Risk Tolerance, Benefit Preferences, and Treatment Priorities

The Duchenne community has entered a new era where more clinical trials are coming online and the possibility of treatment is becoming a reality. Upon completion of a trial, the FDA weighs the evidence presented. But at the end of the day, it is often a value judgment about potential benefits and risks to the patient.

PPMD has launched a program – Benefit/Risk in Duchenne Therapies – to help inform the FDA and other government agencies, biopharmaceutical companies, and others about the treatment priorities and risk tolerance of the Duchenne community.

PPMD is constantly exploring where data gaps exist that could help to move the field forward. The data we are collecting in this program addresses one of those gaps. We have been working with experts in the field to ensure the data we collect is both meaningful and useful.

Phase 1

Here is what we are doing in the first phase of this project:

  1. A parent/guardian survey. The survey is online and anonymous, meaning that no one knows who took the survey or what they said. The goal of the survey is to learn about priorities for treatment benefits and risks, risk tolerance, and worries and concerns. We need certain parents and guardians to take this survey now.

    If you are parent or guardian of a child with Duchenne who:
    • was diagnosed in the past 2 years and still walks well, OR
    • walks independently inside but needs to use a wheelchair outside, OR
    • uses a wheelchair full time

    and you live in the United States, we need your input on the survey now. We need about 30 more parents/guardians whose children meet the criteria above to participate in the phase 1 survey. Click here to take the survey now.

  2. People with Duchenne and family members: Share your story of what you want the FDA to know about Duchenne, potential treatments, and your family.

    Share the story you would like the FDA to hear.
  3. Focus groups of parents and guardians of individuals with Duchenne – coming in late winter and spring

  4. Continue our discussions with the FDA about how to give them the information they need

Through 2013, we will continue to engage our community about risk tolerance, benefit preferences, and treatment priorities.


The next phases will include getting additional information about treatment preferences and risk tolerance from more of the community.  We will keep you informed as the project moves forward!


Benefit/Risk in Duchenne Therapies - Survey Ad

  • Update 2/13/13: We need 10 more parents and guardians of adults with Duchenne to take this survey now

  • Update later on 2/13/13: Thanks to a few savvy survey participants who caught a mistake- parents OR legal guardians are invited to take the survey; if you are a parent of an adult with Duchenne (even if you are no longer his/her legal guardian), you do qualify!  Please click "yes" to the legal guardian question. We are almost done with the first survey data collection.  If you want to participate, please do it today so you don't miss the chance. There will be more opportunities this year to share your treatment priorities!  And don't forget to "tell your story" (see below); we expect to keep taking stories for a few more weeks, at least.

Holly Peay
Vice President, Education & Outreach

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Comment by MEHMET ASIK on July 18, 2013 at 5:30pm

What can we do?

Comment by MEHMET ASIK on July 18, 2013 at 5:30pm

My Son is 3.5 years old. DMD Exon 44 deletion.

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