Issue Position: FDA & Rare Diseases

Issue Position

I am on the frontlines leading the effort to reform the FDA and to increase drug and medical device approvals. New drug approvals are especially important for individuals suffering from a rare disease. There are 30 million Americans who have one of 7,000 rare diseases but only about 250 FDA approved treatments for these rare diseases.

I introduced the Unlocking Lifesaving Treatments for Rare-Disease Act or ULTRA (HR 3737) and the Fast Access to Specialized Treatments or FAST Act (HR 4132). These bills help reform, codify and apply the FDA's Accelerated Approval pathway to the rare disease community. FAST rewrites and modernizes the 20-year-old Accelerated Approval pathway and includes explicit instructions for the FDA to issue guidance applying this pathway to the rare disease community. FAST has the support of over 150 rare disease organizations. Due to the small population of many rare diseases, traditional clinical trials with a large diverse population are challenging if not impossible.

I am also the lead cosponsor of the Expanding and Promoting Expertise in Review of Rare Treatments Act (HR 4156). This bill would facilitate consultation by the FDA with external experts like academic researchers, health professional organizations, patient advocates and care providers about rare disease issues. This will allow FDA reviewers to obtain access to specialized and up-to-date information on rare disease treatment from experts while they do drug review and evaluation.

Both FAST and EXPERRT was included in the bipartisan FDA Reform Act (HR 5651).

Additionally, as Chairman of the Subcommittee on Oversight and Investigations, I held a hearing into the FDA's slow approval of medical devices. As a result of this hearing, Title VII of the FDA Reform Act is comprised of many reforms to FDA's device approval designed to streamline the process.

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