Principles and Standards for Pharmaceutical Industry Interactions

Overview

The mission of the Cystic Fibrosis Foundation (CF Foundation) is to cure cystic fibrosis and to provide all people with cystic fibrosis the opportunity to lead long, fulfilling lives by funding research and drug development, partnering with the CF community, and advancing high-quality, specialized care. Engaging with biotechnology and pharmaceutical companies and other healthcare professionals 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. Employees of the CF Foundation are prohibited from holding employment, consulting, or any other type of remuneration arrangement with a member of Industry.

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, clinical and research expertise, and rights to use intellectual property to pharmaceutical and biotech companies to stimulate and accelerate the development of CF drugs and therapies by entering into research agreements and other agreements. As part of this innovative model, the CF Foundation has and may in the future receive a financial return on therapies resulting from our support, which are used to enhance the funding of our mission.
  • Helps ensure effective monitoring of the safety and welfare of individuals who participate in clinical trials through the Therapeutics Development Network (TDN) Protocol Review Committee and Data Safety Monitoring Board.
  • Licenses data to 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. In connection with such licenses and upon request, assists Industry researchers in interpreting Registry data to aid in designing, analyzing, and interpreting real world studies in CF.
  • 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 Community Voice members on behalf of Industry, regarding topics including but not limited to clinical trial protocol design, through direct communications, focus groups, surveys, or other feedback methods. Interactions with Community Voice members are facilitated by the CF Foundation Community Partnerships staff, unless Industry requests continuous and more detailed insights from a Community Voice member, in which case the CF Foundation will no longer be involved. This service is provided to Industry at no cost and will only be done if the CF Foundation believes that such work is in the best interest of the CF community. All information generated in this category will be available to all Industry companies in a company-agnostic manner.
  • 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.
  • Conducts, at the Cystic Fibrosis Foundation Therapeutics Lab, testing of Industry therapeutic agents, both alone and in blinded testing against other Industry therapeutic agents to evaluate effectiveness. This testing and, when applicable, possible subsequent unblinding of results, upon Industry consent, is company-agnostic, part of an arms-length transaction, and available to any Industry company which has an applicable therapeutic agent.

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 with Industry:

  • 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 the CF Foundation's programs or initiatives in cases where we determine that such support might be perceived to inappropriately influence the programs or initiatives (e.g., advocacy and access efforts such as Compass; clinical trial development; guidelines development; educational materials; our network of accredited care centers; volunteer conferences; or medical/scientific grants).

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.
Venture Philanthropy

The CF Foundation is committed to bringing the best scientific minds and technology into our mission to cure CF and help all people with the disease live long, fulfilling lives. More than 20 years ago, the organization pioneered a model for collaborative drug discovery and development that is now known as venture philanthropy. 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. In some cases, the CF Foundation may also receive the right to milestone-based payments, equity interests, royalties on the net sales of therapies, and/or other forms of consideration in return for our CF research funding. This provides a potential financial return which helps advance our mission, including funding new research to discover and develop drugs that will help more people with CF. Nearly every CF drug available today was made possible because of our model.

The most significant of those investments was research funding to Aurora Biosciences (now Vertex Pharmaceuticals, Inc.) to identify and develop treatments for the underlying cause of CF. Those investments resulted in multiple transformative therapies to treat CF and the sale of royalty rights related to those medicines to Royalty Pharma for $3.3 billion in 2014. Under terms of the agreement with Royalty Pharma, the CF Foundation maintained a stake in future royalties. On October 30, 2020, we sold our remaining stake in royalties from Vertex CFTR modulators to Royalty Pharma for an upfront payment of $575 million. Under the terms of the agreement, the CF Foundation may also receive a potential future payment of $75 million from Royalty Pharma; that payment is not tied to product sales.

Since the sale of royalty rights in 2014, we have supercharged our mission, investing in CF research, care, and community programs at an unprecedented rate. We are investing in virtually every aspect of CF including a $500 million Path to a Cure initiative to accelerate progress towards treatments for everyone with CF; helping to address challenges that people with CF continue to face today with a $100 million Infection Research Initiative, a $40 million Lung Transplant Initiative, and a significant focus on complications. Following the sale, we also established a Community Partnerships function to provide educational and support programming, as well as listening efforts and mentoring, particularly for the growing adult population; and broadened community support through our Compass assistance program, which fields an average of 11,000 calls from CF patients and families every year.

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

For the year ending December 31, 2020 and excluding the one-time royalty sale to Royalty Pharma, total revenue and investment income for the CF Foundation was $667.8 and revenue from Industry comprised approximately 5 percent of the CF Foundation's total revenue and investment income. Find a full list of Industry contributions in 2020 here.

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