Implementing a pragmatic framework for authentic patient–researcher partnerships in clinical research

By clearly outlining the roles of a patient–researcher partnerships, we are laying out a framework for this new approach to research (see Table 1), which is designed to be put into practice by AMCs with active PFACs. We have outlined each stage of the research process and the actions taken by researchers and PFAs, as well as the facilitating PFAC leader (in Box 1), to ensure equal and collaborative participation from both parties.

This framework is built on the Brigham and Women's Hospital (BWH) Center for Patients and Families’ experience engaging PFAs in research. The outlined processes have been implemented in multiple clinical research studies, including those involving a wide range of clinical fields (e.g., sleep apnea, care transitions, geriatric fall prevention) and in inpatient and outpatient settings. This framework has supported patient/family engagement in a wide range of study designs, such as stepped-wedge and randomized control trials. BWH studies designed to engage PFAs using this framework have received funding from multiple organizations promoting patient/family engagement in research, including PCORI and the Agency for Healthcare Research and Quality. This framework has been developed and adjusted based on lessons learned from our experience engaging advisors in research.

To help illustrate our framework, we will provide examples at each stage of implementation of a PCORI-funded study, “Relative patient benefits of a hospital Patient-Centered Medical Home collaboration with an Accountable Care Organization (ACO) to improve care transitions” (Easygrants ID 811; called the ‘Care Transitions’ study henceforth), led by Dr J Schnipper. The Care Transitions study's long-term goal was to assess the benefits and harms of a novel collaborative transitions intervention between the Partners Healthcare Pioneer ACO and several of its patient-centered medical homes. To do so, the main aims of the study were to: develop, implement and refine a multifaceted, multidisciplinary transitions intervention; evaluate the effects of this intervention on post-discharge adverse events, functional status, patient satisfaction and emergency department and hospital utilization within 30 days of discharge; and understand the barriers to and facilitators of successful implementation of this intervention across practices. Staff from the Center for Patients and Families and an experienced PFA were engaged during the proposal synthesis stage to develop a patient-engagement plan. Additional PFAs were engaged from the launch to the study's current stage of results analysis as of February 2016.

In order to provide the necessary infrastructure to recruit PFAs whose medical problems and lived experiences of care are congruent with the research being conducted, the AMC's research leadership must form a partnership with existing PFACs and their PFAs. To do so, researchers need to seek out the leader operationally responsible for the PFACs; at BWH, this is the executive director of the Center for Patients and Families, whose operational role includes leading PFACs, recruiting advisors and supporting their engagement in hospital activities. A natural alliance can be established between research and PFAC leadership to recruit and train interested PFAs.

To begin this process, the principal investigator (PI) summarizes the proposed research study to the PFAC leader and describes ideal attributes of PFAs. The PFAs can be advisors that work within a certain clinical field that are brought in once the researcher has requested them, or they can be a group that is already working with the researcher; regardless, PFAs will participate only as part of the research team and cannot be research subjects once engaged in the study. The ideal situation is to involve PFAs as early as possible in the process, even before a proposal has been written and submitted or the research question decided upon, but advisors can still be matched later in the research process if needed. The PFAC leader creates or uses a narrative matrix of the PFA's lived healthcare experiences. This matrix is a detailed, qualitative database designed to keep track of all interested PFAs and their stories, thus facilitating the pairing of PFAs with relevant clinical research projects. This matrix can also record logistically helpful information such as past participation in research studies and which advisors have received annual Institutional Review Board trainings.

The PFAC leader acts as a neutral facilitator supporting the implementation of the framework. The facilitator role is especially important initially as the researchers learn how to engage the PFAs in each phase of the research study (see Box 1 for details about the PFAC leader's role throughout the study). The PFAC leader coordinates the initial team meeting and ensures appropriate accommodations are made to facilitate PFA involvement, such as a frequency of and location for meetings which can be sustained, flexibility to address specific patient/family needs and methods of communication which work for PFAs (which may not be traditional researcher means of communication). The PFAC leader helps researchers understand that involving PFAs successfully in research requires acknowledging and addressing their needs, including assessing their level of experience with research, ensuring their understanding at each stage and providing an adequate stipend to support their involvement [10,20–21].

