Only 5% of rare diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, forming connections between experts and drug developers, advocating for changes to regulation to expedite research, facilitating patient registries, or other initiatives.
A rare disease, by definition, affects only a small number of individuals, but together, all of the approximately 7000 known rare diseases affect an estimated 350 million patients worldwide (including approximately 30 million people in the United States). Only 5% of these diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, forming connections between experts and drug developers, advocating for changes to regulation to expedite research, facilitating patient registries, or other initiatives.
A newly published paper in Orphanet Journal of Rare Diseases describes efforts to address unmet needs for guidelines on day-to-day decision making by rare disease patient advocacy organizations when working with biopharmaceutical companies. The researchers convened an independent expert panel, comprising 9 stakeholders from patient advocacy organizations and the biopharmaceutical industry (and 2 facilitators) who were experienced in such collaborations. The researchers had 3 objectives for this panel meeting: creating guidelines for day-to-day interactions between advocacy organizations to use in working with industry, to capture comments and suggestions for use in developing a white paper on interacting with industry, and determining next steps to finalize and share the manuscript and guidelines.
The panel was provided with a guidance document used by the Fibrodysplasia Ossificans Progressiva Association (IFOPA), which provided a foundation upon which the panel was asked to build guidelines for the entire rare disease community. Based on the panel’s input, the researchers drafted guidelines, then revised on the basis of further panel comments. A second draft was reviewed by 6 experts from advocacy organizations and industry before being finalized.
The resulting document, titled “Guidelines for Interactions Between Patient Advocacy Organizations and Biopharmaceutical Companies,” comprises 4 sections, highlights of which include the following:
Identification and engagement with companies. The patient organization should seek contact with pharmaceutical companies (which are conducting ethical and high-quality research), and seek insight into objectives and plans of the biopharmaceutical company. In exchange, the patient organization should provide insight appropriate to inform development efforts. The patient group should discuss its goals and expectations with the drug developer, and no member of the pharmaceutical company should sit on the board of the patient group.
Patient engagement and patient privacy. Any engagement between the biopharmaceutical company and the advocacy organization should be conducted in advance of research and have a clear objective. Direct contact between the biopharmaceutical company and patients should be conducted with the awareness of the patient organization, and are best conducted in group settings rather than individual conversations. All learnings and outcomes should be shared openly for mutual benefit, and patient information should be protected.
Financial contributions. The patient organization should maintain good records of requests for financial support from the pharmaceutical company, accept those financial contributions that allow the group to maintain autonomy, accept only named (not anonymous) contributions, and seek donations in a fair and transparent way. Industry should not accept honoraria to speak on behalf of the patient organization in question.
Clinical trial communication and support. The patient organization should serve as a conduit for information by educating patients, but should not seek to influence patients’ choices of whether to participate in trials. The organization should provide accurate and fair information about trials without adding commentary that could influence opinions, and should provide the pharmaceutical company with information on any community-wide observations about needs or barriers to participation. Board and committee members must represent the organization in their conduct (including conduct on social media). The organization should seek a summary of trial results for the patient community in timely way.
The researchers say that they hope these guidelines can help steer ethical, transparent collaboration between patient groups and industry for the greater good of patients with rare diseases.