The Flexibility Papers, No. 2: Beyond Listening—Making Patient Input Matter By James E. Valentine & Sarah Wicks & Jason Mercer, Ph.D. & Mary Bass & Gayatri R. Rao —

The Flexibility Papers, No. 2: Beyond Listening—Making Patient Input Matter By James E. Valentine & Sarah Wicks & Jason Mercer, Ph.D. & Mary Bass & Gayatri R. Rao — In the first installment of The Flexibility Papers, we outlined several principles that we believe should guide FDA’s new Rare Disease Framework Initiative. Among them was a fundamental shift in the starting point for rare disease drug development: rather than treating conventional development approaches as the default and requiring sponsors to justify departures, FDA and sponsors should begin with the characteristics of the disease, available evidence, unmet medical need, and patient perspectives to identify a fit-for-purpose development pathway. https://www.thefdalawblog.com/2026/11/the-flexibility-papers-no-2-beyond-listening-making-patient-input-matter/?utm_source=rss&utm_medium=rss&utm_campaign=the-flexibility-papers-no-2-beyond-listening-making-patient-input-matter The Flexibility Papers, No. 1: First Principles for a Rare Disease Framework October 29, 2026 By James E. Valentine & Gayatri R. Rao & Frank J. Sasinowski — Yesterday, FDA and the Reagan-Udall Foundation for the FDA convened a public meeting to kick off stakeholder engagement on FDA’s new Rare Disease Framework Initiative. And while there is no shortage of opinions about how FDA should approach rare disease drug development, this time the Agency is asking stakeholders to help build the framework itself. https://www.thefdalawblog.com/2026/10/the-flexibility-papers-no-1-first-principles-for-a-rare-disease-framework/?utm_source=rss&utm_medium=rss&utm_campaign=the-flexibility-papers-no-1-first-principles-for-a-rare-disease-framework