Prior to the launch of the Care Transitions study, the PFAC leader provided guidance to the PI regarding practices that would best support PFA involvement. This included recruiting an experienced advisor to be engaged early on and working together to provide input into the patient-engagement plan in the grant proposal. The PFAC leader recommended advisors be paid US$50/h and offered reimbursement for expenses incurred to attend meetings. The patient-engagement plan included monthly meetings between the PI, research staff and PFAs as well as PFA representation on the study Steering Committee. The PFAC leader then led the recruitment of six advisors based on relevant experiences of care as a patient or caregiver (see Table 2 for PFA characteristics) and met with the researcher prior to meeting as a group, in order to brainstorm aims and expectations for each stage of the study.

During this initial phase of the research study, the newly formed team must build a foundation for mutual understanding, collaborative communication and shared decision-making throughout the study. To broker this partnership, it is vital for the researchers, PFAC leader and PFAs to clearly discuss the research process and to establish a common level of language and wording, in order for the PFAs to understand more clearly and be comfortable participating. From the beginning, researchers should actively listen to and value the PFAs’ input in order to facilitate the formation of this partnership. In the Care Transitions study, one way researchers showed this was by eliminating degrees from nametags and introductions, thereby signaling that all contributions were equally valid.

In this stage, components of a grant application are introduced and basic concepts and tenets of clinical research including rigor, validity and reliability are explored. A basic understanding of the scientific process is necessary for PFAs to participate in research. Varying degrees of knowledge and experience with research concepts will require researchers to adopt a new role as teachers to effectively convey these and other concepts, such as power, bias and chance, many of which can be addressed as the research progresses (see Supplementary Data for an example of the tutorial process). The PFAC leader should be available prior to the launch to discuss tutorials with researchers; the PFAC leader should also guide the researchers to clarify concepts and provide further examples during the meetings.

The PFAC leader can further facilitate initial conversations and trust building by asking questions that an advisor might not know the answer to. In many cases, PFAs may not understand why researchers would seek their opinion or what they can contribute with their potentially limited knowledge of research. During the Care Transitions study, it helped PFAs to learn what inspired the researchers to work in this field. The PFAC leader can facilitate this by asking: Why is the researcher conducting research about this topic specifically? These conversations provide the motivational drive for all members to partner together, as the researcher's answer is often closely connected to the realities of care that the PFAs may have experienced. This led the Care Transitions team to start each meeting by having one team member share their story and role, so that both researchers and PFAs had the opportunity to learn about one another.

Another important question the PFAC leader can ask the PI is: once the study is over and the results are disseminated, what happens when your peers read the article? Answers which describe the process of peer review and challenges to the conclusions of newly published research are often met with surprise from PFAs if they do not have a research background. As lay people, PFAs may come into this process regarding researchers as unquestionable experts; sharing this vulnerability helps even out the power dynamic, creates an allegiance between PFA and researchers and helps PFAs understand the importance of scientific rigor. After this conversation, the advisors in the Care Transitions study felt motivated to understand study fundamentals, as they grasped the importance of helping the PI create a rigorous study. Introducing the idea of using valid and reliable tools to do so will help make this more concrete for PFAs. To ensure understanding, it is always essential to explain each concept, why it is important to the research process and what it looks like to have a rigorous, valid and reliable study in practice.

To facilitate PFA involvement in the development of the research question for a CER study, the researcher provides exemplars to illustrate this approach (which can be adapted to other methodologies). PFAs should understand that CER studies seek to compare different treatments and their outcomes, with the goal of providing patients and clinicians with results that can help guide treatment decisions [22]. In the Care Transitions study, several examples were provided, such as a prostate study evaluating difference in outcomes of prostatectomy as compared with other options like watchful waiting [23].

