Suggested Changes to ‘Underrepresented’ Draft Guide
The Duke-Margolis Center for Health Policy says it supports any notion suggesting that several guidances issued by FDA on real-world data (RWD) and real-world evidence (RWE) and early conversations with the relevant agency review divisions should form the basis around uses of RWD/E to answer research questions in postmarket settings about medical product safety and efficacy. The center suggests that FDA provide more specific guidance on the use and appropriateness of RWD in postmarket settings to obtain RWE on treatment outcomes in populations underrepresented in clinical trials, regardless of the reasons or basis for the underrepresentation.
“Overall,” the letter says, “we believe FDA could provide more specific guidance on postmarket study mechanisms it might support to more closely observe medical product safety and efficacy among underrepresented populations in real-world treatment settings.”
The Biotechnology Innovation Organization says its members would appreciate clarity on how FDA will ensure that the issuance of PMRs/PMCs (postmarketing requirements/postmarketing commitments) around diversity and hence its overall approach to inclusive research is consistent across oncology and non-oncology review divisions. It also asks FDA to describe how sponsor development teams can receive agency feedback from reviewers on the progress toward meeting enrollment goals throughout the development program.
Finally, the National Organization for Rare Disorders (NORD) recommends that the agency provide more nuanced guidance on the effective and efficient use of postmarket studies to close key data gaps about the safety and/or effectiveness of a product for historically underrepresented populations. It asks for more detailed and tangible guidance on how to appropriately integrate pre- and post-market data collection into ongoing rare disease development programs to appropriately incentivize rare disease drug development. And it urges FDA to develop and publish best practices and lessons learned to provide examples of successful and potentially unsuccessful pre- and post-market data collection on safety and effectiveness in historically underserved populations.
A second overarching recommendation is for additional guidance to help sponsors meet premarket trial diversity goals and ensure post-market data collection is not abused as a substitute for appropriate pre-market efforts to ensure clinical trials accurately represent the whole patient population