note: Multi-Parameter Epitope-Centric Data Used to Elucidate Mechanisms of Action of Antibodies
• Publication: Wec et al, PNAS 2020, Longitudinal dynamics of the human B cell response to the yellow fever 17D vaccine
• Publication: Cairnes et al, J Virol 2019, Surface plasmon resonance (SPR) reveals direct binding of HSV glycoproteins gH/gL to gD and locates a gH/gL binding site on gD.
• Publication: Awasthi et al, Vaccine 2019,  Antibody responses to crucial functional epitopes as a novel approach to assess immunogenicity of vaccine adjuvants.
• Publication: Hook et al, PLoS Pathog 2018, Vaccine-induced antibodies to herpes simplex virus glycoprotein D epitopes involved in virus entry and cell-to-cell spread correlate with protection against genital disease in guinea pigs.
• Publication: Yeung et al, Nature Commun 2016, Germline-encoded neutralization of a Staphylococcus aureus virulence factor by the human antibody repertoire
• Publication: Chukwuma et al, PLoS One 2018, Increased breadth of HIV-1 neutralization achieved by diverse antibody clones each with limited neutralization breadth

Immuno-Oncology (IO)/Cancer

Immuno-oncology (IO) leverages the body’s own immune system to fight cancer and is revolutionizing the way we think about medicine because it is enabling long-term remission or in some cases “cures” to cancer patients. Common IO approaches involve antibody modulation of T-cells by various mechanisms, for example targeting T-cell checkpoints such as CTLA-4, PD-1, and PD-L1, redirecting T-cells via bispecifics antibodies or replenishing the immune system with “super-charged” T-cells, known as chimeric antigen receptor T-cellular (CAR-T) therapies. The clinical portfolio of antibodies in this space is diverse in format, engineering, and target, and requires deep knowledge of the mechanism of action (MOA) to ensure its safety and efficacy.

Carterra’s LSA provides a highly parallel way of screening hundreds of antibody binding interactions at once in terms of their binding kinetics, affinity, and epitope specificity. These binding properties underpin an understanding of antibody’s MOA and as such, are used as metrics to triage an antibody library to leads.

• Webinar: Discovery of Potent anti-PD-L1 Antibodies
• Publication: Brown ME, et al. PLoS One. 2020,  Assessing the Binding Properties of the Anti-PD-1 Antibody Landscape Using Label-free Biosensors
• Poster: Use of High Throughput SPR to Characterize Antibodies To Common Cancer Biomarkers

Monoclonal antibodies are providing attractive alternatives to traditional small molecule therapies for treating chronic pain based on their high affinity and exquisite specificity that can minimize the unwanted off-target binding and adverse side-effects that seriously limit small molecule approaches. Due to their hydrophilicity and large molecular size, antibodies barely cross the BBB, so do not cause the lethal addictions of small molecule drugs. While the large size of antibodies limit their bioavailability via oral administration, necessitating injection via intravenous, intramuscular, or subcutaneous routes, this inconvenience to patients is somewhat offset by the naturally long serum exposure of antibodies, allowing for less frequent dosing, with some therapeutic antibodies requiring only a single injection per three months. From a practical perspective though, the high cost of developing and manufacturing therapeutic antibodies remains a major hurdle.

Therapeutic antibodies under clinical investigation and on the market aimed at treating chronic pain target a variety of molecules associated with pain pathways,  including calcitonin gene-related peptide (CGRP), ion channels, nerve growth factor (NGF), tumor necrosis factor-alpha (TNF-alpha), and epidermal growth factor receptor (EGFR). Understanding how we can explore different mechanisms for treating pain is essential to bringing new medicines to market. Currently it takes about 12 years to progress a therapeutic antibody from bench to market, and antibodies for treating chronic pain are scrutinized in long and rigorous clinical trials.

The Carterra LSA is a high throughput screening and characterization tool for measuring antibody binding interactions, enabling the analysis of hundreds of interactions at once. By delivering high quality data quickly in the assessment of key binding parameters such as kinetics, affinity, and epitope specificity it can speed up research and ultimately help to converge upon clinically-ready leads faster.

Auto-Immunity

Therapeutic antibodies have proved highly successful in treating a variety of autoimmune diseases, as demonstrated by Humira (adalimumab), one of the biggest blockbuster drugs of all time. Like several other anti-inflammatory antibodies on the market, Humira targets and blocks tumor necrosis factor alpha (TNF-alpha), which relieves the pain and inflammation associated with some autoimmune diseases. Discovering “the next Humira” requires deep knowledge of biology to understand how to target other pathways relevant to autoimmune disease beyond TNF-alpha, such as T- and B-cell viability or activation, IL-1, IL-6, IL-17, and others. Understanding a drug’s mechanism of action (MOA) underpins the clinical success of any drug program.

Carterra’s LSA is a high throughput analytical platform that accelerates the screening and detailed characterization of large antibody libraries in terms of their binding kinetics, affinity and epitope specificity to provide crucial information about antibody binding properties that are used to assess and converge upon the most promising clinical candidates.

• Publication: Bednenko J, et al. MAbs. 2018, A multiplatform strategy for the discovery of conventional monoclonal antibodies that inhibit the voltage-gated potassium channel Kv1.3.

Heart Disease

When developing a new biotherapeutic, understanding the binding interactions that underpin its mechanism of action (MOA) are key to its success in the clinic. The Carterra LSA is a high throughput tool that enables interaction analysis measurements to be performed on hundreds of antibodies at once, allowing researchers to characterize the binding properties of antibody libraries quickly. The ability to generate kinetic, affinity and epitope specificity information at scale accelerates the library-to-lead triage, enabling the prioritization of resources to those antibodies with the most desirable binding properties.

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Therapeutic areas

Society and industry—still the only provider of approved medicine—share the desire to treat important diseases. Governments too, since some diseases’ high prevalence affect the economic output of society. Yet the decisions as to which drugs will be developed are strictly in private hands. The risks required to treat some really important, and often increasingly prevalent, diseases mean that the desires of society and the industry are diverging. This is not boding well for our society.

The understanding and willingness of governments to step in as a partner and financier is not apparent. Will governments recognize their role in this? Since the economic cost of these diseases, in terms of reduced productivity and medical costs to treat the disease, is enormous, understanding should be forthcoming. Government has invested for many decades in an enormous program of cancer research, which now shows dividends. The importance of further government investment in basic research is apparent when industry does not want to or cannot anymore afford it. As Big Pharma drops out of important therapeutic areas, it is not promising to assume that government can step in to the vacancy. There has to be a serious effort to find a formula by which the government gently or not so gently forces the industry to continue its efforts because it is so important.

Perhaps the most important of these neglected diseases is Alzheimer’s disease (AD) because of its dehumanizing nature and its predictably massively increasing prevalence. Yet, none of the currently approved Alzheimer drugs can be used to prevent, cure, or even significantly slow the progression of this disease. It is predicted that by 2030 50% of the over 80s will have the disease. Almost 100% of people with an inheritable trait will develop the disease unless we succeed in finding a preventive therapy. Only recently has a preventive trial been announced.

It is often thought that insulin-dependent diabetes is a well-treated disease and needs no further diagnostic and drug development efforts. This could not be further from the truth. The long-term complications of insulin-treated diabetes add to the economic burden, especially as the growth in prevalence of the disease expands with the waistlines of the world’s population. Three hundred million may have type 2 diabetes by 2030. Pharmaceutical control of weight loss has so far been vexed by serious side effects. When it comes to diabetes drugs, we start to recognize that the control of blood glucose can be further improved; the biggest contribution is not by drugs, but by self-diagnosis with point of care glucose measuring devices.

Morphine is still the most effective medicine against pain. It has been around for 5,000 years and we haven’t come up with anything better. But it can be ineffective against neuropathic pain and cancer pain. New pain medicines are difficult to find because of the subjective measurement and poor animal models. Since many current pain killers are abused, there are other hurdles. Despite the many difficulties, society needs new pain killers at the same time that notable pharma are pulling out, despite the economic opportunity.

