Cure CMD's Externally-Led Patient Focused Drug Development (PFDD) Meeting
Friday, July 1 - 1:00pm U.S. Central Time (CDT)
If you are already registered to attend 2022 SciFam, you don't need to register for this PFDD meeting. Virtual attendees will receive information about how to connect to the meeting via email. In-person attendees may join us at the Renaissance Nashville Conference Center, 2nd Floor, Broadway 2 ballroom.
If you are not registered to attend 2022 SciFam, we'd be delighted to welcome you at this meeting! Please register to attend this PFDD meeting. Registration is free, and you will receive an immediate, unique link to join the meeting upon registration.
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, conduct 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 learn about the patient experience, 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. If you’re unable to attend 2022 SciFam, either virtually or in person, you can still attend the PFDD meeting virtually, free of charge. Register to join the meeting if you aren't already registered to attend 2022 SciFam.
All times listed below are U.S. Central Time (CDT)/Nashville