Briefings & Testimonies

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Congressional Briefing Showcases CF Foundation Drug Development and Care Center Models


Senator Edward Markey (D-Mass.) speaks about the benefits of the CF model at a CF Foundation-hosted Congressional briefing.

On February 11, 2014, the Cystic Fibrosis Foundation hosted a Congressional briefing to discuss the Foundation’s model of specialized, coordinated care and its innovative strategies for drug development, and how they can serve as an example for other rare, chronic diseases.
The event, entitled “Personalized Medicine and Quality, Coordinated Care: The Cystic Fibrosis Model,” included a panel discussion of health care experts who specialize in CF, moderated by Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation.

The briefing was co-sponsored by Senators Edward Markey (D-Mass.) and Kelly A. Ayotte (R-NH) and Congressional Cystic Fibrosis Caucus co-chairs Representatives Tom Marino (R-PA) and James McGovern (D-Mass).  Panelists included Terence R. Flotte, M.D., dean of the University of Massachusetts Medical School; Carlos Perez, M.D., director of the Cystic Fibrosis Care Center at Geisinger Medical Center in Danville, Penn.; Paul Negulescu, Ph.D., vice president for research at Vertex Pharmaceuticals; and Megan Stacy, who has cystic fibrosis.

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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 vital to those with cystic fibrosis. Below, you will find statements 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 speed 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 bi-partisan 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-OR), Orrin Hatch (R-UT), Sherrod Brown (D-OH) and Edward Markey (D-MA) as well as Representatives Lloyd Doggett (D-TX), Tom Marino (R-PA) and Jim McGovern (D-MA), 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 towards 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.

  • 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 letter sent to House members here and Senate members here.

  • 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 letter sent to House sponsors here and Senate sponsors here.


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 to those with cystic fibrosis.

  • 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 (CF)

  • Centralized Institutional Review Board (IRB) – Provides comments on a proposal to create a central IRB system through the National Institutes of Health (NIH) 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 HHS to consider these issues in creating regulations for clinical researchers. 

  • Patient-focused drug development meetings – Requests that the Food and Drug Administration (FDA) 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 in the drug development process, including patient participation in advisory committees as well as utilizing patient expertise in the development of genetically targeted therapies for rare diseases.  

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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.

  • Benefit and Payment Parameters – Outlines the needs of the cystic fibrosis community in updating the health benefit and payment system and commends HHS for moving toward greater transparency in health plan benefits to help those with CF and other chronic diseases make informed decisions regarding their health care.

  • 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.


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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. A final decision regarding these changes is expected from the Social Security Administration in 2015.

Learn more about our briefings and testimonies prior to 2014. 

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Updated: 5/8/2015