Inside Health Policy
By Maaisha Osman
A bipartisan group of lawmakers is pushing forward new legislation to require FDA to meet quarterly with external experts and advocates to discuss approval processes for rare disease therapies, aiming to improve the agency’s review of products for small patient populations. Titled the Scientific External Process for Educated Review of Therapeutics (EXPERT) Act, it was reintroduced Monday by Reps. Doris Matsui (D-CA) and Gus Bilirakis (R-FL) along with Sens. Amy Klobuchar (D-MN) and Roger Wicker (R-MS).
The announcement comes after House Republicans left renewal of FDA’s expired pediatric priority review voucher (PRV) program out of the six-month continuing resolution they unveiled over the weekend. Democrats are attempting to advance the PRV program as part of a “hotline” of the bipartisan, bicameral health care package that stalled in December. However, Sen. Rand Paul (R-KY) is blocking the effort, making the year-end package the most likely opportunity for passage, the Alliance for Regenerative Medicine told Inside Health Policy. Still, the group is working to push it through sooner.
With over 90% of rare diseases lacking FDA-approved treatments, advocates argue that the existing approval process does not adequately account for the unique challenges posed by these conditions. Small patient populations and limited clinical trial data often make it difficult to assess treatment efficacy using traditional standards, they say. Furthermore, experts on rare diseases frequently have conflicts of interest that prevent them from advising FDA, leaving regulators without crucial disease-specific insights.
The EXPERT Act aims to solve this problem by formalizing quarterly “Externally-Led Scientific-Focused Drug Development” (EL-SFDD) meetings at FDA. These sessions will provide a structured forum for scientists, drug sponsors, patient advocates, and regulatory officials to discuss best practices for evaluating rare disease therapies. Each meeting will focus on a different rare disease challenge, and FDA will be required to report annually on how these discussions influence its review process.
“We need more formalized processes for engagement to balance the urgent needs of rare disease patients, who overwhelmingly lack treatment options, with the mission of the FDA,” Matsui said in the press release Monday (March 10). “This bill will ensure that rare disease experts and FDA reviewers can exchange knowledge and have productive conversations about how to move therapies forward.
Bilirakis echoed those sentiments, emphasizing the power of collaboration. “We’ve seen that tangible progress happens when all stakeholders work together,” he said. “Our bipartisan legislation will foster that collaboration to drive innovation for the rare disease community.”
More than 30 million Americans live with a rare disease, and advocates say the bill could help unlock desperately needed treatments. “Our bipartisan bill would help find cures and save lives by supporting more collaborative rare disease research and encouraging communication between the FDA, patients, and scientists,” Klobuchar said.
The bill is already drawing support from major rare disease organizations, including the EveryLife Foundation for Rare Diseases, the Rare Disease Company Coalition, and Global Genes. Advocates stress that while progress has been made in drug development, bureaucratic hurdles continue to delay life-saving therapies.
“Patients are not getting treatments fast enough,” Stacey Frisk, executive director of the Rare Disease Company Coalition, said in a statement Monday. “The EXPERT Act ensures the FDA can incorporate disease-specific expertise to accelerate progress.”
During his confirmation hearing last week, President Donald Trump’s FDA nominee, Marty Makary, committed to making the approval of new cures a key priority.
Separately, the rare disease advocacy group Haystack Project told Inside Health Policy in October that they are working with Senate and House lawmakers including the House Energy & Commerce Committee to develop legislation to allow alternatives to randomized controlled trials (RCTs) for rare disease products.
Haystack does not back existing legislation that would create a limited-term conditional approval pathway; instead, it’s arguing that FDA’s overarching “substantial evidence” standard should remain intact, but that action from Congress is needed to ensure alternative evidence can be used instead of RCTs.