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The Cystic Fibrosis Foundation maintains a strong presence in Washington, hosting events in Congress that highlight the Foundation’s successful drug development model and raise awareness about the disease.
CF Foundation Sponsors Congressional Briefing on Cystic Fibrosis Research
Mitch Greenberg, a 38 year old with CF, spoke at the briefing about his experiences living with the disease.
On October 16, the Cystic Fibrosis Foundation, the American Thoracic Society, the National Heart, Lung and Blood Institute (NHLBI) and the Children’s Interstitial Lung Foundation (CHiLD) presented a briefing on Capitol Hill to underscore the value of investments in biomedical research by highlighting the considerable successes of research into CF and neonatal lung diseases.
Panelists included Susan B. Shurin, MD, Deputy Director of NHLBI; Stephanie Davis, MD, Professor of Pediatrics at Indiana University Health; and Thomas Ferkol, MD, Professor of Pediatrics at Washington University School of Medicine. Mitch Greenberg, a 38 year old with cystic fibrosis who has undergone two double lung transplants, also spoke at the briefing about his experiences living with the disease. Representatives Tom Marino (R-PA), Cliff Stearns (R-FL) and Edward Markey (D-MA), Co-Chairs of the Cystic Fibrosis Caucus, were the briefing’s Congressional sponsors.
Congressional Roundtable Discussion with NIH Director Francis Collins
On June 14, 2011, the Cystic Fibrosis Foundation and Vertex Pharmaceuticals, Inc. hosted a congressional roundtable discussion with National Institutes of Health Director Francis S. Collins, M.D., Ph.D., and members of the U.S. House of Representatives. The event focused on the NIH’s strategies for accelerating drug development for rare diseases and highlighted the CF Foundation’s drug development approach as a model for new NIH programs.
Speakers included Collins; Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation; Vertex Chairman and CEO Matthew W. Emmens; and Vertex Vice President and CF Franchise Lead Eric Olson, Ph.D. Reps. Edward Markey (D-MA), Brian Bilbray (R-CA), Tom Marino (R-PA) and Jim McGovern (D-MA) also participated in the discussion.
Learn more about the roundtable.
Leveraging Federal Investment to Speed the Development of Promising Therapies for Patients
On May 20, 2010, the Cystic Fibrosis Foundation, Faster Cures/The Center for Accelerating Medical Solutions, and Sens. Richard Shelby (R-AL) and Richard Durbin (R-IL) hosted a Senate briefing with Francis S. Collins, M.D., Ph.D., director of the National Institutes of Health (NIH). This event highlighted the Cystic Fibrosis Foundation’s success in bridging the gap between basic science discoveries and creating new therapies for patients.
“There is great progress being made in cystic fibrosis research,” said Collins, who noted how advancements in treatments and care have transformed cystic fibrosis from a pediatric illness to one in which nearly half of people with the disease are age 18 or older.
Collins said his goal is to take the promise of recent research developments in life-threatening diseases such as cystic fibrosis “and turn that into real results.”
Watch the Briefing
Learn more about the briefing.
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In order to ensure that 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 critical 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:
- Read testimony submitted by the CF Foundation to the Labor, Health and Human Services, Education and Related Agencies Subcommittee which urges sufficient funding for important biomedical and translational research; requests support for the National Center for Advancing Translational Sciences (NCATS) to promote the use of innovative methods and technologies to improve the development, testing and implementation of new diagnostics and treatments; requests that Congress support collaborative efforts between the FDA, the NIH and stakeholders in drug development and review; and urges funding for programs that promote affordable access to quality, specialized CF care.
- Read the CF Foundation’s testimony to the Subcommittee on Agriculture, Rural Development, Food and Drug Administration and Related Agencies requesting robust funding for drug review at the FDA, particularly for rare diseases; highlighting Kalydeco’s swift approval as a model of success in FDA drug review; and encouraging adequate funding for the implementation of the Food and Drug Administration Safety and Innovation Act (FDASIA). The Expanding and Promoting Expertise in Review of Rare Treatments Act (EXPERRT), a bill championed by the CF Foundation that encourages the FDA to consult with outside experts when evaluating rare disease drugs, was signed into law as part of FDASIA in July 2012.
The Development and Review of CF Treatments:
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Read testimony, provided by CF Foundation President and CEO Robert J. Beall, Ph.D, regarding:
Health Care Reform and 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.
- Eligibility for Social Security Disability Benefits – Opposes the Social Security Administration’s proposed changes in eligibility for people with CF cautioning that eligibility criteria must be consistent with patterns of care as outlined by providers who understand and treat cystic fibrosis.
