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Policy Statements

NORD advocates on behalf of the entire rare disease community. Read key testimony, letters, and position papers.

2015

  • August 14: Comments to the NIH regarding the NIH Strategic Plan Framework
  • August 10: Comments to the FDA regarding the Duchenne Muscular Dystrophy and Related Dystrophinopathies Developing Drugs for Treatment Draft Guidance
  • July 31: Letter to the Centers for Medicare and Medicaid Services (CMS) on Orphan Exclusion from the Medicaid Line Extension Rebate
  • July 31: Citizen Petition to the FDA on the use of modern testing methods for pre-clinical research
  • July 28: Amicus Curiae Brief filed in support of Spectrum Pharmaceuticals in Spectrum v. Burwell
  • July 27: Comments on the Medicaid Managed Care Proposed Rule as part of the Regulatory Education and Action for Patients (REAP) Coalition
  • July 15: Testimony and Presentation at the FDA Prescription Drug User Fee Program Reauthorization Stakeholder Meeting
  • July 13: Testimony and Presentation at the FDA Medical Device User Fee Program Reauthorization Stakeholder Meeting
  • June 24: Letter to U.S. Senate Committee on Finance regarding incentives for rare disease therapies as related to trade negotiations in Trans-Pacific Partnership (TPP) and President Trade Promotion Authority (TPA)
  • June 24: Letter to House Energy & Commerce Committee from NORD and Member Organizations supporting 21st Century Cures Act
  • June 23: Letter to U.S. House of Representatives supporting Children’s Access to Specialty Care Act of 2015 (H.R. 1859)
  • June 22: Letter to Senate Finance Committee as part of Medicare Access for Patients (MAP) Rx Coalition
  • June 17: White Paper (co-authored by NORD and BIO) , “Impact of the Orphan Drug Tax Credit on Treatments for Rare Diseases,” and accompanying patient stories
  • May 21: Videos from Caucus Briefing on the 21st Century Cures Initiative and the Priorities of the Rare Disease Community
  • May 29: Final Comments to House Energy & Commerce Committee on 21st Century Cures Act
  • April 13:  Comments to FDA regarding Draft Guidance to help simplify “Individual Patient Expanded Access Applications: Form FDA 3926: Draft Guidance for Industry” for patients and physicians
  • April 13: Letters to House and Senate to reauthorize the Rare  Pediatric Disease Priority Review Voucher (PRV) Program
  • April 13: Letter to FDA regarding draft guidance for the Patient Expanded Access Form (Form 3926)

Previous Years

Letter to Commissioner Hamburg thanking the FDA for its commitment to the Pediatric Device Consortia and urging increased investment in this program, October 9, 2014.

Letter from Nord and other organizations urging the Medicare Payment Advisory Commission (MedPAC) to analyze the Medicare Part D exceptions and appeals process and issue recommendations on how to improve the appeals system, October 10, 2014.

NORD Backgrounder on Expanded Access prepared in conjunction with Senate briefing on this topic on Sept. 10, 2014. Slides from the briefing co-hosted by NORD and The Leukemia & Lymphoma Society.

Letter from NORD to the Massachusetts Senate Committee on Ways and Means in support of S.2096, July 16, 2014

Testimony by Marshall Summar, MD, on behalf of NORD for the U.S. House of Representatives Energy and Commerce Committee “21st Century Cures: Incorporating the Patient Perspective”, July 11, 2014

Letter from NORD to Governor Jindal of Louisiana thanking him for the passage of Bill 165, regarding access to specialty tier drugs, July 2, 2014

NORD’s Comments to Representative Upton on the 21st Century Cures White Paper, June 13, 2014

Letter from NORD to Governor O’Malley of Maryland thanking him for the successful passage of the Maryland specialty tier bill (HB761), June 4, 2014

Letter from NORD to Margaret Hamburg, Commissioner, The Food and Drug Administration regarding biosimilars, June 3, 2014

NORD Comments on 21st Century Cures Initiative’s first white paper titled, “A Call to Action”, May 30, 2014

NORD Comments on CMS’s Interim Rule on Third-Party Payments within Exchange Plans, May 13, 2014

Letter from NORD and other organizations on the Newborn Screening Saves Lives Reauthorization Act, May 13, 2014

Letter from NORD thanking Representative Delaney for introducing the Medical Foods Equity Act (H.R.3665), April 30, 2014

Letter from NORD and other health organizations to Congress in regards to the Fiscal Year (FY) 2015 302(b) allocation to the Labor, HHS, Education and Related Agencies Appropriations Subcommittee, March 13, 2014

