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Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseases

  • Susan Stein1,
  • Elizabeth Bogard2Email author,
  • Nicole Boice3,
  • Vivian Fernandez4,
  • Tessa Field5,
  • Alan Gilstrap6,
  • Susan R. Kahn7,
  • Jane Larkindale8 and
  • Toni Mathieson9
Orphanet Journal of Rare Diseases201813:18

https://doi.org/10.1186/s13023-018-0761-2

Received: 12 October 2017

Accepted: 4 January 2018

Published: 22 January 2018

Abstract

Background

Rare diseases are a global public health concern, affecting an estimated 350 million individuals. Only 5% of approximately 7000 known rare diseases have a treatment, and only about half have a patient advocacy organization. Biopharmaceutical companies face complex challenges in developing treatments for rare diseases. Patient advocacy organizations may play a major role by positively influencing research and development, clinical trials, and regulations. Thus, collaboration among patient advocacy organizations and industry is essential to bring new therapeutics to patients.

Methods

We identified an unmet need for guidelines on day-to-day decision-making by rare disease patient advocacy organizations when working with biopharmaceutical partners. We convened an Independent Expert Panel experienced in collaborations between patient advocacy organizations and biopharmaceutical companies (April 2017) to develop consensus guidelines for these relationships. The guidelines were based on an original version by the International Fibrodysplasia Ossificans Progressiva Association (IFOPA). The Expert Panel reviewed and broadened these to be applicable to all patient advocacy organizations. Comments on the draft Guidelines were provided first by Panel participants and subsequently by six independent experts from patient advocacy organizations and industry.

Results

The Panel comprised four experts from the rare disease community who lead patient advocacy organizations; three leaders who perform advocacy functions within biopharmaceutical companies; and two facilitators, both having leadership experience in rare diseases and industry. The finalized Guidelines consist of four main sections: Identification and Engagement With Companies, Patient Engagement and Patient Privacy, Financial Contributions, and Clinical Trial Communication and Support. The Guidelines address the daily considerations, choices, and consequences of patient advocacy organizations as they engage with biopharmaceutical companies, and offer recommendations for volunteer/paid leaders of the organizations on how to interact in a thoughtful, responsible, ethical way that engenders trust.

Conclusions

These Guidelines recommend best practices and standards for interactions between patient advocacy organizations and industry that will ultimately have a positive effect on the development of novel treatments. Patient advocacy organizations will be provided free access to these Guidelines to help bring clarification to day-to-day decision-making around their interactions, and for use as a living document with the potential for regular revisions and updates.

Keywords

Best practicesBiopharmaceuticalGuidelinesPartnershipPatient advocacyRare diseaseStandards

Background

Rare diseases are a global public health concern, affecting an estimated 350 million individuals. The definition of a rare disease varies internationally [1]. In the United States, a disease is considered rare if it affects fewer than 200,000 individuals at any given time; in the United Kingdom, rare disease is defined as a disease that affects fewer than 50,000 individuals. Although the number of patients with each specific rare disease is small, collectively, rare diseases affect about 30 million people in the United States and another 30 million in Europe [1].

In the United States, the Orphan Drug Act was designed to provide incentives to manufacturers to conduct research and development for rare disease therapies [2]. Although the submission and approval rates for orphan drugs in the United States have increased since the passage of the Act in 1983 [2, 3], only 5% of the approximately 7000 rare diseases identified currently have a treatment approved by the US Food and Drug Administration (FDA) [1].

Rare diseases are confronted with some of the most complicated scientific and medical challenges of today. There are numerous barriers to the development of new and meaningful treatments, including unanswered scientific questions; a lack of data on disease epidemiology, pathophysiology, and natural history; a lack of clearly defined biomarkers to measure disease activity; and difficulty in identifying clinical trial endpoints and measurable and validated disease outcomes [1, 4, 5]. The technical challenges are compounded by limited research funding for rare diseases compared with that provided for more common diseases, and by difficulty in finding and recruiting patients to participate in clinical studies [3]. Finally, patients with rare diseases and academic experts are often geographically dispersed, making it more difficult for drug developers to locate and engage with them. Expertise in the care and management of a particular rare disease is often concentrated in a small number of academic centers that may not be experienced in clinical research or development, adding to the time and costs of starting and implementing clinical trials. Patient advocacy associations are a key interface between patients and providers.

Solving the challenges posed by rare diseases requires the collaboration of multiple stakeholders: biopharmaceutical companies, academic researchers, clinicians, patient advocacy organizations, patients, and regulators. Yet collaboration, while essential, is often challenging. The highest personal, professional, and business stakes are at play; considerable ethical and legal issues exist; incentives can be misaligned; and the environment is both incredibly complex and rapidly evolving.