Drugs against cancer have had many high-profile and expensive failures in recent years, with all major companies having at least one failed candidate. Biotech companies have made much progress, however, and they are either collaborating with or being taken over by Big Pharma. Oncology drug development has been crucially enabled by decades of government investment. The key is the many characterized drug targets that other disease areas do not have.

Government intervention in oncology and HIV provide the model for treatment of AD, neuropathic pain, and schizophrenia, among other therapeutic areas. Increasing governmental direct funding of translational research is not the solution many hope it will be.

Diverging views of society & industry

There are a few especially important diseases and therapeutic areas from the viewpoint of society, as well as from the viewpoint of the industry. These are especially important because these diseases affect a very large number of patients, have high prevalence, and thus, they affect the economic output of society and the distribution of expenditures within healthcare and, in a broader sense, within the whole society.

Some of these diseases are able to disrupt the workings of our society. Most countries recognize that epidemics are politically and legally highly destabilizing and cause huge economic losses. Even before modern globalization brought enormous and rapid flows of goods and people from all corners of the world together, epidemics of the plague, cholera, polio, and so forth showed the difficulties in isolating microbes, stopping outbreaks, and halting the spread of disease. Recently avian influenza (bird flu), severe acute respiratory syndrome (SARS), and the fights to restrict methicillin-resistant Staphylococcus aureus , and multidrug-resistant tuberculosis (MDR-TB), have confirmed modern vulnerability to outbreaks. The response to microbial epidemics needs to transcend national borders and ignore differences in the payer systems of national healthcare. This is clearly recognized, with the cooperation within the World Health Organization, with the setting up and use of the Global Fund, 76 but it is not sufficient just to share diagnostics and epidemiological data: we need vaccines and drugs developed for the whole world. Even with multinational cooperation, friendly countries such as the UK and the United States can be enveloped in arguments about delivery of paid-for flu vaccines.

Two large epidemics are slowly encompassing the whole world, not only Africa: obesity/type 2 diabetes and Alzheimer’s disease (AD). These epidemics threaten abrupt and almost unstoppable reorganization of the economy and societal structures unless we bring them under medical and pharmacological control. The obesity/diabetes epidemic is going to affect countries like China and India dramatically, while countries with an aging population like Japan, Germany, and the UK will be hit by the consequences of AD the hardest. Models of the consequences show that up to 15% of the work force will be involved in the 24/7 care of AD patients. These trends should be shaking governments into action.

The health economics that deal with the economic effects of disease burden and the effects of healthcare cost in society is, together with the study of public health, becoming increasingly important in political discourse in all countries; there are superb books on the topic from different political viewpoints. We will only deal with aspects of these important branches of the economy and of medicine that affect the choices of pharmaceutical companies.

Companies try to look forward 10–12 years and to foresee not only the biology and epidemiology, but also the economic and political landscape at the time when any new drug would enter clinical practice, would be paid for, and should start to produce revenue.

The development of drugs to prevent, or at least to treat, these strategically important diseases constitutes from society’s view the most important task for the industry, yet today in all countries’ pharmaceutical industry even vaccine production is in strictly private hands and the decisions are privately made. Governmental organizations weigh in on the safety of what is produced and, in cases of vaccines (and insulin and antipsychotics), about who should pay for the goods if proven safe and effective. Governments, by recognizing the societal importance of vaccine development and vaccination programs, have also influenced outcomes by limiting the liabilities of the private vaccine producers. Active involvement of nongovernmental organizations (NGOs) and governments in what drugs to develop is restricted to the trickle of funds devoted as charity to organizations working on diseases predominantly afflicting poor populations, such as malaria, leishmaniasis, sleeping sickness, and MDR-TB, which is now shared by all of Asia and prevalent in countries like Russia, China, and India.

The drugs that will treat these large diseases also potentially represent the largest incomes. Yet, because some of the diseases represent large drug development risks, even over the unusually high risk the whole industry carries, the big companies are abandoning work in some of these disease areas. The declining interest in AD and pain are examples. This does not bode well for our society.

The understanding and willingness of governments to step in as a partner and financier or limiter of liabilities is not apparent when it comes to the several large and potentially very profitable diseases we are discussing. Governments have always been involved as regulators and distributors, and have previously been involved in the development of some pediatric vaccines and drugs, for example, to control outbreaks or treat epidemics of infectious diseases. These increasingly neglected diseases are just as important for our society as are the infectious diseases.

The human & economic cost of major diseases

Among these large strategically important therapeutic areas/diseases that affect very large numbers of patients and have large human and economic costs are the following:

• 1. New antibiotics, especially for treatment-resistant Pseudomonas , Staphylococcus , and tuberculosis
• 3. Obesity and type 2 diabetes, and associated kidney and retinal complications
• 4. Neuropathic pain
• 5. Schizophrenia
• 6. Ovarian, prostate, lung, and gastric cancer, and glioblastoma
• 7. Hundreds of neurological diseases that collectively affect approximately 10 million patients, that are chronic, and where the scientific understanding of the pathophysiology is good but the market size is judged by the industry’s present standards as too small

Data are available in widespread sources for the estimated economic costs of these diseases. For example, the American Diabetes Association published 77 these data in 2008:

The total estimated cost of diabetes in 2007 is $174 billion, including $116 billion in excess medical expenditures and $58 billion in reduced national productivity. Medical costs attributed to diabetes include $27 billion for care to directly treat diabetes, $58 billion to treat the portion of diabetes-related chronic complications that are attributed to diabetes, and $31 billon in excess general medical costs

Table 4.1 and Figure 4.3Table 4.1Figure 4.3 describe the gap between the number of pharma-supported projects and the existing medical need. The list above is just a summary of the position in 2012. Most diseases are under-researched. Even though research is broad for many, it is just that it is not proportional to the need.

Of the diseases listed, only type 2 diabetes is being adequately addressed by resources of the industry. For the rest, we are not doing very well. While progress in the treatment of cancers, especially leukemias and breast cancer and in the past 2 years melanoma and non-small cell lung cancer, has been substantial, it is thanks to government-funded basic research since the 1960s. 78 Because of this large long-term investment, scientists are aware of many drug targets and thus the industry, which largely dropped its own basic research by the end of the 1990s, makes steady progress.

Each of these areas will be discussed in purely economic terms because, along with having disease-specific aspects, they also provide some very general points of view about the development of pharmaceuticals, about the interplay between government and private industry, and about the importance of government investment in basic research when industry does not want to or cannot anymore afford it.

AD: the most loomingly threatening disease

The most important example is AD. This neurodegenerative disease, which leads to cognitive decline, loss of memory, and inability to recognize faces or names, is, in short, one of the most horrible diseases in many senses. Many argue that all our humanity depends on our memories, and our memories determine who we are. When we lose these we are losing ourselves.

Although Alois Alzheimer diagnosed the first patient more than 100 years ago, AD has not been an important issue of research or of healthcare discussions, and certainly was not discussed in terms of a major threat to society as it is recognized today. The reason for this is that the major risk factor for AD is age. When the average life span of the population was 50–60 years, the frequency of AD was relatively low. Since definitive diagnosis still requires postmortem neuropathological examination of the brain, not many cases were identified. In contrast, the prediction for the United States is that in 2030 every second person aged 80 or above will have AD. The proportion of these people in Japan and the whole Western world is rapidly increasing.

What it really means is that up to 10% or more of the 2030 population in Japan, North America, and Europe may suffer from AD, and this is now being modeled by think tanks and insurers everywhere. Taking care of an Alzheimer patient, if they are taken care of individually, requires three full-time working persons each working 8 hours per day. This translates to a very large proportion of the productive labor force being devoted to taking care of patients with advanced AD, who cannot themselves be relied upon for the most elementary of functions. This is only the economic consideration. The societal implication is huge; there is currently a shortage of care givers. 79

The 2011 estimate is that $600 billion is being spent on taking care of AD patients. Yet there is no drug available that prevents or cures the disease, or even significantly modifies the rate of progression of the disease. The only drugs today approved are giving a relatively small—although in large trials statistically significant—slowing of the loss of cognitive function in mild to moderate AD and in one case are approved to treat moderate to severe AD. Memantine (Namenda from Forest Laboratories) is a 30-year-old German drug (from Merz) originally used for neuropathic pain treatment. It produces very small changes in the rate of decline in already seriously demented patients. It is liked because of its relative safety, not its efficacy. The total number of approved, available drugs is five and none of them can be used to prevent, cure, or even significantly slow the progression of this disease.

