What matters to whom: patient and public involvement in research

Abstract

We outline a call to action for reproductive health researchers to include patient and public involvement (PPI) in research. PPI prioritizes the patient perspective from study design through dissemination of results which centers the people research intends to serve. PPI highlights the patient as an expert in their own condition. PPI that includes groups harmed by healthcare disparities can draw attention to these harms and generate novel approaches to address them. Numerous frameworks exist for the use of PPI in research. Because obstetrics and gynecology conditions can be particularly sensitive, PPI is crucial in our field.

Introduction

Obstetrics and gynecologic health care is inherently sensitive. Reproduction and genitals are culturally wrought and can be targets of abuse. Patients often feel particularly vulnerable when seeking care in these areas. In the modern era, we prioritize culturally sensitive ¹ and trauma-informed care ², although this is admittedly a work in progress. We strive to center the patient and provide personalized care based on their values and their experiences.

Centering the patient is at least equally important when conducting research in obstetrics and gynecology. Research has a long history of abuse ^3,4, with abuse particularly common in historically maltreated populations including women and nonbinary people, people of color, and people with disability. We now have institutional measures to systematically review research proposals and monitor conduct of research, such as Institutional Review Boards⁵. Although IRBs require community members to participate, they are predominantly composed of clinicians and scientists who are members of the institution. Thus, the institution remains centered.

The counterpoint to this is the inclusion or engagement of patients and the public throughout the research process, not simply at the point of review. Although it goes by many names, this concept is gaining popularity. Patient and public involvement (PPI), patient participation, and patient engagement all aim to incorporate the patient perspective throughout the research process. In this paper we will discuss why this is important, how to do it, and challenges in the process. For consistency, we will refer to all processes as PPI. We see this paper as a call to arms for the obstetrics and gynecology research community to prioritize PPI in their future and ongoing projects. We also encourage readers of the literature to engage in thoughtful assessment of studies to determine level of PPI.

The What and the Why of PPI

PPI is the formal involvement of patients and the public in the research process. “Involvement” is a vague term and ultimately can be interpreted widely. A variety of levels of PPI have been described. A useful categorization schema can conceptualize levels of PPI as consultation, collaboration, and patient or public leadership, with ascending levels of patient and public involvement and control (Figure 1)⁶. Consultation involves simply asking patients and the public to provide opinions and then considering them in the decision-making process. This is the most common model of PPI currently in use. Collaboration involves ongoing partnership with shared decision-making power. Patient or public leadership is primarily directed by the public with final decision-making power in their hands. It is generally accepted that best practice involves at least collaboration level in order to avoid “tokenism” of patient opinions. If patient opinions are superficially solicited and/or discounted, the patient-research relationship can be damaged.

Figure 1.

Schema of varying levels of patient and public involvement in research.

The researcher motivation for integrating PPI is primarily twofold. Arguably the most important motivation is moral. Research is meant to benefit the patient and the community. When developing a research question, we ask where there is a meaningful knowledge gap. Who decides what is meaningful? If the definition of meaning remains solely in academic hands who have not experienced the disease or condition in question, there is bias that does not benefit the patient. Thus, people with lived experience (patients) should inform what research is done and what questions are prioritized ⁷. As both the public funders and intended benefactors of research, they have a vested interest and right to input. Patient input has been documented to meaningfully alter planned research. For example, input from stroke survivors and caregivers led to complete abandonment of an original research plan for a stroke care intervention⁸. The intended population did not support the idea and the researchers listened, saving time, money, and potential harms (remember, no study is without possible harm). In less dramatic changes, a planned randomized-controlled trial of osteopathy for cerebral palsy included early input from parents of children with cerebral palsy. Although the intervention remained the same, the trial enrollment was changed to a waitlist design based on the feedback of the parents⁹. In both cases, PPI changed the research plan in meaningful ways.

Secondly, there is a long overdue acknowledgment of the patient expert^10,11. We are not simply asking for patient opinions to mollify them or because it is the right thing. We ask also because patients have expertise that researchers lack. There is no amount of education and studying that can replicate the experience of living with a specific condition, in a specific body. Thus, PPI enriches the quality of research both in goals and design^12,13. Seemingly subtle changes in study design can lead to better recruitment and retention, particularly when patients who have experienced the condition of interest are involved^14,15. There may also be improved dissemination of research when the patient populations of interest and public are engaged throughout the process.

