Pragmatic Clinical Trials (PCTs) are designed to inform decision makers about the benefits, burdens, and risks of health interventions in real-world settings. PCTs often use for research purposes data collected in the course of clinical practice. The distinctive features of PCTs demand fresh thinking about what is required to act properly toward people affected by their conduct, in ways that go beyond ensuring the protection of rights and welfare for “human research subjects” under conventional research ethics regulations. To stimulate such work, we propose to distinguish among categories of research participants in PCTs as follows: Direct participants: (1) Individuals being directly intervened upon and/or (2) individuals from whom personal identifiable data are being collected for the purposes of the PCT. Indirect participants: Individuals who are (1) not identified as direct participants and (2) whose rights and welfare may be affected by the intervention through their routine exposure to the environment in which the intervention is being deployed. Collateral Participants: Patient groups and other stakeholder communities who may be otherwise affected by the occurrence and findings of the PCT. We illustrate these distinctions with case examples and discuss the distinctive responsibilities of researchers and PCT leadership toward each type of participant. We suggest that PCT investigators, Institutional Review Boards (IRBs), health systems leaders, and others engaged in the research enterprise work together to identify these participants. For indirect participants, risks and benefits to which they are exposed should be weighed to ensure that their rights and welfare are protected accordingly, and communication strategies should be considered to help them make well-informed decisions. Collateral participants could provide input on the design, planning and conduct of a PCT, and offer insights regarding the best way to communicate the trial’s results to their constituencies.

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Introduction Patient group engagement is increasingly used to inform the design, conduct, and dissemination of clinical trials and other medical research activities. However, the priorities of industry sponsors and patient groups differ, and there is currently no framework to help these groups identify mutually beneficial engagement activities. Methods We conducted 28 qualitative, semi-structured interviews with representatives from research sponsor organizations (n = 14) and patient groups (n = 14) to determine: (1) how representatives define benefits and investments of patient group engagement in medical product development, and (2) to refine a list of 31 predefined patient group engagement activities. Results Patient group and sponsor representatives described similar benefits: engagement activities can enhance the quality and efficiency of clinical trials by improving patient recruitment and retention, reduce costs, and help trials meet expectations of regulators and payers. All representatives indicated that investments include both dedicated staff time and expertise, and financial resources. Factors to consider when evaluating benefits and investments were also identified as were suggestions for clarifying the list of engagement activities. Discussion Using these findings, we refined the 31 engagement activities to 24 unique activities across the medical product development lifecycle. We also developed a web-based prioritization tool (https://prioritizationtool.ctti-clinicaltrials.org/) to help clinical research sponsors and patient groups identify high-priority engagement activities. Use of this tool can help sponsors and patient groups identify the engagement activities that they believe will provide the most benefit for the least investment and may lead to more meaningful and mutually beneficial partnerships in medical product development.

Patient group engagement is increasingly used to inform the design, conduct, and dissemination of clinical trials and other medical research activities. However, the priorities of industry sponsors and patient groups differ, and there is currently no framework to help these groups identify mutually beneficial engagement activities. Methods: We conducted 28 qualitative, semi-structured interviews with representatives from research sponsor organizations (n=14) and patient groups (n=14) to determine: 1) how representatives define benefits and investments of patient group engagement in medical product development and, 2) to refine a list of 31 predefined patient group engagement activities. Results: Patient group and sponsor representatives described similar benefits: engagement activities can enhance the quality and efficiency of clinical trials by improving patient recruitment and retention, reduce costs, and help trials meet expectations of regulators and payers. All representatives indicated that investments include both dedicated staff time and expertise, and financial resources. Factors to consider when evaluating benefits and investments were also identified as were suggestions for clarifying the list of engagement activities. Discussion: Using these findings, we refined the 31 engagement activities to 24 unique activities across the medical product development lifecycle. We also developed a web-based prioritization tool (https://prioritizationtool.ctti-clinicaltrials.org/) to help clinical research sponsors and patient groups identify high priority engagement activities. Use of this tools can help sponsors and patient groups identify the engagement activities that they believe will provide the most benefit for the least investment and may lead to more meaningful and mutually beneficial partnerships in medical product development.

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