Introduction
The patient advocacy organization seeks the highest level of ethical conduct in engagement with biopharmaceutical companies. The goal of engaging with biopharmaceutical companies is to help enable the development of therapies while maintaining autonomy as a patient advocacy organization. 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.
There are four main areas of engagement between the patient advocacy organization and biopharmaceutical companies described in the following Guidelines:
1.
Identification and Engagement With Companies
2.
Patient Engagement and Patient Privacy
4.
Clinical Trial Communication and Support
1. Identification and Engagement With Companies
The patient advocacy organization desires mutually beneficial dialogue and information exchange with biopharmaceutical companies developing potential therapies for the rare disease of interest. Dialogue is mutually beneficial when it is aligned with and advances the mission of the patient advocacy organization as well as the biopharmaceutical company.
1.1. The patient advocacy organization proactively seeks contact with biopharmaceutical companies that show interest or activity in drug discovery, preclinical research, or clinical research in the rare disease of interest. The patient advocacy organization may also contact companies that are not yet working on the rare disease of interest but have a relevant technology.
1.2. The patient advocacy organization seeks insights into the objectives and plans of the biopharmaceutical company and the potential therapy being evaluated, as appropriate. In exchange, the patient advocacy organization provides the biopharmaceutical company with community-wide insight and perspective as needed and appropriate to inform the development efforts and strategic decisions of the company.
1.3. The patient advocacy organization collaborates with biopharmaceutical companies that are conducting ethical, high-quality research in a responsible manner according to industry, national, and international regulatory standards. Collaboration can include a wide range of activities such as information exchange, access to disease experts, access to tools and infrastructure (e.g., natural history data and biologic samples), and exchange of resources.
1.4. The patient advocacy organization strives to collaborate with multiple biopharmaceutical companies to ensure the sustainability of its initiatives and to allow for a diversity of views and therapeutic approaches.
1.5. The patient advocacy organization discusses goals and expectations of the collaboration at the outset to ensure they are mutual. The patient advocacy organization reserves the right to disengage with a biopharmaceutical company if the goals of the two organizations are not aligned.
1.6. In order to avoid conflicts of interest, the patient advocacy organization does not allow representatives of biopharmaceutical companies actively developing or selling therapies for the disease to sit on the board of directors of the patient advocacy organization.
2. Patient Engagement and Patient Privacy
The patient advocacy organization encourages and enables direct dialogue and information exchange between patients and biopharmaceutical companies developing potential therapies for the rare disease of interest. The voice of the patient is crucial throughout drug development by promoting disease awareness and sharing patient/caregiver perspectives. The patient advocacy organization ensures the privacy of health data provided to the organization by its membership.
2.1. Any engagement between a biopharmaceutical company and a patient advocacy organization should be done to advance understanding of the disease or research efforts and should have a clearly stated purpose or set of objectives.
2.2. Direct interactions between specific patients and biopharmaceutical companies are best arranged with the involvement or general awareness of the patient advocacy organization. The patient advocacy organization can choose from a range of approaches regarding these interactions, from actively facilitating such dialogues to passively providing training and education for patient community members on best practices.
Including the patient advocacy organization in these dialogues accomplishes the following:
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Ensures fairness and transparency within the patient community. Information provided by the biopharmaceutical company to one patient is shared with all patients who have a right to that information, and patients outside of the conversation have an equal opportunity to express their opinions to the biopharmaceutical company.
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Ensures that the patient community is adequately and well represented to the biopharmaceutical company. The voices of individual patients must be considered in the context of the community as a whole; one patient’s experience may not reflect the experiences of other patients.
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Allows for access to professional advisers, such as financial experts and attorneys, who may advise the organization, inform the dialogue, and help individual patients avoid financial and legal risks.
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Helps to avoid misunderstandings in conversations between individual patients and biopharmaceutical companies.
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Allows the organization to advise patients on the protection of their health and personal privacy in any data collection activities.
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Allows the patient advocacy organization to better understand the needs and intentions of both the patient and the biopharmaceutical company in order to best move the field forward for the patient community as a whole.
2.3. The patient advocacy organization encourages biopharmaceutical companies to obtain disease insights from group discussion rather than from one-on-one conversation with single individuals. One best practice is the formation of advisory boards composed of at least three patients. An advisory board format helps to ensure that community views are adequately represented and that work is not unduly requested of any one individual. The patient advocacy organization may offer guidelines and training to patients and families on best practices for effective interactions with biopharmaceutical companies as part of an advisory board.
2.4. The patient advocacy organization expects that learnings and outcomes from all interactions will be shared openly between both parties for mutual benefit. At the outset of these collaborations, the patient advocacy organization may offer the companies guidelines or expectations for how learnings and outcomes can best be shared with their particular community.
2.5. Leaders of the patient advocacy organization (i.e., staff, board members, and committee members) or individuals representing the disease community may be invited by biopharmaceutical companies to speak at internal company meetings, public events hosted by the company, or meetings with regulatory agencies. The patient advocacy organization evaluates each invitation and accepts invitations that promote disease education or awareness and elevate the voice of the patient in a manner that is consistent with the points outlined in these Guidelines.
2.6. The patient advocacy organization takes proper steps to protect all personal and confidential patient information both within the organization and when shared with outside entities, in accordance with applicable laws and regulations. The patient advocacy organization undertakes the following:
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Assists individual patients in thinking through their decisions about providing information or consent.
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Helps patients convey their expectations about privacy.
