Principles and Standards for Pharmaceutical Industry Interactions

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Engaging with biotechnology and pharmaceutical companies is essential to reaching our goal of a cure for all people with cystic fibrosis. These principles and standards for Industry interactions safeguard our independence, objectivity, and ability to make decisions that are in the best interest of people with CF.

Overview

The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead long, healthy lives. Engaging with biotechnology and pharmaceutical companies that are advancing CF-related research, therapies, and devices (referred to here as “Industry”) is essential to reaching our goal of a cure for all people with CF, as they develop and manufacture FDA-approved CF therapies and lead efforts to advance the next generation of treatments for CF.

We are committed to ensuring that the CF Foundation is free from undue Industry influence, and avoiding potential conflicts of interest.

We regularly evaluate our potential and ongoing interactions with Industry to ensure they comply with regulatory guidelines as well as our own internal standards for integrity and ethical behavior. In 2017, we implemented an enhanced set of principles and standards for Industry interactions to further safeguard our independence, objectivity, and ability to make decisions that are in the best interest of people with CF -- and to ensure that these interactions are transparent to the CF community and the public.

Our Principles

The CF Foundation, including our employees, officers, and trustees, are committed to the highest standards of conduct, integrity, ethical behavior, and compliance in all that we do. Our interactions with Industry are guided by the following principles to ensure consistent and clear application of these standards across our operations and to safeguard our independence:

  • Mission Focus: The needs of people with CF are at the heart of all that we do. Any interaction with Industry must further our mission and be in the best interest of people with CF and their families and caregivers.
  • Transparency: We value the trust that our community has instilled in us. Our interactions with Industry are transparent to the CF community and the public.
  • Compliance: All interactions with Industry are conducted in compliance with regulatory guidelines as well as our own internal standards for ethical behavior and integrity.

Summary of Interactions

Below is a summary of the ways we interact with Industry and for what purpose. We have extensive policies and procedures in place that govern all interactions with Industry to avoid and manage potential conflicts of interest. 

Research and Care

The CF Foundation interacts with Industry to find new therapies and a cure for CF in the following ways:

  • Provides early-stage funding and clinical and research expertise to pharmaceutical and biotech companies to stimulate and accelerate the development of CF drugs and therapies by entering into research agreements. As part of this innovative model, the CF Foundation has and may in the future receive royalties on therapies resulting from our initial support, which are used to enhance the funding of our mission.
  • Ensures effective monitoring of the safety and welfare of individuals who participate in clinical trials by reviewing and approving the clinical trial protocols of Industry researchers through the Therapeutics Development Network (TDN) Protocol Review Committee and Data Safety Monitoring Board.
  • Licenses data from the CF Foundation Patient Registry to Industry researchers to monitor drug safety as part of the U.S. Food and Drug Administration's (FDA) required Phase 4 clinical trials process and to encourage research aimed at improving the care of people with CF, while maintaining our obligation and commitment to protect the privacy of our registry participants.
  • Hosts the annual North American Cystic Fibrosis Conference (NACFC), a collaborative forum to advance research and care for the treatment of CF by engaging clinicians with the latest evidence-based updates. The conference offers educational sessions targeted to CF clinicians and other CF experts from around the world, which may include presentations by experts who are affiliated with Industry. The CF Foundation receives payments for NACFC from exhibitors and supporters, which include members of Industry. Agreements for support opportunities are executed at arm's length, and benefits are consistent across all supporters, including members of Industry. Supporters have no ability to influence the content and faculty of the educational session.
  • Solicits feedback from Research Voice, a subset of Community Voice members who support CF research initiatives. Specially-trained members of Research Voice, or “Community Insight Advisors,” consult with the CFF TDN Consulting Services Program. Community Insight Advisors have no direct interactions with pharmaceutical or other study sponsors, and all feedback is provided under a Consulting Service Agreement with Seattle Children's Hospital.
  • Receives, at the Cystic Fibrosis Foundation Therapeutics Lab, biological material from a variety of sources. Pursuant to a valid arms-length agreement, and depending on availability, the Lab may distribute biological material to any company for the benefit of cystic fibrosis research. Additionally, the Lab may execute agreements with Industry to assess Industry therapeutic agents for effectiveness in treating cystic fibrosis. Any agreement executed between the Lab and Industry is company-agnostic and part of an arms-length transaction.

The Foundation also participates in the following activities that are relevant to Industry, but free from Industry influence and do not encompass specific agreements or involve the exchange of funds:

  • Encourages widespread participation in clinical trials to further the development of CF therapies.
  • Facilitates CF Clinical Care Guidelines to ensure that people with CF continue to experience steady gains in length and quality of life, which may reference specific therapies. The guidelines, which are developed by an independent committee of subject matter experts and published in peer-reviewed journals, help our accredited care centers provide CF care based on the latest research, medical evidence, and best practices. There is no Industry involvement in the guidelines development process, and a rigorous conflict of interest policy is in place for potential guidelines committee members. 

