PDUFA V Promotes Research and Development of Treatments for Rare Diseases

Rare Disease Day with text.jpg

Today is the fifth-annual international Rare Disease Day, an international day of advocacy to bring recognition to the challenges faced by those with rare diseases. In the United States, a rare disease is defined as a disease that affects fewer than 200,000 people, and there are currently over 6,000 rare diseases identified. About thirty million Americans suffer from a rare disease of some kind--that's almost one in ten people.

Rare disease patients face a wide variety of challenges, but one of the biggest common problems is the limited array of treatment options. The Rare Disease Program Initiative included in the PDUFA V reauthorization agreement holds great promise for advancing scientific knowledge about rare diseases and thus expanding the treatment possibilities for the millions suffering from a rare disease. It will also improve the communication and outreach to the patient community, and allot resources to train FDA staff and new drug sponsors on rare disease drug development.

We've already seen that the resources provided to the FDA by PDUFA have helped the agency review and approve innovative drugs--of the 35 new treatments the FDA approved last year, 10 were for rare diseases that previously had few or no treatment options. The timely reauthorization of PDUFA V would ensure that the FDA would be able to continue promoting an environment that encourages innovation and investment in breakthrough new treatments for rare diseases.

For patients who currently have very few treatment options for their condition, the swift reauthorization of PDUFA is more important than ever. We are urging Congress to pass a clean PDUFA by July 4th so that the FDA can deliver innovative new cures and treatments to the patients who need them. For more information about how you can help, follow us on Facebook or Twitter, or sign up for email updates.