By Gary Zammit | Forbes Books Author for Forbes Books | AUTHOR POST | Paid Program
Clinical trials are supposed to drive advances in medicine to benefit patients. When there is a lack of diversity in clinical trials, we compromise data quality, miss critical safety signals, and develop treatments that may not work for everyone.
Different groups of people—men, women, children, and people of different races or ethnic backgrounds—respond differently to different drugs. One of the best-known examples of this is BiDil®, a medicine used to treat congestive heart failure. In clinical trials, the medicine didn’t have the desired efficacy across the entire population. However, when a sub-analysis was done, and subject samples were differentiated by race, it was found that Black people responded very well to the drug. BiDil subsequently became the first drug approved by the FDA for a single racial-ethnic group.
Recognizing the risks posed by a lack of diversity in clinical trials, the Food and Drug Administration (FDA) introduced an action plan to address the issue. Unfortunately, these guidelines are insufficient, in my view. It’s thus on the people who design clinical trials to fill the gaps—starting with implementing mandatory minimum racial and ethnic inclusion requirements directly in trial protocols.
The Importance of Diversity in Clinical Trials
If we are going to develop treatments that are safe and effective for all patients, we need to test them in populations that reflect the diversity of people who will use them. Unfortunately, we are currently seeing significant variability in trial enrollment.
In early-stage phase I studies, when drug safety is the primary concern, clinical trials tend to include a higher proportion of people of color. In later-phase trials—phase II and phase III—where documentation of efficacy is a primary concern, clinical trials tend to have higher proportions of Caucasian participants.
As a result, you have cases where massive later-phase clinical trials have been run, only for safety signals (information about a new adverse event warranting further investigation) to show up in these populations, previously underrepresented in the early development process, very late in development.
How Trial Design Can Help Promote Clinical Trial Diversity
Clinical trial protocols represent the methodology used in drug or device development. Protocols are detailed documents that outline key characteristics of a trial, including type of study design, placebo control, blinding, randomization, sample size, and subject selection (who is included in the trial). This is where diversity considerations arise.
Inclusion and exclusion criteria are used to select which subjects to include in clinical trials. Inclusion criteria are key clinical features of the target population that patients must have to be included in the clinical trial. Exclusion criteria are key clinical features that patients must not have to be included in the trial.
Inclusion and exclusion criteria impact the generalizability of a study’s results—the degree to which the results of a research study can be applied to a broad population of people beyond those included in the clinical trial. It is one factor that determines how meaningful, or useful results will be to patients who might receive treatment in clinical settings after a new treatment has been approved.
Subject selection and generalizability represent opposing sides of a double-edged sword. You want homogenous samples that maximize subject similarity at baseline, but you don’t want to define samples so tightly that the results are not generalizable. One approach is to establish minimum racial and ethnic inclusion requirements directly in trial protocols.
Diversity Benefits Patients Today and Advances Medicine Tomorrow
Without diversity in clinical trials, it’s impossible to develop safe and effective treatments for all patients. Failing to address this issue also impedes future scientific progress, like precision medicine. In the future, patients may walk into a doctor’s office and receive a simple biomarker or DNA test that determines which medication will work best for their unique genetic makeup. Understanding drug safety and efficacy in diverse groups brings us closer to that ideal. Without this knowledge, we will be unable to achieve the holy grail of precision medicine, limiting the promise of future advances for tomorrow’s patients.
By Gary Zammit, AUTHOR POST | Paid Program. Gary Zammit, PhD, founded Clinilabs in 2000, driven by his vision to improve central nervous system drug and device development through specialized contract research. Now an award-winning entrepreneur, he is widely recognized for his expertise in both neuropsychiatric drug development and sleep medicine. As president and CEO of Clinilabs, Zammit leads a team dedicated to advancing treatments for conditions affecting the central nervous system. He simultaneously serves as a clinical professor of Psychiatry at the Icahn School of Medicine at Mount Sinai in New York and is a distinguished Fellow of the American Academy of Sleep Medicine.
Zammit earned his PhD from the University of Toledo, where his work in biological psychiatry earned him both the Turin Service Award and the Leckie Scholar Award. His postgraduate training included an internship and clinical research fellowship at the New York Hospital-Cornell University Medical College, where he was recognized with the Alumni Award for Excellence. Throughout his career, Zammit has authored two books and over 250 articles and abstracts related to clinical practice, sleep, and CNS drug development. His professional mission remains steadfast: developing innovative drugs and devices to treat psychiatric and neurological disorders, ensuring patients have access to better, more effective, and safer treatments that improve health outcomes and quality of life.
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