Cases such as the Tuskegee syphilis study, in which Black men with syphilis had treatment withheld from them by white researchers, or the New Zealand cervical cancer study, in which people with carcinoma in situ of the cervix were followed but not treated are a few of the better known examples of egregious research abuse in recent history^16,17. People who hold less systemic power due to their identities (whether based on race, gender, class, sexual orientation, ability, carceral status, or disease state) are at increased risk of abuse when participating in research. By including people from these populations in PPI efforts, we can help ensure cultural sensitivity of research design and increase study participation. Representative study participation is important to ensure people from all backgrounds can benefit from research outcomes, but we must conduct research in a way that minimizes harm. We will later describe examples of effective engagement with patients to address these disparities.

Although those reading this article most likely have an intrinsic understanding of why scientists choose to engage in research, it may be less clear why someone would partake if not part of their career path. The RAND Institute found that patients and the public choose to participate in PPI projects due to general interest in the topic, a desire to improve healthcare, goals of highlighting the patient perspective, and a broad motivation to advance science⁶. Essentially, echoing the same reasons as scientists. Patients and community members engaged in PPI typically receive a small compensation for their time, but this is certainly not their primary motivation. Participants in PPI are passionate about improving research, care for their conditions of interest, and the possibility of better quality healthcare for others in the future.

PPI is increasingly prioritized by funders as well¹⁸. In the United Kingdom (UK), the majority of funding bodies require patient and public involvement throughout the research process and the National Institute for Health Research (NIHR) developed a resource center for these projects⁶. Similarly, the Canadian Institutes of Health Research offer guidelines and tools for PPI¹⁹. In the United States, The Patient Centered Outcomes Research Institute has led the formal funding initiative for PPI in the United States²⁰. However, community engagement has long been valued by the US National Institute of Health (NIH), such as the National Institute of Allergy and Infectious Disease division support for community engagement in HIV prevention trials since 1990^21,22.

NIH designated Centers for Clinical and Translational Science Award programs (CTSAs) are required to engage the community and encouraged to incorporate PPI²³. However, one study found that the extent of community engagement was low and often superficial, with only 42.6% reporting any community engagement, and only 17.3% reporting use of advisory boards or input from community representatives during studies²⁴. Rather, the majority of engagement was described as sharing study conclusions with the community. Moreover, there was only a 40.4% response rate for the survey, so it is hard to know if nonresponders incorporate any community engagement.

Cancer research foundations have lead the way for PPI in research and the United States, although they typically use the term community based participatory research (CBPR)^25,26. The majority of national clinical trial sites have a patient advisory committee. In 2009, a model for community engagement in cancer research was developed using a committee of stakeholders including investigators, research sponsors, cancer patients advocates, advocacy organizations, community-based organizations, and experts in community engagement²⁷. The recommendations generated by this group were circulated to the research community and public, and were open for comment. Although some comments were skeptical of the rationale for CBPR, many people found the project exciting. After this feedback, the stakeholders again met and ultimately created recommendations highlighting the goals of CBPR, including improving IRB review, informed consent, inclusion of community and patients in trial design, recruitment and attention, community support, and dissemination of trial outcomes. They emphasize the importance of including and reaching historically excluded people.

It is not surprising then that the areas of OB/GYN research with the most literature on PPI are in cancer research or at least oncology adjacent. One group engaged a community advisory committee (CAC) of five Latina parents of female adolescents and four young adult Latinas to advise on modification of Teen VaxScene, a web-based intervention to educate parents about HPV vaccines²⁸. The research group planned to use this program in a Latino population and anticipated need for modifications of the platform. Ultimately, the CAC gave feedback on logo, color scheme, and content over four total meetings. This modified Teen VaxScene product was then presented to six 90 minute focus groups of 20 Latina young adults and 27 Latino parents of adolescents. Transcripts of these groups were then analyzed by the study team to further modify content. The manuscript describing this process includes screenshots of before and after changes to the website, and the difference is compelling.

Another group specifically used community engagement to address the racial disparities in 5-year mortality with endometrial cancer, which is 55% higher for white women²⁹. The authors had previously shown that 40% of the excess mortality in Black women was due to stage at diagnosis and receipt of surgery. Looking to understand why this was happening, they created a partnership with community members and worked with them to create a qualitative study and apply for a grant. Throughout their work, the authors used the Public Health Critical Race praxis to determine how race and racism was present throughout the partnership and research development. The participants (researchers and patients) were radically transparent with expressing their identities, previous experiences, and motivations. In summarizing their finders, the authors state, “Researchers should acknowledge that most academic research institutions are not set up to support community-engaged research and have histories of discrimination.” They go on to highlight the many ways that Black women, in particular, face barriers in engaging with PPI given institutional racism. Reading this study is a master class in PPI, and we strongly encourage interested readers to pursue it when developing their own studies, particularly those intended to address disparities.

