News

Washington, D.C., June 4, 2018 — The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement on the increasing use of copay accumulator programs in health plans: “Recently, a number of health plans have started implementing, what are broadly known as, ‘copay […]

Patients speak out as Congress moves forward with a tax bill that would negatively impact individuals with rare diseases Washington, D.C., November 28, 2017 –  Today, the National Organization for Rare Disorders (NORD) is hosting a Save the Orphan Drug Tax Credit Rally for the rare disease community in response to the tax reform bill being debated in […]

Washington, D.C., November 2, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to the release of the Tax Cuts and Jobs Act, and the proposed repeal of the Orphan Drug Tax Credit (ODTC): “Today, the House Ways and […]

Orphan Drugs Account for Only 7.9% of Pharmaceutical Spending in the U.S. Washington, D.C., October 17, 2017—Orphan drugs accounted for only 7.9% of total drug sales in the U.S. in 2016, according to a study issued today by the QuintilesIMS Institute. The study, commissioned by the National Organization for Rare Disorders (NORD), analyzed the role of the […]

Hurricane Harvey has left thousands rid of their belongings and forced out of their homes. For individuals living with a rare disease, the impact of such natural disasters can be even more detrimental. Together, NORD staff worked with our Texas Rare Action Network State Ambassador, Debbie Skolaski to create the Hurricane Harvey Emergency Relief Resources packet, a list of emergency […]

To help drive the efforts of the Rare Action Network℠, NORD launched the State Ambassador Program. State Ambassadors are volunteers in a leadership position responsible for establishing and building a strong grassroots network of rare disease advocates within their state to help increase awareness of rare diseases and the challenges patients and their families face. […]

Senate in Deliberation TODAY!  Yesterday, the Senate voted 51 to 50 to move forward with debate on repealing and replacing the Affordable Care Act (ACA). This is concerning because every proposal put forward publicly so far does not adhere to our Principles for Health Coverage Reform. Following this vote to move forward, the Senate considered a […]

As members of our South Carolina and North Carolina Rare Action Network, we hope you can join us for one of our upcoming Rare Action Network (RAN) Luncheons. These are free meetings that will give you the opportunity to connect, learn, and share with other rare disease advocates. More details are listed below. Together, we can make a [...]

The new administration and Congress are re-examining the United States health insurance system with the goal of repealing the Affordable Care Act, and replacing it with a new system. Before the Affordable Care Act was enacted in 2010, rare disease patients faced discriminatory insurance practices due to their pre-existing condition. Patients were denied coverage to […]

Washington, D.C., July 13, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement regarding the release of a revised draft of the Senate Leadership’s Better Care Reconciliation Act of 2017: Senate Leadership today released a revised draft of the Better Care Reconciliation […]

Washington, D.C., July 12, 2017 – The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) commend the House of Representatives for passing the FDA Reauthorization Act (FDARA) with overwhelming bipartisan approval. Passing this bill is a crucial step in reauthorizing four user fee programs that ensure the Food and Drug Administration (FDA) […]

NORD is pleased to share the Rare Action Network’s Summer 2017 State Policy Legislative Tracker.  This document shows, state-by-state, which legislation we are tracking to help improve the lives of people and families with rare diseases.  Together, we are taking action in 42 states and the District of Columbia.  Let’s keep going – together, we are strong!   […]

Washington, D.C., June 29, 2017 — The following statement was issued today by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), following the FDA’s announcement of its Orphan Drug Modernization Plan: “NORD is grateful to FDA and its recently-appointed Commissioner, Dr. Scott Gottlieb, for its commitment, announced June 29, to eliminating the orphan designation […]

Washington, D.C., June 27, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to the delay in consideration of the Senate Better Care Reconciliation Act: “Today, Senate Leadership announced they will not hold a vote this week on the Better […]

Washington, D.C., June 22, 2017 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement regarding the release of the Senate Leadership’s Better Care Reconciliation Act of 2017 discussion draft: “Senate Leadership today released a discussion draft of the Better Care Reconciliation Act of […]

Washington, D.C., June 20, 2017 – The National Organization for Rare Disorders (NORD), an independent nonprofit organization representing the 30 million Americans with rare diseases, has launched a nationwide campaign to urge the U.S. Senate to protect lifesaving health care for the millions of children and adults with rare diseases by opposing any legislation that would […]

Washington, D.C., May 8, 2017—On Friday, the National Health Council (NHC) and the National Organization for Rare Disorders (NORD), submitted a letter from more than 120 patient advocacy organizations to members of Congress urging them to swiftly reauthorize the FDA user fees. The letter, in part, reads, “The chronic and rare disease and disability patient […]

Washington, D.C., May 4, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to the passage of the American Health Care Act: “Today the House of Representatives passed American Health Care Act (AHCA). We are very disappointed the […]

CALL TO ACTION: Congress could vote this week – please take a minute to phone your legislators and ask them to vote no (click)  Washington, D.C., May 1, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to […]

Washington, D.C., September 30, 2016 – Today, President Obama signed the Advancing Hope Act (S. 1878), extending the Rare Pediatric Disease Priority Review Voucher (PRV) program until December 31. “On behalf of the 15 million children with rare diseases, we thank the President for signing the Advancing Hope Act so that some of our most vulnerable […]

Washington, D.C., September 29, 2016 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, released the following statement from President and CEO Peter L. Saltonstall following President Obama’s signature of the Continuing Resolution today. “On behalf of the 30 million Americans with rare diseases, […]

Unanimous vote extends the Rare Pediatric Disease Priority Review Voucher Program with important policy changes through December Washington, D.C., September 23, 2016 – On Thursday, the Senate reached an important compromise to accelerate new treatments to help 15 million sick children with rare diseases. Led by Senators Robert Casey (D-PA), Johnny Isakson (R-GA), and Lamar Alexander […]

