Thanks to this incredible community urging lawmakers, well over 100 Members of Congress signed and submitted a letter to Dr. Woodcock at the FDA asking that the agency use flexibility and all available tools at their disposal when reviewing Duchenne-specific therapies.
This letter was drafted by a coalition of Duchenne organizations (including PPMD, The Race to Yes, Duchenne Alliance, and Center for Duchenne Muscular Dystrophy) working tirelessly to ensure that the FDA understands the vital importance of approvals in our community.
Thank you to everyone who participated in our Action Alert - the response was overwhelming!
Read the press release:
Fitzpatrick, Keating & King Call on FDA to Increase Efforts to Treat Duchenne
WASHINGTON, D.C. – Congressmen Mike Fitzpatrick (R-PA), Bill Keating (D-MA) and Peter King (R-NY) led a letter sent Wednesday to the Food and Drug Administration urging the agency to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. They were joined by more than 100 other bipartisan members.
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5 and leads to progressively worsening disability and is usually fatal by age 25.
“As the FDA continues its review of a potential new therapy for Duchenne muscular dystrophy, we urge the agency to utilize all available tools, resources, and authorities to accelerate the process of delivering safe and effective treatments to patients diagnosed with this 100 percent fatal disease,” wrote Fitzpatrick, Keating and King. “In particular, we urge the agency to fully utilize its authorities and the tools Congress included in the Food and Drug Administration Safety and Innovation Act (FDASIA) to provide for new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially when no satisfactory alternative therapy exists, as is the case in Duchenne.”
"Our community is heartened to know that our congressional champions remain committed to ensuring the FDA uses the tools, authorities, and latitude necessary to review and clear safe, effective treatments," said Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy (PPMD).
"Eteplirsen is the perfect example of a safe, efficacious drug that deserves the flexibility and approach provided to the agency in FDASIA," said Christine McSherry, Founder & Executive Director of the Jett Foundation. "Duchenne is a deadly and rare disease and we hope that the FDA will quickly complete its review and grant accelerated approval to this potential life-saving therapy."
The full letter is attached and below:
Dear Dr. Woodcock:
Thank you for your ongoing commitment to use the tools and authorities of the Food and Drug Administration to expeditiously review candidate therapies for Duchenne muscular dystrophy. In recent years, Congress and the FDA have made tremendous progress toward achieving the goal of approving the first-ever disease-modifying treatments for Duchenne, an aim we hope will be achieved in early 2016.
As the FDA continues its review of a potential new therapy for Duchenne muscular dystrophy, we urge the agency to utilize all available tools, resources, and authorities to accelerate the process of delivering safe and effective treatments to patients diagnosed with this 100 percent fatal disease. In particular, we urge the agency to fully utilize its authorities and the tools Congress included in the Food and Drug Administration Safety and Innovation Act (FDASIA) to provide for new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially when no satisfactory alternative therapy exists, as is the case in Duchenne.
The risk of doing nothing for a child with Duchenne is guaranteeing his or her death at a tragically young age. But the benefit of approving Duchenne therapies, seemingly efficacious drugs with clean safety profiles, could alter the lives of all Duchenne patients in a very positive way—by giving them a chance to live a longer, better life.
That is why we write to underscore the focus FDASIA has on accelerating the approval of drugs that treat unmet medical needs, prioritizing the patient perspective in evaluating new drugs and treatments, and providing regulators with flexibility when evaluating drugs for a life-threatening illness. The accelerated approval pathway outlined in Section 901 of FDASIA recognizes the limitations of developing drugs for rare diseases and gives the agency the flexibility to grant approval to rare disease treatments that have been shown to be safe and effective in fewer and smaller trials, while still requiring a larger confirmatory trial post-approval to confirm efficacy. This allows demonstrably safe therapies that treat an unmet medical need and appear to be efficacious, even with some uncertainty, to avoid the years of regulatory barriers and become accessible earlier to patients who otherwise have no other option.
Consistent with FDA regulations, we believe it is “appropriate [for the FDA] to exercise the broadest flexibility in applying the statutory standards, while preserving appropriate guarantees for safety and effectiveness” to new therapies intended to treat persons with life-threatening and severely-debilitating illnesses. As the FDA further notes, “the benefits of the drug need to be evaluated in light of the severity of the disease being treated.” As Members of Congress representing constituents battling Duchenne, we wholeheartedly agree with this viewpoint and urge the FDA to ensure this flexibility is applied in reviewing all Duchenne candidate therapies.
FDA has been successful at applying flexibility in oncology and HIV/AIDS to speed patient access to apparently safe treatments, and the need and opportunity to adopt innovative and flexible approaches to the review of rare disease drugs has never been greater than it is today. Patients are waiting.
Furthermore, it is critical the FDA take into account the views and experiences of patients as part of the review process. It is our understanding that the community worked collaboratively with regulators and benefit-risk experts to ascertain quantifiable patient-preference data, has collected narratives from a broad spectrum of the community that have been published and shared with the FDA, and produced a comprehensive draft guidance to industry that informed FDA’s development of its own draft guidance. We urge the FDA to consider the perspectives offered through these many Duchenne-specific patient-focused drug development tools, as well as the testimony and experiences of those in contact with the agency and your representatives, including patient representatives on the advisory committee and patients and expert clinicians who treat them as they testify during the open public hearing portion of the upcoming advisory committee meeting.
We remain committed to ensuring FDA has the tools, the authority, and the latitude necessary to speed treatments for rare disease to patients as quickly as possible, as was the intent expressed by Congress in passing FDASIA in 2012. We hope the agency will embrace the tools it has in order to provide patients and physicians with treatments for Duchenne.
Thank you for your attention to this important matter.