The FDA rightly demands that pre-market clinical trials provide information on the safety and effectiveness of drugs for all gender, age, and racial subgroups in order for drugs to be approved. The population being studied should match the demographics of the disease, and under-represented patients should have reliable data for their care. We know that efficacy and toxicity of some medications can vary in different races, ethnicities, genders, or ages. However, the concern with enrolling a sufficient number of under-represented patients has long been about delaying development and approval of new therapies for everyone.
The FDA has now proposed that if best efforts are made in the pre-marketing registration trials to include underrepresented patient populations, the Agency will consider other post-market approaches to data collection on these additional populations. The FDA provides guidance on two approaches. First, the Agency asks if there are other data sets already being collected that could be mined for data on underrepresented patients (e.g., adverse event data). And second, it proposes post-marketing data collection on subpopulations. This data could be collected after approval, thus addressing the concern of slowing treatments to market, while also ensuring all patients have needed data on safety and efficacy. A separate clinical trial, or even pooling (meta-analysis) of data from multiple trials, and/or using real-world evidence were all proposed.
The full details of the FDA draft guidance can be found here: Postmarketing Approaches to Obtain Data on Populations Underrepresented in Clinical Trials for Drugs and Biological Products: Guidance for Industry.
CLL Society applauds the FDA for approaching this long standing problem with flexibility that allows patients access to potentially lifesaving medications sooner, while still requiring drugs be appropriately studied in all populations that might benefit.