October 28, 2026

Doctors for America Calls for Consistent Evidence Standards, Transparency, and Public Engagement in Rare Disease Drug Review

WASHINGTON, D.C. – Doctors for America’s FDA Task Force sought to offer comments at today’s FDA Rare Disease Framework Initiative Public Meeting, convened in partnership with the Reagan-Udall Foundation, but high demand for public comment exceeded the available speaking slots. The overwhelming interest in speaking underscores the need for and value of open dialogue and transparency. In that spirit, we are sharing our planned comments and recommendations for today’s important discussion.

DFA welcomes the FDA’s goal of greater clarity, consistency, and predictability in rare disease regulatory decision-making. For patients and physicians, predictability means a stable, transparent standard of evidence applied consistently across products, review divisions, and leadership changes. Flexibility in how evidence is generated is appropriate for rare diseases. Flexibility in whether a drug must be shown to work is not. Our patients with rare diseases and their families deserve better.

DFA’s FDA Task Force recommendations emphasize three priorities for the agency’s approach to rare disease drug development:

Consistent, publicly explained standards. Maintain the substantial-evidence standard for effectiveness, clarify how different forms of evidence will be weighed, and publicly explain departures from prior agreements on trial design or endpoints.

Transparency and accountability. Develop policy through public notice and comment, publish complete review records, report on consistency across review divisions, and support the experienced FDA workforce needed for rigorous review.

Independent, public expert scrutiny. Routinely convene advisory committees for rare disease applications involving substantial uncertainty, ensure relevant expertise and rigorous conflict-of-interest screening, and explain FDA decisions that depart from committee recommendations.

Patients with rare diseases need therapies that work, and their physicians need confidence that FDA approval means a drug has been shown to work. Consistent evidence standards and meaningful public engagement are essential to that trust. DFA urges the FDA to fully consider the perspectives of those unable to speak today and welcomes further discussion of these recommendations.

Full comments can be read here.

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About Doctors for America:

Doctors for America (DFA) is a national nonprofit that mobilizes over 40,000 physicians and medical trainees to advocate for policies that improve the health of patients and communities. Through advocacy training and action at the state and federal levels, DFA works to expand access to affordable care, strengthen community health and prevention, and advance health justice and equity. We do not accept funding from pharmaceutical, insurance, or for-profit health care entities, ensuring our work remains fiercely independent and patient-centered. DFA puts patients over politics–and over profits. Find out more at doctorsforamerica.org and on X @drsforamerica or Bluesky drsforamerica.bsky.social.

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