It is also critical that PFAs understand how the framing of the research question can affect knowledge gained from the study. Discussions could include concepts like absolute versus relative benefit. Giving the example of oncology treatment that prolongs survival by 2 weeks without taking quality of life into account will help PFAs understand this difference. Understanding this concept will support PFAs to express what knowledge would be worth gaining based on their clinical experience. Having this conversation allows researchers to learn what PFAs want to know and gives PFAs meaningful input into the direction the study will take, while underscoring the importance of their feedback.

While the steps described in this section ideally occur simultaneously with the launch of the research study and prior to the synthesis of the study's research question, relationship brokering and research tutorials are essential first steps to PFA engagement regardless of when PFAs join the research team.

After the PFAs have been oriented to general research concepts, it is the researcher's undertaking to teach the PFAs about the specific area of interest. Together, the team determines the most appropriate and useful research question to be studied and hypothesis to be tested. The question must resonate as a significant area of study for both the researchers and the PFAs, and represent the interests of the patient population. To do so, the researchers must get the PFAs’ input on questions that will direct the focus and mechanics of the study. The team works together to ensure that the research question yields the most beneficial answer for the constituency the PFAs represent and the primary outcomes will address realistic situations.

While we believe engaging advisors during the design of the research question is one of the most significant and novel ways the patient/caregiver voice can be included in research, we also recognize that this can present logistical challenges, as grant request for applications may require specific criteria or researchers may anticipate their next project. With the support of the PFAC leader, researchers can actively manage this tension by explicitly explaining to PFAs which components are negotiable and which are predetermined. Researchers should make an effort to remain open to and seek input on fundamental aspects of the study, as PFAs often offer insights that are not anticipated or expected.

The PFAC leader and original advisor initially provided input on topic solicitation and hypothesis framing during the Care Transitions proposal writing stage. They gave feedback on the importance of this topic and whether the proposed research question was important to the relevant patient population. Once the proposal was accepted and the other PFAs recruited, the hypothesis was revisited, explained and placed in context of the existing work on care transitions.

After the research question is agreed upon, the researcher should fine tune the hypothesis based on a literature review. It is important for the researcher to determine what information is already available and where the study should aim to add to the field. It is also the researcher's responsibility to collect PFA perspectives on specific aims which will elicit the information they are interested in. As such, the researcher ensures that the study is focused on both the interests of the affected patients and that the right aims are outlined to test the hypothesis.

For the Care Transitions study, this stage took the form of sharing existing discharge practices within the Partners ACO so that PFAs could understand the baseline practice and outcomes; this helped PFAs understand why the general aims had been chosen by the researchers. Though the general aims of the study were predetermined, they remained broad at the outset so advisor input focused on narrowing their scope by revising specific aims to include those that were specifically meaningful in the lives of patients. For example, the aims of reducing clinically meaningful measures such as post-discharge adverse events and rehospitalization rates were supplemented with the aims of improving the ability of the patient to return to work, if applicable, and decreasing the extent to which patients reported feeling like a burden to family and other caregivers.

During this stage, the study methods and outcome measures are explored together by the researchers and PFAs. Researchers should begin by teaching PFAs about additional research concepts, which will prepare the PFAs to discuss patient-facing interventions and data collection methods, such as surveys and interviews. The key is for PFAs to be inspired by how challenging it can be to produce high-quality research and to have a central role in molding the way researchers interact with patients when implementing study methods.

The researcher begins by teaching the PFAs about chance, bias and confounding (see Supplementary Data for a tutorial example), the major threats to the internal validity of a study. It is important for PFAs to understand that researchers must be aware of and address each of these factors when planning a study's methods and measures. Defining each concept, giving concrete examples of how each occur in research and describing how to lessen the impact of each of these factors during the study design and analysis ensures that the PFAs grasp these concepts. Explaining these concepts in plain language builds a common understanding between PFAs and researchers. In the Care Transitions study, some PFAs felt they did not need to know this information in order to fulfill their role; however, the PFAC leader felt that a full understanding would ensure PFA confidence in their grasp of the study, allow them to provide informed input regarding the analysis plan and prepare them for the results and interpretations stages.

