The Jett Foundation Fighting Duchenne Muscular Dystrophy

Jett Foundation to Present Patient Centered Outcomes Report To FDA Advisory Committee on Duchenne Treatment

April 25, 2016


(April 25, 2016) – The Jett Foundation, a Massachusetts-based organization committed to finding treatments for Duchenne muscular dystrophy, will present a first-of-its-kind Patient Centered Outcomes report today April 25, 2016 at the Food and Drug Administration’s Peripheral and Central Nervous System Drug Advisory Committee in College Park, Maryland. This report details patient and caregiver outcomes of those enrolled in clinical trials of eteplirsen, a potential Duchenne therapy manufactured by Sarepta Therapeutics, and will be included in the sponsor’s presentation before the Advisory Committee convened to discuss and review eteplirsen’s New Drug Application (NDA.) While the sponsor has donated core presentation time to Jett Foundation for the presentation of this report, the report is the product of an independent study unsolicited by Sarepta.

Jett Foundation’s Patient Centered Outcomes Report resulted from discussions with high-ranking officials at the FDA’s Center for Drug Evaluation and Research (CDER) and their request to see quantified data that reflects patients’ experiences. It also aligns with Section 1137 of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), which requires the FDA to consider the perspectives of patients during the regulatory process of specific products.

“The Patient Centered Outcomes Report demonstrates the meaningfulness of eteplirsen’s clinical trial results,” said Christine McSherry, Executive Director of Jett Foundation, which conducted the research and developed the report. “It provides the patient perspective on eteplirsen’s impact by quantifying collective patients’ and caregivers’ experiences before beginning therapy and while on drug. It gives clear information on endpoints not included in Sarepta’s protocol that are important to patients, detailing how treatment with eteplirsen impacts those unreported endpoints. This data provides additional context to the relative stabilization seen on the 6-minute walk test (6MWT), the primary clinical outcome measure used in study 201/202.”

Several key findings in this report highlight the limitations of the 6MWT as a clinical trial endpoint for Duchenne. However, the 6MWT is currently the only FDA-recognized clinical endpoint used to measure the rate of decline for Duchenne patients, and the primary clinical endpoint in eteplirsen’s study 201/202.

The key findings of this report highlight that boys treated with eteplirsen experience:

  • A decrease in or absence of spontaneous falls
  • Universal maintenance of ambulation after recovery from lower limb fractures
  • A decrease, stabilization or smaller increase than would be expected in the use of mobility devices (wheelchairs, scooters, etc.)

An unexpected maintenance or increase in the ability to participate in activities of daily living (ADLs)

“The results of this report dovetail the strength of eteplirsen’s clinical data and reveal the true impact of the therapy on the patients, their families, and their everyday activities,” said McSherry. “We hope that the results of this report will inform the panel, the FDA, and the public of what is really important to patients and families affected by Duchenne and how eteplirsen affects those significant aspects of daily life.”

A complete copy of the report, including Methods and Results, can be found here (LINK).

Background on the report: This report provides FDA with quantitative data to evaluate the anecdotal accounts of patients on eteplirsen with input from their caregivers and parents. Interview questions were designed to measure aspects of quality of life valued by patients. The questions and analysis were designed three years after the start of study 201. The report was executed by Christine McSherry, Jenn McNary (Director of Patient Advocacy and Community Outreach at Jett Foundation), and outside consultants, including Mindy Leffler. This study was prepared at the request of the FDA and was not initially developed for the purpose of an advisory committee meeting. It is intended only to further inform scientific data collected and quantified by drug manufacturers. Jett Foundation hopes that it will help the Duchenne community, the FDA, and the PCNS advisory committee better understand the risks and benefits of eteplirsen from the unique perspective of the patients themselves. We at the Jett Foundation also hope our efforts may serve as a model for future patient-centric drug development and benefit-risk assessment efforts.

About the Jett Foundation: Since 2001, Jett Foundation, located in Kingston, MA, has worked to find treatments and a cure for Duchenne muscular dystrophy (DMD) while improving the lives of those affected by DMD.

Over the past decade, Jett Foundation has raised over $15 million dollars for Duchenne research and programs to enhance the lives of those living with Duchenne. The Jett Foundation also provides resources for treatments and adaptive activities, serves as an educational resource, and provides camp opportunities through Camp Promise.

The Jett Foundation is a registered charity with 501(c)(3) status from the IRS; all donations are tax deductible.

Media Contact: Al Jackson,


Show Buttons
Hide Buttons