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Policy Statements

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

2018

  • October 17: Coalition comments to CMS regarding Alabama’s Section 1115 waiver
  • October 16: Coalition letter thanking Congress for increasing funding for research
  • October 9: Coalition letter asking Congress to reauthorize loan repayment for pediatric subspecialists
  • October 1: Coalition letter to Secretary Azar regarding the patient protections in H.R.2077 that ought to be incorporated when implementing step therapy in Medicare Advantage plans
  • October 1: Coalition statement regarding Maine’s SPA to expand Medicaid
  • October 1: Coalition letter asking Secretary Azar and Administrator Verma to approve Maine’s State Plan Amendment (SPA) to expand Medicaid
  • September 26: Coalition letter to Secretary Azar regarding the recent decision to allow step therapy in Medicare Advantage plans
  • September 26: Coalition comments to South Dakota regarding its Section 1115 waiver
  • September 25: NORD comments to FDA regarding its definition of low-prevalence in a recent draft guidance
  • September 21: NORD comments to FDA regarding innovation and competition in the biological products marketplace
  • September 19: Coalition letter to the Senate regarding its recent bill on pre-existing conditions protections
  • September 11: NORD comments to FDA regarding patient-focused drug development
  • September 5: Coalition letter asking Congress to continue supporting the medical expense deduction
  • September 4: Coalition statement addressing the Senate’s recent bill on pre-existing conditions protections
  • August 31: NORD comments on Oklahoma’s Section 1115 waiver
  • August 31: Coalition comments to CMS regarding New Hampshire’s Section 1115 waiver
  • August 30: NORD comments on Alabama’s revised Section 1115 waiver
  • August 22: Coalition letter asking Congress to refrain from funding agriculture association health plans (AHPs)
  • August 17: NORD comments on Kentucky’s Section 1115 waiver
  • August 17: Coalition comments to CMS regarding Mississippi’s Section 1115 waiver
  • August 14: NORD comments to FDA regarding its Humanitarian Device Exemption (HDE) program
  • August 9: Coalition comments to CMS regarding New Jersey’s Section 1332 waiver
  • August 3: Coalition comments to CMS regarding Maryland’s Section 1332 waiver
  • August 1: Coalition statement expressing serious concerns with the Administration’s decision to expand the use of “short-term, limited-duration insurance
    plans.”
  • July 24: Coalition letter to CMS regarding the recent court ruling on Kentucky’s work requirement in Medicaid
  • July 18: Coalition letter to insurance commissioners across the nation expressing concerns with copay accumulators
  • July 17: NORD comments to the Administration on its “Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs”
  • July 16: Coalition letter requesting Michigan to release additional data regarding its recently updated Section 1115 waiver
  • July 11: Coalition statement urging the Administration to resume paying the risk adjustment payments for 2017
  • July 10: Alliance for a Stronger FDA letter to Congress regarding FDA appropriations
  • July 6: Coalition comments to CMS regarding Maine’s Section 1332 waiver
  • June 27: NORD comments to New Hampshire regarding its Section 1115 waiver
  • June 26: NORD comments regarding Managed Care Organizations in Texas’ Medicaid program
  • June 26: Coalition comments to Washington regarding its short-term limited-duration medical plan rulemaking stakeholder draft
  • June 25: NORD comments to CMS regarding the inpatient prospective payment system (IPPS) proposed rule
  • June 21: NORD comments regarding funding for newborn screening in Texas’ Legislative Appropriations Request
  • June 20: Coalition letter in support of copay assistance in Massachusetts
  • June 19: Coalition statement regarding the expansion of Association Health Plans (AHPs)
  • June 19: Coalition letter in opposition to legislation in Pennsylvania that would seek to implement work requirements in Medicaid
  • June 14: Coalition comments to CMS regarding Ohio’s Section 1115 waiver