When the PFAs are comfortable with these concepts, researchers and PFAs collaborate to determine methods and measures that will produce a high quality study and accommodate patient needs. Researchers lead this process, proposing methods and measures and are responsible for making sure each PFA understands and is able to contribute their perspective as to how these choices will impact patients. This process should include the design or refinement of the study intervention itself. Even a predetermined clinical protocol can have room for the PFA perspective; for example, the PFA might weigh in on how communication with the patient is approached. In other cases, the PFA can play an active role in the development and refinement of the intervention itself, providing feedback on the needs of the patient population and pilot testing versions throughout the process. PFAs should also be given the chance to pilot patient-facing data collection instruments for ease of use, clarity in instructions and language and applicability to the patient population. Together, the researchers and PFAs refine the study intervention, methods and measures to allow for the best possible interactions with patients, while maintaining the integrity of the study.

Advisors played an important role in refining the Care Transitions intervention, participating in decision-making, document and tool tailoring and training of research staff executing study enrollment. PFAs gave input on specific components of the interventions, such as ‘Who would you want to be told to call with questions/concerns after discharge and what is the best way to present this information in the discharge summary?’ They were provided with all patient-facing documents and asked to review and provide feedback on clarity and presentation. PFAs also refined tools created for clinicians involved in the intervention, such as a Discharge Advocate Checklist, which was designed to remind clinicians to complete and document important tasks at discharge. PFAs supported research implementation, as well. The Research Assistant who enrolled patients in the study performed a mock intake interview with a PFA in front of the group; the PFAs then discussed and provided feedback to the Research Assistant on patient-friendly language and behaviors.

PFA input also proved to be essential to the Care Transitions’ data collection methods. PFAs played an active role in refining the proposed survey content and administration process. Advisors pilot tested the questions and ensured that they were clear and direct, providing feedback that led to meaningful edits. PFAs felt that it was important to assess which piece of the intervention patients liked most, rather than solely assessing the most effective component, as proposed by researchers. They believed this measure would reveal intervention components that patients found most valuable, least intrusive, and easiest to adhere to, among other possibilities. This new perspective led the team to add a focus group with intervention patients as a qualitative measure to assess which intervention component patients liked most. The focus group with six randomly selected patients who participated in the intervention was led by a PFA and the PFAC leader, with a qualitative researcher listening in and study staff transcribing the dialogue for further qualitative analysis.

To ensure PFAs continue to provide feedback and are prepared to understand the results of the study, regular meetings should be planned throughout the implementation period of the study. These meetings allow researchers to continue to solicit the patient/family perspective on barriers that may arise during implementation and support continuing education for the advisors. Researchers can facilitate PFA engagement and understanding by creating tables and graphs with mock results or current data and have PFAs practice reading and summarizing data from these tables. It is also important to explain the meaning of each component of these tables and concepts that arise in interpreting them, such as n collected, CIs, adjusted analysis, p-values, correlation and causation. These meetings also serve to keep advisors updated on progress, allowing them to build realistic expectations for the study results.

The Care Transitions study is currently in this stage of the study, as enrollment has finished and results are being analyzed. Meetings during the intervention period covered enrollment progress, troubleshooting of intervention challenges and monitoring input from patient participants. Selected patient subjects were interviewed by the team as a means to gather qualitative data and feedback on the intervention; concerns or issues that surfaced in these interviews were addressed by researchers and PFAs together. PFAs also practiced reading preliminary results and discussing their interpretations in preparation for the final stages of the study.

