The non-profit organization Cure SMA has published the video and transcription of an event recently organized, titled “Clinical Regulatory Webinar.” On Monday, November 9th, Cure SMA hosted a one-hour webinar to discuss clinical trial design, expanded access, New Drug Application (NDA) processes, among other topics. Jill Jarecki, the research director at Cure SMA, was the host of the webinar along with a panel of three drug development experts in the field of spinal muscular atrophy (SMA).
The webinar was open to the entire community free of charge. Now, the organization has made a new an extended version of the Q&A portion of the webinar available, since there wasn’t time to answer all of the questions. The information covers topics like the functioning of the different phases in a clinical trial and its anonymity, as well as recent efforts to find a cure for SMA, FDA requirements to approve a drug for the disease, and the importance of clinical and regulatory issues.
Jarecki was joined by specialists on drug development processes Tim Franson, MD, Thomas Murray, PhD, and Tim Miller, MD. Franson is the Chief Medical Officer at YourEncore, a position he gained after serving as principal in FaegreBD Consulting’s health and biosciences practice. Murray is the President Emeritus at The Hastings Center, and he was formerly the Director of the Center for Biomedical Ethics and Susan E. Watson Professor of Bioethics in the School of Medicine at Case Western Reserve University. In addition, Miller is the VP & Head, Medical Affairs, North America Rare Diseases at Genzyme, and has already worked in numerous biotechnology and pharmaceutical programs, including cross functional and leadership roles in medical affairs, regulatory, and clinical research with direct synergies in all business unit operations.
The organization also emphasized that information on research, clinical trials, recruitment and scientific results is available through Cure SMA’s regular post updates and their news section. Additionally, the organization offers other resources to increase understanding about the disease, including the downloadable copy of Learning About Clinical Trials, the Cure SMA’s FDA Engagement Initiative, the “Voices of SMA,” an article on the patient voice, an announcement about a partnership with District Policy Group, as well as the clinical trials section of the website.