Sunday, April 29, 2012

Developing Rare Disease Therapies

The Senate Health, Education, Labor, and Pensions Committee approved Senator Whitehouse’s bill “Expanding and Promoting Expertise in Rare Treatments (EXPERT) Act of 2012”. The legislation would give patients and experts a role in the FDA’s review of new treatments for rare diseases.

As cited by Senator Whitehouse’s legislation, FDA would be able to take advantage of the wisdom and insights of rare disease experts that would speed up the development of therapies for patients with rare diseases. The bill also gives rare disease patients and their advocates a role in consulting with the FDA on the severity of a rare disease, discuss unmet medical needs, and discuss the benefits and risks of therapies to treat the disease.

“We’ve seen that when FDA gets the technical and scientific assistance it needs from rare disease experts, incredible progress can be made,” said Senator Whitehouse. He specifically cited the Cystic Fibrosis Foundation’s recent work with Vertex Pharmaceuticals.

At the end of January 2012, Vertex’s New Drug Application was approved by FDA for Kalydeco™. The drug is used to treat people with Cystic Fibrosis who have a specific genetic mutation.

According to Senator Whitehouse, the FDA was able to approve Kalydeco ™ in just three months. It was one of the fastest review times in FDA’s history and was aided by the input from the Cystic Fibrosis Foundation and experts on the disease.

The legislation is supported by 64 rare disease groups including the Rhode Island Rare Disease Foundation. The EXPERT Act will enable the FDA to have a more viable partnership with experts in the rare disease community who are most knowledgeable on treatments,” said Patricia Weltin, Executive Director of the Rhode Island Rare Disease Foundation.