Once the data have been collected and analyzed, the research team prepares tables and graphs of the findings. The researchers present the data to PFAs in an organized way and explain the meaning of each result in order to support the PFAs’ understanding. To do so, link values shown in the tables and graphs back to the concepts PFAs were taught during the methods and measures phase. The PFAs now seek to apply the hard data to the research framework they have learned throughout this process, considering whether the findings answer the research question.

After the findings have been organized, the research team interprets the results for applicability and generalizability, determining recommendations for clinical practice or further research. The researchers explain their interpretations to the PFAs, describing factors that threaten the study's external validity and explaining the applicability and generalizability to various populations or contexts. Together, researchers and PFAs should discuss limitations and opportunities for future research.

The PFAs’ understanding of this is important, as it will help them to translate the conclusions and recommendations into lay terms to allow for the broader dissemination of the study findings, especially to the populations of patients affected by the results. The researchers and PFAs work together to create a narrative that the PFAs can use to explain the study design, methods and results in an accessible way.

While the Care Transitions study has not yet reached this phase, the patient-engagement plan outlines a process for translating results into a rigorous and accessible message for dissemination. Researchers will present the results and their scientific interpretations and give PFAs a chance to provide input. The team will discuss whether the conclusions of the study make sense or are as expected. The PFAs will provide input on how to best explain the results to other patients and the public so that they resonate and are comprehensible. Together, the PFAs and researchers will decide how to convey the study's conclusions for both scientific and popular audiences.

Once the results have been interpreted, the researchers and PFAs work together to create a dissemination plan that includes professional and lay publication. Researchers will present their plan for writing manuscripts for peer-reviewed journals and presenting with PFAs at professional conferences. In addition to the usual scientific dissemination channels, our framework provides a unique avenue for the results to be disseminated among lay people. PFAs will recommend media outlets that are accessible to those outside the research community, such as targeted popular media, news outlets, state and national stakeholder associations and patient affinity organizations. In these publications, study findings will be shared in a narrative form, using language determined by the PFAs and researchers. As results are disseminated, researchers and PFAs should include and give credit to the others’ contributions. Through these complementary dissemination methods, the study conclusions will reach scientists, researchers, medical professionals and a targeted portion of the general population who may be most interested in or affected by the results.

While final decisions cannot be made until this phase of the study, conversations about dissemination can begin earlier in the research process. This ensures that researchers have considered how they will present methods or results and that advisors have actively thought about the best means to communicate results to the public prior to the end of the study. The Care Transitions study has begun preparing for this stage by discussing potential avenues for dissemination and what information the team hopes to convey in scientific and lay publications.

As detailed earlier, this framework is forged from our experiences engaging PFAs in research through the BWH Center for Patients and Families. This model of engagement has been implemented successfully in a variety of settings, study designs and clinical specialties. In its ideal form, PFA involvement begins prior to the study launch, as described by our framework, but PFAs have also been meaningfully integrated later in the process. At present, the BWH studies using this framework are still in progress. The Care Transitions study is furthest along and in the results analysis phase; as such, formal evaluation to measure the impact of PFA engagement in the research process has not been conducted, which we acknowledge as a limitation to our framework. Nonetheless, we are encouraged by the framework's success in practice and continued requests by PIs for PFA involvement in their projects.

Furthermore, others have proposed evaluative criteria to determine the success of stakeholder engagement in the absence of quantitative analyses. The PCORI conceptual model of PCOR outlines a culture of patient-centeredness and meaningful and effective partnerships between team members as the near-term outcomes of successful stakeholder engagement [14]. Lavallee et al. identified respect, trust, legitimacy, fairness, competence and accountability as measures for optimal stakeholder engagement [24]. In our experience, these factors are critical in determining the success or failure of PFA engagement in research. To assess whether our framework has been successful by these measures, we have collected qualitative feedback from PFAs and researchers that illustrates their perspectives on the culture and values fostered by this process. For example, in the Care Transitions study, advisors reported positively on key measures of partnership between them and the researchers: they felt the expectations outlined were met, communication was respectful and open, their input was valued throughout the study and changes were made based on their feedback. Similarly, researcher feedback also reflected a positive experience. They felt PFAs contributed meaningful insights that resulted in positive changes to every stage of the study, were actively engaged and were important members of the research team.