  • June 14: Sign-on letter from patient organizations in support of Michigan House Bill 5983, an act to establish a Rare Disease Advisory Council.
  • June 8: NORD letter of support for New Jersey Assembly Bill 2431, bipartisan legislation to limit out-of-pocket prescription costs.
  • June 8: Coalition comments regarding Wisconsin’s Section 1332 waiver
  • June 1: NORD comments to the American Medical Association expressing concerns with their resolution proposing reforms to the ODA
  • May 23: NORD letter in support of New Hampshire House Bill 1791, an act calling for prescriber communication with regard to biosimilars
  • May 21: NORD comparison of two versions of the Right to Try Act
  • May 21: NORD letter in support of Ohio Senate Bill 56, an act pertaining to step therapy protections
  • May 21: Coalition letter in opposition to the Right to Try Act (S.204)
  • May 18: NORD comments to FDA regarding the development and submission of proposed draft guidance relating to patient experience data
  • May 16: NORD comments in opposition to Rhode Island Senate Bill 2532, a bill pertaining to copay accumulators
  • May 14: Coalition letter urging CMS to rescind invitation to states to include work requirement policies in their 1115 demonstration waiver applications
  • May 8: Coalition letter to Congress requesting continued support for the defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at the Department of Defense (DOD)
  • April 23: I Am Essential coalition comments to the Administration in opposition to its proposed rule regarding short-term, limited-duration plans (STLDs)
  • April 23: Coalition comments to the Administration in opposition to its proposed rule regarding short-term, limited-duration plans (STLDs)
  • April 23: Coalition letter to the Administration in opposition to its proposed rule regarding short-term, limited-duration plans (STLDs)
  • April 18: Sign-on  letter from over 100 rare disease patient organizations in support of the RARE Act of 2018
  • April 18: NORD letter in support of Connecticut House Bill 5297, an act requiring continuing medical education in screening for rare gastrointestinal cancers
  • April 17: Coalition letter asking Congress to voice opposition to the proposed rule regarding short-term, limited-duration plans (STLDs)
  • April 12: Coalition statement expressing concerns with the 2019 Notice of Benefit and Payment Parameters final rule
  • April 11: Coalition letter in opposition to Michigan Senate Bill 897, a bill requiring the state to implement a work requirement in its Medicaid program
  • April 9: Coalition letter to Secretary Azar regarding Medicare Part D’s protected classes policy
  • April 4: Coalition  comments regarding Colorado’s proposed permanent rule on cost-sharing
  • April 3: Coalition letter asking Governors to voice opposition to the proposed rule regarding short-term, limited-duration plans (STLDs)
  • April 2: NORD comments to the Alabama Medicaid Agency on its Section 1115 Demonstration Application
  • March 27: Coalition letter asking Congress to appropriate funds for the Pediatric Device Consortia (PDC) Grant Program
  • March 26: NORD letter in support of West Virginia House Bill 4524, an act calling for prescriber communication with regard to biosimilars
  • March 26: Coalition statement regarding Congress’ failure to include a market stabilization package in the spending deal
  • March 26: Coalition letter in opposition of Rhode Island Senate Bill 2532, an act that would prevent copay assistance from being applied to out-of-pocket expenses
  • March 22: Sign-on letter to the Senate from 106 patient and provider organizations in opposition to the Right to Try Act
  • March 19: Coalition letter regarding recent policy changes proposed by the administration
  • March 19: Updated NORD one-pager detailing our opposition to the Right to Try Act
  • March 19: Updated sign-on letter from over 100 patient and provider organizations in opposition to the Right to Try Act
  • March 13: Coalition statement calling on Congress to pass a market stabilization proposal
  • March 12: Sign-on letter from 82 patient organizations in opposition to the newest version of the Right to Try Act
