How can we best advance the science of patient input to improve the efficiency of the drug development and regulatory review process?


PPMD and other leaders in the rare disease community were asked by PhRMA to participate in a blog conversation about how to advance the science of patient input in drug development. 
PPMD’s Vice President of Advocacy and Community Outreach, Ryan Fischer, provides the Duchenne example as a model of how the patient voice can impact the development of potential therapies.

How can we best advance the science of patient input to improve the efficiency of the drug development and regulatory review process?

Parent Project Muscular Dystrophy has long held the belief that new approaches are required to expedite the development of treatments for rare diseases like Duchenne. Patient advocates have worked tirelessly with Congress, over time through legislation, to increase the engagement of the patient voice within the regulatory review process for therapeutics.  Passed in 2012, The Food and Drug Administration Safety and Innovation Act (FDASIA) was a culmination of those advocacy efforts by patients.  The legislation included a number of provisions under the patient-focused drug development and patient preference headings that aim to more systematically gather patients’ perspectives on their condition and bring the patient voice to the table like never before.  Within the user fee package, FDA agreed to hold 20 disease specific hearings to begin creating a framework for incorporating the patient voice into the review process. Along with over 7,000 other rare diseases, Duchenne was not one of the “chosen” conditions to be taken up by the agency within the five-year authorization. With a Duchenne pipeline full of potential therapies in or heading to clinical testing, we knew we had little choice but to try and come up with novel approaches to inform the FDA about our patient population and the current state of research.  In 2013, PPMD embarked on the first-ever rigorous scientific survey of benefit/risk.  While patient testimonies and stories are critical to educating industry and the FDA about diseases, this study aimed to translate the patient voice into usable data for the agency to consider when reviewing potential drug applications. PPMD worked closely with industry and the patient community to create a meaningful survey that reflected the current state of research and drug development. The data from the study illustrates a relatively high level of risk tolerance if the potential benefit is slowing or stopping the progression of muscle weakness. The preliminary survey data was presented to FDA leadership and published in Clinical Therapeutics in June. With encouragement from the agency regarding this approach we are now looking to expand the study into harder-to-reach, less connected caregivers, and adults living with the disease.

In another effort to enhance the science of patient input to improve the efficiency of developing therapies, this year PPMD led the development of the first ever patient-initiated draft guidance on Duchenne for Industry, submitted to the FDA in June following an intensive 6-month process that involved over 80 stakeholders from the Duchenne community.  The FDA is currently reviewing the document and have indicated they will produce their own Duchenne guidance in 2015.  PPMD plans to publish the methodology for drafting the document to help inform other rare disease groups who hope to use the same model, and the FDA has again publicly acknowledged the efforts of the Duchenne community as an example of how best to work with industry and the agency to provide much needed data.  How these will translate into impact on approvals remains to be seen. The agency will hopefully have several Duchenne-specific new drug applications in front of them in the coming year.


PPMD is now engaging Congress on the 21st Century Cures Initiative and proposing the development of a patient engagement assessment tool in order to quantify how FDA reviewers use – or do not use – available patient-focused drug development tools and data. The patient community needs transparency about whether increased engagement is yielding an impact on outcomes. Otherwise, we are wasting time and resources that could be directed elsewhere in drug development. In a catastrophic illness like Duchenne, time is too precious of a commodity to lose.

Stay tuned for more updates from PPMD on our participation in the 21st Century Cures Initiative.

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