HELP Committee Advances Bipartisan Bill to Help Patients by Facilitating Faster Development of Drugs for Rare Diseases

Bennet-Burr-Warren-Hatch bill would help pave the way for advancing new drugs for rare diseases

Washington, D.C. - The Senate Health, Education, Labor and Pensions (HELP) Committee today approved a bipartisan bill help advance the development of targeted drugs for patients with serious or life-threatening rare genetic diseases. The Advancing Targeted Therapies for Rare Diseases Act of 2015 was introduced by U.S. Senators Michael Bennet (D-CO), Richard Burr (R-NC), Elizabeth Warren (D-MA), and Orrin Hatch (R-UT) in September.

"Our conversations with Coloradans suffering the effects of Duchenne's muscular dystrophy, cystic fibrosis, and other life-threatening rare diseases, have made it clear we need to do more to remove red tape and improve access to cutting edge treatments," Bennet said. "Bioscience companies are developing targeted therapies that are saving and lengthening lives, and this bill will allow them to expand the scope of their testing to help save even more lives. Allowing these researchers to safely test new therapies on patients with the same rare disease but a different mutation can help unleash a host of lifesaving new breakthroughs."

"As a new therapy for a rare disease becomes available, there's a chance that Americans who suffer from a number of similar diseases could benefit from it," said Burr. "We need to cut the red tape that stands in between patients and the medicines that could save their lives. I'm pleased that this bill has cleared this hurdle and thank my colleagues on both sides of the aisle for their support."

"Targeted therapies are a promising form of treatment for people living with rare diseases. We should use all the tools we can to help bring these new therapies to market, while maintaining the FDA's strong safety and effectiveness standard," Warren said. "I've heard from families in Massachusetts who often have no other treatment options. Today's committee vote on this bipartisan bill is a step toward helping innovators advance the next generation of precision medicines, which will make a real difference for these families and for many families across the country."

"As a long-time champion for helping those with rare diseases, I am encouraged by the amazing progress we are making in precision medicine. If we want to realize the full potential of cutting-edge advances and address serious unmet needs of many patients, the Senate must pass the Advancing Targeted Therapies for Rare Diseases Act," said Hatch. "This legislation will incentivize drug development, help overcome the difficulties of conducting conventional trials for many rare diseases, conserve FDA's limited resources, bring needed therapies to patients faster, and make treatments available where there otherwise would be no other options."

Many rare diseases like Duchenne's muscular dystrophy, cystic fibrosis, and certain cancers have genetic origins. A variety of mutations within a gene can result in the same disease, further fracturing diseases into a number subtypes. Advances in medicine have made it possible to develop treatments targeted to a particular genetic subtype, but the use of these targeted therapies is limited to patients with an exact mutation. Targeted therapies are usually developed first for the most common genetic subtype.

Developing drugs for rare diseases is particularly difficult because of the small patient populations available for clinical trials. Therefore, there would need to be dozens of therapies to treat the full spectrum of certain genetic rare diseases.

The Advancing Targeted Therapies for Rare Diseases Act of 2015 affirms the Food and Drug Administration's (FDA's) authority to allow innovators to use their own data supporting the approval of a targeted therapy to help facilitate additional targeted therapies to treat patients with the same rare disease.

This bill does not change the FDA's current approval standards and has the support of the National Organization for Rare Disorders (NORD), Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association, and the Duchenne Alliance.