Scientific EXPERT Act of 2025

This bill would set up regular, science-focused meetings, led by an outside foundation, to help speed up the development of treatments for rare diseases. These meetings would bring together FDA reviewers, doctors, patients, and drug developers to work through tough science questions—like how to design studies, which endpoints to use, and when biomarkers or natural history data can stand in for longer trials—so promising medicines can move forward faster and more safely.

The Reagan-Udall Foundation would host at least four of these meetings each year, each focused on a different rare disease or group of rare diseases. A steering committee—including FDA centers, medical experts, patient representatives, and industry—would help choose topics based on need and potential impact. FDA reviewers must participate. After each meeting, within 180 days, a transcript and a plain-language summary must be posted, highlighting what people agreed on, what’s still unclear, and next steps.

The FDA would also publicly say, when it approves a relevant drug or biologic, whether and how it used input from these meetings, and it must publish a yearly summary of the meetings and their impact. The bill allows up to $1,000,000 per year from 2026 through 2030 to support this work and clarifies that it doesn’t change confidentiality protections or force the FDA to meet with specific people.

• Who is affected: People living with rare diseases and their families; medical experts and researchers; drug developers; FDA staff.
• What changes: At least four outside-led meetings per year; a steering committee to pick topics; FDA participation; public transcripts and summaries after each meeting; FDA statements on how meeting input shaped approvals; an annual report on impact.
• When: Meeting summaries posted within 180 days of each meeting; funding for 2026–2030.