27-29 February 2024
The U.S. Food and Drug Administration (FDA) has published a draft guidance on postmarketing approaches to obtain data on typically under-represented populations in clinical trials (CTs). The purpose of this document is to describe FDA requirements and provide recommendations for obtaining safety and effectiveness information on drug and biological products in the postmarketing setting.
FDA regulations require sponsors to present information from premarket clinical trials on the safety and effectiveness of drugs. These CTs should include patient populations that are historically underrepresented in clinical research (e.g., populations based on ethnicity, sex, or age). The agency strongly recommends sponsors to obtain information from a diverse, representative patient population early in drug development. However, if, despite the sponsor’s best efforts, these populations are not adequately represented in premarket CTs, it may be appropriate to collect such data in the postmarketing setting.
There are various mechanisms for obtaining postmarketing data on underrepresented populations. The FDA may require an applicant to conduct postapproval studies or CTs as a postmarketing requirement (PMR), or the agency may enter into a written agreement with the applicant to collect these data as a postmarketing commitment (PMC). However, before requiring a postmarketing CT, the FDA must find that a postmarketing study will not be sufficient to meet those purposes.
Moreover, the agency may approve a marketing application based solely on foreign clinical data if the data are applicable to the U.S. population and U.S. medical practice. If a sponsor submits a marketing application comprised of patients enrolled predominantly outside of the United States, data and rationale should be submitted to support applicability to the U.S. population and medical practice. The agency may request studies or trials in addition to further characterize the efficacy or safety of the product in subpopulations relevant to the U.S. population.
More Information is avalaible in FDA's draft guideline entitled “Postmarketing Approaches to Obtain Data on Under-Represented Populations in Clinical Trials”.