The number of diagnosed Alzheimer patients in 2011 in the United States was 5.3 million, and a similar number applies to Western Europe, but, including the nondiagnosed patients, the total is estimated at some 30% higher. Where do we stand with the development of drugs to be used in the therapy of AD patients? The scientific background is such that in this disease there are both sporadic forms, accounting for 99.8% of cases, and clear familial (or inherited) forms, which is to say, cases that have an early onset of disease at about 40 to 45 years based on a genetic predisposition; these account for 0.2% of cases. There are several other genetic vulnerabilities identified. A very frequently occurring vulnerability is being a carrier of a protein isoform called ApoE4. One can carry two of these gene copies when one is homozygotic. One can carry one ApoE4 and one ApoE3 , which is more common in the population, when one is heterozygotic. But, the vulnerability for AD is significantly higher in the ApoE4 homozygotes, who make up 2% of the U.S. population. 80

Incidentally, in 2007–2008, the first two full-genome sequences of notable individuals were published: those of Craig Venter, 81 who published “The Sequence of the Human Genome” 82 coincidently with the Human Genome Project’s parallel study, 83 and Jim Watson, 84 the co-discoverer of double helix. 85 In the first case, Venter’s sequence showed increased risk of AD 86 and in the second case the ApoE3/4 encoding sequence was omitted from Watson’s data at the specific request of Dr. Watson. He did not want to know if he were susceptible to an incurable disease to which a grandmother had succumbed. 87 Interestingly, the cost of the Human Genome Project was $3 billion over 13 years, Venter’s sequence cost some $100 million, and Watson’s was variously reported at $1–2 million in 2–4 months. 88 The costs are dropping in a dramatic manner: Complete Genomics, Life Technologies, and Illumina have disclosed that from January 2012 they will provide a full genome sequence for anyone for $1,000. We have yet to start a discussion about what cognitively happens to individuals carrying both the ApoE3 and ApoE4 genes—one from each parent—as their age increases.

There are more serious, but luckily less frequent, genetic causes of AD, such as the “Swedish” and “Dutch” mutations in the amyloid precursor protein. These mutations and mutations in another associated protein, presenilin, and a combination of these mutations lead, in a small group of people, to very early onset (about 40 years of age) rapid progression AD. These patients, who will have “familial forms of AD,” make up a small portion, 0.1–0.2%, of all AD patients. Most of the AD patients endure the sporadic form, yet the discovery of patients with familial AD suggests the possibility of highly focused trials for AD preventive medicines in a group whose risk is very high. Almost 100% will develop the disease unless we succeed in finding a preventive therapy.

It is worth explaining that in diseases where there is a large sporadic group of patients yet a small “familial group” or “genetically determined group” the understanding of the familial forms had great importance, in almost all cases, for finding treatments for the sporadic cases. This is because when people with familial disease are treated, we are fairly sure of the causative mechanism. It may be compared to the increasing introduction of companion diagnostics in oncology: treating those who have a genetic cause of the tumor improves responder rates from 5% to over 60%. 89 Yet we have great difficulties in finding any (preventive AD) trials involving family members of patients who have or have died from familial AD. The patient associations that “organize” these individuals have good lists of who would be eligible, but basic research and pharma have failed these people and missed this opportunity for two reasons. One is pure greed: “Why wait 4–5 years to show in familial cases that a therapy works, and then do another 4–5-year study in the real market representing sporadic patients?” goes the argument. It is added that “it is hard to recruit for familial case clinical trial,” which does not seem to be true, or it has to do with the second factor: the drugs we so far have put into trial are associated with such bad side effects that it is hard to convince people presently fully healthy, even if they know that they are likely to have early AD, to endure the side effects for an uncertain delay of the onset or for the prevention of the disease. We need to improve science and pharmacy, and we need governments, not NGOs, to pay for the more focused trials. 90

It is important that in the new healthcare legislation in the United States, upheld by the Supreme Court in June 2012, one cannot be excluded or penalized for a pre-existing condition or a genetic predisposition. This will become more and more important, especially as genome sequences are becoming affordable as part of diagnostics, not just as a research tool.

In general, the presence of genetic forms of the disease accelerates the understanding of the disease and indirectly accelerates the development of drugs to treat the disease. This is also the case with AD. The early onset disease has been linked to mutations in the metabolism of the amyloid precursor protein leading to the appearance of amyloid plaques. These are amorphous aggregates formed from several proteins. The major component is a fragment of the amyloid precursor protein. Research directed toward the proteolytic enzymes that produce this fragment of amyloid protein, the amyloid beta (or Aβ or Abeta) peptide, led to an investigation to dismantle the plaques formed from this peptide. The last 15 years have seen a tremendous effort by the industry to develop inhibitors for the two major APP-hydrolyzing proteases. Beta-secretase and gamma-secretase produce the amyloidogenic Abeta 1-40 or 1-42 peptides, but, so far, no peptidase inhibitor has reached clinical use. A gamma-secretase inhibitor (semagacestat or {"type":"entrez-nucleotide","attrs":{"text":"LY450139","term_id":"1258021836","term_text":"LY450139"}} LY450139 ) from Lilly came closest; it reached phase 3 trials and then was withdrawn in 2010 because of toxicities. This additionally shook the confidence of large pharma in Alzheimer therapies. 91 It is worth examining which companies try to develop AD drugs, the intensity of their effort, and the major issues that affect the intellectual and economic investment in the development of Alzheimer drugs.

Early diagnostic imperatives

The start of each treatment is diagnosis of the disease. Today, AD diagnosis heavily relies on neuropsychology test batteries, which have become better and better. They are now able to detect mild cognitive impairments and early AD. But nobody really knows how much damage has already occurred in the brain when cognitive impairment becomes obvious to relatives, friends, and colleagues. It is, therefore, very important that 2011 has seen the clinical entry of several imaging agents. 92 When working well, these permit the visualization of amyloid plaques in the living brain, and might, therefore, be important in establishing whether a drug aimed at inhibiting the production of amyloid peptide is really successful in reducing the plaque load. It is, however, evident that nobody is bothered by carrying amyloid plaques. When it is conclusively shown that the amyloid plaque load predicts cognitive impairment, we will then be able to convince people who exhibit no cognitive evidence of AD to take drugs that prevent the formation of amyloid peptide and amyloid plaques. But then these drugs will have to be very, very “clean” with no or a very low incidence of serious side effects or even annoying side effects.

If we had succeeded in producing a drug candidate with a strong scientific rationale suggesting it would prevent AD, without major and minor side effects, then which people would be convinced to take it? Firstly, those with a very high risk of developing familial AD would take it, preferably before showing any symptoms. Later, those who, through aging and some first signs of mild cognitive impairment, appear to have the risk of developing AD in a few years would take it. Some of these candidates would have to be prepared to test this drug in trials that will take 3–5 years. The principle is very similar to the way we have convinced millions and millions of people to take the cholesterol-lowering statins. High cholesterol per se is not a disease, but it is a harbinger of many cardiovascular diseases. If amyloid peptide and amyloid plaques can be detected and shown more rigorously to be a harbinger of AD, then volunteers would be easier to find. But for this to happen one will need much cleaner and much safer Alzheimer drugs than those we presently have.

Based on earlier neuropsychological diagnosis, patients are selected for the clinical trials of proposed Alzheimer drugs. These trials are becoming longer and longer. The reason for this is that we are entering into these trials based on the success of neuropsychological assessment that now captures early Alzheimer patients, in ever milder forms of the disease.