  • March 6: Coalition comments on the Department of Labor’s proposed rule regarding Association Health Plans (AHPs)
  • March 6: NORD letter in support of Connecticut Senate Bill 398, an act relating to the enforcement of Connecticut’s step therapy law
  • March 5: Coalition comments to CMS regarding the Medicare prescription drug benefit and beneficiary access
  • February 27: Coalition letter to Congress regarding medical research within the fiscal year 2019 National Defense Authorization Act
  • February 23: Coalition letter to Congress in support of the Medical Nutrition Equity Act (S.1194/H.R.2587)
  • February 22: Coalition comments to CMS on Wisconsin’s Section 1115 BadgerCare demonstration waiver extension application
  • February 22: NORD comments to CMS on Wisconsin’s Section 1115 BadgerCare demonstration waiver extension application
  • February 22: Coalition comments to CMS on Mississippi’s Section 1115 revised demonstration waiver application
  • February 22: NORD comments to CMS on Mississippi’s Section 1115 revised demonstration waiver application
  • February 21: Coalition sign-on letter to Medicaid Directors on the importance of Medicaid formulary access for rare disease patients
  • February 20: NORD comments in support of Missouri House Bill 2407
  • February 20: Coalition statement in response to a proposed rule that would expand the use of “short-term limited duration” insurance plans
  • February 18: NORD comments to FDA regarding the designation of pediatric subpopulations
  • February 16: NORD comments to FDA regarding patient-focused drug development
  • February 16: NORD comments to the Food and Drug Administration (FDA) regarding targeted therapies
  • February 13: Coalition letter to Secretary Azar regarding Idaho’s recent insurance bulletin
  • February 13: NORD one-pager emphasizing our opposition to the Right to Try Act
  • February 12: NORD letter in support of Ohio Senate Bill 56, an act relating to step-therapy
  • February 12: NORD comments in support of Kansas Senate Bill 304, an act relating to step- therapy
  • February 6: NORD letter in support of Oregon House Bill 4156, an act relating to out-of-pocket costs
  • February 6: Coalition letter to Congress in opposition to the Right to Try Act
  • February 5: NORD comments to the Food and Drug Administration (FDA) regarding pediatric drug development guidance
  • February 5: Coalition comments to CMS on Arizona’s Section 1115 demonstration waiver request
  • February 5: NORD comments to CMS on Arizona’s Section 1115 demonstration waiver request
  • February 2: American Plasma Users (APLUS) coalition letter regarding the Patient Notification System (PNS)
  • January 30: Coalition letter to Congress regarding a desire for a hearing on ensuring patient safety and access in the midst of the Biologics Price Competition and Innovation Act (BPCIA)
  • January 30: Coalition comments to CMS on New Mexico’s Centennial Care 2.0 1115 Waiver Renewal Application
  • January 26: Coalition comments to CMS on Kansas’ KanCare 2.0 Section 1115 Demonstration Renewal Application
  • January 26: NORD comments to the Centers for Medicare and Medicaid Services (CMS) on Kansas’ KanCare 2.0 Section 1115 Demonstration Renewal Application
  • January 25: Coalition response to the Department of Health and Human Service’s (HHS) request for information (RFI) titled  “Promoting Healthcare Choice and Competition Across the United States”
  • January 18: NORD letter in support of South Dakota Senate Bill 75, an act calling for prescriber communication with regard to biosimilars
  • January 17: NORD comments to the Centers for Medicare and Medicaid Services (CMS) on Next Generation Sequencing (NGS)
  • January 17: Coalition comments to the Centers for Medicare and Medicaid Services (CMS) on Next Generation Sequencing (NGS)
  • January 17: NORD letter in support of adding SMA to Georgia’s newborn screening panel
  • January 17: NORD letter to Pennsylvania’s Rare Disease Council regarding the state’s newborn screening panel
  • January 16: Coalition letter to the Centers for Medicare and Medicaid Services (CMS) regarding direct and indirect remuneration (DIR)
  • January 16: Coalition comments to the Centers for Medicare and Medicaid Services (CMS) regarding Medicare Part D