Patient organizations are playing an increasingly important role in addressing and overcoming the challenges of drug development. About half of all known rare diseases are represented by a disease-specific patient advocacy organization [1]. This number is growing. Although the history of patient advocacy organizations lies in grass roots efforts and family fundraising events, the sophistication of the patient community has increased to match its ambition to find effective therapeutics. Many patient advocacy organizations today are led by professionals, are meaningful and empowered stakeholders in the development of treatments, and have a large impact on research [57].

Indeed, today, one of the most important factors in the development of new therapies is the positive influence of patient advocacy organizations on research and development, clinical trials, and governmental regulations [7].

Patient advocacy organizations perform many functions that enable drug development and access, including the following [57]:
  • Educating patients, physicians, and the community about a disease and innovations in its management and treatment

  • Pushing the pace of research by championing and directly funding efforts to increase the understanding of the cause of a disease and to develop new therapies

  • Forming connections between disease experts and drug developers

  • Providing drug developers with relevant insights into the patient community to enable the development of therapies that best meet the community’s needs

  • Advocating for and influencing changes in regulations to expedite research

  • Facilitating and/or sponsoring patient registries and natural history studies to aid in the development of treatments

  • Helping to ensure that patients have access to treatments

With the evolution of patient advocacy organizations and the increasing activity of biopharmaceutical companies in the development of novel therapies for rare diseases, collaborations between patient advocacy organizations and biopharmaceutical companies have become more common. However, many organizations face uncertainty in addressing the complex and important day-to-day issues that arise within the context of these collaborations.

Although guidance documents exist, most either are directed at the biopharmaceutical industry, are specific to the European healthcare system [8, 9], or are too general to serve as a roadmap for day-to-day decision-making for patient advocacy organizations. Given the important and dynamic nature of their work and the complexity of collaboration in the development of treatments for rare diseases, many patient advocacy organizations are seeking clarity and guidance concerning approaches to industry collaboration.

Guidelines are needed now more than ever, especially with healthcare regulatory bodies increasingly restructuring themselves to put the needs of patients first. The European Medicines Agency (EMA) established the Patients’ and Consumers’ Working Party (PCWP) in 2006 to provide a platform for information exchange between patients and the Agency. The US FDA enacted the US twenty-first Century Cures Act in December 2016 [10]. The Act seeks to make improvements throughout the entire research and development system, from discovery, to development, to delivery of new medical products [10]. The policy reforms in the Act are meant to strengthen patient centricity in the areas of biomedical product development and regulatory approval and to catalyze innovation in clinical trials and regulatory approval. Such innovations would include promoting FDA qualification of biomarkers and other drug development tools and the study of how best to use innovative clinical trial designs and the real-world evidence that is generated during product development and regulatory approval [10, 11]. In 2016, the EMA and the FDA established a new ‘cluster’ on patient engagement. This cluster is intended to provide a forum to share experiences and best practices on the way the two agencies involve patients in development, evaluation, and post-authorization activities related to medicines [12].

The great need for guidelines was most recently emphasized by the publication of an article in March 2017 in The New England Journal of Medicine addressing conflicts of interest that may arise when patient advocacy organizations interact with industry [13].

In order to address the need for guidelines, we convened an Independent Expert Panel of leaders from patient advocacy organizations and the biopharmaceutical industry to develop guidelines on effective collaborations between these two stakeholders. This report presents the objectives and guiding principles developed by the Independent Expert Panel, the process used to develop the guidelines, a summary of points raised during the Panel discussion, and the finalized guidelines.

Objectives

The objectives of the Independent Expert Panel meeting, titled “Principles for Interactions With Pharmaceutical Companies: Guidelines for Patient Organizations,” were as follows:
  • To create guidelines directed at day-to-day decision-making for rare disease patient advocacy organizations for use in working with industry partners

  • To capture comments and suggestions from participants for use in developing a white paper on the principles of interactions with industry to support the guidelines

  • To determine next steps regarding finalization and dissemination of the manuscript and the guidelines

Methods

Nine experts chosen for their expertise and availability participated in the Independent Expert Panel: four leaders of patient advocacy organizations and three leaders in the pharmaceutical industry who interact with patient advocacy organizations. The Panel also included two facilitators, one with experience in such collaborations at both a patient advocacy organization and a biopharmaceutical company (EB) and one from a medical communications company specializing in rare diseases (SS).