How to

For research groups interested in incorporating PPI in their projects, there are a number of organizations providing guidance^18,19,30,31(Table 1). To mention a few: the INVOLVE project by the UK NIHR offers briefings on different aspects of design and conduct of PPI⁶. The Cochrane Training group has a webinar series “Patient and public involvement in research: What, why and how?” that can aid in training and inform protocols³². The RAND Corporation, a corporation focused on research to guide decision making, particularly with regards to national defense, has authored a report that acts as a guide for PPI, detailing motivations, approaches, and challenges to this work. They encourage a systematic approach and transparency in reporting methodology³³. Regardless of which starter kit one employs, it is important to note that there is also a checklist for standardized reporting of PPI in research, the GRIPP2³⁴. This checklist includes aims, definitions, context, impact, outcomes, economic assessment, and reflections. Given the importance of sharing how PPI is used meaningfully, we encourage people both to report and to use the GRIPP2 in doing so.

Table 1.

Selected resources for implementing PPI in your own research.

Reference	Key information
How to incorporate patient and public perspectives into the design and conduct of research. Opinion article. Hoddinott 201818.	• Definitions and terminology • Links to additional resources • Comparisons of methods of PPI • Ethics of PPI • Areas of research to incorporate PPI
National Institute of Health Research (NIHR) Involve Project. Nationally sponsored project with educational website6.	• Definitions and terminology • Motivations for PPI • How to involve members of the public • Who to involve and how to find them • Approaches to PPI • Challenges and possible solutions • Payment and recognition for participants • Training and support
National Institute of Health Research. A brief guide to public involvement in funding applications31.	• Description of how the Research Design Service (a component of the NIHR) can aid with PPI in funding applications • Similar how-to guide as with Involve but particularly targeted towards PPI prior to funding • Geared towards NIHR but concepts can be globally applicable
Canadian Institutes of Health Research. Patient Engagement in Research Resources. Nationally sponsored project with educational website19.	• Interactive tool to help research teams plan PPI • Checklist to support communication with patient partners • Budgeting tools • Evaluation tool • Links to other resources
Designing a tool to support patient and public involvement in research projects: the Involvement Matrix. Methodology paper. Smits 202030.	• Description of reasons to incorporate PPI • Report of development of the Involvement Matrix – this was a collaborative project with patients and caregivesr • The Involvement Matrix, a tool to support establishment of roles and expectations in groups conducting PPI; the goal is to develop sustainable partnerships
Cochrane Training. Patient and public involvement in research: What, why and how? Webinar 201932.	• Videos and slides of presentations explaining the basics of PPI • Useful for training research team
Patient and public involvement in research: enabling meaningful contributions. RAND Corporation research report. Ball 201933.	• Report on existing literature and expert interviews on PPI • Motivations for participation in PPI • Approaches to conducting PPI • Challenges and support for PPI • Recommendations to consider prior to embarking on PPI

A comprehensive review of published frameworks for PPI found 65 separate published models of PPI including tools, checklists, and guides³⁵(19). This review grouped frameworks by their focus: power imbalance investigation, research priority setting, study quality and efficiency focused, reporting focused, and partnership focused. The authors propose that groups investigate published frameworks and adapt them to their purposes instead of developing new frameworks for each project. They emphasize the importance of working with patients to develop your PPI framework. One group proposed a metric for evaluating programs in each of the following areas: “1) focus on local relevance and determinants of health, 2) acknowledge the community, 3) disseminate findings and knowledge gained to all partners, 4) seek and use the input of community partners, 5) involve a cyclical and iterative process in pursuit of objectives, 6) foster co-learning, capacity building, and co-benefit for all partners, 7) build on strengths and resources within the community, 8) facilitate collaborative, equitable partnerships, 9) integrate and achieve a balance of all partners, 10) involve all partners in the dissemination process, and 11) plan for a long-term process and commitments, all measured on a 1–5 Likert scale³⁶. Although this assessment is subjective, it can help remind researchers what to look for when choosing a framework for PPI. A few differing frameworks are described below.

