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 Comments on the Consideration of Adding Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel
Published: Sep 15, 2025
Tagged in: Access to Diagnostics
Newborn Screening
NORD and 189 other patient organizations send letter to Congress urging reauthorization of the Rare Pediatric Disease Priority Review Voucher Program
Published: Sep 11, 2025
Tagged in: Access to Innovative Medicines and Therapies
Access to Pediatric Therapies
Rare Disease Clinical Trials
NORD Comments on FDA Rare Disease Innovation, Science, and Exploration Public Workshop Series
Published: Aug 27, 2025
Tagged in: Access to Innovative Medicines and Therapies
Advancing Rare Disease Research and Regulatory Science
NORD Comments on Reauthorization of PDUFA VIII
Published: Aug 14, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
NORD Joins Statement Urging Congress to Reauthorize Enhanced Advance Premium Tax Credits (eAPTCs)
Published: Jul 30, 2025
Tagged in: Access to Affordable, Adequate Coverage
Affordable Care Act
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Joins Six Organizations in Statement on Federal Newborn Screening Legislation
Published: Jul 8, 2025
Tagged in: Access to Diagnostics
Newborn Screening
NORD Joins Letter Warning Congress Against Dangerous Cuts to Health Care for Patients
Published: Jul 1, 2025
Tagged in: Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Joins Statement Opposing Senate Passage of HR1
Published: Jun 28, 2025
Tagged in: Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Rare Disease Centers of Excellence Directors Urge Congressional Leadership to Reject Medicaid Cuts in HR1
Published: Jun 27, 2025
Tagged in: Access to Affordable, Adequate Coverage
Access to Innovative Medicines and Therapies
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Urges Ohio Governor Mike DeWine to Line-Item Veto Rare Disease Advisory Council (RDAC) Repeal in the Final Budget
Published: Jun 26, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Submits Comments to CMS on MDPNP IPAY 2028
Published: Jun 26, 2025
Tagged in: Access to Affordable Medicines
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Submits Comments to FDA on “Clinical Data Interchange Standards Consortium Dataset-JavaScript Object Notation”
Published: Jun 9, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
Research
NORD Signs-On to Letter Asking Congress to Support the NIH
Published: Jun 5, 2025
Tagged in: Ensuring Adequate Funding for NIH
NORD Submits Testimony in Opposition to the Proposed Elimination of the Ohio Rare Disease Advisory Council in State Senate Budget Proposal
Published: Jun 4, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Asks Governor Stitt to Sign Senate Bill 207 (OK) Into Law Creating the Oklahoma Rare Disease Advisory Council
Published: May 30, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Joins Statement Urging CMS to Deny Georgia’s Pathways to Coverage Waiver Extension Request
Published: May 30, 2025
Tagged in: Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Submits Testimony to House Appropriations Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies
Published: May 23, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
Appropriations
Public Health
NORD Joins Statement Urging Congress Not to Pass Devastating Health Care Cuts
Published: May 20, 2025
NORD Urges Governor Dunleavy to Sign SB 133 (AK) Step Therapy Reform Bill
Published: May 19, 2025
Tagged in: Access to Affordable Medicines
Access to Innovative Medicines and Therapies
Utilization Management
NORD Submits Statement for the Record in Opposition to Cuts to Medicaid
Published: May 13, 2025
Tagged in: Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Joins Statement for the Record in Advance of Ways and Means Markup
Published: May 13, 2025
Tagged in: Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Joins Leading Patient Organizations in Letter to HHS Secretary Urging CMS to Reject Arkansas ARHOME 1115 Demonstration Amendment
Published: May 9, 2025
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
Medicaid Section 1115 Waivers
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Joins Leading Patient Groups in Opposition to Arizona AHCCCS Waiver
Published: May 9, 2025
Tagged in: Access to Affordable, Adequate Coverage
Medicaid Section 1115 Waivers
Medicare, Medicaid, and Private Health Insurance Coverage
NORD Joins 271 Organizations in Community Letter to HHS on the Termination of the Advisory Committee on Heritable Disorders in Newborns and Children and Proposed Elimination of other Federal Newborn Screening Infrastructure
Published: May 5, 2025
Tagged in: Access to Diagnostics
Newborn Screening
NORD Sends Letter to Bipartisan Senate Hearing Titled, “Biomedical Research: Keeping America’s Edge in Innovation”
Published: Apr 29, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