The first Alzheimer drug clinical trials in moderate to severe patients could show that the first class of drugs, the acetylcholine esterase inhibitors (AChEIs), could slow the decline of cognitive abilities significantly after a 6–12 month treatment (trial) period. The latest trials with AChEIs have, however, taken 18 months to show a significant difference in cognitive decline between drug-treated and placebo patients. The milder, and earlier the AD cases enrolled into the trials, the longer we have to dose the drug to show a significant difference in the cognitive performance between drug-treated and placebo patients. This is a clear recognition by pharma that, in order to show efficacy, they will need longer trials. These are a major expenditure, but, more importantly, the results are further and further away from what affects the stock price in the 2–3-year period upon which present pharma executives focus or are obliged to focus.

Another issue regarding Alzheimer patients and clinical trials is that the average age of these persons is above 65 and they suffer from a great number of other diseases, for which they are also being treated. This means that the drugs to be used in Alzheimer therapy have to be devoid of interactions with a very, very large number of drugs. It also means that because of age and other diseases, a relatively large number of these people will drop out of the trial or may die during the 18- or 24-month length of the trial. This means that these trials, at least at phase 3, will have to be very long and very large. The industry has conducted such long and large trials in cardiovascular disease and in type 2 diabetes. However, most large and long trials—usually post marketing, i.e., post approval, phase 4 trials—are carried out by governmental agencies in the UK, United States, and so forth. But the repeated failure of Alzheimer drugs over the last 10 years is making more and more companies silently withdraw from the effort to develop therapeutic agents for the treatment of AD, which would slow disease progression, prevent the disease, or cure the disease.

This is understandable from the point of view of profit within organizations, but it is not acceptable for our society. As we recognize AD as a serious risk for our society, we cannot afford to give up trying to make drugs that achieve effective treatment. Today, there are no seriously capable organizations except the pharma industry to develop such a drug. So, when they withdraw, openly or silently, and are permitted so to do, we will end up with the horrific prospect of every second over 80-year-old citizen suffering from AD, but not being offered any kind of treatment that would help the patient, the family, and society as a whole.

Translational research

When we say that the only seriously capable complex, sophisticated large organizations to develop therapeutics are the pharmaceutical companies, we are not disregarding the expensive efforts government-supported institutions, such as the National Institutes of Health (NIH) and corresponding European institutions, put into what they call translational research , that is, research that is directed toward affecting medical practice.

Translational research, which is the modern equivalent of applied research, is an increasingly important part of their task. However, in retrospect, one can today clearly state that these organizations have not discovered any major drugs in the past 40 years. They have, during these last 40 years, been the major contributors to the basic science breakthroughs that are the scientific basis of drug development in the world’s private pharmaceutical companies, which all abandoned in-house basic research from 1980 to 2000. The government-sponsored research institutions, together with some major NGO-sponsored research entities (e.g., the Wellcome Trust, the Howard Hughes Medical Institute, and the Bill and Melinda Gates Foundation), have introduced a multitude of extremely important tools to preclinical and clinical research. These permit the regulatory agencies and the industry to measure and improve the success of drug discovery, drug development, and the drug approval processes. Starting in 2012 the NIH collaborated officially with the Food and Drug Administration (FDA); in principle they are both government sponsored. This will assist the FDA, which has so far relied on ad hoc panels of academics for scientific advice, to lean more on scientists in government pay. NIH scientists have a much stronger set of rules to keep them from receiving payments from the industries that apply for drug approval. This will help dilute the requirement of Congressional Committees periodically to uncover high-profile examples of the FDA receiving advice from academics with a conflict of interest. Conflict of interest is a serious matter that can undermine all public trust in the impartiality of FDA decisions regarding drug approvals, no matter if the decision itself was impartial.

Thus, the recent, long-awaited official agreement, pushed by the Obama Administration, to improve the quality and image of FDA decisions by having two U.S. tax-payer financed organizations, the NIH and the FDA, sharing scientific competence, bodes well. The NIH is the world’s powerhouse for preclinical discoveries through its intramural and extramural programs, and it is important in academic medicine in small proof-of-concept trials; however, as a clinical research organization, it is small to medium sized. The roughly $5 billion spent by the NIH on clinical projects and the $700 million proposed for translational research in 2011–2012 is dwarfed by the estimated $92 billion research budgets of major pharma and major biotech spent in the same year on R&D, over 85% of which is translational research (see Table 5.1 , Table 5.2 ). The effort is also dwarfed by the collective experience that exists in these companies in the development and testing of clinically useful drugs.

Top 15 R&D budgets of the major pharma companies in 2009

Compare to the NIH budget of $29.5 billion in 2009. 1 Around 70% ($22B) of the NIH budget and 20% ($20B overall) of the companies’ budget is for preclinical research. This is not basic research per se, but research to select the drug candidate to enter into clinical trials. Almost all basic research is now left to government and NGO financing. Internationally, governments fund more than 60% of basic research in large national institutes and universities; the rest is from private sources to universities. NGOs fund 2% and industry the remaining approximately 30%. Eighty percent of all private investment is for translational research in the hope of developing diagnostics and drugs that will generate profit. We do not have precise figures, but the indications are that the budgets would have fallen by a collective 15% in the two subsequent years (2010 and 2011).

Top 15 R&D budgets of the major biotech companies in 2009

Around 80–90% of biotech budgets ($6.9B from the above) is allocated to translational research.

It is clear that the NIH and its international counterparts bear huge responsibility for the “real” science in AD. When one compares funds poured into HIV/AIDS and cancer research to those put into AD, one is shocked. It is clear that funding initiatives can bring large numbers of scientists to work on a problem, and that it is what we badly need. Instead, AD research is conducted by a small club; this we cannot afford.

Therefore, it is not a promising route to let private industry back out of trying to develop Alzheimer drugs, saying: “it’s too hard; it’s too risky; it’s too slow; it’s too expensive,” and assume that governments will step in to the vacancy. There has to be a serious effort to find a formula by which governments, gently or not so gently, force the industry to continue its effort in developing Alzheimer drugs. Intervention can come from tax breaks or other incentives, as well as somewhat punitive legislation for pharma to release data from programs they have dropped, in order to support this highly risky activity because it is so important . We have seen how U.S. and other governments can enhance and semi-force the development and production of antidotes to biological weapons, and how it can incentivize and encourage the production of vaccines.

AD is not as acute as bird flu or the risk of a bioterrorist attack, but it is as, or more, threatening for the individual and for our society.

Obesity & type 2 diabetes

The first biological to be used on a large scale was insulin, albeit preceded by some vaccines and antidotes to snake toxins, and so forth. The first clinical trial of insulin involved one patient: a child. We often forget this. As discussed earlier, production of insulin is now made with recombinant technology in bacteria or yeast. The three major producers, Novo Nordisk, Lilly, and Sanofi-Aventis, are producing both acutely active and delayed, slowly, or long-acting depot or retard insulin preparations.

One may think that insulin-dependent diabetes is, therefore, a well-treated disease and needs no further diagnostic and drug development efforts. This could not be further from the truth: up to 30% of patients are resistant to the most widely prescribed diabetes drug, metformin (see below); most patients are becoming increasingly resistant to insulin; and we have ongoing exacerbations with the patients who are not becoming resistant to either of these two drugs.

Even with well-controlled diabetic patients who, by judicious use of insulin, do not let their blood glucose rise, the development of renal illness, blindness resulting from retinal vascularization problems, diabetes-induced neuropathies, and pain in the extremities is very high. On top of this comes the world’s increasing obesity, the most obvious risk factor for type 2 diabetes. Thanks to a rising standard of living and global flows of food staples, fewer and fewer people in the world are starving, or, rather, more and more people are not starving. Obesity growth is linked with the change of diet in Asia. In India and China, where more bountiful food, coupled with wealth, is added to some genetic predisposition in these two huge countries, there has been a dramatic increase in obesity and in type 2 diabetes. It should be noted that not all obese people will develop type 2 diabetes, only about 20% do, but the cost of treatment for these people and the cost of treatment of the complications from diabetes is staggering and comparable to that of AD.

