Clinical Trials Day
CDER Center for Clinical Trial Innovation (C3TI)
C3TI Champion Mitchell Psotka Reflects on the Importance of Innovating Clinical Trial Enrollment
Mitchell Psotka, MD, PhD, is a Medical Officer in the Division of Cardiology and Nephrology (DCN) in the Office of New Drugs (OND) in the Center for Drug Evaluation and Research (CDER). He is a C3TI Champion and chairs the Digital Health Working Group for the Heart Failure Collaboratory, a public-private partnership with FDA. He also serves as the System Section Chief of Heart Failure and Transplant and Director of the Reinsch Pierce Cardiomyopathy Center at Inova Schar Heart and Vascular in Falls Church, Virginia. Clinically, he specializes in heart failure, heart transplant, mechanical circulatory support, pulmonary hypertension, and infiltrative cardiomyopathies.
My time treating and counseling patients about cardiovascular clinical trials inspired me to want to make trials more efficient, which ultimately led me to join efforts like the CDER Center for Clinical Trial Innovation (C3TI). In my work as Director of the Infiltrative Cardiomyopathy Center at Inova Fairfax Hospital, before coming to FDA in 2020, I regularly advised patients to consider clinical trial participation when they exhausted other therapeutic options or were seeking access to potential novel therapies. I often heard patients express concerns about randomization, the possibility of receiving placebo therapy, and the uncertainty around whether an experimental treatment would help them. Even when patients were randomly assigned to receive a novel therapy, there was no guarantee they would respond. It could take many years of perseverance before anyone knew whether their participation had a meaningful effect on them or for others with the same disease.
Patients also faced logistical challenges, such as coming in for regular appointments, completing additional testing and questionnaires, and adhering to the rules of study participation. However, I noticed patients would be motivated when I emphasized their participation could help generate evidence to support drug development and potentially benefit future patients. Even so, enrollment in cardiovascular clinical trials remains low today, despite the significant burden heart disease places on society.
Seeing these enrollment challenges firsthand drew me to C3TI and specifically to programs like Selective Safety Data Collection (SSDC), one of C3TI’s Demonstration Project Areas where I now serve as a co-lead. SSDC aims to reduce the collection of certain types of data in clinical trials for products with well‑characterized safety profiles. Among other benefits, SSDC can lessen the burden on clinical trial participants. Ultimately, I see programs like these paving the way for more efficient—and hopefully faster—evidence generation, which can lead to new therapies and new options for patients.
Clinical Trials: A Critical Part of Drug Development
We celebrate Clinical Trials Day on May 20, in recognition of what is often considered the start of the first randomized clinical trial. In 1747, James Lind began a clinical trial on this day to study the effects of different remedies for scurvy among sailors at sea. His research famously provided evidence of the benefits of citrus fruits to treat this disease. Clinical trials have undergone many changes since the time of James Lind, not only in respect to medical, scientific and technological advances, but also in terms of ethical and legal requirements. Regardless, clinical trials remain a critical part of drug development, as a means of generating evidence to support the regulatory decision-making for safe and effective new medicines.
Celebrate Clinical Trials Day
May 20 is an opportunity celebrate the individuals who participate in clinical trials, as well as those professionals who help run clinical research and the FDA staff who review and interpret results from these trials. It is also a chance to emphasize the importance of innovating clinical trials. Despite the tremendous progress we have made, there are new medical challenges and quandaries we have not solved — both of which require innovation and creative thinking. That is why through the CDER Center for Clinical Trial Innovation (C3TI) and other initiatives, CDER is working to further existing clinical trial innovation programs and spur future activities.