Amazing things happen when this community works together, when our messages are consistent, and our goals are in sync. We work together, we change the landscape, we make history.
Today the FDA has released their draft guidance on Duchenne. This is the first time that a patient community has proposed and drafted a guidance for FDA to use as a jumping off point for their own drug development guidance – an official map for how companies can move their Duchenne-focused innovation through the regulatory approval process.
In December 2013, we joined hands in a Policy Forum. FDA brought along 19 members of the Agency to listen, learn, and dialogue. That meeting set the stage for the development of a community-led draft guidance, submitted to FDA in June of 2014. Drafting guidance was no simple matter. Our community explored Duchenne, from diagnosis, to biomarkers, to trial design and extrapolation (the need for broad labeling). The Community Advisory Board (CAB) of patients and caregivers was loud and clear about what was important to them, to us – prioritize slowing or halting disease progression, recommend no or minimal exposure to placebo, encourage trials that include all individuals (young, adult, walking and non-ambulatory), use regulatory tools such as Accelerated Approval – a loud urgent voice for unmet needs.
And today, FDA released its draft guidance. In a meeting with us on May 28, they said the guidance was complete and going through the FDA clearance process. We were told that the documents would differ on the level of specificity, but that the themes and intent were consistent. FDA also told us that our draft guidance will continue to be a useful tool for industry sponsors and the agency. This means our draft guidance retains relevance and visibility—that gives us the opportunity to revise it whenever the need arises and submit those updates to FDA.
Why does all of this matter so much? FDA cannot discuss what happens in meetings with companies. Data submitted to FDA is confidential. Comments about how they weigh things, their willingness to listen to the voice of the families, their sense of urgency, are often tossed around, but we have few windows into the thinking of FDA decision-makers. With the availability of FDA guidance, any sponsor will have easy access to FDA’s perspectives on Duchenne. This should substantially lower the bar for companies to initiate faster and more efficient Duchenne drug development programs.
This community’s collective efforts, from meetings with FDA leaders, producing landmark benefit/risk data, telling our stories, convening the policy forum, and submitting guidance has all led to FDA drafting this guidance. This now gives all of us insights into how they are thinking about Duchenne, what is important to know and do.
Like you, PPMD is excited to dive in and find out what the FDA guidance says (and doesn’t say). And the process isn’t over. We, indeed anyone in the Duchenne community, can comment to the agency for the next 60 days. FDA will review those comments, make final tweaks, and then release a final guidance. And, in the meantime, we are told that both our draft guidance and theirs are available now to the review division to inform the review process. Our voice continues to matter.
Today we should celebrate. No more guessing games. We now have insight, directly from the source, about how FDA thinks about Duchenne. The guidance is here for us to read, think about, learn…then comment.
And together we will continue to change the landscape. Together, we will end Duchenne.