NORD: The Voice of the Community - November 2017

News from NORD and its Member Organizations

Hundreds of Patient Organizations Join NORD in Supporting Orphan Drug Tax Credit

In letters sent this week, NORD and its advocacy partners are urging key Congressional policymakers not to abandon the Orphan Drug Tax Credit (ODTC) as they consider tax reform legislation. NORD’s key messages to Congress are that the tax credit is extremely important to the 30 million Americans who have rare diseases, and that one-third fewer orphan drugs would be brought to market if the tax credit were eliminated.

In addition to its outreach to Congress, NORD has provided public education about the importance of the tax credit through media stories, including one on the website of the Today Show.

More than 200 nonprofit patient organizations have signed onto NORD’s letter to the leadership of the House of Representatives and its Ways and Means Committee. In the letter, NORD expressed “strong concern” about the repeal of the ODTC in the House Tax Cuts and Jobs Act.

“The Orphan Drug Tax Credit is one of the only tax credits that saves lives,” NORD’s letter stated. “With 95 percent of individuals with a rare disease still waiting for a treatment, we implore you to maintain this critical incentive for orphan drug development.”

In a similar letter to the leadership of the Senate, NORD and 36 policy partners said they “stand united in opposition to the Senate Finance Committee’s proposal to substantially weaken the Orphan Drug Tax Credit.”

The ODTC was one of the key incentives for orphan product development in the Orphan Drug Act of 1983, for which NORD played a key role in advocacy. A study conducted in 2015 concluded that there would be one-third fewer treatments developed for people with rare diseases if the tax credit did not exist.

Nominations Are Open for 2018 NORD Rare Impact Awards
January 12th is the deadline to submit nominations for 2018 NORD Rare Impact Awards. These awards honor individuals, organizations or companies that have had a positive impact in the fight against rare diseases.

The awards are presented each year in May at NORD’s annual gala celebration and fundraiser in Washington DC. In 2018, the program will have special significance because it will mark the 35th anniversary of NORD and the Orphan Drug Act.

Awards will be given in four categories to match the four pillars of NORD’s mission: Honorees have included patients, caregivers, healthcare professionals, researchers, legislators, and government agency staff. Nominations may be submitted through the NORD website.

NORD’s New Documentary Highlights Importance of Community Support
A new documentary video released by NORD shows how a small Kansas community came together to turn night into day for an 11-year-old boy who has a rare condition that requires him to avoid sunlight.

Peyton Madden has xeroderma pigmentosum (XP), a rare inherited skin disorder characterized by a heightened sensitivity to ultraviolet radiation. As a result, he is not able to do many of the things, such as swim in the community pool, that other kids his age enjoy.

For the documentary, NORD partnered with Peyton’s hometown, El Dorado, Kansas, to plan an after-dark community celebration as a surprise for Peyton.

“More than 400 people came out to show their support and interest in getting to know one young boy struggling with a rare disease a little better,” said Lisa Phelps, director of marketing and community. “We are so grateful to have had the opportunity to film his experience and share it with the world. We hope it serves as a springboard for conversations about what individuals and communities can do to make those impacted by rare diseases feel less isolated and more supported.”

The trailer for the documentary can be seen here: 

NORD Issues Statement on Nomination of Alex Azar to Serve as HHS Secretary
Noting that the Department of Health and Human Services is critical to the health and social service needs of the nation, NORD President and CEO Peter L. Saltonstall issued a statement in support of the nomination of Alex Azar to head the department.

“In our view, Alex Azar brings superior credentials for this job, based on his extensive management experiences and deep understanding of our healthcare system,” Saltonstall said. “He understands the needs of the patient community and particularly the challenges of the rare disease community and the urgency of orphan drug development.”


Adrenal Insufficiency United (AIU) Conference
Registration is now open for AIU’s first-ever conference. The event will take place March 23-25 in Kansas City. Leading experts will speak on a wide variety of topics important to the AI community. Read more

HCU Network America
HCU Network America, for those affected by homocystinuria, will host its first patient/family conference, Taking the Lead for HCU, from April 21-22, 2018 in Westford, Massachusetts. The first day of the event will focus on best practices and what to expect with homocystinuria, while day two will focus on research and looking to the future. This event intends to bring patients, families, doctors, researchers and industry from around the country together. Register today

National MPS Society
Abstracts for the International Symposium on MPS and Related Diseases, which is in San Diego, California, from Aug. 2-4, 2018 are now being accepted until January 8. The symposium will include a scientific program and will also feature a one-day workshop, Clinical and Translational Research Opportunities in Lysosomal Disease, on Wednesday, Aug. 1. For more information, visit, or email regarding abstracts.
A Research Collaboration with NCATS, and Retrophin that will focus on the development of assays for small molecule high-throughput screening in an effort to better understand the biology of the disorder, and identify potential small molecules to be developed as a therapeutic for patients living with NGLY1 Deficiency was recently launched. Retrophin will seek to continue development of compounds through pre-clinical and clinical phases, with the ultimate goal of delivering a treatment option for the NGLY1 community. Read more.

Pulmonary Hypertension Association
As part of PHA’s Pulmonary Hypertension (PH) Awareness Month campaign, a Chronic Thromboembolic Pulmonary Hypertension (CTEPH) Awareness Day will be observed on November 21. PHA will take to social media to share informational facts and memes, video stories, and a live interview with Dr. Manreet Kanwar, a PH specialist at the PH Center at Allegheny General Hospital, Pittsburgh, PA, – a center that has earned PHA’s esteemed PHCC accreditation. Learn more

Stay tuned each month for more news from NORD.
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