Ellayna and Becky Gallis (CMTC-OVM US president) attended the NORD Rare Diseases and Orphan Products Breakthrough Summit, held October 21 & 22 in Alexandria, Virginia. The FDA keynote address, “The Patient’s Role in the Approval Process” was delivered by Janet Woodcock M.D., Director for the Center of Drug Evaluation and Research (CDER), FDA. She focused on the drug evaluation as a continuum, where stakeholders need to agree on acceptable levels of benefit/risk/uncertainty. Another point Dr. Woodcock stressed was that to achieve the full potential of adaptive licensing for public health and drug development, licensing decisions should be aligned with payer, prescriber, and patient decisions.
The conference held 3 summits and 3 tracks from which to choose. On day one, the summit Becky chose was, “Transforming Diagnosis – Physician Engagement and Disease Diagnosis.” The emphasis of this session was directed toward the fact that most rare diseases affect a small patient population who are geographically dispersed. The panel agreed that rare diseases, while being NOT contagious = NO public awareness.
On the final day of the Breakthrough Summit, the chosen track was, “Advancing Patient Engagement and Support.” This session defined the value of the patient’s perspective. Here, the panel talked about the initiative to include the patient in the drug development and regulatory process. While considering the pros and cons it is important to observe how a drug impacts the daily life of a person with a rare condition, and what the implications could be if treatment is not available or accessible.
The conference was informative and placed hope in the possible advancement toward treatment of CMTC and other vascular malformations.