# The Scientific EXPERT Act: Making It Easier to Develop Drugs for Rare Diseases

This bill creates a new process to help speed up the development of medications for rare diseases and conditions. Right now, when pharmaceutical companies are developing drugs for uncommon illnesses, they often work in isolation with the FDA, and different experts may have conflicting ideas about what kind of evidence is needed to prove a drug works. This bill tries to fix that by bringing everyone together in organized meetings to align on the best scientific approaches.

Here's what the bill actually does: It requires the FDA to work with an independent organization called the Reagan-Udall Foundation to hold at least four public meetings each year focused on specific rare diseases. These meetings would bring together drug companies, medical experts, patient advocates, and FDA staff to discuss the scientific challenges in developing treatments. For example, they might discuss what kind of clinical trials make sense for a disease with very few patients, or what measurements should count as proof that a drug is working. The goal is to reach agreement on the best path forward before companies invest millions in development that might hit roadblocks later.

The meetings would be open and transparent—the FDA would publish transcripts, recordings, and summaries of what was discussed within six months. When the FDA eventually approves a drug for one of these rare diseases, it would have to publicly explain whether it held a relevant meeting and how the discussion influenced its decision. The bill also requires annual reports to Congress about how many meetings happened, who participated, and whether the meetings actually helped speed up drug approvals.

This is primarily good news for people with rare diseases and the companies trying to develop treatments for them, as clearer guidance could mean faster access to new medications. However, the bill includes safeguards to make sure it doesn't create unnecessary delays—it explicitly states that these meetings won't increase the number of review cycles drugs have to go through, and it protects confidential business information from being disclosed.

Scientific EXPERT Act of 2025

Matsui, Doris O.

Scientific External Process for Educated Review of Therapeutics Act of 2025 or the Scientific EXPERT Act of 2025This bill requires the Food and Drug Administration (FDA) to facilitate and participate in externally led, science-focused drug development meetings to discuss the development of treatments for rare diseases and conditions.&nbsp;The FDA must enter into an arrangement with the Reagan-Udall Foundation for the FDA under which the foundation agrees to convene such meetings. Meetings must be held at least four times a year, and each meeting must focus on a different rare disease or condition.&nbsp;The foundation must establish a permanent steering committee to review and recommend topics for each meeting. In evaluating potential topics, the committee must consider unmet therapeutic needs, patient population sizes for different diseases and conditions, and whether a disease or condition would benefit from clarity and alignment on drug development questions, among other factors.&nbsp;In planning each meeting, the foundation must develop a list of medical experts, drug sponsors, scientific organizations, patient organizations, and other entities to be invited to participate. Representatives of the FDA’s review divisions must attend each meeting.&nbsp;After each meeting, the foundation must make available a summary of the meeting noting areas of consensus, areas where additional clarification or information is needed, and next steps agreed upon with the FDA.The bill also requires the FDA to indicate whether it incorporated any input from these meetings when approving a new drug or biologic.&nbsp;