It is clear that it is in the interest of patients, society, and the pharmaceutical industry to find drugs that prevent the rapid increase in the number of obese people and, therefore, reduce the risk that 20% of these will develop type 2 diabetes. It is also clear that we have to develop better treatments for this very large group, which is estimated to include close to 300 million people by 2030, all of whom will have type 2 diabetes. Unfortunately, it is very difficult to develop safe drugs that prevent or cure obesity when the patients are unwilling or unable to switch diet and to exercise. Diet control and exercise are both becoming increasingly difficult in the stressful world of rising standards and rising expectations of work and other achievements.

The pharmaceutical industry has seen some spectacular successes in this area. Unfortunately, these then turned into spectacular failures, sometimes taking with them the entire company, no matter how big it was. Most in the industry clearly remember the successful development of Fen-Phen (fenfluramine + phentermine), a very active (amphetamine-like) mixture of appetite control drugs that has shown clearly visible, tangible results of large rapid weight loss. The beauty of trials on obesity drugs is that the patients can, by checking their waistlines or stepping on to a balance, see for themselves whether the drug is efficacious. The fallacies of these trials are many, however. While it is relatively easy to achieve a 5–10% weight loss in the first 6–8 weeks, it is very hard to keep it over 12 months. This is why the FDA now insists that drugs that might be approved achieve and maintain a 5% or higher weight loss over a 12-month treatment because less than that is not clinically meaningful. Also, drug treatment is not comparable to other ways of achieving dramatic weight loss such as bariatric surgery, itself not without dangers. Surgery is, however, very expensive and not acceptable or appropriate for every patient, but bariatric surgery can produce a 30% or higher weight loss that remains after 12 months.

Fen-Phen had been a spectacular success for the company American Home Products until it turned out that a large number of patients develop valvulopathy—changes in the heart valve—as a result of taking this drug. As this is an irreversible disease state leading to death in many cases, the subsequent lawsuits brought down this very large pharmaceutical conglomerate. History should record that Fen-Phen as a combined drug was never approved by the FDA and was the result of an off-label development at some U.S. academic institutions.

Similar amphetamine-based weight loss drugs by Servier were questioned and were recently withdrawn in France. Other weight-loss drugs have fallen on other safety issues. Some of the few approved and “safe” ones have very unpleasant side effects. For example, orlistat (Xenical) blocks uptake of fat by the intestine, which causes hard to control diarrhea with almost inevitable soiling. Despite this, GlaxoSmithKline entered the generic drug market with orlistat (Alli) as its first generic (see Chapter 04).

The development of weight loss drugs is very attractive but very difficult; the last 3 years have seen the industry bringing forward compounds or combinations of compounds that showed efficacy of 6–10% weight loss over 12 months, but which contributed to an increase in social isolation, suicidal ideation, and suicides. It forced the FDA to demand that companies specifically examine these side effects, and it finally asked its outside academic–clinical experts whether obesity is such a life-threatening condition that we should permit the taking of drugs that might significantly increase suicidality. The answer was no; we have to be able to find drugs that achieve the same weight loss without these serious side effects.

Other weight loss drugs have shown large increases in blood pressure and heart rate, and were deemed not safe. Presently, the industry is wondering if it is possible to make a truly safe obesity drug, while several biotechs are hampered by their financial inability to afford the long cardiovascular safety study rightly requested by the FDA.

But, unlike in the case of AD, the industry is not abandoning the area because it seems that the development of these drugs is easier in the sense that the scientific basis to fight obesity is better understood, and that the chance of proving that one has an efficacious safe drug is better than achieving the same in AD. In particular, the efficacy of the drug is shown and seen by the patients themselves within weeks. The other problem is maintaining this efficacy. It is so clear that there is a great desire in our world to achieve the ideal body weight determined by fashion and by health advisories of a body mass index (BMI) around 22–24. Despite a period of 3 years of efficacious drugs not being approved because they were not safe enough, it is less likely that the industry will abandon its search for obesity drugs.

When it comes to diabetes drugs, we start to recognize that the control of blood glucose can be further improved. The biggest contribution to improved control is not by drugs, but by continuous, daily, or repeated daily self-diagnosis of high blood glucose levels. These levels can be measured at home using the glucose meters developed in the last 30 years, which have become so cheap and reliable that all patients can use them. This so-called point of care device , of which we will see many in other indications, enables patients to control their own therapy, which in this case is of great importance. The first glucose meter developed by Boehringer Mannheim was as big as a refrigerator and required 2–4 ml blood. Today’s machines use just a drop of blood, are pocket-sized, and, in most countries, given free of charge. This locks the patients into buying the measuring strips, much like Kodak practically gave away the instamatic camera so you would buy its film. That this marketing works shows how widespread self-monitoring of blood glucose has become.

Secondly, we realize that there are drugs like the GLP-1 (glucagon-like peptide-1) analogs and inhibitors of the GLP-1 degrading enzyme DPP-4 (dipeptidyl peptidase-4), which can fine-tune glucose control and will postpone some of the diabetic complications.

The problem for those who are developing diabetes drugs is that we have a very large number of relatively efficacious, cheap, generic drugs such as metformin, 93 which have side effects but work quite well. For the largest number of patients these drugs will remain the mainstay of their diabetes treatment because they are well known and because they are cheap. The new drugs have to demonstrate significant improvements over the presently available insulin releasers, insulin sensitizers, and so on. In this quest for better control of blood glucose, we have also learned metabolic disease patients are accepting of injection, not only of insulin but also of other drugs. This opens the route for the use of biologicals in an area where this was not widely foreseen. Today, the GLP-1 analog exenatide (Amylin/Lilly’s Byetta—a synthetic version of exendin-4) and its competitor liraglutide (Novo Nordisk’s Victoza) are injectable biologicals generating revenues of more than a billion dollars each per annum. Together with insulin, we have now accepted in clinical practice other injectables to control blood glucose. In contrast, the inhibitors of DPP-4 are oral drugs: sitagliptin (Merck’s Januvia) and saxagliptin (BMS’s Onglyza).

In summary, it has to be said that the impact of the fight against obesity as a major risk factor for type 2 diabetes cannot be overstated. The problems of finding efficacious and safe obesity drugs are great, both practically and theoretically, because eating is so deeply and intricately programmed in our brain. So many processes of mental well-being are controlled by parts of the neuronal circuits that control repeated, pleasurable activities such as eating. Nevertheless, it is clear that here the industry continues to stand at the gates of the FDA with new drugs and drug combinations that can achieve significant weight loss.

Surgical versus drug interventions

A comparison of surgical procedures such as bariatric surgery to drug treatment is an important one. Those who work for insurance companies and major health plans calculate the risk–benefit ratios of major surgeries. One often finds that bariatric surgery is only recommended for those who are relatively young and who are enormously difficult to treat with drugs, diet, and exercise. Whether this will change with improvements in surgical procedures or not is hard to foresee. But it is clear that many patients will prefer any available oral or injectable drug if it comes close to the efficacy of the weight loss produced by a surgery where today mortality is over 1%.

Neuropathic pain

Morphine is one of our oldest drugs. The ancient Egyptians called it “God’s own medicine.” This was not because of its marvelous pain-killing properties, but because it effectively stops diarrhea, which is one of the great killers in the world. The existence and knowledge of how to dose morphine and other opiates has been the basis for the effective treatment of pain for a long time. Many thought that opiates could be useful in the treatment of all pain states. Neither the patients nor the anesthesiologists held this view and it is manifestly not the case. There is a very large group of pain syndromes—neuropathic pain resulting from neuronal injury and cancer pain—which are often not responsive to opiate painkillers. On top of this, opiates have huge safety problems that include constipation, alcohol interaction, and so forth, as well as their well-documented addiction potential. Relieving pain is one of the great dreams and aspirations of mankind. Yet the introduction of new efficacious pain medications is very, very slow. The inadequacy of all pain treatments is best illustrated by the following:

Most clinical trials for pain killers evaluate the efficacy of the medication using a visual analog scale. Patients judge for themselves the intensity of the pain on a linear analogue scale from “0” = no pain to “10” = excruciating pain. The gold standard of non-opiate, non-morphine-type pain drugs is gabapentin. Gabapentin is a more than $2 billion-selling drug that in its clinical trial reduced pain from an average of somewhat above “7” to an average of “4,” not to zero. The FDA suggests that patients may be recruited for pain trials if they assess their own pain to be above “4.” This means that even after having taken the best available drug many patients would still be eligible to enroll in a new trial for a painkiller.

