Building Partnerships and Teams to ensure a successful trial
Section 1
Introduction
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 partner groups throughout the research process is a core feature of comparative effectiveness research and particularly important in the context of PCTs. Partner 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.
Partner (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 partner engagement will be revisited throughout the Living Textbook and also merits upfront discussion. The NIH Pragmatic Trials Collaboratory has a working group dedicated to supporting partner engagement and developing best practices: the Health Care Systems Interactions Core. Lessons gleaned from the NIH Collaboratory PCT experiences, along with additional resources on partner engagement in PCTs, are described in this chapter.
Partner engagement in pragmatic research is not automatic—it must be mindfully established. According to Eric Larson, MD, formerly of the NIH Pragmatic Trials 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 partners 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 partners 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 partner 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 partners throughout the research process.
There are multiple methods of partner 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 partners, geographic dispersion, and resources. One method for facilitating engagement is to establish an advisory board or steering committee with members from different partner 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.
Engagement should begin with the selection and clarification of a research question and continue through all phases of the research. Rather than asking potential partners, “How can we answer the question I have already selected?”, investigators should ask, “What questions are most important to your health system and the people you serve?” 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 partners are fully prepared to participate and are fairly compensated for their time and effort (see the PCORI Compensation Framework for additional guidance on compensating partners).
Case Study: IMPACT-AFib Trial
A good example for the importance of collaboration comes from the IMPACT-AFib trial. A large number of collaborators led to multiple iterations of the protocol, substantial discussion, and a lengthy review process (Cocoros et al. 2019). Branding, logos, and details related to how the intervention looked required review and approval from all partner sites. These tasks and many other essential tasks could not have been performed by external investigators, and internal champions and were instrumental in ensuring the proposed protocol could be executed (Garcia et al. 2020). Throughout the process of the trial, patient representatives also provided indispensable guidance (Cocoros et al. 2019).
SECTIONS
Resources
Quick Start Guide for Researcher and Healthcare Systems Leader Partnerships
This Quick Start Guide is designed to help clinical investigators partner with healthcare system leaders to support the successful conduct of an ePCT within their healthcare system. It provides advice from the Collaboratory and serves as an annotated table of contents, pointing readers to essential content in the Living Textbook regarding partnering to conduct an ePCT.
Contains guidance on compensating partners
REFERENCES
Cocoros NM, Pokorney SD, Haynes K, et al. 2019. FDA-Catalyst—Using FDA’s Sentinel Initiative for large-scale pragmatic randomized trials: approach and lessons learned during the planning phase of the first trial. Clin Trials. 16:90-97. doi:10.1177/1740774518812776. PMID: 30445835
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.
Garcia CJ, Haynes K, Pokorney SD, et al. 2020. Practical challenges in the conduct of pragmatic trials embedded in health plans: lessons of IMPACT-AFib, an FDA-Catalyst trial. Clin Trials. 17:360-367. doi:10.1177/1740774520928426. PMID: 32589056
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. www.pcori.org/sites/default/files/Engagement-Rubric.pdf. 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.