21st Century Cures Initiative: Priorities for the Rare Disease Community
Room: B318
- Dormant Therapies and MODDERN Cures Act, Rep. Leonard Lance (R-NJ)
- Orphan Product Extensions Now, Accelerating Cures & Treatments (OPEN ACT), Rep. Gus Bilirakis (R-FL)
- “Patient Focused Drug Discovery” Kim McCleary, Director of Strategic Initiatives, FasterCures
- Genetically Targeted Platform Technologies for Rare Diseases
- Priority Review Voucher, Nancy Goodman, Executive Director, Kids V. Cancer
- Rare Disease Patient Organization Perspective, NORD
- NIH Funding Provisions, Ellie Dehoney, Vice President of Policy & Advocacy, Research!America
Moderator: Max G. Bronstein, Senior Director, Public & Goverment Affairs, EveryLife Foundation for Rare Diseases
Briefing hosted by:
Ask your Members of Congress to Join the Caucus
It will only take 2 minutes: RDLA will automatically find your legislators for you. All you need to do is enter your address and add a personal note about why it’s important to you. If your legislator is already a member of the Caucus then you can send a thank you letter to your legislator instead.
Caucus Briefings:
September 14th, 2016 – Strengthening Medical Innovation in America for Rare Disease Patients
May 18th, 2016 – The NIH and FDA: Vital Agencies in the Fight Against Rare Diseases
Nov. 5th, 2015 – Precision Medicine: New Frontiers for Rare Diseases
May 21st, 2015 – 21st Century Cures Initiative: Priorities for the Rare Disease Community
Feb. 26th, 2015 – Urgent Healthcare Policy Needs of the Rare Disease Community
Sept. 17th, 2014 – Implementation of Rare Disease Provisions in FDASIA
May 7th, 2014 – Access to Care & Therapies in the New Healthcare System: A Rare Disease Perspective
Feb. 28th, 2014 – Science Behind Rare Disease Policy, Feat. Special Guest Jonny Lee Miller