Current practical treatment of pain may use multiple drugs. Physicians and patients learn to use several drugs to achieve some pain relief. The biggest problem is not that our basic pain research is lacking in effort, but that the animal models, in which we try to assess the efficacy of new pain medications, are not very reliable. Therefore, many clinical trials that were initiated after successful animal experiments have failed. There are additional big problems with developing new pain medications. This includes the potential for misuse of painkillers. People take them recreationally rather than because they have pain. Often these drugs, which were made for oral use, are being ground up and injected intravenously when their effect is much faster and much more robust in the brain, sometimes causing deadly accidents. So, whoever tries to introduce a novel serious painkiller has to prove or has to safeguard against abuse potential. This is difficult.

Painkillers are also the most often overdosed drugs. Patients in pain start taking the recommended dose then readily take more or more often when the pain does not abate. Therefore, these drugs have to be very safe even at 5 to 10 times the recommended dose. Another problem of many painkillers is their interaction with alcohol.

Despite these inherent difficulties, we cannot stop trying. In many ways painkiller development is ideal for the pharma companies as the trials are short: dental pain, 72 hours; postoperative pain, 3–7 days; and post-herpetic neuralgic pain, 7–14 days. It is just that what works in all the animal models consistently fails in patients, explaining why the industry has openly stopped trying in some very notable cases. Some of the largest pharmaceutical companies, such as Roche—the most profitable pharma company today—Novartis, Astra-Zeneca, and others, have openly closed their pain research units, saying that it is too difficult. This leaves the field to small companies or to companies such as Merck and Pfizer (previously active in COX-2 inhibitors, see Chapter 03) that are mostly only interested in one type of pain, inflammatory pain, for which we already have relatively good medication, as everybody who has been given ibuprofen by the dentist knows.

Pain is an example of an area of disease where the number of people affected is tremendous. The suffering is chronic. The economic opportunity therefore exists, yet the companies openly withdraw from competing in this area. This is calling for serious governmental entry into the field at the level of both basic and clinical research, which would help to define more valid, more reliable, and more translatable models, so that the existing argument of “whatever works in animals may or may not work in humans, and we will only know it $500 million dollars and 3 years later” will no longer be valid. Directive research by governments could change the situation and would, therefore, change the daily lives of tens of millions of people with chronic pain.

The huge medical need and market opportunity in neuropathic pain is thus dominated by gabapentin, a drug developed as the anti-epileptic Neurontin by Parke-Davis (now Pfizer), which is now more than 18 years old. It was approved in 1994 and has spawned many generic forms such as Fanatrex, Gabarone, Gralise, Nupentin, and so forth. Those who work in pain clinics are using a mixture of dedicated painkillers, anti-inflammatory drugs, tricyclic antidepressants, and anti-epileptic drugs to treat neuropathic pain, but their success is very limited.

In the 1970s and 1980s, the 20 large pharma companies, of which today exist less than 10 because of mergers and acquisitions, were not very interested in the area of oncology. The argument was that cancer, leukemias and solid tumors, are often discovered too late to do anything and that many cancers will not be treated easily because breast cancer, prostate cancer, lung cancer, renal carcinoma, pancreatic cancer, and so on, are clearly different diseases of different organs that will require completely different medications; therefore, the market is very fragmented. So, not only are the patients diagnosed late but there are also too few patients for each cancer form, maybe with the exception of some leukemias and lymphomas (see Table 5.3 ). However, it turns out we knew too little about some of the underlying cellular pathways and that certain controls of the cell division that runs amok in cancer are general in many cell types; thus, there will be carry over from developing a drug for one cancer indication (see the section “Effective governmental & societal intervention”). We were too organ focused, and not focused on cellular pathways.

Estimated cancer prevalence in the United States in 2007

Data include the 23 most prevalent cancers; totals include other less prevalent cancers, of which there are many.

In any clinical trial, the industry would not want to use, as the comparison, the most commonly used chemotherapy: the cytotoxic agents. These nucleotide analogs are still among the most efficacious cancer drugs, but with terrible side effects like hair loss, nausea, and weight loss. They are still used frequently, and these are drugs that are very cheap to make and very cheap to buy.

Big companies are not leaving the therapeutic area of oncology, although 2010–2011 was full of great disappointments when it came to clinical trials. This has shaken some of the best-selling cancer drugs such as bevacizumab (Roche-Genentech’s Avastin), which has been approved for treatment of non-small cell lung carcinoma and for colorectal cancer, but which has failed to show efficacy in breast cancer. This also may be initiating a possible re-evaluation of the original approval in non-small cell carcinoma, thereby endangering one of the best-selling cancer drugs from Roche.

Several of the spectacular clinical trial failures of 2010 were in oncology ( Table 5.4 ), but so were three of the most spectacular successes ( Table 5.5 ). Oncology remains a highly successful area, both medically and economically, for pharmaceutical companies. We also saw the first new therapeutic modalities such as the antibody-drug conjugate (ADC) brentuximab vedotin (Seattle Genetics’ Adcetris), approved in August 2011 for anaplastic large cell lymphoma (ALCL) and Hodgkin’s lymphoma; many others are in trials.

The top 10 phase 3 failures of 2010

Drug list is as reported by FierceBiotech. 1 The average phase 3 trial costs $400–750 million. All major pharma are present on the list. These companies have extensive drug development and clinical trial experience. The only small and new company is Novelos Therapeutics, Inc. of Madison, WI.

Advances in cancer therapies: new approvals 2009–2011

New drugs, new antibody-drug conjugates, and new orphan drug strategies by both small and large companies; phase 3; and approvals in 2009–2011. It is important to note three trends in this table: (1) companion diagnostics appearing and improving responder rate from 5–10% to 60%; (2) we have become—after 10 years and dozens of failures—good at designing protein kinase inhibitors that are less toxic than the first ones were (by not binding to the ATP-binding site of the enzyme), and we can often design a selective compound for the mutated protein kinase that leaves the non-mutated, non-cancer-related form free from inhibition; and (3) the therapeutic antibodies are coming in different formats and armed with different cell-killing mechanisms on top of selectively recognizing some marker of the cancer cell. Most of the entries in this table are first in class drugs, proving that we can, when we put the resources into it, innovate with cancer drugs. Why can we not in AD or pain?

Effective governmental & societal intervention

There are 40 times as many drugs being tested for cancers than for schizophrenia. Why? In short, the explanation is government-sponsored basic research. Today oncology 94 is the most drug target-rich area, whereas large chronic diseases, which affect up to 1–2% of the population over their entire lifetime, like schizophrenia, are extremely poor in drug targets.

The reason for the richness of drug targets in oncology is twofold. One is the decision by governments and NGOs, such as cancer societies, to engage large-scale sponsoring of basic research over several decades starting in the 1960s. Firstly, this research has shown that many molecular mechanisms are common in very different cancer forms. Therefore, even though on the surface prostate and ovarian cancer are clearly different and occurring in different genders, there are molecular pathway similarities. Effective drugs may affect basic underlying mechanisms of the repair of DNA, or the formation of vasculature, which is needed to supply the tumor with blood, and so on. These putative drugs might be used to treat more than one cancer form. Secondly, the discoveries of drug targets in leukemias and then in solid tumors have accelerated as a result of major public spending. When considering the importance of this, one has to remember that until the 1960s the pharma industry had been responsible for the basic research in most areas of life sciences (except embryology), which was the foundation for most of the drug developments. Since the 1960s, government-sponsored research in physical and life sciences in the United States and Western Europe has increased (the “Sputnik effect”) and has almost completely replaced the basic research efforts of big pharmaceutical companies. It is true that pharma spent $92 billion on research and development in 2009, but a very small portion, maybe $10 billion, of this was considered basic research. This is to be compared with the more than $60 billion spent annually on basic research by public and other sources. The NIH alone in the United States has a budget of over $30 billion.