The Moorfields National Institute for Health Research Biomedical Research Centre held a two day event for PPI focused on inflammatory ocular diseases in 2012 open to the public³⁷. They included information on the diseases, patient stories, expert panels of professionals and patients, presentation of recent research, and working groups. Working groups were tasked with selecting, fine tuning, and prioritizing research questions and then outlining study designs for the top questions. The event resulted in prioritization of psychological interventions for patients with Grave’s orbitopathy, which was surprising to the researchers.

PPI need not be as extensive as a two day workshop to still be impactful. A series of two hour interviews with six patients resulted in impressive modifications to a randomized controlled trial of a cycling and education intervention for osteoarthritis³⁸. Patient input was incorporated into communication about the trial, modifications to the intervention, assessment of the primary outcome, and the results dissemination plan. The authors of this trial described patient input as “invaluable.”

For projects including an advisory board, it is important to decide if patients should be included in a general advisory board along with practitioners and scientists or if patients should have their own separate advisory board. The benefit of the former is that all stakeholders are working together, theoretically on even footing. The benefits of a separate patient advisory board include typically increased number of patients and decreased risk that patient voices will be overshadowed by non-patient participants. An example of a trial using an advisory board that intermingles patients with practitioners, scientists, and policy makers is a multi-center trial of understanding prenatal opioid exposure on brain development ¹². They focused on inclusion and exclusion criteria, recruitment, retention, barriers due to COVID-19, and ethical and legal issues. The investigators highlight the importance of patient inclusion in their study design given the particular vulnerability of a pregnant person with a substance use disorder.

Cautions and Challenges

Although participation in PPI has overall minimal risk, there are potential adverse effects. Participants use their time with no promise of future benefit to themselves or to others. If the research involves a sensitive subject (as many health topics are), patients must balance the benefit and meaningful nature of their involvement with the potential emotional trauma of participation. Researchers can find the additional process of PPI and the funding needed to support this process taxing¹². If done poorly, PPI can be conducted in a superficial way that simply “checks a box” without truly engaging the patients and community. Clear expectations and process guidelines can help attenuate these potential harms.

Developing a PPI program as an integral part of a research center helps avoid these adverse effects. Formalized programs with established processes, patient communication in plain language, and clear expectations of roles facilitates incorporation of PPI into studies routinely³⁹(23). This requires investment and resources at the institution level. PPI leaders should be appointed and their time supported. Training in PPI should be included in general research training. Any PPI initiative should include routine formal evaluation and restructuring. All participants (researchers, patients, and public) should be given the opportunity to evaluate the process.

Admittedly, it takes time and money to effectively engage in PPI in research, particularly prior to grant funding. In the UK, there is are Research Design Services (RDS) units providing small funds (up to 500 per application) to engage in PPI prior to submission of grant applications⁴⁰. Systemic support such as this, either at the national or institutional level, could strengthen the use of PPI. Institutional support should also include a cultural acknowledgment that time is necessary to meaningfully have PPI in research. Academic culture encourages speediness with rapid grant submissions and publications; taking a breath is necessary to properly prioritize patients throughout the research process.

Reading the Literature

As yet, there are few studies in obstetrics and gynecology that specifically report their PPI processes as stand alone studies or is informing methodology, such as are detailed above. However, there are other ways to assess the centering of patient perspectives within research. Many institutions have patient outreach organizations and there exist countless patient advocacy groups in areas such as preeclampsia, amniotic fluid embolism, placenta accrete, PCOS, and endometriosis, to name a few. These organizations may be referenced, either tangentially within the methods or directly in the acknowledgments. Although this will not necessarily explain the process of patient involvement, it will let the reader know that the patient perspective was considered. The type of study will also inform you of PPI in some instances. Surveys, focus groups, and interviews by their nature elicit the patient perspective. For these studies, read to see if there was PPI in the development of the questions. Lastly, it is reasonable to subjectively assess whether patient centered outcomes are included. There are no strict criteria for what is or is not a patient centered outcome. Certainly, all outcomes involve patients. However, evaluating things like patient satisfaction, patient preference, comfort and pain, side effects of treatments, and unintended consequences such as impact on lactation indicate a consideration of the patient perspective as important when evaluating interventions.

Summary

The use of PPI in obstetrics, gynecologic, and sexual health research is crucial given the emotional, sensitive, and intimate topics at the heart of these inquiries. Representative PPI involvement is also necessary to heighten the translatability of the research to the greater public. Involved patients and public must also have a sense of ownership of what is investigated and disseminated in order to gain empowerment from the results. Although incorporating PPI takes time and resources, it ultimately has the potential to make research more meaningful and impactful.