Previous Years

  • December 20: Coalition statement in response to Congress repealing the ACA’s individual mandate within the Tax Cuts and Jobs Act
  • December 14: NORD comments to the Massachusetts Senate Joint Committee on Financial Services regarding fail first legislation
  • December 12: Coalition letter to tax reform conferees regarding the ACA’s individual mandate
  • December 8: Coalition letter to the conference committee in strong support of the medical expense deduction
  • December 7: Coalition letter urging Congress to protect funding for the Medicare State Health Insurance Assistance Program (SHIP) for FY18.
  • December 7: Coalition letter from over 160 patient organizations to the tax reform conference committee in strong support of the Orphan Drug Tax Credit
  • December 1: NORD letter of opposition regarding Arkansas Board of Pharmacy’s proposed rule
  • November 30: Coalition letter to the Senate in support of the Collins Amendment
  • November 30: Coalition statement regarding a Congressional Budget Office (CBO) score confirming that the Alexander-Murray bill is not a fix for repealing the individual mandate
  • November 27: Coalition comments on Health and Human Services (HHS) Notice of Benefit and Payment Parameters for 2019
  • November 20: Coalition statement in opposition to the House of Representatives’ vote to repeal the Orphan Drug Tax Credit
  • November 15: NORD letter to IL Department of Healthcare and Family Services regarding Medicaid DUR for nusinersen and other orphan drugs
  • November 14: Coalition statement urging Congress to not include a repeal of the individual mandate within tax reform
  • November 14: Coalition statement by 36 patient organizations in opposition to the Senate’s proposed weakening of the Orphan Drug Tax Credit
  • November 8: Coalition letter from over 200 patient organizations imploring Congress to maintain the Orphan Drug Tax Credit **Updated letter as of November 10**
  • October 31: Coalition letter in support of the proposed substitute for Michigan House Bill No. 4472
  • October 26: Coalition statement urging Congress to act on the Bipartisan Health Care Stabilization Act of 2017 in light of the Congressional Budget Office (CBO) report
  • October 20: NORD comments to the Centers for Medicare & Medicaid Services (CMS) regarding the MassHealth Section 1115 Demonstration Amendment Request
  • October 18: Coalition statement in support of the HELP bipartisan stabilization deal
  • October 18: Coalition letter to the Centers for Medicare & Medicaid Services (CMS) regarding the MassHealth Section 1115 Demonstration Amendment Request
  • October 12: Coalition letter to Congress in support of the Ensuring Children’s Access to Specialty Care Act of 2017
  • October 12: Coalition letter urging Congress to include support for loan repayment for pediatric subspecialists as part of the effort to reauthorize the Title VII health professions workforce programs
  • October 12: Coalition letter urging the House to adopt the Senate Appropriations Committee’s funding levels for outreach and enrollment in marketplace coverage under the Affordable Care Act
  • October 12: Coalition statement regarding President Trump’s executive order released today
  • September 29: Coalition letter urging Congress to reject any proposal to increase Medicare Part D copayments for beneficiaries receiving the low-income subsidy (LIS)
  • September 25: NORD comments to the Institute for Clinical and Economic Review (ICER) on a proposed adaptation to its value framework for the assessment of treatments for ultra-rare conditions
  • September 22: Coalition statement regarding the Senate Finance Committee Hearing to Consider the Graham-Cassidy-Heller-Johnson Proposal
  • September 19: Coalition letter to the House Energy and Commerce Committee in opposition to S.204, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, and H.R. 878, the Right to Try Act of 2017
  • September 18: Coalition statement in opposition of Graham/Cassidy Bill
  • September 14: Sign-on letter from 140 patient organizations urging Congress to keep the Orphan Drug Tax Credit in place
  • September 11: NORD comments to the Centers for Medicare & Medicaid Services (CMS) regarding the “14-Day Rule”
  • September 11: NORD comments to the Centers for Medicare & Medicaid Services (CMS) regarding biosimilars
  • September 8: Coalition letter urging Secretary Price to retain the six protected classes under Medicare Part D
  • August 30: NORD letter voicing concerns over California Assembly Bill 265
  • August 21: NORD comments on the Massachusetts Executive Office of Health and Human Services’ (EOHHS) proposed Amendment to the MassHealth Section 1115 Demonstration
  • August 16: NORD letter to Congress outlining short-term and long-term stabilization and improvement proposals for the Affordable Care Act (ACA)
  • July 18: Coalition letter urging the House to restore Medicare State Health Insurance Assistance Program (SHIP) funding to FY16 levels