More than 300 organizations join forces to ask Congress to provide robust, sustained, and predictable budget increases for the NIH Washington, D.C., September 22, 2016— The National Organization for Rare Disorders (NORD) is one of more than 300 organizations participating in the Rally for Medical Research Hill Day on September 22, uniting millions of Americans […]

The Hill, a top U.S. political website, read by Congressional offices, the White House, political pundits, and more, today published the following op-ed by NORD President and CEO, Peter L. Saltonstall, urging Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program. The PRV program encourages the development of therapies for children with […]

The National Organization for Rare Disorders submitted comments to the Institute for Clinical and Economic Review (ICER) in response to their call for comments on how to improve their value framework development process. You can find NORD’s comments here. People with rare diseases have a uniquely important role to play in value framework development. Almost every input […]

By Peter L. Saltonstall One of the most successful pieces of legislation ever passed by the Congress is the Orphan Drug Act of 1983 (ODA).  It was intended to stimulate the research and development of new therapies for rare diseases, which were generally neglected by the research community and the drug industry. Since 1983, more […]

NORD has published a new guide to help people and caregivers living with rare diseases understand expanded access and how to access unapproved medicines. In the guide, NORD answers frequently asked questions including how to determine if you are eligible for expanded access, how to apply, and what you can expect throughout the application period and […]

On July 15, the Food and Drug Administration released the performance goals letter for the reauthorization of the Prescription Drug User Fee Act, also known as PDUFA VI.  The PDUFA program is the agreement between the FDA and the pharmaceutical industry to pay user fees to facilitate the review of pharmaceutical products. PDUFA must be reauthorized […]

UPDATE:  July 11, 2016 Illinois HB 4576 is close to passing!  This bill would establish a Rare Disease Commission that would give patients – many of whom are too young or sick to speak for themselves – a voice in the state government.  The bill is awaiting Governor Bruce Rauner’s signature before it is passed […]

The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), as the Senate announced it would not vote on its Cures legislation, also known as the Senate Innovations for Healthier Americans Initiative, until September at the very earliest. “On behalf of the 1 in 10 […]

Time is running out – today may be our last chance before September for anything to happen on the critical Zika package.  This Friday, Senators will depart D.C. for their 7-week summer recess.  This is problematic because July and August in the U.S. are the worst months for mosquito and potential Zika activity. Today, July 13, […]

Save the date for Sept. 21-22 for a nationwide day of action!  Join NORD and 300+ partner organizations as we ‘rally’ for medical research as part of the annual Rally for Medical Research Capitol Hill Day. Federal funding for medical research is critical to the health and well-being of all Americans, especially for the 1 in 10 living […]

NORD has recently become a member of a coalition of patient groups in Oregon seeking to reduce out-of-pocket drug costs for patients. The Oregon legislature recently created a Prescription Drug Cost Working Group which will be meeting for the first time this month. As part of its participation in this meeting, NORD is supporting the […]

NORD, the leading, independent advocacy organization committed to helping the 1 in 10 Americans with rare diseases, applauds the Senate for authorizing medical foods coverage in the National Defense Authorization Act for Fiscal Year 2017 (S.2943) on Tuesday.  The bill now must be harmonized with the House version passed last month. “This is an important […]

Today, June 7, join rare disease advocates around the country and tell your Senators to support the Senate Cures Legislation. This month is the last chance for Senate Cures to pass before the Senate is out of session until after the November election. Senate Cures (also called the Senate Innovation Act for Healthier Americans) is the companion […]

RareAction continues to make strides around the country for the 1 in 10 Americans with rare diseases. In this version of the State Policy Legislative Tracker, there is an increase in the bills being supported in both Massachusetts and Tennessee. Learn more about the bills that RareAction is tracking and supporting to improve the lives of […]

Greetings from Oklahoma, The awesome folks in the NORD marketing department created the snazzy logo and imagery that you see above (and in all previous posts). Driving through Oklahoma today, it felt like I was on that very road. Absolute nothingness for miles in every direction. So far the trip has covered about 3,000 miles, with […]

Greetings from Tallahassee, As Einstein famously postulated, time is relative. I will make use of this fact to take you back in time to my visit to Tallahassee, Florida (schedules prevented our advocate and me from posting a few days ago). I like Tallahassee, it is one of few state capitals with the vibe of […]

Greetings from Denver, As the road tour heads further west, the distances between stops grow longer and longer. More time in the car means less time to blog. But, 10 hours and 632 miles later I made it to Denver. It was somewhat of a tedious drive with nothing much to gaze at, however, the payoff was worth it; […]

Greetings from Atlanta, Georgia. I am happy to introduce NORD’s State Ambassador for Georgia, Beth Nguyen. Beth is a registered nurse and Executive Director of Worldwide Syringomyelia & Chiari Task Force (a NORD member organization).  As a NORD State Ambassador, Beth is hoping to advance provider education for Syringomyelia and all rare diseases. Beth will be working to […]

Today, the National Organization for Rare Disorders (NORD), Biotechnology Industry Organization (BIO), and Ernst & Young released “Impact of the Orphan Drug Tax Credit on Treatments for Rare Diseases,” a new white paper examining the role of the Orphan Drug Tax Credit (ODTC) on rare disease drug discovery.   Patients share their experiences in profiles that […]

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. NORD congratulates the House Energy and Commerce Committee for unanimously approving the 21st Century […]

NORD’s Assistant Director of Public Policy, Paul Melmeyer, authored this great piece (below) in the March 2015 issue of the NCC Collaborator, the monthly publication from the National Coordinating Center for the Genetic Service Collaboratives (NCC).  NCC focuses on bringing quality genetic and newborn screening services to local communities, and building bridges between public health, […]