Briefings, Testimonies, and Regulatory Comments

By educating elected officials and other government decision makers on the needs of the cystic fibrosis community, the Cystic Fibrosis Foundation works to shape public policy to help our efforts to cure CF and provide all people with CF the opportunity to lead long, fulfilling lives.

Briefing

On Dec. 8, 2015, the Cystic Fibrosis Foundation hosted a congressional briefing, Precision Medicine & Cystic Fibrosis: Using Genetics to Treat Disease, to educate members of Congress and their staff on the role of precision medicine and genetically targeted treatments and therapies. 

The event included a panel discussion moderated by William R. Skach, M.D., senior vice president for research affairs at the CF Foundation. The panelists included Eric D. Green, M.D., Ph.D., director of the National Human Genome Research Institute (NHGRI) at the National Institutes of Health (NIH); Noreen Roth Henig, M.D., chief development officer of ProQR Therapeutics NV; and Mara Cray, a 19-year-old with CF.

The briefing was co-sponsored by the Congressional Cystic Fibrosis Caucus co-chairs, Representatives Tom Marino (R-Pa.) and Jim McGovern (D-Mass.). 

Testimonies

To ensure the voices of the CF community are heard in Washington, the Cystic Fibrosis Foundation submits testimony, comments, and letters to Congress and federal agencies on health care policies that affect or have the potential to affect those with cystic fibrosis. Following are statements submitted by the Foundation that advocate for the CF community's interests in three areas: federal investment in cystic fibrosis research and drug review, the development and review of CF treatments, and health care reform.

Federal Investment in CF Research and Drug Review

21st Century Cures Initiative

Energy and Commerce Committee Chair Fred Upton (R-Mich.) and Representative Diana DeGette (D-Colo.) are spearheading the 21st Century Cures Initiative, an effort aimed at developing policies to accelerate the discovery, development, and delivery of treatments and biomedical innovation. Through this initiative, the Energy and Commerce Committee is soliciting input from industry, patients, researchers, government agencies, and other stakeholders about ways Congress can incentivize and accelerate the development of cures. The CF Foundation has provided expertise in a number of areas to help inform this effort.

Ensuring Access to Clinical Trials Act of 2015 (EACT)

The bipartisan Ensuring Access to Clinical Trials Act of 2015 makes permanent the Improving Access to Clinical Trials Act of 2009 (IACT), a law enacted in 2010 that allows patients with rare diseases to participate in and benefit from clinical trials without fear of losing vital benefits. Sponsored by Senators Ron Wyden (D-Ore.), Orrin Hatch (R-Utah), Sherrod Brown (D-Ohio), and Edward Markey (D-Mass.) as well as Representatives Lloyd Doggett (D-Texas), Tom Marino (R-Pa.), and Jim McGovern (D-Mass.), this bill will permanently allow patients with cystic fibrosis and other rare diseases to receive up to $2,000 in compensation for participating in clinical trials without that compensation counting toward their income eligibility limits for SSI and Medicaid. The Cystic Fibrosis Foundation actively supported the Improving Access to Clinical Trials Act when it was introduced and advanced in 2009, and we are working to ensure that the law continues to eliminate barriers to clinical trial participation for the CF community.

  • In support of the Ensuring Access to Clinical Trials Act, the CF Foundation's Senior Vice President of Therapeutics Development Dr. Michael Boyle provided testimony during a congressional hearing. Read Dr. Boyle's testimony here.
  • The Foundation sent letters thanking the sponsors of the Ensuring Access to Clinical Trials Act and outlining the importance of this legislation for the cystic fibrosis community. View the letters sent to House members her and Senate members.
  • The Foundation formed a coalition in support of the Ensuring Access to Clinical Trials Act and collected the signatures of more than 75 organizations on a letter explaining the bill's positive impact on clinical trial participation for the rare disease community. View the letters sent to House sponsors and Senate sponsors.

Drug Development and Review

Read the Cystic Fibrosis Foundation's comments and proposals to the National Institutes of Health (NIH), Food and Drug Administration (FDA), and other related agencies on streamlining the drug development process to speed safe and effective therapies for those with cystic fibrosis.

  • Prescription Drug User Fee Act: Under the Prescription Drug User Fee Act, the FDA is entitled to collect application fees from drug manufacturers when a New Drug Application (NDA) is submitted. This document provides comments to the Food and Drug Administration on its framework of priorities for using these funds to improve and streamline the drug development process.
  • Federal Policy for the Protection of Human Subjects: This policy comments on the federal effort to improve oversight of human subjects research and suggests further improving and streamlining the informed consent process and requiring use of a central IRB for multi-site studies.
  • Patient-Focused Drug Development for Nontuberculous Mycobacterial (NTM) Lung Infections: Discusses the risk and significant burden of NTM infections for people with cystic fibrosis and commends the Food and Drug Administration for its public meeting on this issue. 
  • FDA Draft Guidance on Rare Disease Drug Development: Commends the Food and Drug Administration for providing guidance on significant challenges associated with the development of drugs for rare diseases and suggests additional areas where further guidance is needed in this area.
  • Use of Biomarkers in Drug Development: Discusses the critical importance of advancing the identification of biomarkers in the process for developing new, targeted cystic fibrosis therapies and medications.
  • FDA Advisory Committee Meeting on New Drug Application for Lumacaftor/Ivacaftor Combination: Provides comments to the Food and Drug Administration in support of the application of lumacaftor and ivacaftor for treatment of individuals with F508del homozygous cystic fibrosis.
  • Centralized Institutional Review Board (IRB): Provides comments on a proposal to create a central IRB system through the National Institutes of Health and commends the NIH for undertaking this important initiative.
  • OHRP Standards of Care: Describes the impact of new standards for disclosing risks in research participation on clinical trials for cystic fibrosis therapies and asks Health and Human Services (HHS) to consider these issues in creating regulations for clinical researchers. 
  • Patient-Focused Drug Development Meetings: Requests that the Food and Drug Administration host a meeting about the specific needs and experiences of people with cystic fibrosis as part of the FDA's Patient Focused Drug Development initiative. 
  • Patient-Centered Review: Proposes a number of ways that the FDA can involve patients throughout the drug development process, including patient participation in advisory committees and using patient expertise in the development of genetically targeted therapies for rare diseases. 

