We are thrilled to announce that the Advancing Targeted Drugs for Rare Diseases Act bill dropped in the Senate today (see press release below)! PPMD has been working with the Senate HELP Committee and Sarepta for the last several months on this 21st Century Cures provision which morphed into stand-alone legislation (view PPMD's most updated endorsement of this legislation).
According to PPMD President Pat Furlong, "It is our expectation that this bill will enable sponsors developing therapies for Duchenne - and other devastating rare diseases and disorders with profound unmet medical needs – to leverage similar or closely related underlying technologies and/or data to accelerate the development pathway for additional targeted therapies.”
The bill has the support of Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association, the Duchenne Alliance, and the National Organization for Rare Disorders (NORD).
Read today’s announcement to learn more about the bill and stay tuned for ways you can help this important legislation move forward:
Bennet, Burr, Warren, Hatch Introduce Bill to Help Patients by Facilitating Faster Development of Drugs for Rare Diseases
Bipartisan bill would help pave the way for advancing new drugs for rare diseases
Washington D.C. – Today U.S. Senators Michael Bennet (D-CO), Richard Burr (R-NC), Elizabeth Warren (D-MA), and Orrin Hatch (R-UT) introduced the Advancing Targeted Therapies for Rare Diseases Act of 2015. This bipartisan bill will help advance the development of targeted drugs for patients with serious or life-threatening rare genetic diseases.
“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.”
“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.”
“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.”
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 diseases are further fractured into various 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 Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association, the Duchenne Alliance, and the National Organization for Rare Disorders (NORD).