International Clinical Trials Day on May 20: Reflect. Recognize. Applaud

Not all heroes wear capes. The world celebrates International Clinical Trials Day on May 20 to commemorate clinical research professionals and express reverence to James Lind, whose experiment with citrus fruit (in 1747) was one of the first reported clinical trials in medicine. It exhorted that oranges and lemons were a cure for scurvy. His service provided solace and hope to clinical investigators who strive to explore new treatments in medicine.

Timeline of significant developments in clinical trials over the last 30 years:

1990s	2000s	2010s	2020s
The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) was established in 1990 to develop guidelines for the pharmaceutical industry to ensure safety, efficacy, and quality of medicinal products. The US Food and Drug Administration (FDA) launched the Accelerated Approval Program in 1992, which allows faster approval of drugs for serious or life-threatening illnesses with preliminary evidence of clinical benefit. In 1997, the FDA issued guidelines on the inclusion of women and minorities in clinical trials to address the under-representation of these groups in clinical research.	The ClinicalTrials.gov database was launched by the National Institutes of Health in 2000, which allows researchers and the public to access information on clinical trials. The FDA issued guidance on the use of adaptive designs in clinical trials in 2006, which allows for modifications to the study design based on interim analysis. The FDA Amendments Act of 2007 required registration and results reporting of clinical trials on ClinicalTrials.gov, and mandated post-marketing safety monitoring for approved drugs.	The FDA issued guidance on the use of master protocols in clinical trials in 2013, which allows for the evaluation of multiple treatments and/or disease subtypes within a single trial. The Precision Medicine Initiative was launched by the US government in 2015, which aims to develop targeted treatments based on individual patient characteristics, including genetic information. The FDA issued guidance on the use of real-world evidence in clinical trials in 2018, which allows for the use of data from sources other than traditional clinical trials, such as electronic health records.	The COVID-19 pandemic has led to a rapid increase in the use of virtual and decentralized clinical trials, as well as accelerated approval pathways for vaccines and treatments. The FDA issued guidance on the use of digital health technologies in clinical trials in 2020, which allows for the use of devices and software to collect and analyze patient data remotely. Overall, the last 30 years have seen significant advancements in clinical trial design, data collection and analysis, and regulatory oversight, aimed at improving the safety, efficacy, and efficiency of clinical research.

 

As the science of clinical trials has evolved, clinical testing progression and design have garnered headlines. Clinical trials need to be given the green signal to be started, and even children can partake in the research. In the United States, for instance, drug developers must submit an Investigational New Drug (IND) application to the FDA before beginning clinical research. Clinical trials are conducted across different stages, known as phases.

Phase I	Focus on safety and dosage	15-50 participants
Phase II	Focus on effectiveness and side effects	<100 participants
Phase III	Comparison of new and existing treatment/drug	Hundreds of participants
Phase IV	Focus on approval and availability. Long-term effects are observed	Thousands of participants

 

Pulse of Clinical Research in Rare Diseases

Science mandates curiosity, grit, and tenacity-factors that are relatable to rare disease clinical trials and research. An estimate suggests that more than 300 million people are living with rare diseases globally.

Scientific innovation, enhanced rare disease awareness, and public policies that underpin research and development are a breath of fresh of air. Fostering clinical research in rare diseases will require patient communities, specialist clinicians, and key opinion leaders to adopt a patient-centric approach. Reducing the diagnostic journey could be an invaluable step toward addressing patients’ unmet needs.

In January 2024, the U.S. FDA reportedly issued final guidance on rare disease drug development. The finalized guidance covers trial design, nonclinical pharmacology and toxicology, endpoint selection, drug manufacturing issues, and the evidence standards for establishing safety and effectiveness. With rare diseases becoming a global health priority, accurate diagnosis and timely access to treatment will transform patient lives.

As pharmaceutical and biotech companies strive to prevent delays and reduce costs, an emphasis on clinical trials procurement could lead to achieving supply chain flexibility in manufacturing, sourcing, labeling, packaging, and distribution. For instance, a quick response to changes in trial arms, formulations and dosing levels can propel clinical trials logistics supplies. Besides, focusing on sourcing strategies could be a robust move to boost cost-effective solutions and bring treatments to patients faster.

Combating Stigma-filled Challenges through Mental Health Trials

New research from the U.S. CDC (quoted by CNN in June 2023) found that almost 15% of children in the U.S. were treated for mental health disorders in 2021. The finding claimed that mental health disorders, including attention-deficit/hyperactivity disorder or anxiety, are prevalent among school-age children. The stigma around mental health problems and how communities and cultures perceive mental health have furthered the significance of pediatric clinical trials.

