CERSI Collaborators: Jennifer Miller, PhD; Joseph Ross, MD, MHS; Sakinah Carter Suttiratana, PHD, MPH, MBA; Jason Schwartz, PhD; Reshma Ramachandran, MD, MPP; Harlan Krumholz, MD, SM; Michelle Mello, JD, PHD; Cary Gross, MD; Marcella Nunez-Smith, MD, MHS; Kiana McDavid

FDA Collaborators: Lola Fashoyin-Aje, MD, MPH; Asma Dilawari, MD; Bindu Kanapuru, MD; Timil Patel, MD

Project Start Date: October 2022

Regulatory Science Challenge

Policy efforts to improve representation and diversity in clinical research span decades, yet disparities in enrollment persist. New cancer therapies are often tested in clinical trials that enroll patients who are younger, healthier, and more likely to report White race than patients with conditions who are not in a research study. Women are under-represented in trials for some types of cancer too. When the demographics of the trial participants do not represent the demographics of patients for whom treatment is indicated, clinicians, payers, and patients can be left wondering how to interpret the results from the trial and how to apply them to different groups of patients. Research examining diversity in clinical research has shown some pharmaceutical companies are better than others at enrolling representative participants. This research group proposes to study the factors that distinguish these exceptional performing sponsors from others, using a positive deviance approach, to determine shared behaviors, strategies and contexts enabling them to perform better that can be generalized and implemented by the broader research community. Findings are anticipated to help advance health equity through better inclusion of under-represented populations in cancer drug research and development.

Project Description and Goals

Researchers will use a positive deviance approach (an approach that focuses on studying the positive deviants, or those who have achieved success, to understand the strategies, behaviors, and attitudes that contributed to their success) to identify shared strategies enabling trial sponsors to perform exceptionally, that is better than their peers, on enrolling diverse and representative participants in cancer research, that can be generalized and implemented by the broader research community to produce similar results. This will involve, first, identifying positive deviant sponsors, those demonstrating exceptionally high performance on adequately representing older adults, women, Asian, Black and Latinx identifying patients, via a cross sectional study. The study will compare trial participant demographics, for pivotal trials supporting novel cancer drugs approved by the FDA in 2012 through 2021, to those of the patients with the disease or condition targeted in a study, by constructing a participant-to-prevalence ratio (PPR) for each trial. Second, researchers will conduct a qualitative study to identify practices, strategies, and organizational contexts positive deviant trials and sponsors share, allowing them to achieve top performance.