For decades, NORD has pursued federal and state policies to improve the lives of Americans impacted by rare diseases. This page is updated daily to provide timely information on our policy efforts. Please contact [email protected] if you have questions or need additional information.
NORD Issues Position Statement: State Implementation of Medicaid Community Engagement Requirements (H.R.1)
Published: May 18, 2026
Tagged in: Access to Affordable, Adequate Coverage
Medicaid Engagement Requirements
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Comments on FDA’s Proposed Plausible Mechanism Framework Guidance
Published: Apr 27, 2026
Tagged in: Access to Affordable Medicines
Access to Innovative Medicines and Therapies
Access to Pediatric Therapies
Advancing Rare Disease Research and Regulatory Science
Gene Therapies
Genetic Testing
Protecting FDA Standards for Safe and Effective Drugs
Rare Disease Clinical Trials
NORD Rare Disease Centers of Excellence Letter to HHS on Termination of ACHDNC
Published: Apr 6, 2025
Tagged in: Access to Diagnostics
Newborn Screening
NORD CEO Statement on the Termination of the Advisory Committee on Heritable Disorders in Newborns and Children
Published: Apr 4, 2025
Tagged in: Access to Diagnostics
Newborn Screening
Public Health
NORD joins 21 patient organizations in submitting comments to the Senate HELP committee on including protections for individuals with chronic conditions in the Pandemic and All-Hazards Preparedness Act (PAHPA) reauthorization
Published: Jul 21, 2023
Tagged in: COVID-19
NORD joins 215 organizations and Children’s Hospitals in encouraging members of Congress to support the Accelerating Kids’ Access to Care Act
Published: Jul 20, 2023
Tagged in: Access to Affordable, Adequate Coverage
Out-of-State Care for Children on Medicaid
NORD and 80+ patient advocacy and provider organizations in the All Copays Count Coalition (ACCC) request the Senate to include HELP Copays Act in any PBM reform package that comes to vote
Published: Jul 19, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD joins leading patient and provider organizations in urging NY Governor Hochul to promptly sign S1196
Published: Jul 17, 2023
Tagged in: Access to Innovative Medicines and Therapies
NORD joins 64 organizations in urging Ohio House of Representatives to move HB24 forward without the addition of unnecessary medical necessity criteria and an arbitrary monetary cap on testing costs
Published: Jul 17, 2023
NORD joins other 19 other patient organizations in providing comments to CMS on Arkansas’ ARHOME Section 1115 Amendment
Published: Jul 14, 2023
Tagged in: Access to Affordable, Adequate Coverage
Medicaid Section 1115 Waivers
NORD submits comments on “Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act”
Published: Jul 13, 2023
Tagged in: Access to Affordable, Adequate Coverage
Access to Innovative Medicines and Therapies
NORD joins 30 other patient organizations in opposition to H.R. 824, the Telehealth Benefit Expansion for Workers Act of 2023
Published: Jul 12, 2023
Tagged in: Telehealth
NORD joins 35 other patient organizations in submitting comments to White House leadership conveying concerns about nationwide losses in Medicaid coverage
Published: Jul 10, 2023
Tagged in: Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
NORD and 36 patient organizations applaud the Biden administration’s proposed rule to protect patients from short-term health plans
Published: Jul 7, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD provides comments regarding Docket No. FDA-2023-D-0026- “Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making”
Published: Jul 5, 2023
Tagged in: Access to Innovative Medicines and Therapies
Advancing Rare Disease Research and Regulatory Science
Clinical Trials
NORD joins leading patient organizations in providing comments on New York’s Section 1332 State Innovation Waiver
Published: Jul 5, 2023
Tagged in: Access to Affordable, Adequate Coverage
Affordable Care Act
Section 1332 Waivers
Final Inflation Reduction Act Guidance Risks Stifling Innovation for Rare Disease Community
Published: Jun 30, 2023
Tagged in: Advancing Rare Disease Research and Regulatory Science
Medicare, Medicaid, and Private Health Insurance Coverage
Orphan Drug Act
Research
NORD submits comments to the Institute for Clinical and Economic Review (ICER) on proposed revisions to its Value Assessment Framework
Published: Jun 30, 2023
Tagged in: Access to Affordable Medicines
Value-based Pricing Assessments
NORD and leading patient organizations submit comments to CMS on the Medicaid and Children’s Health Insurance Program (CHIP) Managed Care Access Proposed Rule
Published: Jun 29, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD joins 25 other patient organizations in submitting comments to CMS on the Medicaid Access Proposed Rule
Published: Jun 29, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD joins patient organizations in sending a letter to the House of Representatives regarding concerns about the detrimental impact of policies included in HR 2868, the Association Health Plans Act; HR 2813, the Self-Insurance Protection Act, and HR 3799, the CHOICE Arrangement Act
Published: Jun 21, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD joins 22 other organizations in sending a letter to the House Energy & Commerce Committee conveying patient community concerns about the detrimental impact of HR 824, the Telehealth Benefit Expansion for Workers Act
Published: Jun 21, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD submits comments to CMS on the proposed counteroffer phase of the Medicare drug price negotiation program
Published: Jun 20, 2023
Tagged in: Access to Affordable Medicines
NORD joins many patient organizations in thanking those who helped protect Medicaid for millions of Americans
Published: Jun 14, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD submits comments to CMS on the FY2024 Inpatient Prospective Payment System (IPPS) Proposed Rule
Published: Jun 9, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD sends letter to CMS supporting Part D Out-of-Pocket Caps
Published: Jun 5, 2023
Tagged in: Out of Pocket Prescription Drug Costs
National Organization for Rare Disorders Urges Congress To Protect Funding for Rare Disease Research, Commends Exclusion of Work Requirements for Medicaid in Debt Ceiling Deal
Published: Jun 2, 2023
Tagged in: Advancing Rare Disease Research and Regulatory Science
Appropriations
Ensuring Adequate Funding for NIH
Medicare, Medicaid, and Private Health Insurance Coverage
NORD and leading patient organizations urge the U.S. Department of Health & Human Services to protect Medicaid coverage by pausing redetermination in states with high procedural termination rates
Published: May 31, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD submits comments to the FDA on Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics
Published: May 26, 2023
Tagged in: Access to Innovative Medicines and Therapies
Advancing Rare Disease Research and Regulatory Science
FDA’s Accelerated Approval Pathway
NORD provides comments in support of the FDA’s Patient Focused Drug Development Listening Meeting ‘Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products’
Published: May 25, 2023
Tagged in: Access to Innovative Medicines and Therapies
Advancing Rare Disease Research and Regulatory Science
Research
NORD Submits Comments to the NJ Governor Regarding the Establishment of the NJ Rare Disease Advisory Council (RDAC)
Published: May 23, 2023
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD and 11 national patient advocacy organizations submit comments opposing work requirements in Arkansas’ ARHOME 1115 Amendment.
