April 25, 2012

HELP Committee Approves Whitehouse Legislation to Expedite Review of Rare Disease Treatments

RI Senator’s Proposal Included in Drug Safety Bill

Washington, DC – Today, a key Senate Committee approved legislation by U.S. Senator Sheldon Whitehouse (D-RI) that would promote access to rare disease treatments by providing opportunities for rare disease experts and stakeholders to share their expertise with FDA.  The Whitehouse bill was considered as part of a legislative package to reauthorize the Food and Drug Administration’s (FDA) ability to approve prescription drugs, medical devices, and other medical products.  The bill was cleared by the Senate Health, Education, Labor, and Pensions (HELP) Committee, of which Whitehouse is a member.

Whitehouse’s legislation, the Expanding and Promoting Expertise in Rare Treatments (EXPERT) Act of 2012, would give patients and experts a role in the FDA’s review of new treatments for rare diseases – a practice which has resulted in faster approval times for drugs when it has been used in the past.

“We’ve seen that when the FDA gets the technical and scientific assistance it needs from rare disease experts, incredible progress can be made,” said Whitehouse, who specifically cited the Cystic Fibrosis Foundation’s recent work with Vertex Pharmaceuticals.  “I hope that the EXPERT Act will lead to more good stories for other Rhode Island patients and families afflicted with rare diseases.”

In the example cited by Whitehouse, the FDA approved a new cystic fibrosis treatment in just three months.  It was one of the fastest review times in the FDA’s history, and was aided by the input of the Cystic Fibrosis Foundation and renowned experts on the disease. 

Whitehouse’s legislation specifically encourages the FDA to take advantage of the wisdom and insights of rare disease experts, in order to speed the development of therapies for rare disease patients.  The bill also gives rare disease patients, and their advocates, a role in consulting with the FDA on topics like the severity of a rare disease, unmet medical need, and the benefits and risks of therapies to treat the disease. 

The legislation is supported by 64 rare disease groups, including the Rhode Island Rare Disease Foundation.  “The EXPERT Act would allow the FDA a more viable partnership with those in the rare disease community who are most knowledgeable in rare diseases and their treatments,” said Patricia Weltin, Executive Director of the Foundation.  “With over 7,000 rare diseases, utilizing information provided by rare disease experts will create a review process that is more effective and timely, which would greatly benefit the rare disease community.”


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