Since its establishment in 1983, the National Organization for Rare Disorders has advocated for policy changes to improve the lives of Americans impacted by rare diseases at the federal and state levels. NORD continues this critical work to this day. Read more about our policy positions on this page.
Learn more about NORD’s policy priorities for 2024
As many as half of all people living with a rare disease are children, and rare pediatric disease priority review vouchers (PRVs) offer a crucial incentive for companies to develop therapies for these particularly challenging patient populations. NORD is urging Congress to reauthorize this vital program in its current form before the September 30, 2024 deadline in order to maintain an important tool in ongoing efforts to address the significant unmet treatment needs of the pediatric rare disease population.
Learn more about NORD’s position on Rare Pediatric Disease Priority Review Vouchers (PRVs) here
For the first time, due to the Inflation Reduction Act, the Centers for Medicare & Medicaid Services (CMS) will be able to negotiate the prices of certain prescription drugs. This will have significant impacts for some rare disease patients on their ability to afford needed treatments but could also impact broader rare disease drug development by disincentivizing orphan drug research. NORD is taking a leading role to ensure this law ultimately benefits patients and families living with rare disease. We sent a letter to CMS signed by more than 100 member organizations, detailing the concerns of the rare community. We hosted a webinar with senior leadership from the Centers for Medicare & Medicaid Services where we asked some of our most pressing questions about the implementation of the Inflation Reduction Act. And NORD President and CEO Peter L. Saltonstall wrote an op-ed in the journal STAT about the need for Congress to create exemptions for orphan drugs.
View NORD’s positions on the Inflation Reduction Act and download our helpful infographic here
The Promising Pathway Act 2.0 (PPA 2.0) would create a new conditional approval pathway at FDA, allowing companies to sell certain rare disease drugs for children and adults before appropriate clinical trials are completed to show a product is safe and effective. NORD is concerned the bill jeopardizes patient safety and will negatively impact current and future patients’ ability to access safe and effective treatments. We issued a statement of opposition on an earlier version of the bill, submitted a statement for the record to the Senate Aging Committee, and have hosted listening sessions with our patient advocacy member organizations to explain our position. While NORD opposes PPA 2.0, we are committed to collaborating with any individual or organization in pursuit of effective public policy that will increase patient access to safe and effective rare disease treatment options.
Learn more about NORD’s position in our one-pager.
More than 90% of rare diseases lack an FDA approved treatment. NORD was founded by advocates who helped to pass the Orphan Drug Act, which provides key incentives to spur the development of treatments for rare diseases. It is critical that the research and regulatory environment helps to address continued barriers for rare disease drug development and foster a successful bench to bedside pathway for rare disease treatments. NORD advocates on policies such as:
Recently, in The Milbank Quarterly, NORD President Peter L. Saltonstall, Vice President of Policy and Regulatory Affairs Heidi Ross, MPH, and former Henry Waxman staffer Paul Kim documented the renaissance in rare disease drug development sparked by the Orphan Drug Act and responded to subsequent critiques of prescription drug pricing that followed the launch of “blockbuster” orphan drugs. Read the full article here.
Patients with rare disorders often find their financial lives upended by the debilitating nature of their diseases, and on their behalf, NORD is committed to ensuring access to affordable, comprehensive health care coverage. NORD advocates on policies such as:
Learn more about our work on access to diagnostics by visiting our policy statements here
Many rare disease patients face years of a difficult “diagnostic odyssey” before receiving their correct diagnosis. Obtaining an early and accurate diagnosis is critical for allowing patients to seek effective treatment sooner. For instance, newborn screening programs have been improving and saving the lives of children across the United States through the early identification of diseases that cause permanent disability or death if not treated early. For other patients with rare disorders, genetic testing holds the hope of a timely diagnosis and effective, targeted treatment, but access and affordability is currently a limiting factor. NORD advocates on policies such as:
Learn more about our work on access to diagnostics by visiting our policy statements here
A Rare Disease Advisory Council (RDAC) is an advisory body providing a platform for the rare community to have a stronger voice in state government. RDACs address the needs of rare patients and families by giving stakeholders an opportunity to make recommendations to state leaders on critical issues including the need for increased awareness, diagnostic tools and access to affordable treatments and cures. NORD’s Project RDAC is designed to optimize the existing RDACs and to increase the number of RDACs across the country. Under this initiative, NORD provides opportunities for the RDACs to collaborate with each other on a regular basis, creates educational resources to guide RDACs at every step of their journey, and helps states pass legislation to create high functioning RDACs.
Learn more about Project RDAC and learn how to get involved here
People with rare diseases face many challenges, including traveling significant distances, often across state lines to find a health care provider knowledgeable about their specific rare disease and the need to take time off to attend numerous medical appointments or care for a loved one with a rare disease. Additionally, the COVID-19 pandemic has had significant impacts on our community. NORD advocates on policies such as:
