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 Wednesday, April 29, a hearing will be held in the House of Representatives to consider funding for the Departments of Labor, Health and Human Services, and Education. This is an opportunity to weigh in on the importance of federal funding for medical research and public health preparedness.
At NORD, our goal is to help develop safe, innovative treatment options to fight rare diseases. We need Congress to provide adequate funding to support medical research that can lead to lifesaving cures.
Join Research America in a day-long social media campaign letting Congress know that Americans care about medical progress and want a stronger public health system.
Related news: Read NORD’s Assistant Director of Public Policy Paul Melmeyer’s blog post, “Medical Research Should Be Everyone’s Priority“
We are excited to be featured on America’s Charities “Making Impact” blog today! Visit the America’s Charities website or read below to find out what director of development Derek Gavin does at NORD and how being a parent advocate shapes his work.
As posted on America’s Charities
In this Q&A, Director of Development, Derek Gavin, shares his role in executing NORD’s mission. While Derek’s official role is the Director of Development, his unofficial role is parent advocate. This February marked the 14th anniversary of his son Trevor’s passing. Being born with Metachromatic Leukodystrophy, his life expectancy was 3-5 years, but Trevor lived miraculously to 2 months shy of his 10th birthday.
Q&A With Derek Gavin:
What attracted you to this job & particular cause?
Since my son’s passing I have wanted to get involved in advocacy for people with rare diseases and their families. It took many years to overcome the devastating pain and emptiness of losing my son. I couldn’t talk about him without sobbing like a child. We were introduced to NORD as a resource upon getting his diagnosis. The people were nice and helpful and pointed us to another patient organization that represented the Leukodystrophies. As time went on, the need to advocate and give of myself overcame my emotional tenderness such that I could actually work professionally in this community which would help me make the most impact.
How are you making an impact through your work?
I was the owner of a small company when an opportunity opened at NORD. As the director of development, my overarching responsibility is to bring revenue into the organization from both the fundraising, individual giving side, and the industry side providing programs to industry that benefits industry and our patients.
It has been a blessing for me personally to work in this role as NORD is a truly independent advocacy charity and has been doing amazing work on all fronts for 32 years.
Many of the programs I promote to industry help the patients directly and in that way inspires hope and community. One of the overarching feelings one gets when diagnosed with a rare disease is one of isolation and the feeling of being alone. NORD’s motto “Alone we are rare, together we are strong” exemplifies the mindset of the organization and some of the goals of its programs. read more >
Recently, NORD’s partners at Frontline Medical Communications (FMC) shared the results of a survey conducted among physicians who received a Rare Neurological Disease Special Report co-published by FMC and NORD.
Nine out of 10 respondents indicated that the content was somewhat or very useful, with 91% saying it would help them improve patient care, screen patients more proactively and treat them more effectively.
These findings are consistent with responses to a survey conducted by FMC among physicians and other healthcare professionals last year in which 88% of 1,632 respondents agreed that there is a need for more professional content on rare diseases. In that survey, 75% of the HCPs acknowledged that they have patients with rare diseases and 78% said they had searched the medical literature for information about rare diseases during the previous 12 months. read more >
Talk about inspiring–today, members of the Running for Rare Diseases Marathon Team will pound out 26.2 miles in chilly temps while running the Boston Marathon on behalf of rare disease patients!
Each runner is partnered with an individual rare disease patient – see their amazing stories here – and this effort raises money, awareness and support for all 7,000 rare diseases. We are beyond thrilled and proud to partner with such an amazing and inspiring group of people.
Running for Rare Diseases has grown from a grassroots team of four runners to this year’s record-breaking team of 100+ runners. Over the years, the team has raised nearly $500,000 for NORD’s educational programs and, as of 2013, our Undiagnosed Diseases Patient Assistance Fund. The fund pays for medical testing for undiagnosed patients who cannot afford the basic medical work-up needed to make them eligible to apply for the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP). The team says, “We are thrilled to be able to provide this patient assistance program to support patients who have exhausted all other avenues to seek a diagnosis!” 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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