Cure SMA and our partners in the SMA Industry Collaboration are pleased to announce the release of the Spinal Muscular Atrophy Voice of the Patient (VoP) Report. This report is a thorough written account of the Patient-Focused Drug Development (PFDD) Meeting with the FDA, in the spring of 2017.
Patient Focused Drug Development Meeting with FDA
On April 18, 2017, over 400 individuals in the SMA community, including families, clinicians, academic researchers, industry and regulators, gathered for the PFDD meeting, in Washington, D.C. The goal was to provide the FDA with an overview of the impact of SMA on individuals and families, and of the expectations and priorities for current and future treatments.
Twenty different panelists representing both patients and caregivers of all types, ages and stages of SMA, provided vivid accounts of what life is like with a progressive, degenerative disease, and spoke on managing SMA’s most debilitating, oftentimes life-threatening symptoms with courage, grace and hope for better treatments and enhanced quality of life. Each round of panelists’ testimonies was followed by a period of facilitated discussion, including the larger audience, and polling questions with all participants. Participation included in-person and webcast audiences.
In addition to the typical meeting synopsis, a detailed description of discussions and polling results, and a summary of results from a benefit-risk study in SMA is also included in the VoP Report. This study was conducted to better assess what specific risks our community would be willing to accept in exchange for certain treatment benefits, to help inform regulators in the drug-approval process.
The recent FDA approval of a treatment for SMA was a landmark one for the community and is having a major impact on the disease, particularly when patients are treated early. Even so, there are still many unmet needs when addressing the complexities of this disease.
The SMA VoP report will be shared with senior leaders at FDA to inform future drug approvals in SMA. Cure SMA will directly share this report with key members of the FDA Division of Neurology Products, Rare Disease Program, Office Strategic Programs within CDER and the Office of Tissues and Advanced Therapies (CBER). It is our hope, that this report will also be included in the FDA website of submitted externally-led Voice of the Patient Reports under PDUFA VI and that regulators will consult with the report as part of their decision-making process when future SMA drugs are submitted for approval. The report will also be useful for outreach to researchers, clinicians, payers and other related organizations, to provide a better understanding of the needs, hopes and goals of our community.
“What we hear [from the SMA community] will help us to think about clinical trial design, what outcome measures to use in clinical trials, what really matters to patients, and how we as regulators should think about the balance of risks and benefits for patients with SMA,” said Wilson Bryan, MD, director of the Office of Tissues and Advanced Therapies in the FDA’s Center for Biologics Evaluation and Research, in closing remarks at the PFDD meeting. “Most of our medical education comes from patients. This gives us as regulators the opportunity and the privilege to continue our education by listening to you.”
A video playlist of each panel, including opening and closing sessions, is available on Cure SMA’s YouTube channel.
Thank you to all the FDA members who attended the PFDD meeting, and particularly to Drs. Dunn, Bryan and Goldsmith for providing the opening and closing remarks. Thanks also to Dr. John Day for providing an opening overview of SMA, and to James Valentine, JD, MHS, for moderating the discussion.
Thank you to our panelists for their incredible generosity with their time and their stories: Gina Cannady, Debbie Cuevas, Scott Ellis, Kristen Farrell, Christine Getman, Grace Grutter, Kelly Jankowski, Patti Kemp, Rio Landa, Kristen Lasko, Jungin Angie Lee, Kathryn McBride, Christina Murray, Brad Nunemaker, Kevin Schaefer, Danyelle Sun, Hugo Trevino, Lyza Wiesman, Brynne Willis and Jessica White. Thanks also to the in-person and webcast audiences for their valuable and thoughtful contributions.
Thank you to the members of the SMA Industry Collaboration for their support of this meeting and of the Voice of the Patient Report: Astellas, AveXis, Biogen, Novartis, Roche/Genentech, Cytokinetics, Ionis, MDA and the SMA Foundation.