  • July 12: Coalition comments in response to Health and Human Services (HHS)’s Request for Information (RFI) on ways to reduce regulatory burdens and improve health care choices to empower patients
  • July 11: Coalition letter urging Congress to negotiate a bipartisan budget deal that raises the sequestration caps
  • July 6: Coalition letter opposing proposed budget cuts to newborn screening programs
  • June 15: Coalition letter urging Senate leadership to protect Medicaid
  • June 12: Coalition letter urging Congress to reauthorize the Children’s Health Insurance Program (CHIP)
  • June 12: Coalition comments to FDA on the creation of an Office of Patient Affairs
  • June 9: NORD letter in support of the Medical Nutrition Equity Act of 2017
  • June 1: Coalition letter in support of New York S.4788/A.7509, acts regarding the substitution of interchangeable biological products
  • May 23: Zika Coalition letter to the House Committee on Energy and Commerce
  • May 17: Oregon coalition letter regarding out-of-pocket costs
  • May 9: NORD letter in support of the Enhancing Clinical Trial Design Act
  • May 8Sign-on letter from 120 patient organizations urging Congress to reauthorize FDA User Fee Agreements
  • May 2: Coalition principles for access to unapproved therapies
  • April 28: NORD comments on FDA Reauthorization Act of 2017 discussion draft
  • April 28: NORD letter in support of the Rare Disease Expert Provision in Iowa House File 653
  • April 24: MAPRx Coalition letter to the Centers for Medicare & Medicaid Services (CMS)
  • April 19: Coalition comments on manufacturer communications regarding unapproved uses of approved or cleared medical products
  • April 4: Coalition letter urging Congress to provide funding for newborn screening in FY 2018 appropriations
  • April 3: NORD comments on proposed changes to ICER’s value framework
  • April 3: NORD comments on ICER’s Patient Participation Guide
  • March 28: NORD letter in support of California Senate Bill 643, a bill to add Duchenne muscular dystrophy to the list of medical conditions eligible for the Genetically Handicapped Persons Program
  • March 28: NORD letter in support of Iowa House File 233, an act regarding step therapy protocols
  • March 27: Coalition letter urging Congress to include funding to combat Zika in Fiscal Year 2018 appropriations
  • March 21: NORD letter in support of Florida House Bill 963, an act regarding newborn screening
  • March 20: Coalition letter to Congress expressing concern over proposed changes to Medicaid
  • March 20: NORD letter in support of Nevada Assembly Bill 245, an act calling for prescriber communication with regard to biosimilars
  • March 16: NORD letter in support of Oregon Senate Bill 237, an act regarding cost sharing
  • March 16: NORD letter in support of Kansas House Bill 2107, an act calling for prescriber communication with regard to biosimilars
  • March 7: American Plasma Users Coalition (APLUS) letter to Congress regarding patient protections
  • March 7: Coalition for Accessible Treatments (CAT) letter to Congress regarding ACA Cost Sharing
  • March 6: Coalition healthcare reform principles
  • March 1: Coalition letter in support of CT HB 7118, an act calling for prescriber communication with regard to biosimilars
  • February 27: Coalition letter to President Trump regarding rare diseases and the hiring freeze
  • February 23: NORD Principles for Health Coverage Reform
  • February 22: NORD State Ambassador testimony before the Connecticut General Assembly’s Public Health Committee supporting HB 6009, an act to establish a Rare Disease Advisory Council
  • February 15: NORD letter in support of Alabama House Bill 82, an act calling for prescriber communication with regard to biosimilars
  • February 10: Coalition letter urging Congress to preserve funding for programs that provide critical support to state newborn screening programs
  • February 10: Coalition letter urging Congress to provide ample funding to combat the Zika virus
  • February 10: Coalition letter to New Jersey Legislature regarding patient out-of-pocket costs for prescription medications
  • February 10: NORD letter in support of New Mexico Senate Bill 180 and House Bill 260, acts calling for prescriber communication with regard to biosimilars
  • February 9: NORD letter on preexisting conditions insurance protections
  • February 7: Coalition letter to President Trump regarding vaccine safety
  • February 3: NORD letter in support of South Carolina Senate Bill 299 and South Carolina House Bill 3438
  • February 3: NORD letter in support of Nebraska Legislative Bill 481
  • February 3: NORD letter in support of Alaska Senate Bill 32
  • January 24: NORD letter in support of Kansas House Bill 2107
  • January 18: NORD letter in support of Montana House Bill 233
  • January 6: NORD comments to FDA on the Center for Devices and Radiological Health’s (CDRH) establishment of the Patient and Care-Partner Connection (P&CC)