Published: May 23, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and 53 advocacy organizations urge the Department of Children and Families (DCF) to pause Florida’s current Medicaid redetermination process
Published: May 23, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD submits comments on the Information Collection Request (ICR) Form for Negotiation Data Elements under Section 11001 and 11002 of the Inflation Reduction Act
Published: May 22, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and other health organizations applaud Stay on Braidwood v Becerra lawsuit to protect coverage for preventive services
Published: May 15, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and patient organizations concerned by the loss of Medicaid coverage for nearly 75,000 Arkansans
Published: May 11, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and leading patient organizations urge the Senate HELP committee to pass meaningful PBM reform
Published: May 10, 2023
Tagged in: Access to Affordable Medicines
NORD and 78 patient organizations send letter to the HELP committee supporting the Retaining Access and Restoring Exclusivity (RARE) Act
Published: May 2, 2023
Tagged in: Access to Affordable, Adequate Coverage
Access to Innovative Medicines and Therapies
Advancing Rare Disease Research and Regulatory Science
NORD releases a statement for the record in support of S. 1214, the Retaining Access and Restoring Exclusivity (RARE) Act
Published: May 2, 2023
Tagged in: Access to Affordable, Adequate Coverage
Access to Innovative Medicines and Therapies
Advancing Rare Disease Research and Regulatory Science
NORD joins numerous organizations in requesting the Senate to continue supporting and funding the Congressionally Directed Medical Research Programs (CDMRP) at the DoD
Published: May 2, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and many patient organizations send a letter to the House of Representatives encouraging continued support and funding for the Congressionally Directed Medical Research Programs (CDMRP) at the DoD
Published: May 2, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and critical patient advocacy organizations support passing Expanded Access to Biomarker Testing in New York (A1673/ S1196)
Published: May 2, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD submits comments on “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products Guidance for Industry” (Docket No. FDA-2022-D-2983-0002)
Published: May 2, 2023
Tagged in: Advancing Rare Disease Research and Regulatory Science
Research
NORD joins patient organizations in filing an amicus curiae brief in support of the reversal of the Texas district court’s April 7, 2023 order
Published: May 1, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and 24 organizations convey concerns regarding AHPs and benefit exemption for telehealth-only plans
Published: Apr 26, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and numerous patient organizations urge Congress to protect Medicaid from funding cuts during budget negotiations
Published: Apr 24, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD and 29 patient organizations strongly oppose adding “work requirements” to the Medicaid program
Published: Apr 24, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and 32 patient and provider groups urge courts to preserve FDA’s authority and applaud Supreme Court ruling
Published: Apr 24, 2023
Tagged in: Access to Affordable, Adequate Coverage
NORD and 200+ advocacy organizations urge HHS to protect Medicaid continuous coverage
Published: Apr 24, 2023
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
NORD and 38 other patient organizations submit a letter to the TX Senate Health and Human Services Committee urging them to support SB 1408, Rare Disease Advisory Council legislation
Published: Apr 19, 2023
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD submits comments to the TX House Insurance Committee on HB 3188, biomarker legislation
Published: Apr 18, 2023
Tagged in: Access to Diagnostics
Founding member NORD issues fiscal year 2024 budget request with the NCATS Alliance
Published: Apr 18, 2023
Tagged in: Appropriations
Ensuring Adequate Funding for NIH
NORD and leading patient organizations urge Congress to reject adding a ‘work requirement’ policy to the Medicaid program
Published: Apr 17, 2023
Tagged in: Access to Affordable, Adequate Coverage
Public Health
NORD and 100 rare disease patient organizations send letter to CMS regarding Medicare Drug Price Negotiation Program Guidance
Published: Apr 14, 2023
Tagged in: Access to Affordable, Adequate Coverage
Please complete this form to access the requested resource.
