Washington DC (May 21, 2015)—-NORD President and CEO Peter L. Saltonstall today issued the following statement on the approval by the House Energy and Commerce Committee of the 21st Century Cures legislative initiative and the introduction of the OPEN Act in the United States Senate. read more >
Today, NORD is joining the Everylife Foundation, Genetic Alliance, Global Genes, and the rare disease community in voicing our support for the OPEN Act (H.R.971), and requesting its inclusion within the 21st Century Cures Act.
On today’s day of action, we are asking you to call your Congresspeople, and ask them to support the OPEN Act, and to ask them to tell the Energy and Commerce Committee that they should include this important legislation in the 21st Century Cures Act.
Here is a sample script for your message:
Hello, my name is XXXX. I’m calling to urge you to support the OPEN ACT, HR 971, and to ask you to urge your colleagues in the Energy and Commerce Committee include it in the 21st Century Cures Act.
The OPEN Act could drastically increase the number of rare disease therapies, and could solve many of the off-label reimbursement problems that many rare disease patients face.
[please explain why the legislation is important to you as a patient]
Thank you for your time and consideration.
To call your Congressperson, call the Capitol Switchboard at: (202) 224-3121.
Together we are strong!
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 following statement was issued today by Peter L. Saltonstall, President and CEO of NORD, following the release of the second discussion draft by the House Energy and Commerce Committee for its 21st Century Cures initiative:
The second discussion draft of the 21st Century Cures initiative released today includes a number of encouraging ideas on how to advance the development of new medical therapies. The Committee and its staff deserve praise for efforts to bring these ideas together and for their months of work to make this a collaborative and transparent process.
We are encouraged by many of the ideas included in the second discussion draft. Since the initiative was launched one year ago, NORD has advocated for several provisions included therein, including incorporating the patient perspective through the patient-focused drug development initiative, developing registries of natural history data of rare disease patients, increasing transparency in the expanded access process, and increased funding for NIH, among others.
Nothing is more important to the rare disease community than providing an environment conducive to the development of new therapies and access to them. We will conduct a thorough evaluation and will continue to work closely with the Committee and members of the House and Senate as legislation moves forward.
Peter L. Saltonstall
President and CEO, National Organization for Rare Disorders (NORD)
On behalf of the millions of Americans who have diseases with little or no treatment, the National Organization for Rare Disorders (NORD) has submitted a seven-step plan to the 21st Century Cures Initiative outlining ways to improve the discovery, development, and delivery of medical treatments in the U.S. 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.
Translate This page:
SUBSCRIBE TO OUR E-NEWS