Ask the FDA about their priorities around rare diseases: At the largest and most impactful rare disease conference of the year, NORD’s Rare Diseases & Orphan Products Breakthrough Summit (Oct. 21-22 in Washington, DC), the heads of FDA, CDER, CBER and CDRH will share their insights on current issues including Patient-Focused Drug Development, gene therapy, pediatric drugs and devices, natural history studies and more. Attendees can take the unique opportunity to ask questions directly of FDA senior staff during a special town hall conversation. The Summit offers numerous networking opportunities with 95+ speakers, 6 breakout sessions, leaders from multiple stakeholder groups, expansive poster sessions, roundtable discussions and much more.
As technology in rare disease accelerates, and with topics like drug pricing being a top priority in government, we must act now to drive innovation and collaboration to develop stronger resources, better outcomes and find cures for the community. In 2019, we believe The Time is Now to prioritize action in making positive changes for the millions of Americans living with rare diseases.
Some key speakers include:
- Norman E. “Ned” Sharpless, MD, Acting Commissioner, FDA
- Janet Maynard, MD, MHS – Director, Office of Orphan Products Development, FDA
- Pooja Merchant, Head, Patient Partnerships and Engagement Oncology Global Medical Affairs, Bayer Pharmaceuticals
NORD, a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.