The example of oncology shows that when politicians recognize the need either for popular or other reasons to support research, then they can initiate sustained and large-scale efforts that eventually produce scientific breakthroughs. HIV treatment is another area serving as a good example of the joint forces of patient interest groups, relatives of patients, celebrities, and governments to put pressure on a pharma industry that had not been in general very successful in developing antiviral agents. Yet, in the relatively short period of 20 years, HIV has switched from being a 100% deadly disease to a chronic disease that can be managed at several levels. This is one model, in which society can influence pharma by spending on basic research (to provide basic functional understanding and drug targets). Society would then rely on different sized companies, small ones first and bigger ones later, to pick up the scientific breakthroughs and apply them in the development of novel drugs. This is a very long-term, indirect way to find new drugs, but it has worked and it does not rely on specifying the commercial entities that will use the scientific results; instead it hopes that several will, and that they will do it in competition with one another.

There is also post-drug development encouragement and incentive by allocating NGO budgets to guarantee the purchase of a very large number of doses for developing countries. They exert a push–pull mechanism: the push on development and pull on production.

The examples of oncology and HIV cannot be underestimated. They are extremely important if we are not to give up work on Alzheimer therapies and on finding drugs against neuropathic pain and, for example, against schizophrenia, where the present drugs have relatively low response rates and are associated with severe metabolic side effects in many cases leading to early type 2 diabetes in young schizophrenics.

There are many good reasons to protect the freedom of researchers and not to direct all research, mostly because we really are not that good at predicting the truly innovative breakthroughs, many achieved through studying systems such as Escherichia coli , yeast, nematode worms, sea slugs, and fruit flies, and leading to cancer drugs. Nevertheless, one must not shy away from directing a much larger portion of the research budget to AD and antibiotics resistance than is happening presently. NIH and European research agencies already have large directed programs, but they are not large enough. Biotechs would be most helped if phase 2 and phase 3 clinical trials in AD were made possible with the partnership of government as an alternative to partnering to Big Pharma, which is currently not interested in these trials.

76 The Global Fund to Fight AIDS, Tuberculosis, and Malaria.

77 American Diabetes Association (2008) Economic costs of diabetes in the U.S. in 2007. Diabetes Care . Mar; 31(3):596–615.

78 See Figure 4.2.

79 See also Chapter 11.

80 See also Chapter 09.

81 Levy S et al. (2007) The diploid genome sequence of an individual human. PLoS Biol . Sep 4; 5(10):e254.

82 Venter JC et al. (2001) The sequence of the human genome. Science . Feb 16; 291(5507):1304–1351.

83 International Human Genome Sequencing Consortium; Lander, ES et al . (2001) Initial sequencing and analysis of the human genome. Nature . Feb 15; 409(6822):860–921.

84 Wheeler DA et al. (2008) The complete genome of an individual by massively parallel DNA sequencing. Nature . Apr 17; 452(7189):872–876.

85 Watson JD and Crick FHC (1953) A structure for deoxyribose nucleic acid. Nature . Apr 25; 171, 737–738.

86 In addition to antisocial behavior, cardiovascular disease, and wet ear wax (see http://en.wikipedia.org/wiki/Craig_Venter ).

87 See http://www.nature.com/news/2007/070528/full/news070528-10.html .

88 See http://www.nature.com/news/2008/080416/full/452788b.html .

89 See Chapter 08.

90 See also Chapter 11 where we may reveal that our argument is vindicated: one trial, designed in the way we recommend is now being conducted. For those readers who wish to “fast forward” you should look, for example, at http://www.multivu.com/mnr/56128-banner-alzheimer-s-institute-genentech-nih-prevention-trial-genetics .

91 Confidence has not been improved by the poor results from Lilly’s therapeutic antibody trial of solanezumab (see Chapter 06).

92 The approval for the imaging agent florbetapir (Amyvid) to image amyloid plaques from Avid-Lilly was, however, delayed over procedures of evaluation.

93 Metformin has had a long history. First synthesized in 1920, then trialled by a French physician in 1957, it was introduced in the UK in 1958 but not until 1995 in the United States. In 2010, 48 million generic prescriptions were filled. See http://en.wikipedia.org/wiki/Metformin .

94 perhaps together with inflammation.

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At Biotrial, we recognize that conducting clinical research studies for rare diseases presents unique challenges in terms of project management, site selection, and patient enrollment and retention. Given that children represent 50% of the rare disease population, the regulatory requirements specific to this age group represent an additional challenge that is mastered by our team of experts. We understand the urgent need to provide treatment options for patients living with rare diseases, and our full-service CRO offers innovative solutions to meet these challenges. Our services include:

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• Published: 15 April 2024

Targeting aging and age-related diseases with vaccines

• Ruochen Wu   ORCID: orcid.org/0009-0007-3379-8134 1 , 2 , 3   na1 ,
• Fei Sun 4   na1 ,
• Weiqi Zhang   ORCID: orcid.org/0000-0002-8885-5104 3 , 5 , 6 , 7 , 8 ,
• Jie Ren   ORCID: orcid.org/0000-0002-6450-3163 3 , 5 , 6 , 7 , 8 , 9 &
• Guang-Hui Liu   ORCID: orcid.org/0000-0001-9289-8177 1 , 2 , 3 , 5 , 8 , 10 , 11 , 12  

Nature Aging ( 2024 ) Cite this article

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Aging is a major risk factor for numerous chronic diseases. Vaccination offers a promising strategy to combat these age-related diseases by targeting specific antigens and inducing immune responses. Here, we provide a comprehensive overview of recent advances in vaccine-based interventions targeting these diseases, including Alzheimer’s disease, type II diabetes, hypertension, abdominal aortic aneurysm, atherosclerosis, osteoarthritis, fibrosis and cancer, summarizing current approaches for identifying disease-associated antigens and inducing immune responses against these targets. Further, we reflect on the recent development of vaccines targeting senescent cells, as a strategy for more broadly targeting underlying causes of aging and associated pathologies. In addition to highlighting recent progress in these areas, we discuss important next steps to advance the therapeutic potential of these vaccines, including improving and robustly demonstrating efficacy in human clinical trials, as well as rigorously evaluating the safety and long-term effects of these vaccine strategies.

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Acknowledgements

We are grateful to Z. Wu for providing critical advice for this Review, and L. Bai for her administrative assistance. This work was supported by the National Natural Science Foundation of China (81921006 to G.-H.L., 92149301 to G.-H.L.), the National Key Research and Development Program of China (2020YFA0804000 to G.-H.L., 2020YFA0803401 to J.R., 2022YFA1103700 to W.Z. and 2019YFA0802202 to J.R.), the National Natural Science Foundation of China (92168201 to G.-H.L., 92049116 to W.Z., 32121001 to W.Z. and J.R., and 31970597 to J.R.), the Strategic Priority Research Program of the Chinese Academy of Science (XDB0570100 to J.R.), the CAS Project for Young Scientists in Basic Research (YSBR-076 to G.-H.L. and J.R., YSBR-012 to W.Z.) and the New Cornerstone Science Foundation through the XPLORER PRIZE (2021-1045 to G.-H.L.). All figures were created with or modified from BioRender.com .

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These authors contributed equally: Ruochen Wu, Fei Sun.

