NORD staff attended today’s 21st Century Cures hearing, following yesterday’s release of the second discussion draft, and there were more than a few highlights for NORD and the rare disease community.
NORD’s Groundbreaking Registry Program
The highlight of the briefing occurred when Rep. Matsui asked about the registries provision and whether the FDA/NIH will be working with NORD. Dr. Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research, agreed entirely, touting her ongoing work with us. You can see the exchange in this video starting at 1:18:45.
Rare Pediatric Disease Pediatric Review Voucher (PRV) Program
Rep. Butterfield entered into the official record NORD’s sign-on letter of 115 patient organizations in support of the PRV program. He spent most of his five minutes touting the program, and questioning the Committee for not reauthorizing the program in the latest discussion draft.
Additional Points of Interest
Rep. Capps called for more rare disease research funding, Rep. Engel called for more attention to rare diseases and biomarkers, and Rep. Bilirakis advocated for the OPEN Act.
The Energy & Commerce Committee featured NORD’s position statement, issued yesterday, in today’s “What They’re Saying” Recap, seen here. We will continue to work closely with the Committee and members of the House and Senate on behalf of our members and all patients with rare diseases as legislation moves forward.
The terms risk, benefit, effectiveness, and safety are key to the approval of all new medical treatments (and of previously approved treatments for new uses). However, there is a vast divide between the concepts of relative risk and relative benefit (for a well defined group of patients) and the concepts of effectiveness and safety. The distinctions between these concepts are particularly important in the world of rare diseases. read more >
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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