December 14: Coalition letter to President-elect Trump and Congressional leadership regarding health care reform

November 28: NORD comments on the FDA’s Medical Device User Fee Amendments

November 18: Coalition letter asking for long term, optimally permanent, reauthorization of the Rare Disease Priority Review Voucher program as part of the 21st Century Cures Act.

November 16: Coalition letter urging Congress to pass the 21st Century Cures Act

November 16: Coalition letter supporting the Pediatric Subspecialty Loan Repayment Program in MH Reform

November 11: NORD presentation for the inaugeration of the NGO Committee for Rare Diseases (CfRD)

November 9: NORD provides testimony during the FDA’s “Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products” Public Hearing

October 19: Coalition comments from PBSA to FDA on the BsUFA Commitment letter

October 6: NORD comments on FDA’s Next Generation Sequencing draft guidances

October 6: Coalition comments from I Am Essential to Secretary Burwell on Notice of Benefit and Payment Parameters for 2018 Proposed Rule

September 28: Coalition letter urging Congress to approve Zika funding immediately

September 12: NORD comments to the Institute for Clinical and Economic Review (ICER) on their proposed Value Assessment Framework 2.0

August 22: NORD comments to FDA on the PDUFA VI Commitment Goals Letter along with our accompanying Office of Patient Affairs proposal and one pager

August 3: Coalition letter to Congress urging for the conference committee to craft a compromise Zika funding package that can be passed upon their return from recess in September

July 22: Coalition letter to NDAA Conference Committee members in support of TRICARE coverage of medically necessary foods

July 13: Coalition letter uring Congress to send bipartisan Zika emergency spending bill to the President

July 12 & 13: PBSA statement before FDA Arthritis Advisory Committee meetings

June 28: Coalition letter to Congress urging for the conference committee to reconvene and produce a new Zika funding bill

June 27: REAP Coalition comments on CMS’s MACRA physician quality payment program proposed rule

June 24: PBSA thank you letter to Dr. Janet Woodcock and the FDA

June 7: Coalition letter endorsing H.R. 1859/S. 2782

May 31: PBSA Coalition comments to FDA on Biosimilar Labeling Guidance

May 24: Coalition letter to Senator Kirk on the REGROW Act

May 18: Coalition letter to the House supporting Zika Supplemental Funding

May 13: Coalition letter to the Senate supporting Zika Supplemental Funding

May 13: Coalition letter in support of Advancing Research for Neurological Diseases Act

May 9: CAT Coalition comments to CMS on CMMI Medicare Program: Part B Drug Payment Model

May 9: NORD comments to CMS on CMMI Medicare Program: Part B Drug Payment Model

May 1: Coalition letter to FDA on Biosimilar distinguishable naming

April 28: NORD releases a position statement on patient assistance programs (PAPs) related to the issue of increases in prescription drug spending and overall health care costs.

