Building partnerships to ensure a successful trial

Section 1



James Fraser

Rachael Moloney, MHS

Ellen Tambor, MA

Leah Tuzzio, MPH


Contributing Editors

Karen Staman, MS

Gina Uhlenbrauck

Pragmatic clinical trials (PCTs) are designed to answer questions that are relevant to patients, clinicians, payers, policy-makers, and other healthcare decision-makers and to identify generalizable, sustainable ways to improve health and care delivery. Engagement of key stakeholder groups throughout the research process is a core feature of comparative effectiveness research and particularly important in the context of PCTs. Stakeholder input helps to ensure that PCTs are designed to answer questions important to them, that they are feasible to conduct with minimal clinical disruption, and that results are interpreted and shared appropriately.

Stakeholder: “An individual or group who is responsible for or affected by health- and healthcare-related decisions that can be informed by research evidence” (Concannon et al. 2012)

Engagement: “A bi-directional relationship between the stakeholder and researcher that results in informed decision-making about the selection, conduct, and use of research" (Concannon et al. 2012)

With PCTs conducted in real-world settings (e.g., hospitals, nursing homes, clinics), productive collaboration among researchers, clinicians, patients, and healthcare delivery organization leaders is needed to ensure that studies can be conducted in ways that support research and the goals of the organization, the clinician, and the patient.

The topic of stakeholder engagement will be revisited throughout the Living Textbook and also merits upfront discussion. The NIH Collaboratory has a working group dedicated to supporting stakeholder engagement and developing best practices: the Health Care Systems Interactions Core. Lessons gleaned from the NIH Collaboratory PCT experiences, along with additional resources on stakeholder engagement in PCTs, are described in this chapter.

Stakeholder engagement in pragmatic research is not automatic—it must be mindfully established. According to Eric Larson, MD, of the NIH Collaboratory’s Health Care Systems Interactions Core,

“the best way to create engagement is for partners to commit to it at the outset so that they learn to trust each other and address problems collaboratively... Such collaborations are the key to solving the unforeseen and inevitable challenges of conducting clinical trials in large healthcare systems” (see full interview).

Guidelines and methods for engagement

Guidelines for meaningfully engaging stakeholders in the research process include core principles such as respect, fairness, co-learning, accountability, transparency, and trust (Lavallee et al. 2012; PCORI 2015; Sheridan et al. 2017). These principles can be demonstrated by ensuring that 1) roles and expectations of all stakeholders are clearly established, 2) everyone is adequately prepared to participate in engagement activities, 3) different viewpoints are encouraged and respected, and 4) feedback is provided regarding how stakeholder input was used or why it was not used.

Seeking to advance the study and practice of engagement in health research, the Patient-Centered  Outcomes Research Institute (PCORI) launched the Engagement in Health Research Literature Explorer. Locating relevant research articles about engagement can be challenging because of a lack of standard terminology. The new tool searches a curated database of peer-reviewed literature on engagement. Articles are included in the database if they describe engagement experiences, report research findings on engagement practices, or present theories, concepts, or views on engagement. The database is updated monthly and is one way PCORI is helping to promote meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders throughout the research process.

There are multiple methods of stakeholder engagement, and decisions about which method or methods to employ should be based on factors such as the stage of research, engagement objective, number and diversity of stakeholders, geographic dispersion, and resources. One method for facilitating engagement is to establish an advisory board or steering committee with members from different stakeholder groups. It may be helpful to build on previous collaborations (e.g., quality improvement champions) or to begin partnerships through a pilot study. Face-to-face meetings are ideal for establishing relationships and trust, but are not always feasible. Although phone and web-based conferences can be a reasonable substitute, special effort is required to facilitate active participation by stakeholders. For some types of input, one-on-one interviews or survey techniques may be more appropriate.

The frequency of engagement may also vary over the course of a study. For example, more frequent engagement may be beneficial during the early stages of planning and implementation and once results from the study are available, while less frequent interaction may suffice during the enrollment and follow-up period. Regardless of the method or frequency of engagement, it is necessary to ensure that all stakeholders are fully prepared to participate and are fairly compensated for their time and effort (see the PCORI Compensation Framework for additional guidance on compensating stakeholders).




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Concannon TW, Meissner P, Grunbaum JA, et al. 2012. A new taxonomy for stakeholder engagement in patient-centered outcomes research. J Gen Intern Med. 27:985–991. doi:10.1007/s11606-012-2037-1. PMID: 22528615.

Lavallee DC, Williams CJ, Tambor ES, Deverka PA. 2012. Stakeholder engagement in comparative effectiveness research: how will we measure success? J Comp Eff Res. 1:397–407. doi:10.2217/cer.12.44.

PCORI. 2015. PCORI Engagement Rubric. Accessed May 9, 2017.

Sheridan S, Schrandt S, Forsythe L, Hilliard TS, Paez KA. 2017. The PCORI Engagement Rubric: promising practices for partnering in research. Ann Fam Med. 15:165–170. doi:10.1370/afm.2042. PMID: 28289118.

Version History

February 27, 2020: Made minor nonsubstantive text corrections (changes made by G. Uhlenbrauck).

December 15, 2018: Added text and revised as part of annual review (changes made by K. Staman).

Published August 25, 2017


Fraser J, Moloney R, Tambor E, Tuzzio L. Building partnerships to ensure a successful trial: Introduction. In: Rethinking Clinical Trials: A Living Textbook of Pragmatic Clinical Trials. Bethesda, MD: NIH Health Care Systems Research Collaboratory. Available at: Updated February 27, 2020. DOI: 10.28929/095.