Appropriations
Ensuring Adequate Funding for NIH
Research
NORD Signs-on to ARPA-H Appropriations Letter
Published: Apr 23, 2025
Tagged in: Appropriations
Ensuring Adequate Funding for NIH
NORD Submits Witness Testimony to Labor HHS Subcommittee for the NIH
Published: Apr 9, 2025
Tagged in: Ensuring Adequate Funding for NIH
NORD Signs-on to Ad-Hoc Letter to Support NIH Funding for Fiscal Year 2026
Published: Apr 8, 2025
Tagged in: Ensuring Adequate Funding for NIH
Twenty-Four Patient Advocacy Organizations Join NORD In Support of Montana HB943 Creating a Rare Disease Advisory Council
Published: Apr 3, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Supports Montana HB943 (Tuss) to Establish a Montana Rare Disease Advisory Council
Published: Apr 3, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Issues Statement Regarding the Department of Health and Human Services Reduction in Force
Published: Apr 1, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
Ensuring Adequate Funding for NIH
Medicare, Medicaid, and Private Health Insurance Coverage
Newborn Screening
Protecting FDA Standards for Safe and Effective Drugs
Public Health
NORD’S CEO Pam Gavin Joins Statement In Support of Pediatric Palliative Care Homes
Published: Mar 27, 2025
Tagged in: Palliative Care
NORD Joins 215 Organizations in Community Letter of Support of the Accelerating Kids’ Access to Care Act
Published: Mar 26, 2025
Tagged in: Access to Affordable Medicines
Access to Affordable, Adequate Coverage
Medicare, Medicaid, and Private Health Insurance Coverage
Out-of-State Care for Children on Medicaid
NORD Joins Other Patient Organizations in Support of Prior Authorization Legislation in Montana
Published: Mar 26, 2025
Tagged in: Utilization Management
NORD and 62 Patient Advocacy Organizations Support A1296-A (Paulin)/S1287-A (Persaud) – Establishing a Permanent Rare Disease Advisory Council in New York
Published: Mar 19, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Submits Testimony in Support of Biomarker Legislation in Oregon
Published: Mar 18, 2025
Tagged in: Genetic Testing
NORD Submits Testimony In Support of S474 (Lawson) – A Bill to Establish a Rare Disease Advisory Council (RDAC) in Rhode Island
Published: Mar 18, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD and 30 Patient Advocacy Organizations Support S474 (Lawson)/H5023 (Kennedy) – Establishing a Rare Disease Advisory Council in Rhode Island
Published: Mar 18, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Joins Letter in Support of HELP Copays Act (S. 864)
Published: Mar 13, 2025
Tagged in: Access to Affordable Medicines
Copay Assistance
NORD Urges New Mexico to Join the Interstate Medical Licensure Compact
Published: Mar 13, 2025
Tagged in: Telehealth
NORD Joins Patient & Provider Organizations in Support of Rhode Island Step Therapy Reform S116
Published: Mar 12, 2025
Tagged in: Access to Affordable Medicines
Access to Innovative Medicines and Therapies
Utilization Management
NORD Submits Comments on “Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway Guidance for Industry”
Published: Mar 10, 2025
Tagged in: FDA’s Accelerated Approval Pathway
Protecting FDA Standards for Safe and Effective Drugs
NORD Urges Support of Arizona House Bill 2380 to Create the Rare Disease Advisory Council in AZ
Published: Mar 7, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Requests Support for Funding for California’s Jacqueline Marie Zbur Rare Disease Advisory Council
Published: Mar 6, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Joins Leading Patient Groups in Urging Administration to Reverse Decision to Exclude Public Comment Periods
Published: Mar 6, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
NORD Submits Comments on Expedited Program for Serious Conditions — Accelerated Approval of Drugs and Biologics Guidance for Industry
Published: Mar 5, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
FDA’s Accelerated Approval Pathway
Protecting FDA Standards for Safe and Effective Drugs
NORD Signs on to Letter to Ensure Robust Appropriations for the NIH and Reaffirm Indirect Cost Reimbursements
Published: Mar 4, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
Appropriations
Ensuring Adequate Funding for NIH
Public Health
Research
NORD Sends Letter of Support for HF 1501 to Fund Minnesota’s RDAC for FY2026 and FY2027
Published: Mar 4, 2025
Tagged in: Rare Disease Advisory Councils (RDACs)
NORD Signs Letter in Support of Federally Funded Research
Published: Feb 26, 2025
Tagged in: Advancing Rare Disease Research and Regulatory Science
Ensuring Adequate Funding for NIH
Funding for Ongoing Science
Public Health
NORD Joins Leading Patient Groups in Urging House Energy & Commerce Health Subcommittee to Pass Meaningful PBM Reform
Published: Feb 25, 2025
Tagged in: Access to Affordable Medicines
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