New Act Creates Formal Scientific Meetings to Speed Up Rare Disease Drug Approvals

The Scientific EXPERT Act of 2025 is designed to tackle one of the toughest problems in medicine: developing treatments for rare diseases. The bill establishes a new, formal process for scientific collaboration called Externally Led, Science-Focused Drug Development (ELSFDD) meetings. Think of it as creating a structured, high-level study group dedicated to figuring out the scientific roadblocks that slow down drug development for conditions that affect few people. The goal is simple: get life-changing drugs to patients faster by solving the hard science problems early.

The Science Huddle: What This Means for Drug Development

Under this bill, the Secretary of Health and Human Services (HHS) must contract with an external organization, referred to as the “Foundation,” to run these ELSFDD meetings. This Foundation is tasked with hosting at least four meetings annually, each focusing on a different rare disease or group of related conditions (Sec. 2). The core idea is to bring everyone to the table—academic researchers, patient groups, drug companies, and medical experts—to agree on the best way forward, whether that’s standardizing clinical trial designs, deciding what measures to use, or even determining if natural patient data can replace a traditional control group.

To keep things on track, a Science-Focused Drug Development Multistakeholder Steering Committee is created. This committee, which must include representatives from key FDA centers (like the Center for Drug Evaluation and Research), practicing doctors, and patient advocates, will advise the Foundation on which diseases to prioritize (Sec. 2). They’ll look at factors like patient need and whether a discussion could help speed up development for similar conditions. This structure ensures that the discussions focus on the diseases where scientific clarity is most urgently needed.

Transparency and Accountability in the Review Process

For anyone invested in the development of these treatments—from patients waiting for a cure to small biotech companies needing clear guidance—this bill mandates a major boost in transparency. No later than 180 days after any ELSFDD meeting, the Foundation must post a full transcript and recording of the entire session on the FDA's website (Sec. 2). They also have to publish a summary analysis highlighting where experts agreed, where they still need more data, and what the agreed-upon next steps with the FDA will be.

Crucially, the bill ties this external input directly to the FDA’s final decision-making process. When the FDA approves a drug that was discussed in one of these meetings, they must publicly state that an ELSFDD meeting was held and describe exactly how the input from that meeting was used in their final risk-benefit assessment for the drug (Sec. 2). This is a big deal, as it forces the agency to formally acknowledge and justify how they incorporated (or didn't incorporate) the consensus scientific advice they received, ensuring that the meetings aren't just for show.

Real-World Impact and the Fine Print

For patients and families dealing with rare diseases, this bill offers a more structured path to potential treatments by clearing up scientific hurdles earlier in the process. For drug developers, it provides a formalized, early-stage opportunity to get scientific alignment with regulators and experts, potentially saving years and millions of dollars on flawed trial designs. Congress has authorized $1 million annually from 2025 through 2029 to fund the organization and follow-up reporting for these meetings.

It’s important to note what this bill doesn't change. It doesn't force the FDA to adopt any advice given, nor does it alter existing rules protecting trade secrets. While the FDA is required to send staff to these meetings, the ultimate regulatory power remains with the agency (Sec. 2). The effectiveness of this process will heavily rely on the external Foundation and the Steering Committee, whose conflict-of-interest rules are set by the Foundation itself. However, by mandating transparency and requiring the FDA to publicly justify its use of the input, the Scientific EXPERT Act aims to integrate external scientific wisdom directly into the regulatory pipeline for rare disease treatments.