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Briefings & Testimonies

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CF Advocate Provides Remarks about Kalydeco, FDA Review at Congressional Briefing

Will Elder Jr

William Elder, Jr., (right) discusses new breakthrough therapies for cystic fibrosis.
On July 24, 2013, the Cystic Fibrosis Foundation participated in a Congressional briefing hosted by Friends of Cancer Research to educate members of the U.S. Senate about new processes developed by the FDA to speed the approval of breakthrough therapies.

William Elder, Jr., a medical student with cystic fibrosis, spoke at the event about the health gains he has experienced since starting Kalydeco, the first oral drug that treats the underlying cause of CF in a small group of people with the disease. He also stressed the importance of keeping the FDA fully funded, telling Senators and their staff about the promise of the new process used by the Food and Drug Administration (FDA) to speed the development of breakthrough therapies, including those for cystic fibrosis.

Elder was joined by Sen. Michael Bennet (D-CO), who provided the keynote address at the briefing, Jeffrey Leiden, the chief executive officer of Vertex Pharmaceuticals, Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research and other representatives from the medical community.

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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.
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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.

  • 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.

  • Protection of Clinical Trial Data – Provides input to the FDA on ways to ensure the anonymity of patients in the clinical trial data submitted to the agency as part of medical product applications.

  • 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.

  • New pathway for expediting drug development –Suggests revisions to the FDA’s proposed new strategies for expediting the development of drugs for life-threatening illnesses, such as encouraging the agency to develop clear and specific guidelines, avoiding policies that could make treatments more expensive and consulting with external rare disease experts during drug review and approval.

  • Evaluating the risks and benefits of new drugs – Evaluates the FDA’s proposed 5 year plan for implementing a new framework for benefit-risk evaluation in the human drug and biologic review process.

  • New incentives for antibiotics development – Provides insight to the FDA on implementing the Generating Antibiotics Incentives Now (GAIN) Act, which encourages the development of new antibacterial and antifungal drugs to treat certain serious infections and creates a list of the pathogens that would be targeted by new products.

  • New breakthrough therapies – Suggests a change to a new FDA program that would accelerate the review of treatments for serious diseases like cystic fibrosis. The comments also commend the agency for making its draft guidance on the new program clear, concise and responsive to the needs of those with CF.

  • Response to RFI – CFF’s Response to the Request for Information on Specialty Practitioner Payment Model Opportunities

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.

  • Official comments from the Cystic Fibrosis Foundation to the Social Security Administration expressing concern about the agency’s proposed changes to disability benefits for people with CF.

  • A letter signed by 128 CF care center directors cautioning against proposed eligibility changes for Social Security disability benefits.

  • Letters to the Social Security Administration from 24 members of the House of Representatives and 13 Senators asking the Social Security Administration to reconsider the proposed revisions to eligibility for Social Security benefits.
  • A transcript of the Social Security Administration’s presentation during a special teleconference for the cystic fibrosis community about proposed changes to disability benefits on May 10, 2013.

Learn more about our briefings and testimonies prior to 2013.

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Updated 4/18/14

The Cystic Fibrosis Foundation is an accredited charity of the Better Business Bureau.