Stakes are high on mental health clinical trials as research funders, ethicists, regulators, and patient groups seek treatment options for depression, anxiety, and schizophrenia. To illustrate, in March 2024, Acadia Pharmaceuticals reportedly announced top-line results from the Phase 3 ADVANCE-2 trial assessing pimavanserin to treat negative symptoms of schizophrenia.

Harnessing Virtual Clinical Trials

Decentralization of clinical trials refers to the use of technology and remote methods to conduct clinical trials, as opposed to traditional in-person methods. Decentralized clinical trials (DCTs) or Virtual Clinical Trials are designed to improve patient access, reduce patient burden, and increase the efficiency of the clinical trial process.

Virtual clinical trials have gradually gained ground to fill the chasm left by traditional trials. The former uses smartphone applications, electronic monitoring devices, and video conferencing. So, what makes online clinical trials stay cut above the rest?

Virtual trials can boost retention, ramp up participants’ recruitment, and propel participant diversity and engagement. Pharmaceutical companies may seek remote trials to minimize drug development costs. As the duration of the study is pruned, data is collated more rapidly and enrollment periods are reduced.

Since the COVID-19 pandemic onslaught, patients, clinical trial sponsors, and pharmaceutical companies have largely relied on virtual clinical trials. For instance, in February 2024, the National Cancer Institute (NCI) announced the rollout of the Virtual Clinical Trials Office (VCTO) pilot program.

• Gamification and remote sample collection are some of the notable advancements that have added a fillip to clinical trial services. Gamification can motivate participants to reach trial milestone and comply with trial protocols.

• A mobile app can be used to track sample collection and transport and get participants to collect samples at home, fostering clinical trials support services.

• Lately, the use of AI has propelled clinical data analytics solutions with the automation of clinical trial processes.

• Stakeholders are wielding the power of generative AI models to detect discrepancies and provide compliance and safety insights.

• Heart diseases have become rampant globally; one person succumbs to cardiovascular disease every 33 seconds in the U.S. Meanwhile, 7.6 million people are living with a heart or circulatory disease in the U.K.

• The prevailing trends have compelled European researchers to build Virtual Physiological Human (VPH) to boost care standards and turn basic science into real medical practice. Researchers, sponsors and other stakeholders are gearing up to overcome challenges in cardiovascular clinical trials.

While patient-centric trials have gained prominence, researchers are expected to foster their efforts to minimize morbidity and mortality. The Strategic Research Agenda for CVD, in partnership with the European Society of Cardiology and the European Heart Network, found out major research priorities:

• Treatment of chronic heart failure and atrial fibrillation;

• Earlier recognition of cardiovascular disease;

• Personalized treatment;

• The interaction between CVD and other disorders;

• Repair of the heart and blood vessels.

In June 2023, the multi-stakeholder group reportedly issued a second joint statement, emphasizing the requirement for complete datasets for reliable research outcomes. It also warned against the risks of datasets underrepresenting specific segments of the population.

An emphasis on reducing patient burden, optimizing study protocols, monitoring efficacy and safety endpoints can underscore cardiovascular clinical trials.

Challenges in HIV Clinical Trials

Drug developers and scientists are developing next-generation HIV vaccines amidst a surge in HIV-infected people. In November 2023, a U.S. government website (HIV.gov) noted that around 39 million people globally had HIV or AIDS in 2022. Since HIV is one of the most lethal infectious diseases and people do not have access to prevention, treatment and care (there is still no cure), challenges keep mounting to boost HIV clinical trials. In January 2024, Novotech claimed that the biopharma industry had begun more than 1,000 HIV clinical trials since 2018 (globally).

Navigating Neurology Clinical Trials

Neurological conditions have become rampant, becoming one of the leading causes of disability and ill health globally. So much so that more than 80% of neurological deaths and health loss have reportedly been witnessed in low- and middle-income countries. Some of the major neurological conditions are:

• Migraine

• Stroke

• Dementia

• Neonatal encephalopathy (brain injury)

• Meningitis

• Diabetic neuropath

• Autism spectrum disorder

• Nervous system cancer

With the prevalence of neurological diseases, drug developers have emphasized investments in neurology clinical trials to enhance treatment and quality of life. The WHO cited the Lancet Neurology’s study, noting that prevention of exposure to lead could minimize the burden of idiopathic intellectual disability by 63.1%.

What Next?

Clinical trial sponsors and pharmaceutical companies will continue to witness traction for brick-and-mortar research facilities and virtual trials. Biopharmaceutical companies will potentially focus on customized drug development activity amidst a surge in data volume and diversity. Indeed, International Clinical Trials Day celebrates invaluable research that takes place to boost healthcare and provide high-quality evidence on health interventions.

Explore more research reports on Clinical Trials

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• Cardiovascular Clinical Trials Market Size, Share & Trends Analysis Report, 2023 - 2030
  

• Rare Disease Clinical Trials Market Size, Share & Trends Analysis Report, 2023 - 2030