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Public Policy Positions

What We Advocate For:

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.

Ensuring Affordability of Prescription Drugs Under the Inflation Reduction Act

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 also 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.

View NORD’s positions on the Inflation Reduction Act and download our helpful infographic here >>

Advancing Rare Disease Research and Innovation

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 polices such as:

  • Protecting the Orphan Drug Act
  • Adequate funding for research and innovation at the NIH, FDA, and other agencies
  • Patient focused drug development
  • Improving clinical trials to reflect the unique needs of patients with rare disorders

Learn more about our work on advancing rare disease research and innovation by visiting our policy statements here

Access to Affordable Comprehensive Health Care

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:

  • Protecting the Affordable Care Act
  • Access to comprehensive care and treatment through state Medicaid programs
  • Access to comprehensive and affordable care and treatment through Medicare
  • Access to expert care across state lines
  • Better medical nutrition coverage at the state level and through advocacy for the Medical Nutrition Equity Act
  • Reducing patient out-of-pocket prescription drug costs
  • Battling against the expansion of “junk insurance”
  • Ending surprise medical billing
  • Protecting patients from inappropriate step therapy and utilization management

Learn more about our work on access to diagnostics by visiting our policy statements here

Preventing a Diagnostic Odyssey

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:

  • Genetic testing
  • Newborn screening

Learn more about our work on access to diagnostics by visiting our policy statements here

Project RDAC

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

Other issues of importance to the rare disease community

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:

Learn more about our work on maximizing patient access to health care and treatment by visiting our policy statements here