We, the Duchenne community, have entered into a new era where more trials are coming online and the possibility of treatments becoming a reality. PPMD remains focused on working with the FDA to inform them about Duchenne so they can better understand the complexities of the disease and its patient population.

We have been working hard to create a survey about perceptions of benefits and risk tolerance of parents/guardians of individuals with Duchenne, together with a forum for you to share your stories and what you want the FDA to know.  Together, this is a compelling set of data to bring to the FDA. It is the sort of data that they have asked for, and the sort of data that the FDA should be receptive to. They need to better understand how parents and guardians weigh potential risks of treatments against the potential benefits that any child with Duchenne or Becker might achieve.

We started with a fairly simple survey, and then we asked for and received a lot of input. This input has made the survey much better, but also more complicated. We are in the final stages of tweaking the survey. It has taken us longer than expected, but we are really excited by the potential of this expanded survey to make a real difference to the FDA.

Here’s who has reviewed and provided formal input into the survey:

• Industry representatives at PPMD’s Duchenne Drug Development Roundtable,
• Parent leaders of our FACES groups,
• A social scientist with experience doing these types of surveys,
• And most importantly: an expert in risk/benefit experiments who has consulted with the FDA on their risk/benefit assessment, and who has been working for years on better ways to get patient/family input to regulators and industry.

These people have expressed a great deal of interest in the survey and a commitment to help us get data that is useful to the FDA, industry, and our community.

You’ll hear more from me about this survey next week. Until then, best wishes for a happy Thanksgiving!

Holly Peay, Vice President, Education & Outreach
Read more PPMD Blogs