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.
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.
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.
Pat Furlong, Founding President, CEO
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