Authors and Affiliations

State Key Laboratory of Membrane Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, China

Ruochen Wu & Guang-Hui Liu

Key Laboratory of Organ Regeneration and Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing, China

University of Chinese Academy of Sciences, Beijing, China

Ruochen Wu, Weiqi Zhang, Jie Ren & Guang-Hui Liu

Department of Cell Biology, Duke University Medical Center, Durham, NC, USA

Institute for Stem Cell and Regeneration, Chinese Academy of Sciences, Beijing, China

Weiqi Zhang, Jie Ren & Guang-Hui Liu

CAS Key Laboratory of Genomic and Precision Medicine, Beijing Institute of Genomics, Chinese Academy of Sciences, China National Center for Bioinformation, Beijing, China

Weiqi Zhang & Jie Ren

Sino-Danish College, School of Future Technology, University of Chinese Academy of Sciences, Beijing, China

Aging Biomarker Consortium, Beijing, China

Key Laboratory of RNA Science and Engineering, Beijing Institute of Genomics, Chinese Academy of Sciences and China National Center for Bioinformation, Beijing, China

Beijing Institute for Stem Cell and Regenerative Medicine, Beijing, China

Guang-Hui Liu

Advanced Innovation Center for Human Brain Protection, and National Clinical Research Center for Geriatric Disorders, Xuanwu Hospital, Capital Medical University, Beijing, China

Aging Translational Medicine Center, International Center for Aging and Cancer, Xuanwu Hospital, Capital Medical University, Beijing, China

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G.-H.L., J.R. and W.Z. designed and supervised the review and reviewed the manuscript; R.W. and F.S. wrote the manuscript and constructed the figures and tables. All the authors read and approved the article.

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Wu, R., Sun, F., Zhang, W. et al. Targeting aging and age-related diseases with vaccines. Nat Aging (2024). https://doi.org/10.1038/s43587-024-00597-0

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PRC has a wealth of experience in first-in-human trials, providing support for pre-study development before the launch of a study drug or biologic product. With numerous therapeutic areas of CRO expertise, PRC will help you navigate the complex regulatory landscape and ensure that your product is ready for clinical trials. We can help you achieve success in your pre-study development efforts.

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CDER Launches a Center for Clinical Trial Innovation

CDER provides scientific and regulatory advice to guide clinical trial development and implementation as a cornerstone in drug development, helping patients and consumers access the therapies they need. As part of an ongoing effort to innovate and enhance clinical trials, CDER is establishing the CDER Center for Clinical Trial Innovation (C3TI). C3TI will foster CDER’s innovation efforts and act as a central point for coordinating, sharing knowledge, and communicating with both internal and external parties.

In this CDER Conversation, Kevin Bugin, PhD, lead for C3TI and deputy director of operations in the Office of New Drugs (OND), explains the purpose of C3TI, the importance of clinical trial innovation, and ways in which C3TI can advance public health.

What is C3TI?

C3TI will be a central hub within CDER to support the implementation of innovative approaches to clinical trial design and conduct. C3TI’s mission is to promote existing and future CDER clinical trial innovation through enhanced communication and collaboration. This will ultimately improve the efficiency of drug development, bringing safe and effective drugs to patients as optimally as possible. C3TI will foster innovation across industry and therapeutic areas, and across new and ongoing initiatives within CDER. The goals of C3TI are to:

• Facilitate the sharing of lessons learned across CDER’s existing and new clinical trial innovation programs.
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• Manage a C3TI Demonstration Program that will expand opportunities for sponsors of drug development programs to work with CDER experts in enhanced ways to illustrate the value and impact of innovative approaches to the design and conduct of clinical trials.

Why did CDER create C3TI?

For years, CDER has supported new approaches in the design and conduct of clinical trials. However, drug development continues to evolve and now, more than ever, trials need to leverage the most fit-for-purpose designs and tools to efficiently and effectively generate evidence to support the regulatory decision-making for safe and effective new medicines. For this reason, CDER opened a public docket in late 2023 and held a two-day public workshop in March 2024, in collaboration with the Duke-Margolis Institute for Health Policy, to better understand opportunities that could improve the development and adoption of innovative approaches in clinical trials.

CDER heard that sponsors want our support integrating innovation into their trials for new drug development. CDER staff noted they would benefit from greater exposure to or experience with the use of innovations in their regular review work. There is a desire across the drug development “ecosystem” to integrate innovation, but there is a need for more direction on how to better bridge the gap between policy and implementation.

Based on this insight, C3TI will aspire to create more opportunities for staff to participate in development programs planning to implement novel trial approaches to gain experience with a given innovation, via a C3TI Demonstration Program. C3TI will also explore how to create new opportunities for CDER staff to receive training and exposure to new clinical trial methods, technologies, and tools. Lastly, C3TI intends to better capture and disseminate lessons learned from implementing a given clinical trial innovation through improved communication and coordination with colleagues (e.g., dedicated rounds to discuss clinical trial innovation) and with external parties (e.g., public meetings or webinars).

How can C3TI leverage opportunities in clinical trial innovation?

With advances in healthcare and technology, there are many ways to innovate clinical trials. C3TI can help us take advantage of these opportunities by fostering collaboration and knowledge sharing. For instance, while guidance documents have been critical to guide clinical trial innovation, real-world examples help us better understand how to implement these innovations. The experience from these examples, either identified by C3TI and its partners or generated through the Demonstration Program, can be shared broadly.

C3TI is also involved in planning public workshops, which can offer a unique forum to share and discuss case studies or examples. Our public workshop in March 2024 with Duke-Margolis identified several driving forces for clinical trial innovation, such as increasing the participation of diverse populations in clinical trials, increasing the number of clinical trials for rare diseases with participants with unique needs, and incorporating decentralized trial elements and digital health technologies. Furthermore, it was clear from this workshop that collectively we all benefit from discussing our real-world experience with implementing clinical trial innovation and should seek out future opportunities for similar collaborative discussions.

How does C3TI impact public health?

Clinical trials are a key step in the drug development process and for generating the evidence needed to support approving safe and effective drugs. By improving the efficiency and effectiveness of clinical trials, we hope to accelerate this process.

C3TI is very interested in increasing diversity and inclusion in clinical trials. This can potentially enhance the quality of clinical trial data and provide more reliable information on populations that may have been previously underrepresented in clinical trials such as racial and ethnic minority and indigenous populations, individuals with underlying genetic conditions, or those affected by environmental factors.

Rare diseases are another challenging area that C3TI will address. Many rare diseases have little or no approved therapies, which means there is rarely a clearly defined clinical roadmap for future drug development. Another potential obstacle is finding enough individuals to participate in rare disease clinical trials. C3TI will help in this area by supporting the broader understanding and adoption of the most fit-for-purpose clinical trial designs, ultimately accelerating the development and approval of new rare disease drugs.

What plans does C3TI have for the future?

As we look to the future, the vision of C3TI is to accelerate the pace of clinical trial innovation through collaboration and the promotion and adoption of useful innovations. In the near future, we’ll focus on our C3TI Demonstration Program , consisting of three initial project areas. The program is for selected sponsors of clinical trials in these specific project areas to serve as case examples. If selected, sponsors will have the opportunity for enhanced engagement with additional CDER staff within the context of their traditional interactions with the review teams. The goal of this program and selecting these case examples is to generate new lessons learned to share more broadly with the clinical trial community.

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Clinical Research Training to Support Capacity Building in Liberia

Building Capacity for Tuberculosis Research in China: The China TB Clinical Trials Consortium (CTCTC)

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Both countries identified a need to sustain the research capacity that was developed during their respective Ebola outbreaks. The partnerships were formed between the U.S. National Institute of Allergy and Infectious Diseases (NIAID) Division of Clinical Research (DCR) and the Ministry of Health (MOH) of each country. Their missions are to:

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From its inception in 2013 through 2020, we provided technical support and guidance to the CTCTC to implement strategies for clinical trial capacity building and partnered with other organizations to participate in the CTCTC, including the University of North Carolina Institute for Global Health & Infectious Diseases.

The CTCTC provides mentoring and advice around the following topics to maintain a successful tuberculosis (TB) clinical trial network in China:

• Training and quality monitoring to follow Good Clinical Practice (GCP) guidelines
• Establishing and maintaining reliable, accurate TB laboratory services
• Conducting ethical, effective patient recruitment and retention
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Read more about FHI Clinical’s role in the CTCTC.

Therapeutic Areas of Expertise

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• Gastroenterology
• Rare Diseases
• Ophthalmology
• Immunology / Autoimmune / Rheumatology
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