U.S. Senators Elizabeth Warren (D-MA), Michael Bennet (D-CO), Richard Burr (R-NC), and Orrin Hatch (R-UT) have introduced the Advancing Targeted Therapies for Rare Diseases Act of 2015. This bipartisan bill will help advance the development of drugs for patients with serious or life-threatening rare genetic diseases by providing innovators the clarity they need to develop targeted therapies.
"Targeted therapies are a promising form of treatment for people living with rare diseases who often have no other treatment options. 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 standards," Warren said. "By clarifying the FDA's current authority to consider research supporting previously-approved targeted treatments, this bipartisan bill will help innovators advance the next generation of precision medicines."
"Companies developing these targeted therapies are saving and lengthening lives, and if we allow them to expand the scope of their current testing we can potentially save even more lives," Bennet said. "We have met with Coloradans suffering the effects of Duchenne Muscular Dystrophy, Cystic Fibrosis, cancer and other life threatening diseases who have asked us to remove red tape and help them access cutting edge treatments. By allowing these innovators to safely test new therapies on patients with the same disease, but a different mutation, we can unleash a host of lifesaving new breakthroughs."
"This is an exciting era of medicine that holds great potential for personalizing treatments to improve and save lives," said Burr. "This bipartisan legislation will help fulfill this potential for patients in North Carolina and across our nation by facilitating the development of targeted drugs for rare diseases. I'm proud of North Carolina's innovators and the work they are doing on behalf of patients, and it is my hope that this legislation will facilitate many life-saving treatments that give hope to families who are battling rare diseases."
"Innovation in treatments is critical for patients with rare diseases. The Advancing Targeted Therapies for Rare Diseases Act will enable more patients suffering from rare diseases to benefit from advances in precision medicine," said Hatch. "By removing difficulties involved in conducting conventional trials for genetic subgroups of many rare diseases, this legislation will incentivize new drug development, create greater efficiency in the drug review process, and bring needed treatments to patients faster. I am proud to work with Senators Bennet, Burr, and Warren on this meaningful, bipartisan legislation."
The legislation is also supported by medical and patient groups in Massachusetts including MassBio and the Jett Foundation.
"On behalf of MassBio, its 650 member companies and the patients we serve, we are thrilled to see the Targeted Therapies legislation filed today," said MassBio President & CEO Robert K. Coughlin. This bill will have a real and significant impact, allowing companies in Massachusetts and around the country to develop new therapies for serious rare diseases faster and more efficiently, while still maintaining the highest standards for safety and efficacy. We thank Senator Warren for working with Senators Bennet, Burr and Hatch and for all of their efforts and leadership in addressing this issue for the benefit of rare disease patients around the world."
"We are extremely grateful to Senator Warren, her colleagues and her staff, for their ongoing and endless support of boys and families battling Duchenne Muscular Dystrophy," said The Jett Foundation's Executive Director Christine McSherry. "The Senator understands that time is the enemy. This bill will ensure FDA has the clarity needed to help advance safe and effective treatments to patients fighting against rare disease."
Many rare diseases like Duchenne Muscular Dystrophy, Cystic Fibrosis, and certain cancers have genetic origins. Different mutations within a gene can result in the same disease, meaning some rare diseases are further fractured into genetic 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.
The Advancing Targeted Therapies for Rare Diseases Act of 2015 reaffirms the Food and Drug Administration's authority to allow innovators to use their own supporting research to expand their successful targeted therapies to treat patients with different genetic mutations of the same disease.
This bill does not change the FDA's current approval standards and has the support of the Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association, the Duchenne Alliance, and the National Organization for Rare Disorders (NORD).