The Independent Expert Panel meeting utilized a roundtable discussion format. The meeting was held via a 2-h web conference, ensuring that each participant was given the opportunity to provide input into the guidelines. The agenda included background and introductions, questions for discussion, a session on other comments and suggestions, and next steps.

Prior to the meeting, the Panel was provided with and asked to review the known guidance documents available on this topic (Table 1) [1317]. The Panel was also provided with a set of guidelines previously developed by the International Fibrodysplasia Ossificans Progressiva Association (IFOPA), a nonprofit patient advocacy organization for this rare disease founded in 19889 (see www.ifopa.org), titled “The International Fibrodysplasia Ossificans Progressiva Association’s (IFOPA) Guidelines for Engagement with Pharmaceutical Companies” [18].
Table 1

Background materials for the Independent Expert Panel meeting

Existing Guidance Documents

 

 • Pharmaceutical Manufacturers of America (PhRMA) (United States): “PhRMA Principles on Interactions with Patient Organizations” [14].

 

 • Clinical Trials Transformation Initiative CTTI Recommendations (United States): “Effective Engagement with Patient Groups Around Clinical Trials” [15].

 

 • European Federation of Pharmaceutical Industries and Associations (Europe): “EFPIA Code of Practice on Relationships Between Pharmaceutical Companies and Patient Organisations” [16].

 

 • BioPontis Alliance for Rare Diseases (United States and Europe): “Integrating Rare Disease Patients into Pre-Clinical Therapy Development; Finding Our Way with Patient Input” [17].

 

Published Literature

 

 • McCoy MS, Carnoil M, Chockley K, Urwin JW, Emanuel EJ, Schmidt H: Conflicts of interest for patient-advocacy organizations. N Engl J Med 2017;376:880–5.

 
The IFOPA guidance document provided a foundation from which the Panel was asked to build a set of guidelines for use by the entire rare disease community. The IFOPA document was chosen because it reflects many of the broader principles described by the known guidance documents listed in Table 1. The facilitators discussed and raised key questions for each of the five sections of the IFOPA guidance document (Table 2) [18]:
  1. 1.

    Identification and Engagement of Companies

     
  2. 2.

    Patient Engagement

     
  3. 3.

    Financial Contributions

     
  4. 4.

    Clinical Trial Communications

     
  5. 5.

    Patient Privacy

     
Table 2

Discussion questions posed to the participants of the Independent Expert Panel meetinga

1. Identification and engagement of companies

 

 1.1. Are there any conditions under which a patient advocacy organization should not be in contact with a pharmaceutical company?

 

 1.2. Are there criteria a patient advocacy organization should use to decide whether or which companies to contact?

 

 1.3. What information should a patient advocacy organization expect a company to share?

 

 1.4. What information should a company expect a patient advocacy organization to share?

 

2. Patient engagement

 

 2.1. Should a patient advocacy organization be involved in direct interaction between a patient and a pharmaceutical company? What does “involvement” in this case mean?

 

 2.2. Are there circumstances under which a patient advocacy organization should not be involved in direct dialogue between patients and companies?

 

 2.3. What are the reasons behind this recommendation?

 

 2.4. For formal disease insight, is an advisory board preferred over individual input? Are there circumstances under which individual input is preferred or acceptable?

 

3. Financial contributions

 

 3.1. Should patient groups accept financial contributions from pharmaceutical companies? If so, under what circumstances?

 

 3.2. By what mechanisms should a patient group receive contributions? What processes should be in place for receiving and reporting the contribution?

 

 3.3. Should patient group leaders accept honoraria for speaking on behalf of their organization?

 

 3.4. Are there circumstances under which a patient advocacy organization could/should operate as a paid service provider to a pharmaceutical company?

 

4. Clinical trial communications

 

 4.1. What is the role of a patient advocacy organization in “supporting” clinical trials?

 

 4.2. Should a patient advocacy organization have criteria for which clinical trials it will “support”?

 

 4.3. What role, if any, should a patient advocacy organization have in guiding or advising conversations about clinical trial participation on social media?

 

 4.4. Should leaders of patient advocacy organizations (board members and other volunteer leaders) follow organizational practices when writing or speaking in personal blogs or other social media as a parent, family member, or affected individual?

 

5. Patient privacy

 

 5.1. What role does a patient advocacy organization have in ensuring that pharmaceutical companies adequately protect patient privacy in surveys, advisory boards, or other community engagement?

 

a The questions listed herein apply to sections 1 through 5 of the Guidelines for Engagement with Pharmaceutical Companies by The International Fibrodysplasia Ossificans Progressiva Association (IFOPA) [18]

During the meeting, the Panel addressed the key questions (Table 2) and provided comments and revisions to the IFOPA document. Based on input from the Panel, the authors developed a first draft guidance document.

