Our Policy Priorities

BEF advocates for policies that improve the lives of people with CF. We engage state and federal policymakers to advance research, support clinical care, accelerate drug development, and enable affordable access to life-extending medications and healthcare options. BEF is informed, involved, and vocal on public policy. We believe strongly that all people with CF, and others living with rare diseases, deserve equitable access to healthcare and innovative medicines, the ability to critique the cost of healthcare, and the right to defend their own needs.

 

Want to get involved in CF policy and advocacy? Reach out to Meredith Marden, Director of Advocacy and Strategy.

Our Policy Priorities

as of September 2025

To ensure affordable innovation

Institutions and policies that promote biomedical research and innovation are key to delivering the best care possible for all patients. To keep innovation affordable, policies must also guarantee undelayed access to generic medicines when they become available. 

A Strong Foundation of Science

  • Ensure adequate funding, personnel, and resources for organizations like the NIH and the FDA in order to advance scientific discovery and ensure the highest quality and safety of medicines for all patients
  • Protect the the ability to freely research, to access to a wide range of scientific resources, to collect robust and comprehensive data, and to openly share information

An Environment for Innovation

  • Revise policies that may impede the innovation and development of lifesaving medicines
    • Establish parity between small molecules and biologics in Medicare price negotiations introduced by the Inflation Reduction Act by passing the EPIC Act
    • Discourage the use of Prescription Drug Affordability Review Boards (PDABs) that can enact price controls on medicines based on their list price, which does not reflect the true value of treatments, particularly for rare diseases 
      • Instead of PDABs, advocate for policies that streamline approval and guarantee undelayed access to generic medicines
  • Increase antibiotic development as proposed in the PASTEUR Act and create market conditions to enable new antibiotic options
  • Reauthorize the Pediatric Rare Disease Priority Review Voucher Program to incentivize the development of rare pediatric disease treatments through the Give Kids a Chance Act

Value medicines based on their value to society, including patients, caregivers, and communities, through adopting cost-effectiveness assessment methods like the Generalized Cost-Effectiveness Analysis in healthcare decision-making

To ensure accessible healthcare options for people with CF and their families

For many in the CF community, breakthrough treatments are only accessible through robust insurance coverage with low out-of-pocket costs. Early diagnosis and treatment, regular screening for age-related comorbidities, and the infrastructure to support new and emerging gene therapies are all crucial to ensure the highest quality of life for all people living with CF. 

Comprehensive Insurance Coverage

  • Ensure that all Americans with CF have access to high quality health insurance
    • 36% of adults living with CF and 52% of children under 10 years old living CF are covered by Medicaid in the United States
      • Ensure necessary funding and ease of enrollment for all state Medicaid programs
      • Advocate for states to be clear, consistent, and patient-centered in the implementation of work requirements for Medicaid to ensure that eligible individuals do not lose coverage
    • Renew premium tax credits for Affordable Care Act marketplace plans to keep premiums affordable for the individuals and families that rely on these plans
  • Create more flexible exemptions for step therapy
    • Support measures included in the Safe Step Act, such as making a clear process for patients to request exemptions from step therapy and clarifying step therapy processes
  • Streamline prior authorizations to ensure that they are efficient, simple, and do not interfere with physicians’ and their patients’ authority of their care 
  • Expand and strengthen the availability, quality, and coverage of mental healthcare services for the CF community

Affordable Healthcare Costs

  • Eliminate copay adjustment programs, which include accumulators, maximizers, and alternative funding plans, to ensure that copay assistance can meaningfully lower out of pocket (OOP) costs for patients
  • Reduce or eliminate out-of-pocket expenses and cost-sharing insurance policies
    • Extend the $2,000 OOP cost cap in Medicare to all insurance plans
  • Discourage using Most Favored Nation policies to set prices for US medicines, which may negatively impact global access, harm consumers in the US, and will not solve the root problem of healthcare affordability 
  • Reform pharmacy benefit managers’ (PBMs’) practices to increase transparency and to ensure that savings negotiated by PBMs are passed down to patients

Continuous Access to Care

  • Promote access to comprehensive and timely newborn screening
    • Advocate for the inclusion of all known CFTR variants on newborn screening panels, as recommended by the Cystic Fibrosis Foundation 
    • Encourage primary care provider education to recognize CF symptoms and provide timely diagnoses
    • Reinstate the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) to ensure that newborn screening remains robust, well-informed, cohesive, and effective
  • Lower the CF-specific minimum screening age for colorectal cancer, which people living with CF are 5-10 times more likely to develop 
  • Ensure timely and affordable access to emerging cell and gene therapies through innovative and thoughtful payment policies 
  • Create regulatory clarity for gene therapies for rare diseases, including streamlined review and post-market surveillance processes and consistent communication and collaboration between regulatory bodies and patient communities
  • Expand delivery infrastructure to deliver cell and gene therapies by increasing the number of accredited centers and promoting policies that facilitate the uninterrupted care of patients across state lines to ensure access to gene therapies 

To protect patients’ ability to critique the cost of healthcare and to defend their own needs

In clinical trials and in the clinic, the patient perspective must be centered and fully integrated to deliver the highest quality of care. 

Centering Patients' Voices

  • Ensure that patients are central to healthcare decision-making in the clinic through education and access to shared decision-making frameworks
  • Reduce barriers to clinical trial participation
    • Create regulatory frameworks to pay clinical trial participants more to promote widespread participation in clinical trials and to address the financial barriers to enrollment
    • Strengthen telehealth flexibilities to streamline clinical trial participation, including remote participation
    • Create paid time off (PTO) or short-term disability leave for people participating in clinical trials

Memberships

BEF is a patient advocacy member of NYBio, BioNJ, and the Lower Out of Pocket Costs (LOOP) Coalition.

Policy and Advocacy Fellowship

BEF’s Policy and Advocacy Fellowship is an online program for college students and young adults designed to equip patient advocates with the tools, knowledge, and confidence to enhance their engagement in key policy and advocacy issues. In weekly meetings, fellows discuss key policy and advocacy topics such as antimicrobial resistance, shared decision-making in the clinic, and insurance benefit design. Fellows also write position papers and produce a capstone project on a topic of their choice.

 

Interested in participating? Reach out to mmarden@esiason.org