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Ensures that biopharmaceutical companies, and other organizations, have in place at least basic guidelines or a policy for ensuring patient privacy prior to any data collection, including surveys, photographs, video and audio recordings, slide decks, and consent forms.
2.7. The patient advocacy organization advises patients and industry that personal health information of patients must not be recorded by the biopharmaceutical company without proper and prior informed consent from the patient.
2.8. The patient advocacy organization advises patients and industry on the value of sharing data with the research community for future research needs, and encourages use of consent documents that allow for secondary research on data as permitted by patients. Patient advocacy groups may encourage sharing of data on completion of studies.
3. Financial Contributions
A robust patient advocacy organization is a vital partner to biopharmaceutical companies in the development of potential therapies. Financial resources are a key need for the growth and maintenance of the patient advocacy organization. Demands on the organization are increased by drug development activities, particularly during the clinical and commercial stages. The following principles guide the patient advocacy organization in the receipt of biopharmaceutical company donations:
3.1. The patient advocacy organization requires and maintains proper documentation of all requests for financial support from a biopharmaceutical company. All requests are documented on the letterhead of the organization and clearly state the mission and activities of the advocacy organization and reasons for the request.
3.2. The patient advocacy organization accepts financial contributions that support its stated mission and allow the organization to maintain its autonomy. The patient advocacy organization assesses the alignment of mission between the two organizations as part of the funding discussion.
3.3. The patient advocacy organization does not accept financial support from biopharmaceutical companies for product promotional activities. The patient advocacy organization avoids taking payment from a biopharmaceutical company that could be perceived as buying special privileges, such as the opportunity to promote a therapy to a patient audience, to direct a meeting agenda, to guide content of educational materials, to promote participation in a specific clinical trial, to influence the outcome of a specific research program, or to provide exclusive support of a particular research program.
3.4. It is ideal that any financial contribution to the patient advocacy organization be made either as (1) unrestricted funding or (2) sponsorship of a specific activity initiated by the patient advocacy organization to support its stated mission.
3.5. All donations must be given in a named manner (i.e., not given anonymously). The patient advocacy organization is transparent and open about its funding sources. Any funding provided by a biopharmaceutical company is disclosed by the patient organization (e.g., “project supported by…”).
3.6. The patient advocacy organization seeks donations in a fair and transparent manner among multiple partners to avoid real or perceived exclusive relationships and to maintain autonomy. Relying on a single partner and/or industry can compromise sustainability and autonomy; therefore, the organization attempts to receive donations from more than one partner and/or industry whenever possible.
3.7. The patient advocacy organization establishes metrics to evaluate the effectiveness of an activity or initiative in which it has collaborated with a biopharmaceutical company and regularly communicates back to the company results of the specific project or use of funds.
3.8. The patient advocacy organization may provide consultation to a biopharmaceutical company if the consultation is consistent with the mission of the organization and allows it to maintain autonomy. Terms of these services will be documented by mutual agreement between the patient organization and the biopharmaceutical company. The leaders (i.e., staff, board members, committee members) of a patient advocacy organization will not operate as independent consultants to a biopharmaceutical company outside of their roles within the patient organization.
3.9. The leaders of the patient advocacy organization will not accept personal honoraria to speak on behalf of the organization but, alternatively, may have the honoraria given to the organization.
3.10. Travel expenses incurred to participate in advisory board meetings or disease awareness activities may be reimbursed directly to the individual patient or to the patient advocacy organization.
3.11. Any transfers of value or benefits provided to the patient advocacy organization by a biopharmaceutical company should be documented by a signed agreement between the two organizations.
4. Clinical Trial Communication and Support
As a representative of the patient community, the patient advocacy organization is committed to providing education and resources about clinical trials to its members. The organization informs the patient community about open and upcoming clinical trials. The organization also educates patients about their vital role during the clinical trial process, from design to conclusion. Overall community participation in clinical trials is essential to advance the science and understanding of the disease.
4.1. The patient advocacy organization acts as a conduit for information about clinical trials by providing education and resources to the patient community.
4.2. The choice to participate in any particular trial is an individual one; the patient advocacy organization does not seek to influence that choice, but rather, assists patients and families in making informed decisions through education and awareness.
4.3. The patient advocacy organization disseminates accurate and fair-balanced information about clinical trials without adding commentary or opinion that may influence an individual’s decision in any way.
4.4. To support optimal clinical trial design and communication, the patient advocacy organization may provide the biopharmaceutical company with community-wide observations, needs, and barriers to participation.
4.5. The patient advocacy organization shall develop and communicate a position on their role in the sharing of individual clinical trial experiences in social media. Disclosing clinical trial experiences in social media can compromise the validity and conduct of a clinical trial and has implications for individual health privacy. However, ultimately, the choice to share information is personal; thus, a patient organization cannot dictate what information clinical trial participants do or do not share in public forums. The patient organization may provide the community with educational materials on the potential implications, both positive and negative, of disclosing clinical trial experiences publicly.
4.6. Board and committee members of the patient advocacy organization have a responsibility to represent the patient organization in their conduct. Information about clinical trials that is accessible to the community through social media, including in personal blogs or other forms of communication, should adhere to the principles outlined in these Guidelines.
4.7. At the end of a clinical trial, the patient advocacy organization asks the biopharmaceutical company to provide a summary of available trial results for trial participants and the patient community in a timely fashion. The patient advocacy organization requests that the company inform patients, in a way that is easily understandable and offers the option to seek clarification, about the ways in which the patients’ participation has resulted in a valuable contribution to the knowledge base or to the development of a therapy.