Results

The Independent Expert Panel reviewed and revised this first draft. A second draft was also reviewed by six independent experts from patient advocacy organizations and the biopharmaceutical industry. The Guidelines were finalized in October 2017.

The Guidelines produced by the Independent Expert Panel, titled “Guidelines for Interactions Between Patient Advocacy Organizations and Biopharmaceutical Companies,” are provided in the Appendix to this article. The Panel adapted the original five sections of the IFOPA guidelines into four sections:
  1. 1.

    Identification and Engagement With Companies

     
  2. 2.

    Patient Engagement and Patient Privacy

     
  3. 3.

    Financial Contributions

     
  4. 4.

    Clinical Trial Communication and Support

     

The Guidelines address the day-to-day considerations, choices, and consequences for patient advocacy organizations in their engagement with biopharmaceutical companies. The Panel agreed that all interactions between the patient advocacy organization, industry, and the disease community should be transparent; should enable trust, accountability, and shared learning; and ultimately should work most efficiently and effectively toward advancing meaningful treatments for patients. The Guidelines strive to achieve these objectives while recognizing that every relationship is unique and that there are a variety of ways to partner successfully.

The recommendations contained within the Guidelines are relevant for any paid or volunteer leader of a patient advocacy organization, including staff, board members, and committee members. The Guidelines are intended to be a living resource that can be revised and expanded as the environment evolves.

Participants in the Expert Panel stressed the importance of having the Guidelines available and published in the literature. The Panel also provided the following overarching comments:
  • Patients and industry cannot “go it alone.” Both need patient advocacy organizations.

  • Patient advocacy organizations may not realize their value and how important they are to industry.

  • Successful collaborations between patient advocacy organizations and biopharmaceutical companies are achieved when they have reciprocal relationships in which both parties recognize the value of the other.

  • Mutual respect is essential, which requires honesty and authenticity. Transparency and commitment from both parties should begin on day one.

The Independent Expert Panel provided Probing Questions for possible use in conjunction with the Guidelines (Table 3). The Panel also recommended that patient advocacy organizations develop educational resources for patients (Table 4).
Table 3

Probing questions from the Independent Expert Panel meeting, for possible use with the Guidelines

Identification and Engagement With Companies

 

 • What changes occur when more than one patient advocacy organization is involved?

 

 • What changes occur when more than one industry partner is active?

 

 • How can the challenges of having only one biopharmaceutical company in the particular rare disease space be addressed?

 

Patient Engagement and Patient Privacy

 

 • What does a great patient advocacy organization look like?

 

 • What are the ways that individual patient advocacy organizations can facilitate bringing back information to the disease community?

 

 • What are some reasons that patient advocacy organizations should be included in conversations with industry?

 

 • How should patient advocacy organizations handle various aspects of confidential information as an organization?

 

Financial Contributions

 

 • Should there be a cap on the total percentage of funding from pharmaceutical companies? Should the cap depend on the size of the group?

 

 • How should public disclosures of pharmaceutical funding be handled?

 

 • Can industry employees serve on the boards of patient advocacy organizations?

 

Clinical Trial Communication and Support

 

 • How should patient advocacy organizations respond to misinformation on the Internet and in social media?

 

 • What changes occur once a drug is commercially available?

 

 • How should negative results of clinical trials be handled? How should that information be disseminated to the patient community?

 

 • How should patient advocacy organizations handle issues surrounding compassionate use?

 

 • Should patient advocacy organizations attempt to educate academic researchers on how to communicate with patients?

 

 • Should patient advocacy organizations provide education and resources to patients about the informed consent process?

 
Table 4

Education and resources that patient advocacy organizations can provide and share

 1. Provide training on these Guidelines.

 

 2. Develop case studies of how patient advocacy organizations have worked with the biopharmaceutical industry in the past.

 

 3. Trade patient-friendly toolkits with other patient advocacy organizations.

 

 4. Provide education and guidance to patients concerning:

 

   Clinical trial participation; informed consent and how to make a decision about participating in a clinical trial

 

   The use of social media around clinical trials

 

   How patients should interact with industry regarding participation in advisory groups

 

 5. Provide education for academic researchers about how to communicate information about clinical trials with patients.

 

Discussion

Collaborations between patient advocacy organizations and the biopharmaceutical industry, which have become increasingly more common, are needed now more than ever to overcome the challenges of developing treatments for rare diseases. The increasing focus on patient engagement by healthcare regulators highlights the need for guidelines to facilitate this collaboration. Although collaboration is essential, it is also poses challenges. The Independent Expert Panel developed Guidelines that are intended to help address these challenges. The Guidelines presented here are directed at day-to-day decision-making for rare disease patient advocacy organizations for use in working with industry partners. Comments and suggestions from the members of the Independent Expert Panel were included in the Guidelines, and the Expert Panel reviewed and finalized the Guidelines. Additional comments on the draft Guidelines were provided by independent experts from patient advocacy groups and the biopharmaceutical industry.