We also recognize the favorable circumstances at Brigham and Women's Hospital, which allowed us to build a robust PFAC program with a diverse and talented group of PFAs, thus facilitating this type of engagement. BWH's institutional prioritization of patient- and family-centered care and our location in Massachusetts, which is currently the only state that legislates a requirement for hospitals to create PFACs, encouraged the early adoption and growth of this feature. While Massachusetts does not mandate PFAC involvement in research, the law has promoted the growth of PFACs into robust and active participants in hospital activities, which reinforces PFAC ability to engage in activities like research. Furthermore, due to the visibility and success of the BWH PFAC program, researchers have sought out the involvement of PFAC members, contributing to our ability to integrate PFAs successfully into research projects.

Nonetheless, because PFACs have become increasingly widespread at AMCs, we see wide applicability of our framework. Legislation requiring PFACs is not a requisite to their growth, as evidenced by the more than 2000 hospitals that have launched PFACs since 1990. As PFACs become well established at hospitals around the country, they increasingly participate in research [25,26]. With the creation of PCORI, incentives to create and use PFACs to engage PFAs and patient and families in research have increased; our framework supports institutions to do so in a comprehensive and successful way. We also see opportunities for further study of our framework, including a formal analysis of its impact on the research process.

With passage of the Affordable Care Act, there has been a significant increase in institutional and financial support for patient/family engagement in research. We have outlined a pragmatic framework to enable successful and impactful collaboration between patients and researchers. As the patient/family perspective is given equal footing to other stakeholder perspectives and their lived experience is incorporated throughout the research process, research outcomes will better meet the needs of relevant patient populations and increase patient-centered practices in research and care delivery. Going forward, we see additional opportunities to expand current healthcare institutional and research engagement of patients and families as well as the need for further studies on the efficacy of engaging them in different ways.

Despite the significant growth in the number of PFACs and their meaningful involvement in healthcare organizations around the country over the last 10 years, PFA engagement in research remains limited [2,25–26]. As PFAC programs continue to grow and mature, this improved infrastructure will support increased PFA involvement in research. PCORI's leadership in funding and promoting patient-centered research will continue to encourage CER and other patient-centered outcomes research that includes patient and family stakeholders as part of the research team. PCORI's recent development of various networks to support sharing of research methodology, data and lessons learned, such as the National Patient-Centered Clinical Research Network and the Evidence to Action Networks, and their dissemination of results from early PCORI-funded studies will further increase the efficacy and profile of this approach[27,28]. Concrete steps for engagement, as outlined in our pragmatic framework, will provide researchers with a blueprint to effectively engage patients and families in research.

Consequently, we hope the next 10 years will see a shift toward patient- and family-centered CER, which, through the exploration of research questions directly relevant to patient and family lived experience, will lead to findings with the potential to directly impact care in novel and dramatic ways. The dissemination of these results in accessible consumer-oriented media will empower patients and families to educate themselves and engage in their care to a new extent. The goal and challenge will be to translate this research and its results into the delivery of patient- and family-centered care on a systemic level. It will be the responsibility of healthcare leaders to push for the integration of new standards and practices into care while keeping patient and family values and needs at its center.

The authors would like to thank Brigham and Women's Hospital and the Brigham Research Institute for their institutional prioritization of patient- and family-centered care, patient and family advisory councils and patient engagement in research. The authors would also like to thank the Patient-Centered Outcomes Research Institute for their groundbreaking work in leading and funding patient-centered outcomes research. Finally, we acknowledge Dr J Schnipper, H Heyison and Dr S Lee for their contributions to revising this manuscript.

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.