FDA Patient-Focused Drug Development Initiative: Affected Community Testimonials at 2022 SciFam


Progress toward treatments for CMD is a battle fought on several fronts. Clinicians work to improve diagnostics, care, and understand the disease course, or natural history. Researchers work to explain a condition’s underlying pathology and which therapies might mitigate symptoms, slowing or even halting disease progression. Pharmaceutical companies take this collective work, run toxicology studies, figure out how to efficiently manufacture a drug, and conduct clinical trials. Regulatory agencies like the FDA and EMA review data collected from all of these areas to make determinations about whether or not a proposed treatment is safe and effective for a given disorder. And advocacy organizations like Cure CMD work with all of these experts to coordinate efforts, fund research, maintain a patient registry, and educate, mobilize and support the affected community.


One of the formal mechanisms used by the Food & Drug Administration (FDA) to gain a better understanding of an affected community’s experience is the Patient-Focused Drug Development (PFDD) meeting. The goal of this meeting is for agency representatives to get a better understanding of the patient perspective, directly from affected individuals and caregivers. At a PFDD meeting, stakeholders share the impact of a condition, symptoms that most impact function, independence, and quality of life, and what treatments (including care optimization) are currently available to improve the condition.


In December 2021, the FDA approved Cure CMD’s application to host an externally-led PFDD meeting for the five primary subtypes of congenital muscular dystrophy (Collagen 6, Dystroglycanopathy, LAMA2, LMNA, and SELENON). This four-hour meeting on Friday, July 1 will take place at 2022 SciFam.


In the coming weeks, CMD community members will be given the opportunity to provide their perspectives through the completion of a survey, comment submission, pre-recorded video testimonials, and the chance to contribute live at the PFDD meeting. So, stay tuned for more information!


Check out the agenda, and join us for this critical piece of the CMD drug development process. If you’re unable to attend 2022 SciFam, either virtually or in person, you can still attend the PFDD meeting virtually, free of charge. More information about how to join the meeting will be shared soon.


For a deeper understanding of the drug development process, check out my four-part series.