Q: What prompted the creation of Parent Project Muscular Dystrophy (PPMD)?
PPMD was incorporated in 1994. It was created in an effort to draw attention to and focus efforts on Duchenne muscular dystrophy (DMD). PPMD was the first DMD-specific organization.
Q: What were the challenges in Duchenne research that you felt PPMD could help address?
PPMD initiated efforts to create Centers of Excellence and to develop interdisciplinary care in DMD. PPMD's advocacy efforts and resulting legislation galvanized the research community, incentivized industry, and has been instrumental in creating widespread awareness for Duchenne. Current challenges include stratification and accreditation of clinics to insure clinical trial readiness and advocacy to insure access and reimbursement for treatments for all.
Duchenne muscular dystrophy faces some of the same challenges that are typical of drug development in a rare disease, including the need for better developed natural history data and endpoints, recruitment challenges, and data silos. In addition, I think we have additional challenges in that Duchenne is not only rare, but it is also a progressive pediatric disease. We definitely feel a sense of urgency that is not compatible with typical timelines for drug development, and this is complicated by the fact that no new drug for Duchenne has ever been approved, so there is no "tried and true" regulatory path in existence. We have concerns about the flexibility of regulatory agencies and their understanding of benefit/risk in Duchenne as applied to their ability to be flexible in their decision-making.
Q: Can you tell us a bit about PPMD's research strategies and how they help overcome the barriers you've identified?
We have thought very carefully about our research strategy to make sure that we are identifying those pivotal points where additional dollars/resources will really make a difference. This thought has resulted in a three-pronged research strategy that we call "Better! Faster! Now!" In practice this means that we focus on putting better quality candidates into clinical testing, increasing the efficiency of that testing, and, in parallel, prioritizing approved drugs for repurposing for Duchenne. We are more and more cognizant that drug development is a complicated ecosystem that requires investment, not just in drug development, but in the tools that make drug development successful, whether that is investment in patient registries, endpoint and biomarker development, or development of the regulatory pathway. For example, we have recently conducted a very rigorous benefit/risk survey in the Duchenne community to provide the FDA with data about risk tolerance on which to base decisions – we did this to help better define the regulatory path for Duchenne and to give each good drug candidate its best chance for success. Another project we worked on a few years ago was the development and adoption of standards of care for Duchenne; we recognized that these are needed, not only to improve care, but to decrease variability in drug trials. So it is an ecosystem, and even things that seem to be primarily care-related can also be about drug development.
Q: What do you consider PPMD's greatest accomplishments to date?
PPMD's greatest accomplishments include a significant $45 million investment in research, seeding and supporting research that is now in preclinical, Phase I, II, and confirmatory studies. PPMD's advocacy efforts resulted in the first piece of legislation focused on muscular dystrophy, the MD CARE Act, which leveraged $500 million into muscular dystrophy research, a regulatory strategy including meeting with CDER/Neurology, a risk tolerance survey, and a white paper focused on regulatory policy and DMD.
Q: What are your top research goals over the next year, and what will it take to reach those goals?
We are collaborating with another group to develop a new paradigm for a clinical research network that is truly industry-friendly. In other words, we want something that meets the needs of industry and not just academic investigators, and we've done extensive interviews with industry to better understand their needs. We will then be able to fund specific drug trials directly, and at the same time provide them with better infrastructure across which to run these trials, hopefully increasing efficiency and reducing cost for all of us. We have also launched the Duchenne Drug Development Roundtable, which is designed to bring together industry, advocacy groups, academic investigators and government representatives around projects in the precompetitive space, such as developing data standards and aggregating placebo arm data.