Fundraising

To date, the CF Foundation has invested more than $3 billion in our fight against CF, resulting in groundbreaking treatments and a care model that is widely recognized as the gold standard for a complex, chronic disease. Achieving our goal of a cure for all people with CF will require continued fundraising, and we accept donations from Industry under the following circumstances:

  • Corporate sponsorship and grants from Industry for fundraising activities, such as Great Strides, CF Climb, Cycle for Life, and others.
  • Employees or directors of Industry companies may also participate in these fundraising activities as individuals.

We do not solicit or accept Industry support for advocacy and access efforts (including Compass), clinical trial development, guidelines development, educational materials, our network of accredited care centers, volunteer conferences, medical/scientific grants, or internal research.

Community Support

In keeping with our mission, we provide support for people with CF and their families to access the high-quality care they need, including FDA-approved therapies. The CF Foundation plays no role in directing people with CF to specific treatments and we do not accept funds from Industry for any of the following activities:

  • Assists people with CF in navigating insurance, financial, legal, and other issues through CF Foundation Compass. To help people with CF access needed treatments as prescribed by their care teams, Compass may connect people with CF to Industry-sponsored programs that provide copay assistance.
  • Provides grants to an independent copay assistance foundation to help individuals living with CF access vitamins and supplements. This support is provided in accordance with the copay foundation's obligations under the Anti-Kickback Statute and Office of Inspector General advisory opinion.
  • Actively advocates for policies and legislation that help advance research and drug development; works to improve access and coverage for CF drugs, therapies, and quality care; and raises awareness about the disease.

Monetization of Royalties

Like many diseases that impact a small population, CF was ignored by the pharmaceutical industry for many years, despite decades of advances in basic science driven by the CF Foundation. In response to this unmet need, the CF Foundation pioneered our venture philanthropy model. Our approach is to provide upfront research funding, as well as scientific and clinical expertise, to reduce the technical and financial risk for pharmaceutical companies developing innovative CF therapies and encourage them to invest in treatments and cures for CF. Nearly every CF drug available today was made possible because of our model.

In some cases, the CF Foundation is eligible to receive funds from the sales of approved therapies or from potential therapies that were financially supported through our venture philanthropy, which enables us to fund new research to discover and develop drugs that will help more people with CF. In cases where these funds are, or are likely to be, significant, our approach is to sell our royalty rights when possible.

The most well-known and significant of these transactions resulted from a series of therapeutic discovery and development awards to Aurora Biosciences/Vertex Pharmaceuticals Inc. over a period of 12 years beginning in 2000. This contracted research resulted in the development of ivacaftor, the first therapy to address the underlying cause of CF, as well as other important disease-modifying treatments. In this case, the CF Foundation sold its royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. to Royalty Pharma for $3.3 billion in 2014. 

The proceeds from the monetization have provided the CF Foundation a tremendous opportunity to accelerate our mission as never before and expand our investments in research, care, and patient programs. In fact, our funding of research and medical activities has more than doubled since 2014, vastly increasing our potential to have a positive impact on the CF community.

Drug Pricing

We are concerned about the current approach to pricing drugs and the impact of cost on access for people with CF. As the price of treatments for CF continues to rise, we are increasingly seeing access restricted by payers.

Drug pricing is a complex issue that will require multiple stakeholders to address. Though we do not have a role in setting or negotiating the prices of individual therapies, our goal is to represent the needs of people with CF in ongoing discussions to define and implement an approach to pricing that will allow patients to have continued access to innovative treatments and support a health care system that achieves:

  • Affordability: Treatments should be priced at a point that does not prohibit access for patients.
  • Adequacy: Pricing and coverage decisions should be rooted in clinical evidence to support patients' access to high quality therapies and care that reflect the latest research, clinical guidelines, and best practices.
  • Availability: Patients should be able to access therapies and care regardless of an individual's income, employment, health status, or geographic location.

We are bringing data to the table to help define value in clinical practice and evaluate the impact of new therapies. We welcome the opportunity to bring the perspective of the CF community to this discussion and look forward to playing a role in finding solutions that support our goal of providing all people with CF with access to a cure and helping them live longer, healthier lives.

Our Policies

The needs of people with CF are at the heart of all that we do. In addition to ensuring we comply with applicable laws and regulations, our Policy on Industry Interactions governs the way we engage with Industry to ensure our commitment to our community and our high ethical standards remain uncompromised.

Sources of Revenue

Revenue from Industry comprised approximately 2 percent of the CF Foundation's total revenue and investment income for the year ended Dec. 31, 2017. Total revenue and investment income for the CF Foundation during that period was $757.3 million.

*Industry includes biotechnology and pharmaceutical companies that are advancing CF-related research and therapies

**Other industry revenue includes revenue resulting from the other arm’s length transactions listed within the CF Foundation’s Principles and Standards for Pharmaceutical Industry Interactions

***Additional detail on 2017 sources of revenue is available in the CF Foundation’s public financial statements

****To achieve compliance with Nevada Senate Bill 539, and to provide increased transparency into our contributions from Industry for the year ended December 31, 2017, please see the following disclosure.