Various limitations to the development of these Guidelines exist: the complexity of collaborations between patient advocacy groups and the biopharmaceutical industry, the dynamic nature of the work of patient advocacy organizations in the development of therapies for rare diseases, and various regional differences such as those between the United States and Europe related to interactions between these types of groups. However, these Guidelines are intended to be freely available for use as a living document, with the potential for regular revisions and updates. Future versions of the guidelines should explore regional differences and needs in how patient organizations interact with pharmaceutical companies, particularly between the United States and Europe.

Conclusions

The Guidelines described here are intended to guide collaborations between patient advocacy organizations and the biopharmaceutical industry in an ethical and transparent way for the benefit of patients who desperately need novel and meaningful therapeutics. The Guidelines recommend best practices and standards for interactions between patient advocacy organizations and the biopharmaceutical industry that will ultimately have a positive effect on the development of novel treatments.

Abbreviations

CTTI: 

Clinical Trials Transformation Initiative

EFPIA: 

European Federation of Pharmaceutical Industries and Associations (Europe)

EMA: 

European Medicines Agency

FDA: 

Food and Drug Administration

IFOPA: 

International Fibrodysplasia Ossificans Progressiva Association

PCWP: 

Patients’ and Consumers’ Working Party

PhRMA: 

Pharmaceutical Manufacturers of America

Declarations

Acknowledgements

The authors thank the following independent external reviewers for their time and comments related to the Guidelines: Jean F. Campbell, Principal, JF Campbell Consultants LLC, and Founding Board Member, Professional Patient Advocates in Life Sciences (PPALS); Jill Dolgin, PharmD, Head of Patient Advocacy, Applied Genetic Technologies Corporation; Sierra Kulas, Patient Advocacy Consultant, AveXis; Chad A. Morin, Editorial Board Member, Life Science Compliance Update; and Carrie Ostrea, Little Miss Hannah Foundation.

Funding

Not applicable.

Availability of data and materials

Data sharing not applicable to this article as no datasets were generated or analysed during the current study.

Authors’ contributions

EB made major contributions to the writing of the manuscript. SS, EB, NB, VF, TF, AG, SRK, JL, and TM comprised the Expert Panel that developed the Guidelines. All authors read and approved the final manuscript.

Ethics approval and consent to participate

Not applicable.

Consent for publication

Not applicable.

Competing interests

SS is President and Chief Executive Officer of Connexion Healthcare, a medical communications company and a Board Member of Global Genes.

EB is Chair of the Research Committee of the International Fibrodysplasia Ossificans Progressiva Association (IFOPA).

NB is Founder and Chief Executive Officer of Global Genes.

VF is Director of Patient Advocacy at REGENXBIO Inc.

TF is Patient Advocacy Lead at Spark Therapeutics.

AG is Executive Director of Advocacy and Policy at Akcea Therapeutics.

SRK is Executive Director of the National Tay-Sachs & Allied Diseases Associations Inc.

JL is Vice President of Research Development at the Friedreich’s Ataxia Research Alliance and Executive Director of the Duchenne Regulatory Science Consortium at the Critical Path Institute.

TM is Chief Executive, Niemann-Pick UK (NPUK); UK Representative, International Niemann-Pick Disease Alliance (INPDA); NPUK Representative, UK LSD Collaborative; and Patient/Public Voice (PPV) Member, NHS England Clinical Reference Group for Inherited Metabolic Disease; and Member of the UK LSD Expert Advisory Group.

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Authors’ Affiliations

(1)
Connexion Healthcare, Newtown, USA
(2)
International Fibrodysplasia Ossificans Progressiva Association (IFOPA), North Kansas City, USA
(3)
Global Genes, Aliso Viejo, USA
(4)
REGENXBIO Inc, Rockville, USA
(5)
Spark Therapeutics, Waltham, USA
(6)
Akcea Therapeutics, Cambridge, USA
(7)
National Tay-Sachs & Allied Diseases Associations Inc, Brighton, USA
(8)
Friedreich’s Ataxia Research Alliance, Tucson, USA
(9)
Niemann-Pick UK, Tyne and Wear, UK

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Copyright

© The Author(s). 2018

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