Partnering with the FDA to accelerate approvals- The question of risk tolerance in the Duchenne community


Friends, we have been actively pursuing a regulatory strategy and have had two meetings with the neurology division at the FDA. We are currently working on the 'to do' list for the community and are trying to understand barriers/obstacles to approval from the FDA side, as well as, from the sponsor side of the issue. We are working a smart, targeted call to action. We have learned through years of advocacy that the most successful approach is to make sure what we are asking is consistent throughout the community.


During the FDA rare disease open meeting in May, Janet Woodcock said - FDA weighs the evidence presented, but at the end of the day, it is often a value judgment about benefit to the patient with a high unmet medical need. During a round table conversation with Dr. Katz, he was asked about the perception that he was 'risk adverse.’ He explained that he views risk on a scale and used a cancer example at one end and migraine headache at the other end. Basically, he said that when someone is dying within a period of six months, he would accept a 50/50 risk. At the other end of the scale, where there is a chronic condition and no imminent threat of death, he would be risk adverse. So that said, in this last meeting we had a similar conversation where our goal was to make sure the neurology division understood that Duchenne, while not exactly in the same category as a stage 4c cancer diagnosis, causes boys to peak in function at age 7–10 (depending on steroid response) and then lose function, so that each loss, each small negative change should be viewed as a 'little death,' from the boy's perspective as well as the increasing burden in terms of required care. A degenerative disease is not at all the same as a chronic disease and should be much closer to the cancer end of the scale than to the migraine end.


Taking Action

The first step is for this community to weigh in on this benefit/risk equation. As we speak, PPMD is setting up a survey that is in development and will be posted in the coming weeks. YOU will have the opportunity to weigh in on risk/benefit and include your own personal story about your situation.Presenting this data to FDA will provide compelling arguments and rationale for the FDA to grant accelerated approval to compounds that demonstrate safety and efficacy.


We have learned, success with our advocacy is the result of a single, compelling message. While every family wants and needs treatment for their son, our message is about successfully navigating the regulatory process in order to get drugs approved – these first candidates currently in trial and all others to come. We are consulting with experts from communities that have been effective in getting drugs approved, as well as, combination therapies.   


We are exploring opportunities with our regulatory advisors and consultants. We are currently organizing meetings with companies to ensure we adequately understand the barriers they face and will come to all of you with an overall strategy and plan for discussion and opinion. In November, we will collect risk/benefit and personal stories –either text or video.  In December, we will lay out a comprehensive strategy for review and discussion. This may also involve contacting your members of Congress.


We have to be measured, strategic, and effective. We can do this. This community has gotten legislation passed and reauthorized, $500 million federal dollars which galvanized research, incentivized industry, and brought us to this moment. Our conversation has changed and we are now focused on a regulatory strategy. We must work together. Our sons are worth it.


This Thursday, October 25, PPMD will participate in the FDA open meeting, this time to respond to a call for public comment. This is part of the PDUFA V reauthorization that PPMD worked so hard to pass in 2012.  


The FDA's notice invites public comments on the preliminary list of disease areas and requests that those who propose additional disease areas for consideration describe how they applied the criteria identified by the FDA. On first glance, we might suggest Duchenne should be at the top of this list, as it certainly fits the criteria outlined below. If you think about it, you might imagine FDA has more than 1,000 disease specific advocacy organizations making the same request. It seems to make more sense to potentially achieve the same goal by focusing on pediatric muscle disease which exactly fits the criteria and provides FDA the ability to accomplish the stated objectives of the patient-focused drug development initiative. We are working with other rare disease groups in order to have the same message to the FDA. They have agreed to review 20 diseases within the 5-year authorization period. That will not be sufficient time to review the many disease indications that exist, thus the reasoning for combining similar diseases that all have similar characteristics. 


Those key targets are made clear in the FDA's public notice – a thorough understanding of the severity of the treated condition and the unmet medical needs so as to set the context of regulatory decision making in regards to benefit and risk. 


  • Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living
  • Disease areas that reflect a range of severity – While the patients with each of these life-shortening disorders are very severely affected, the individual patients with each of them experience a range of severity in such factors as age of onset of symptoms, rate of progression, age at which full-time assistance is needed for activities of daily living, and age at death – those ‘little deaths’ that occur with loss of function
  • Disease areas for which aspects of the disease are not formally captured in clinical trials
  • Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly)
  • Disease areas that represent a broad range in terms of size of the affected population
  • Disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives


We feel that this is the best strategy to move the needle for the disease in order to properly educate the FDA on the risk benefit analysis. At the same time we will be analyzing our own study of the community. Please continue to stay engaged. PPMD will be keeping you informed – in the coming weeks we will be contacting you with the survey we mentioned, as well as, requesting your personal stories to better educate those who make decisions on therapies for Duchenne within the FDA. We will then lay out the plan moving forward.


We are excited for the progress made to date with our community, we must continue this momentum, and with your help, we will do what it takes to end Duchenne.  


Learn more

Pat Furlong, Founding President, CEO
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Comment by Peter on November 13, 2012 at 6:19pm

Dear Pat,

Another supportive voice from Poland.  PPMD gives us hope we can successfully fight with the disease. th.ak you Pat

From Europe we should send requests to EMEA.

Comment by Grzegorz Majchrzak on November 13, 2012 at 4:55am

Dear Pat,
There are priceless efforts and work that you undertake. Thank you on behalf of our children!
I am a father of nice boy suffering from DMD, however we live in Poland. Any way we are tracking every day what is the progress of medicine and sience in this area, and we are aware how important is the process of approval of the new trialed compounds.
My question is if could I support this initiative being not an USA citizen? Would our voice from Poland be substantial in this case, when it is addressed to FDA?
If so, I will sign for it without further questions.

Warm Regards for all wonderfull kids.

Comment by Keith Van Houten on October 28, 2012 at 10:36am

What are the other pediatric muscle diseases you'd want to combine with?  

Comment by Trinh Nguyen on October 25, 2012 at 3:38am


Comment by Melissa Meller on October 24, 2012 at 10:35am

Awesome!!! I can't thank you all enough.. My family, friends and I are willing to do ANYTHING we can to help. <3

Comment by Liisa Underwood on October 24, 2012 at 10:27am

Fantastic work!!!  It is hard as parents to accept the life limiting element of Duchenne. When I've researched Medicaid resources, I found that there is a "hospice" Medicaid waiver, which is now called "Life Limiting" Medicaid Waiver.  Duchenne is Life Limiting and the Medicaid system has approved Connor under this waiver, which had NO wait list for our region in Colorado. So my message is meant to support Pat's message but to inform parents on long Medicaid Waiver wait lists to explore other possible waiver options even if the label is tough to accept.

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