The Scientific EXPERT Act of 2025 mandates the FDA to organize science-focused drug development meetings for rare diseases, facilitated by a Foundation, to improve drug development and regulatory processes.
Amy Klobuchar
Senator
MN
The Scientific EXPERT Act of 2025 aims to improve drug development for rare diseases by mandating the Secretary to develop a process for Foundation-led meetings. These meetings will focus on discussing challenges in developing drugs for rare diseases, involving experts from the FDA, clinicians, patient representatives, and industry. The goal is to facilitate collaboration and gather input to inform the FDA's risk-benefit assessments, with meeting outcomes and FDA responses reported publicly. The act allocates funding for these meetings and requires annual reports to Congress on their impact.
Scientific EXPERT Act Mandates $5M for Expert Meetings to Tackle Rare Disease Drug Development
Here's the deal on the Scientific EXPERT Act of 2025: it's designed to get smarter about developing drugs for rare diseases. The core idea is mandating a series of "science-focused drug development meetings," led by an independent Foundation, specifically to hash out the tricky challenges in creating treatments for conditions that don't affect large populations. The bill requires the Secretary (think Health and Human Services, overseeing the FDA) to set up a process for these meetings, ensuring at least four happen each year, each tackling different rare diseases. To get this rolling, it authorizes $1 million per year for five years, from 2025 through 2029.
Who's Running the Show?
A key piece here is the structure. The Foundation tapped to run these meetings will form a steering committee to guide the topics. This committee is a mix of folks: FDA representatives, medical clinicians, patient advocates, and crucially, industry representatives – all operating under conflict-of-interest rules, according to the bill. Their job is to pick meeting topics based on unmet needs, the size of the patient group, what's already in the pipeline, and where regulatory guidance is fuzzy. The Foundation is tasked with consulting widely – with the FDA, industry, patients, and academics – when planning the specifics of each meeting.
Transparency and FDA Accountability
This isn't just about talking; it's about tracking the results. The bill mandates that the Foundation publish transcripts, recordings, and summary analyses of each meeting on the FDA's website within 180 days. These summaries need to clearly state what everyone agreed on and what the next steps should be. Importantly, FDA drug review staff must participate in these meetings. Furthermore, when the FDA approves a drug relevant to a meeting's topic, the agency has to publicly state if such a meeting occurred and explain how the input gathered influenced their decision-making, particularly the risk-benefit assessment. An annual report to Congress will also track the meetings, FDA participation, and overall impact.
The Real-World Angle
So, what does this mean practically? The goal is clearly to accelerate drug development for rare conditions by bringing key players together to troubleshoot scientific roadblocks. More collaboration and public insight into these discussions could be a big win for patients and researchers. However, the inclusion of industry reps on the steering committee raises the usual questions about potential influence – ensuring the focus stays purely on scientific hurdles rather than specific company interests will be critical. The bill also explicitly allows the Foundation to seek additional funding beyond the $1 million authorized annually, which adds another layer to watch regarding potential conflicts. Ultimately, the success hinges on these meetings genuinely tackling tough science questions and the FDA meaningfully incorporating the insights.