April 28: Letter to The Honorable Jay Nixon, Governor of Missouri, to support SB 875, a bill that will protect patients by including language calling for prescriber communication.

April 27: Coalition letter to the Senate HELP Committee in support of the Innovations initiative

April 19: PBSA Coalition letter to Commissioner Califf on FDA’s implementation of the Biologics Price Competition and Innovation Act (BPCIA)

April 5: Coalition letters to Congress in support of Zika funding

March 25: MAPRx Coalition letter to MedPAC in opposition of proposals related to Medicare Part D

March 17: Letter to House Appropriators in support of funding for the Pediatric Device Consortia Grant Program

March 14: Letter to House Armed Services Committee on TRICARE coverage of medical foods

March 14: NORD comments to FDA on Clinical Outcome Assessment (COA) Compendium

February 29: NORD letter to Congress on supporting Rare Disease Day 2016

February 19: Coalition letter to the Senate in support of Dr. Califf’s Nomination for FDA Commissioner

February 9: NORD statement at the FDA Arthritis Advisory Committee hearing on Celltrion’s biosimilar for Remicade

January 15: Coalition comments from I Am Essential to Secretary Burwell on the Draft 2017 Letter to Issuers in the Federally-facilitated Marketplaces

January 6: NORD comments to OHRP on Federal Policy for the Protection of Human Subjects

January 6: Coalition comments to OHRP on the implications of the proposed changes of the Common Rule for universal newborn screening programs

December 21: A-PLUS Coalition comments to CMS on its benefit and payment parameters 2017 proposed rule

December 21: NORD comments to CMS on its benefit and payment parameters 2017 proposed rule

December 21: Coalition comments from I Am Essential to CMS on its benefit and payment parameters 2017 proposed rule

December 21: CAT Coalition comments to CMS on its benefit and payment parameters 2017 proposed rule

December 9: NORD letter to Chairman Hatch of the Senate Committee on Finance on CMS biosimilar reimbursement

November 13: NORD letter to Congressional Appropriators in support of FDA appropriations for 2016

November 12: Coalition letter to the Senate HELP Committee in support of the Advancing Targeted Therapies for Rare Diseases Act (S.2030)

November 9: Coalition letter from I Am Essential to Secretary Burwell on discrimination in health insurance plan structure

November 9: REAP Coalition letter to Secretary Burwell on non-discrimination in health plans and activities

October 29: Coalition letter to the Senate HELP Committee in support of Dr. Califf’s Nomination for FDA Commissioner

October 27: NORD comments to the FDA on Biosimilar naming

October 23: Patients for Biologics Safety & Access coalition comments to the FDA on Biosimilar naming

October 16: Comments to the FDA on their draft guidance titled “Rare Diseases – Common Issues in Drug Development”

October 12: Letter to Chairman Paul Ryan of the House Ways and Means Committee in support of the Preserving Access to Orphan Drugs Act

September 21: Letter to the Senate Health, Education, Labor, and Pensions (HELP) Committee in support of Humanitarian Device Exemption (HDE) reforms in the 21st Century Cures Act.

September 21: APLUS Coalition letter to Secretary Burwell and letter to House Committee Chairmen expressing support for recent changes to out-of-pocket maximum rules

September 17: Coalition letter to the Senate HELP Committee in support of the OPEN Act

September 8: Comments to CMS regarding reimbursement codes for Biosimilars

September 2: Letter to the Precision Medicine Working Group recommending the inclusion of children in the Precision Medicine Initiative (PMI) cohort

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

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