Benefit/Risk Survey Results to Be Submitted to FDA with a Goal of Accelerating Review of Duchenne Therapies

Results to Be Submitted to FDA with a Goal of Accelerating Review of Duchenne Therapies


PPMD has conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments - information that PPMD is encouraging regulators to use to accelerate review time and, ultimately, to speed access to effective therapies.


PPMD will present an overview of the results during a webinar for the Duchenne community that will be held at 1 p.m. on June 12, 2013.


PPMD President Pat Furlong:

"For patients and parents impacted by Duchenne, there is no more important goal than accelerating access to effective therapies. PPMD has long championed legislative and regulatory efforts to expedite this process, and this first-ever survey is another important step forward. It is our hope that leaders at the Food and Drug Administration and other regulatory bodies will take meaningful actions to ensure the perspectives of our community on benefit/risk are taken into account."

The study suggests that the presence of side effects and risks could be compensated for by a treatment that stops progression of muscle weakness. While parents were not willing to accept unlimited risk, they would be willing to accept risk of a serious side effect if the potential benefit were stopping or slowing the progression of the disease.


The survey is the latest in a string of actions by PPMD to address this important issue. Last year, PPMD championed a number of provisions to accelerate the development of therapies for rare diseases that were incorporated into the FDA Safety & Innovation Act (FDASIA). PPMD has had multiple meetings with agency leaders on this and related issues, and recently released a white paper titled "Putting Patients First: Recommendations to speed responsible access to new therapies for Duchenne muscular dystrophy and other rare, serious and life-threatening neurologic disorders" that called for four specific policy actions.


As part of FDASIA, the FDA issued a draft implementation plan for developing a structured approach to benefit/risk assessment over the five year performance period, selecting 20 diseases to receive a meeting called for in the law. Duchenne was not one of the chosen conditions. "We don't have time to wait for the FDA to gather this critical data that it needs. Time is not on our side," said Furlong.


When invited by PPMD to "share their stories," one parent voiced a common theme of frustration:

"We hear each tick of the clock very loudly in our heads, worried that science will take too long to develop a treatment that will slow/eliminate the progression. Our bigger worry is that science will develop it and the FDA will take too long to approve it."

Another parent expressed a similar point of view:

"Simply put, we don't have years to wait. While I understand the need for FDA approval, and the concerns over possible risks and side effects - we aren't overly concerned with what these drugs could potentially do to our boys in the future. Without the medications, these boys have no future."

The Benefit/Risk in Duchenne Therapies findings must now be leveraged in discussions with FDA and other regulatory bodies, company sponsors, and others to drive change.


Holly Peay, MS CGC, Vice President of Education & Outreach at PPMD, and co-investigator of the study along with an academic collaborator:

"PPMD launched Benefit /Risk in Duchenne Therapies program in late 2012 in response to an FDA request for input on benefit/risk assessment in rare disease. We hope the study will stand as a model for informing benefit/risk prioritization in other rare disease populations. Results from this study will be submitted to scientific journals and widely presented in professional and advocacy forums. And we have been overwhelmed with the enthusiasm of pharmaceutical companies, who are extremely interested in this data."

Webinar Information

Time: June 12, 2013 from 1pm to 2pm eastern

To Participate:

  1. Visit and use participant code 9449985. (Be sure to test your computer beforehand)
  2. Audio Dial-In Information:
  • U.S. & Canada: Dial 866.740.1260 and use the Access Code 9449985
  • Outside the U.S. and Canada: Lookup your number

Join the webinar to learn about: 

  1. Parent survey on treatment preferences and risk tolerance: hear a summary of what we’ve learned about priorities, worries, and risk tolerance from 119 participants
  2. “Tell your story” about Duchenne and potential treatments: learn about the stories we are sharing with the FDA, submitted by families like yours
  3. PPMD’s white paper on FDA’s benefit/risk determination: making the case for a different way to weigh benefits and risks for rare, progressive disorders
  4. Benefit risk considerations in rare disease- how these differ from other diseases
  5. How what we are learning about Duchenne can be used as a model for other rare diseases

All of these efforts are designed to make our case with the FDA—an informed case, supported by data, research, and your stories. Join Holly Peay (Vice President, Outreach and Education) and Tim Fransen (Principal, FaegreBD Consulting) as we describe PPMD’s efforts and how we continue to work with the FDA.


Click here for full details on this week's webinar. 



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