Health Care Reform and Other Regulatory Guidance

View the Cystic Fibrosis Foundation's testimony, comments, and letters highlighting the need for access to sophisticated, specialized care and health coverage for people with cystic fibrosis.

  • Chronic Care Guidelines: Guidelines that examine policy options for improving the quality of care for those living with chronic diseases as set forth by the Senate Finance Committee's Bipartisan Chronic Care Working Group, which highlights the needs and perspectives of the CF community.
  • Stabilizing the Health Insurance Marketplaces: Lays out five suggestions to promote well-functioning health insurance marketplaces to help people with CF access the care they need, including maintaining essential health benefit protections, continuing cost-sharing reduction payments, and establishing a permanent reinsurance program. These policies can help incentivize insurer participation and moderate premium growth in the individual and small group markets.
  • Tax Reform and Its Impact on the CF Community: As both chambers of Congress considered potential reforms to the U.S. Tax Code, the Cystic Fibrosis Foundation sent a letter expressing its concerns about removing several protections that benefit the CF community. Specifically, the Foundation asked Congress to protect the Medical Expenses Deduction and the Orphan Drug Tax Credit.
  • Comments on Graham-Cassidy Health Care Proposal: As the U.S. Senate considered the Graham-Cassidy health care bill, the Cystic Fibrosis Foundation submitted comments highlighting the Foundation's major concerns about the impact that removing protections for patients with pre-existing conditions, dramatically cutting Medicaid, and opening the door to annual and lifetime coverage caps, would have on the CF community's ability to access the adequate, affordable care they need to stay healthy.
  • Renewing Funding for the Children's Health Insurance Program (CHIP): The Children's Health Insurance Program (CHIP) is critical for children with life-threatening, chronic diseases like cystic fibrosis. With Congress allowing funding for CHIP to expire, the Foundation sent a letter in support of renewing CHIP to help ensure children with CF have access to highly specialized care and therapies.
  • Health Benefit Plan Network Access and Adequacy Model Act: Commends the National Insurance Commissioners (NAIC) for updating the managed care network adequacy act, and recommends modifications that reflect the needs of individuals living with CF, as well as others with serious and life-threatening illnesses.
  • Benefit and Payment Parameters: Discusses the proposed changes to health insurance exchanges and standards for qualified health plans, and suggests further revisions to ensure that those with CF receive quality care in-network and more affordable care outside of their provider network.
  • Medicaid Managed Care: Commends the Centers for Medicare and Medicaid Services (CMS) for their efforts to streamline and improve Medicaid managed care systems, and identifies obstacles that still remain for those with CF who receive high-quality, specialized care in accredited CF care centers.
  • Mental Health Parity Requirements: Highlights the importance of addressing mental health needs for those with CF and their caregivers, and addresses the expansion of coverage for these services offered by Medicaid-managed care organizations, Children's Health Insurance Programs (CHIP), and Medicaid Alternative Benefit Plans.
  • CHIP Reauthorization: Underscores the value of the Children's Health Insurance Program for children with CF in ensuring that these patients have access to quality, specialized CF care, and asks Congressional leadership to reauthorize funding so that states may continue to provide this coverage to children with CF and other life-threatening chronic illnesses.
  • The Role of Managed Care Plans in the Medicaid Program: Asks Congressional leadership to consider the needs of medically complex, rare disease populations when determining best practices for the shift to managed care plans in Medicaid programs by limiting specific barriers to treatment and coverage that can be especially detrimental to these individuals.
  • Newborn Screening: Emphasizes the importance of newborn screening programs in diagnosing CF and delivering early, lifesaving treatments, and urges Senate leaders to pass legislation to continue funding for this vital public health program.
  • Specialty Practitioner Payment Model Opportunities: Highlights the CF care center network as a model of efficient, effective care delivery and discusses the challenges and opportunities associated with improving payment and delivery for CF care through episode-based bundles.
  • Medicare Part D Access to Immunosuppressants: Opposes a proposal by the Centers for Medicare and Medicaid Services that would hinder access to immunosuppressant medication for post-transplant patients.
  • Federally Facilitated Marketplaces: Urges the Centers for Medicare and Medicaid Services to ensure affordable access to CF care centers and treatments in federal health insurance marketplaces.

Social Security Disability Programs

In February 2013, the Social Security Administration (SSA) proposed changes to the criteria it uses to determine if a patient with cystic fibrosis is eligible for disability benefits. The CF Foundation is concerned that some of these changes could hinder access to disability benefits for CF patients. View below letters to the SSA from the Cystic Fibrosis Foundation, cystic fibrosis care center directors, and members of Congress expressing concern about the proposed changes.

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