- Letter to the Social Security Administration – A letter signed by nearly 130 CF care center directors cautioning against proposed eligibility changes for Social Security disability benefits.
- Medicaid, CHIP and Exchanges – Cautions that the proposed rule may potentially increase obstacles to care for people with CF and other chronic illnesses by imposing barriers to life-saving treatments and increasing cost-sharing for patients and families.
- Read a letter to Dr. Margaret Hamburg, Director of the Food and Drug Administration, applauding the agency’s swift review and approval of Kalydeco™ from Dr. Robert Beall, President and CEO of the CF Foundation, and Mary B. Dwight, Vice President for Government Affairs.
- Obtaining Patient Perspectives During Drug Development and Review – Read the Foundation’s request that the FDA expand its efforts to obtain and incorporate the patient perspective in considering treatments for orphan genetic diseases.
- Read a letter from Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation, to Senators Harkin and Enzi thanking them for their leadership on the Food and Drug Administration Safety and Innovation Act (S. 2516). This Act included the Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act, which provides the FDA with greater and more consistent access to rare disease experts during the drug review process.
- View thanks from Dr. Robert Beall, President and CEO, to Congressmen Pitts, Upton , Waxman, and Pallone for including the Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act into the Food and Drug Administration Reform Act (H.R. 5651). In doing so, the Committee has demonstrated commitment to providing the FDA with greater and more consistent access to rare disease experts during the drug review process.
- Read a letter from Dr. Robert J. Beall, President and CEO of the Cystic Fibrosis Foundation, to the sponsors of the EXPERRT Act expressing the Foundation’s support for increased collaboration between the FDA and rare disease experts during the drug review process.
- View a letter of support from various patient groups representing millions of Americans with rare and genetic conditions who support the EXPERRT Act.
- Essential Health Benefits Package: Round One – Cautions that allowing each state to determine its own essential health plan package does not adequately protect access to comprehensive care for people with life-threatening, chronic diseases.
- Essential Health Benefits: Round Two – Highlights the importance of access to quality, specialized care for people with cystic fibrosis, including covering critical CF care and treatments, limiting financial burdens for patients, defining habilitative services and ensuring patients are protected by state laws governing coverage and out of pocket costs.
- Read correspondence sent to congressional leaders by CF Foundation President and CEO Robert J. Beall, Ph.D., and Vice President of Government Affairs Mary Dwight regarding attempts to repeal the health care reform law on January 6, 2011.
- Development of Drugs for Combination Therapy – Supports the development of drugs that will be used together in combination therapy.
- Medicaid-Eligible Adults – Urges that Medicaid programs develop more quality standards for people with rare diseases.
- Accountable Care Organizations (ACOs) – Stresses that beneficiaries should be involved in establishing ACOs and ensure that money-saving strategies do not negatively impact patient care.
- Appeals and External Reviews – Opposes current rules that limit access to specialty care when patients need treatment outside of their plan network.
- Companion Diagnostic Devices – Urges that the FDA avoid any unnecessary review of diagnostic devices, which could slow the approval of certain therapies for CF patients.
- Standards for Health Exchanges: Round Two – Advocates that insurance plans must be affordable, and that rare disease experts should be consulted when developing state exchanges.
- Summary of Benefits and Coverage – Recommends that benefits information should be comprehensive, easy to understand and accessible to the public.
- Human Subjects Protections – Commends the Department of Health and Human Services (HHS) for their work to improve complex Human Subjects Research Protections. These comments reinforce the importance of ensuring that that the needs of children who participate in clinical studies are addressed and note the possible high initial costs of making the proposed changes.
- Affordability Tax Credits – Advocates that Medicaid should provide coverage from the date of application for assistance.
- Coverage for Dependents Through Age 26 – Supports coverage for adult children through their parents’ health plans up to age 26.
- Grandfathered Insurance Plans – Advocates for more information about what protections are considered “grandfathered” and reinforces that grandfathered plans must provide patients with adequate coverage.
- Preexisting Conditions & Lifetime Limits – Supports the removal of annual and lifetime limits to insurance coverage by 2014 and promotes patient protections during the implementation of the health care reform law.
- Standards for Health Exchanges: Round One – Urges that states should consult with qualified experts to provide the best care for people with rare and chronic diseases and insists that health plans in the new exchanges include access to specialty care.
- Comment to the IOM on Essential Health Benefits – Stresses that essential benefits packages must balance cost and coverage so patients can afford the care they need, and discusses the importance of coverage for specialty care for people with CF.
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