The Jett Foundation Fighting Duchenne Muscular Dystrophy

24 United States Senators Stand with Duchenne

April 17, 2016


Jett Foundation thanks the 24 United States Senators who have chosen to stand with the Duchenne Community, including patients, families, and clinicians and researchers as we approach the April 25th Advisory Committee Meeting to evaluate eteplirsen. We want to specifically thank Senators Wicker and Klobuchar for spearheading this effort. Our community is eternally grateful for your support.

April 15, 2016
Ryan Taylor (Wicker), 202-224-6253
Colin Milligan (Klobuchar), 202-224-3244

Wicker, Klobuchar Urge FDA to Advance Muscular Dystrophy Therapies Senators Encourage Agency to Utilize Available Resources, Patient Perspective

WASHINGTON – U.S. Senators Roger Wicker, R-Miss., and Amy Klobuchar, D-Minn., are leading a bipartisan effort to encourage the U.S. Food and Drug Administration (FDA) to use all available resources and authorities to accelerate the process of getting safe and effective treatments to patients diagnosed with Duchenne Muscular Dystrophy (DMD). In their letter to Dr. Janet Woodcock, Director of FDA’s Center for Drug Evaluation and Research, the 24 Senators highlight recent congressional action to fast-track the approval process under the “Food and Drug Administration Safety and Innovation Act” (FDASIA).

“For those living with Duchenne and their families, every minute matters,” Wicker said. “Over the past two decades, researchers and medical professionals have made significant strides in the fight against this fatal disease. With modern advancements in science, we have every reason to continue the march toward even better treatments and ultimately a cure. Congress has given FDA new tools to accelerate these life-changing goals. I am hopeful that the agency will leave no stone unturned.”

“For families living with Duchenne muscular dystrophy, any advancement of treatments can make a real difference in quality of life,” said Klobuchar. “While we’ve made progress in recent years, much more needs to be done to help patients with this rare but deadly disease. I’m hopeful the Food and Drug Administration heeds our bipartisan call to use all available tools at their disposal to help those with Duchenne by moving safe and effective treatments forward.”

The letter urges Woodcock to utilize the pathway outlined in FDASIA, which gives the agency improved flexibility to grant approval to rare disease treatments that have proven to be beneficial and allows FDA to impose post-approval studies to confirm the clinical benefit. The Patient Focused Drug Development initiative, also included in the law, asks that the agency consider the views and experiences of patients as part of the drug review process.

In addition to Senators Wicker and Klobuchar, cosigners of the letter include Kelly Ayotte, R-N.H., John Barrasso, R-Wyo., Michael Bennet, D-Colo., Richard Blumenthal, D-Conn., Barbara Boxer, D-Calif., Sherrod Brown, D-Ohio, Maria Cantwell, D-Wash., Shelley Moore Capito, R-W.Va., Bill Cassidy, R-La., Susan Collins, R-Maine, Chris Coons, D-Del., Tom Cotton, R-Ark., Angus King, I-Maine, James Lankford, R-Okla., Edward Markey, D-Mass., Robert Menendez, D-N.J., Lisa Murkowski, R-Alaska, Chris Murphy, D-Conn., Marco Rubio, R-Fla., Jeff Sessions, R-Ala., Charles Schumer, D-N.Y., and Elizabeth Warren, D-Mass.

The full text of the letter follows:

April 15, 2016
Dear Dr. Woodcock,
Thank you for your ongoing commitment to the expeditious review of candidate therapies for Duchenne Muscular Dystrophy (DMD). In recent years, advancements in science have resulted in progress toward advancing the first-ever disease-modifying treatments for DMD, a goal we hope will be achieved soon.
As the FDA continues its review of potential new therapies for DMD, we urge the agency to utilize all available resources and authorities to accelerate the process of getting safe and effective treatments to patients diagnosed with this 100 percent fatal disease. The Food and Drug Administration Safety and Innovation Act (FDASIA) had a strong focus on accelerating the approval of drugs that treat unmet medical needs, prioritizing the patient perspective in evaluating new drugs and treatments and providing reviewers with flexibility when evaluating drugs for a life-threatening illness. We request you fully employ the tools Congress included in FDASIA and the broad regulatory flexibility the agency is granted through federal regulation[1] to help advance new DMD therapies.
The accelerated approval pathway outlined in Section 901(b) of FDASIA gives the agency the flexibility to grant approval to rare disease treatments that “have an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit,” and allows the FDA to impose post-approval studies to confirm the clinical benefit. In FDA’s draft Guidance, Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry, the Agency expanded on this concept specifically in the context of DMD. We request that the agency consider surrogate endpoints and intermediate clinical endpoints to reduce the time and difficulty of performing clinical studies on treatments for rare diseases like DMD and help new therapies become accessible to patients who otherwise have no option as the agency has done with other deadly diseases such as HIV and cancer.
FDASIA includes multiple provisions focused on addressing the challenges of the rare disease patient community. The patient population of a rare disease is by definition small, meaning clinical trials will be conducted with fewer participants than trials for more prevalent conditions. We encourage the agency to utilize advances in regulatory science that can allow clinical trials in a small population able to provide the evidence necessary for accelerated approval of products that treat life-threatening, rare diseases.
FDASIA launched the Patient Focused Drug Development (PFDD) initiative and charged the agency to take into account the views and experiences of patients as part of the review process. As you know, the DMD community worked collaboratively with regulators and benefit-risk experts to ascertain patient-preference data, collect narratives from the community, and produce draft guidance that informed FDA’s development of the draft Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry. In addition, the experiences of patient representatives on the advisory committee and testimony of patients at the advisory committee meetings offer important perspectives and information. We urge the FDA to ensure that all of these perspectives are considered in regulatory review.
Patient perspectives are also important in conducting risk-benefit analyses. The FDA notes “that physicians and patients are generally willing to accept greater risks or side effects from products that treat life-threatening and severely debilitating illnesses, than they would accept from products that treat less serious illnesses” and “that the benefits of the drug need to be evaluated in light of the severity of the disease being treated.” Under Title 21 regulations, it is appropriate for the FDA to exercise broad flexibility when reviewing drugs for certain disease types while ensuring safety and efficacy. We urge the FDA to ensure this flexibility is considered in the review of candidate therapies that meet regulatory requirements, while maintaining the rigor necessary to uphold safety and efficacy standards for new drugs.
The risk of doing nothing for a patient with DMD is their certain death. Treatments that are safe and reasonably likely to produce clinical benefit for DMD patients could meaningfully alter their lives.
Members of Congress remain committed to ensuring the FDA has the tools, authorities, and latitude necessary to review and approve safe and effective treatments for rare diseases as quickly as possible. We hope and expect that the agency will fully utilize these tools and authorities when appropriate to provide patients and physicians with new options to treat rare and deadly diseases like DMD.
# # #

Show Buttons
Hide Buttons