Letter from NORD and partners to Congress in regards to a proposal made public by Chairman Dave Camp to amend the Internal Revenue Code of 1986 would repeal the Orphan Drug Tax Credit, March 10, 2014

Letter from NORD to the Centers for Medicare & Medicaid Services regarding the Contract Year 2015 Policy and Technical Changes to the Medicare Advantage and the Medicare Prescription Drug Benefit Programs, March 7, 2014

Letter from NORD to state officials regarding Rare Disease Day, February 15, 2014

Letter from NORD to members of Congress regarding Rare Disease Day, February 3, 2014

Testimony by NORD VP of Public Policy Diane Dorman beforeFDA Cardiovascular and Renal Drugs Advisory Committee, January 14, 2014

Letter from NORD to the FDA regarding the patient’s role in  advisory committee hearings, January 14, 2014

Letter from NORD thanking Senator Sherrod Brown (lead sponsor) for the passage of the National Pediatric Research Network Act., December 17, 2013

Letter from NORD and other patient organizations to the Budget Conference Committee, regarding the Orphan Drug Tax Credit, November 22, 2013

Letter from NORD to Kathleen Sebelius, Secretary, HHS, regarding Qualified Health Plans under the Affordable Care Act, November 21, 2013

Letter from NORD regarding the government shut-down, October 10, 2013

Letter from NORD regarding the Patients’ Access to Treatments Act (H.R. 460), August 30, 2013

NORD Comments on FDA Draft Guidance on Expedited Programs for Serious Conditions August 26, 2013

Letter from NORD and other patient organizations to the Chairs of the Senate Finance Committee and the Chairs of the House Ways and Means Committee in support of the Orphan Drug Tax Credit, July 25, 2013

Letter from NORD and other patient organizations to Acting Commissioner of Social Security Carolyn Colvin asking for support for the Compassionate Allowances Program, July 12, 2013

Letter from NORD and other members of the Friends of Newborn Screening Coalition to Secretary Sebelius thanking her for the establishment of the Discretionary Advisory Committee on Heritable Disorders in Newborns and Children, May 22, 2013

Letter signed by NORD and several other advocacy organizations: NORD Comments Submitted to FDA on 5-Year Draft Plan Regarding Benefit/Risk, April 30, 2013

Comments delivered by Peter L. Saltonstall at the FDA Commemoration of the 30th Anniversary of the Orphan Drug Act, January 7, 2013

Comments delivered by Peter L. Saltonstall regarding the launch of new public-private partnership to encourage development of innovative medical devices, December 3, 2012

Comments submitted by NORD in response to the Food and Drug Administration’s request for comments regarding the implementation of the FDA’s patient-focused drug development initiative, October 31, 2012

Essential Health Benefits (EHB) Sign on Letter
Submitted to Kathleen Sebelius Secretary, U.S. Department of Health and Human

Policy Statement: Assuring the Continued Development of Life-saving Therapies to Treat Rare Diseases:Enhancing the Accelerated Approval Process; the FAST Act and TREAT Act
March 2012

Letter to HHS commenting on the Essential Health Benefits bulletin
Submitted electronically to EssentialHealthBenefits@cms.hhs.gov, January 26, 2012

NORD Testimony From Congressional Hearing on Prescription Drug User Fee Act Reauthorization
Presented by Diane E. Dorman of NORD to the United States House of Representatives Committee on Energy and Commerce Subcommittee on Health, February 1, 2012

Letter regarding current conflict of interest statutory provisions related to FDA, October 2011

Public Meeting on Prescription Drug User Fee Act Reauthorization
Presented by Diane E. Dorman of NORD to the FDA, October 24, 2011

Public Comments regarding the FDA section 510(k) clearance process for medical devices
Submitted by NORD to the FDA, September 22, 2011

Citizen’s Petition to FDA Regarding Orphan Drug Reviews
Submitted by Peter Saltonstall of NORD to the FDA, September 1, 2011

Comments regarding patient-centered outcomes research
Submitted by Diane Dorman of NORD to the PCORI Board of Directors, July 18, 2011

Supporting our Nation’s Most Vulnerable Patients: Letter to Congressional Leadership Regarding Reclassification of Therapies that Treat Rare Diseases

Letter Sent July 18, 2011

Remarks Presented Before The FDA Pulmonary-Allergy Drugs Advisory Committee
Presented by Diane E. Dorman, June 23, 2011

Supporting a Stronger FDA: Letter to Members of the House Committee on Appropriations
Letter Sent May 31, 2011

Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures
Presented by Diane E. Dorman, July 21, 2010

Considerations regarding FDA review and regulation of articles for the treatment of rare disease
Presented by Frank Sasinowski, June 29, 2010

Testimony before the Senate Appropriations Committee
Presented by Diane E. Dorman, June 23, 2010

Testimony regarding the significance of the The Orphan Drug Act for the development of infectious diseases therapies in the 21st century, before the Food and Drug Administration’s (FDA)Infectious Diseases Planning Committee
Presented by Diane E. Dorman, April 28, 2008

Testimony regarding possible compassionate allowances for people with rare diseases who apply for Social Security disability
Presented by Diane E. Dorman, December 4, 2007

Testimony regarding “Ensuring Safe Medicines and Medical Devices for Children”
Presented by Robert Campbell, Jr., MD, NORD Medical Advisory Committee, March 27, 2007

Letter to Commissioner of Social Security regarding initiative to improve rules for Compassionate Allowances
September 2007

DC Court of Appeals Amicus Brief for Abigail Alliance for Better Access to Developmental Drugs, et al., v. Andrew C. Von Eschenbach, et al.
March 2007

Introductory Remarks, NORD Generic Biologics Conference
Presented by Abbey S. Meyers, March 19, 2003

Wiley Lecture for 2005 presented to FDA Alumni Association
Presented by Abbey S. Meyers, April 6, 2005

Testimony regarding the Food and Drug Administration’s (FDA) approval process, drug safety and the concerns of patients before the U.S. Senate Health, Education, Labor, and Pensions Committee
Presented by Abbey S. Meyers, March 1, 2005

Responses to follow-up questions posed by the HELP Committee chairman

Letter to NIH Director Regarding Public Access to Research Information
By Abbey Meyers, NORD President, October 2004

Speech at Workshop on Ultra-Orphan Genetic Disease Therapeutics
Presented by Abbey S. Meyers, May 8, 2003

In support of a funding increase for FDA Orphan Products Research Grant Program in FY 2003
Testimony submitted for the record, March 2002, Diane E. Dorman, NORD Vice President for Public Policy

Human Research Subject Protection
Written comments prepared for the Senate HELP Committee. May 2002. Abbey S. Meyers

A Tribute to Roscoe Brady, MD
Speech delivered at NIH Symposium by Abbey Meyers, October 2002

Analysis by and Recommendations of the Special Emphasis Panel of the NIH on the Coordination of Rare Diseases Research
Summary — January 2001, Abbey S. Meyers

Unfulfilled Promises and Stem Cell Research
Presented to MIT, November 20, 2001, Abbey S. Meyers

Public Meeting on the Reauthorization of the Prescription Drug User Fee Act
Testimony submitted before the House Energy and Commerce Subcommittee on Health, December 7, 2001, Diane E. Dorman

Evaluating the Effectiveness of the Food and Drug Administration Modernization Act
Testimony submitted before the House Energy and Commerce Subcommittee on Health, May 3, 2001, Abbey S. Meyers

Orphan Drug Development Conference: “Understanding the History of the Orphan Drug Act”
September 2000, Abbey S. Meyers

Position Paper: Genetic Patenting
By Abbey S. Meyers, President NORD. September 25, 2000

Position Paper: Insurance Reimbursement for Orphan Drugs
By Abbey S. Meyers, President, NORD. August 6, 2000

Position Paper: Genetic Discrimination
By Abbey S. Meyers, President NORD. August 11, 2000

Position Paper: Patients’ Bill of Rights
By Abbey S. Meyers, President NORD. November 2000

Position Paper: Patient Protection in Clinical Trials
By Abbey S. Meyers, President NORD. August 14, 2000

The Patient Advocate Perspective. Preparing for FDA Advisory Committee Presentations. Views from Four Perspectives
Abbey S. Meyers, President NORD. June 30, 2000

Collaboration Between the Pharmaceutical Industry and Patient Organization: A Delicate Balance
Abbey S. Meyers, President NORD. February 17, 2000. Spain: International Conference of Rare Diseases and Orphan Drugs.

History of the American Orphan Drug Act
Abbey S. Meyers, President NORD. February 18, 2000. Spain: International Conference of Rare Diseases and Orphan Drugs

Exploring the Role of Patient Organization in the Development of Orphan Products
Abbey S. Meyers, President NORD. September 17, 1999

New Initiatives to Protect Patients in Gene Therapy Trials
Abbey S. Meyers, President NORD

“Priority Orphans” Chart