Recent years have seen increased efforts to engage with patient and participant communities and include them more closely in research. This approach is reflected in the founding of the Patient Centered Outcomes Research Institute (PCORI) as well as statements from the US Institute of Medicine and Food and Drug Administration (FDA) on the importance of including participant perspectives at every stage of the research process—from the design of studies through their implementation and the dissemination of research results.

While these efforts promise to promote public trust and increase research value, they also raise novel regulatory and ethical questions that could impact IRB review. At what point does engaging patients in study design and implementation require IRB review? Are there any special obligations or risks for sponsors that might arise when working closely with patient advocacy groups? What role, if any, should the IRB play in scrutinizing conflicts of interest that may arise for patient partners?

Engagement vs Recruitment

With patients becoming more closely involved in different and new aspects of research, such as study design, it may not always be clear when IRB oversight is required. Patients engaged in the research process fall under IRB purview when (and only when) they qualify as research participants under the US regulations. It may be helpful to distinguish participant “recruitment” from “engagement” in this regard.

Recruitment is any effort to enroll individuals in particular studies, such as through the use of advertisements and screening instruments. Recruitment efforts constitute the beginning of informed consent and require IRB review. Engagement, by contrast, does not necessarily involve efforts to enroll people in studies; rather, it may consist in consulting with patient groups about such things as research priorities and study design. Such activities do not typically require IRB review.

Special Obligations and Risks?

Does working closely with a patient advocacy group give rise to special obligations, such as ensuring that members of the group have an opportunity to participate in a study they helped to design? It would be unfair to restrict enrollment solely to advocacy groups with which the sponsor has a relationship. That said, engaged groups may often have a natural advantage on enrollment, since they will be more likely to have knowledge of such things as the trial’s eligibility criteria and where the trial will be recruiting.

In a similar vein, it may be unclear whether sponsors working closely with patient groups have special obligations to return research results to them or provide them continued access to beneficial therapies. However, arguably, there are already good reasons to return results, in that doing so promotes transparency and public trust in research. In addition, the Declaration of Helsinki and other ethics guidelines support continued access for participants to therapies shown to be effective in the course of clinical trials.

Ultimately, sponsors working closely with patient groups should set clear expectations up front about how recruitment will proceed and whether any priority will be given to patient group members, as well as around return of results and continued access.

Close interaction between patient groups and sponsors may also give rise to risks. Especially in small or rare disease populations, sponsors working with patient groups who form part of the study population may come to know the identity of study participants. The risk of identification should be proactively addressed at the beginning of the engagement process. If members of the patient group understand this risk and accept it, this should remove ethical concerns.

Conflicts of Interest

Empirical research suggests that the patients most accessible to sponsors and investigators for engagement purposes may be affiliated with drug companies or other commercial entities that stand to benefit from the research, which may raise conflicts of interest. What steps, if any, should be taken to mitigate conflicts of interest (COI) among patients engaged in research? Should IRBs apply existing COI policies and practices to patients with conflicts, or are different approaches required?

It is important to remember that, just as non-patient advisors or consultants for clinical trials are not typically within the IRB’s purview, the same holds for patient advisors. It is not the responsibility of the IRB to apply COI mitigation strategies to patient consultants. However, patients that serve in study team roles, for example, as sub-investigators, will fall under the IRB’s responsibility. For patients serving as study personnel, the IRB should apply the same reporting and mitigation standards for COIs that they typically apply to non-patient study team members. This will ensure that patients are not singled out or held to a different standard but treated fairly with regard to COIs.

Conclusion

Patient engagement promises increased value and research relevance but also carries the preceding, and other, ethical challenges. These challenges are by no means insurmountable but do require careful reflection and consistent policies from IRBs and oversight bodies. This will in turn enable confident assessment of the issues and encourage more widespread and successful patient engagement.

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