Definitions of Patient-Centered Care and Patient-Centered Clinical Research

The Institute of Medicine (IOM) defines patient-centered care as: “providing care that is respectful of and responsive to individual patient preferences, needs, and values, and ensuring that patient values guide all clinical decisions,” and included patient-centered care as one of six key aims health care systems should adopt to improve outcomes for patients.⁶ The Patient-Centered Outcomes Research Institute (PCORI) defines PCOR as “research that addresses the questions and concerns most relevant to patients.”⁷

The uptake of a patient-centered approach in medical care and clinical research may be facilitated by a variety of factors including targeted PCOR funding, patient-driven research design, and stakeholder-relevant dissemination strategies that intend to enhance informed clinical decision-making. Patient-centered research funds would be directed to address questions that are high priority for and relevant to patients and other key stakeholders. Patient-centered research design would take into account patient preferences and concerns early in the design phase of a new research study to align research questions with patient needs and enhance research efficiency; for example, patients may have recommendations about optimizing recruitment strategies or selecting the number or mode of administration of patient-reported outcome (PRO) measures used in the study. Dissemination is also critical for increasing patient-centeredness. Dissemination of research findings to health care providers and patients should be timely and transparent, and findings should be communicated in a manner that is clear and understandable to patients. Lastly, patient-centered care relies on patients having the necessary information to make informed decisions about health care choices available to them, linked to health outcomes that are important to them.

Patient-centered care and patient-driven clinical research is intended to result in two primary outcomes: (1) increased satisfaction of patients and health care providers with medical care; and (2) improvement in health outcomes that are most meaningful to patients and clinicians. These concepts are summarized in Table 1-1.

Table 1-1

Goals and benefits of patient-centeredness in research and medical care.

Though well over a decade has passed since the IOM and other stakeholders have called for increased patient-centeredness in medical care and clinical research, researchers are only beginning to quantify the extent to which the desired benefits of this shift are being achieved, and several challenges remain. In particular, challenges during the funding and design phases include the additional time and expense associated with engaging patients and other stakeholders at the beginning and throughout all phases of a research study, as well as the need for effective, documented engagement strategies. Informed decision-making by health care providers and patients is complicated by the availability and accessibility of necessary evidence, including sufficiently “personalized” evidence from population-based studies. Informed decision-making also relies on effective two-way communication between health care providers and patients about all treatment options while considering the range of outcomes that are most important to each individual patient. Fitting this type of patient-centered conversation into the constraints of typical office visit can be challenging.

Evolving Role of Patients in Medicine, Research, and Policy

The growing demand for patient-centered research has led to several new initiatives that seek to actively engage patients. One of the most notable, in terms of its immediate impact, is the establishment of PCORI, an independent nonprofit organization. In 2010, Congress authorized the establishment and funding of PCORI; its mandate is to fund patient-centered comparative effectiveness research that addresses critical evidence gaps in current clinical practice and that produces evidence that can be used by patients, caregivers, clinicians, and other stakeholders to support clinical and health policy decisions.

To increase the likelihood that the research it funds is patient-centered, the hallmark of PCORI’s funding is a requirement for all awardees to actively engage patients and other key stakeholders in all phases of the research project.⁸ To this end, PCORI has created an engagement rubric, a set of methodology standards, and a compensation framework for patients engaged in PCORI-funded studies that serve as basic guides for researchers and their collaborative partners. To reinforce the commitment to incorporating multi-stakeholder perspectives in research and to ensure proposals are responsive to stakeholders’ needs, all research proposals submitted to PCORI are not only peer-reviewed for technical merit by scientific and methodological experts, but also evaluated by patients and other key stakeholders on the quality of “patient-centeredness” and “patient and stakeholder engagement.” This process is critical to establishing and maintaining the credibility of patient-centered research.

In addition to the work of PCORI, a number of recent efforts are encouraging the active participation of patients, their caregivers, and patient advocates in regulatory decision-making. For instance, there has been considerable interest on the part of some regulatory agencies in adopting “adaptive approaches” for drug approval and reimbursement decisions. These approaches hinge on the early and continuous engagement of patients and other key stakeholders throughout the life-span of drug development.⁹ The push for adaptive approaches has been driven in part by patient demands for early access to medications. Innovative clinical trial designs, stronger understanding of disease processes, and improved availability of monitoring tools for post-licensing surveillance using real-world data have also supported the development of adaptive approaches to drug approval and reimbursement.⁹

One example of an adaptive approach is the European Medicines Agency’s (EMA) Adaptive Pathways Program, launched in December 2014 to improve the timely access of new drugs for patients.^9–11 Through this program, the EMA can provide accelerated treatment approval for specific subgroups of patients with unmet medical needs while requiring additional research to be conducted to support future expansion (or restriction if necessary) of the regulatory approval and reimbursement for that drug. Due to the uncertainty and trade-offs involved, the EMA requires that patients and other relevant stakeholders be engaged in the decision-making about the design of the research that needs to be conducted to support future decisions about expansion of the regulatory approval.¹⁰

The U.S. Food and Drug Administration (FDA) has taken several steps to formalize the integration of patient perspectives into the regulatory process, including establishing a Patient Engagement Advisory Committee.¹² The purpose of this committee is to provide advice to the FDA Commissioner on a variety of patient-related topics, such as the inclusion of patient preference and PRO measures in study design, benefit-risk determinations, and areas of unmet clinical need. Another effort to directly incorporate patient perspectives is the FDA’s Patient-Focused Drug Development Initiative, a commitment under the Prescription Drug User Fee Act (PDUFA) to obtain patient views on specific conditions and currently available therapies to treat those conditions. Over 20 public meetings are planned to be held focusing on specific disease areas over the five year authorization period of “PDUFA V”, with the output summarized in “Voice of the Patient” reports to be made available on the FDA’s Web site.¹³

Additionally, the FDA published guidance in 2009 on the inclusion of PRO measures as evidence for treatment benefit, demonstrating its support for the inclusion of patient-centered outcomes as endpoints in industry-sponsored clinical trials that may contribute to regulatory approval and product labeling.¹⁴ Following the release of the initial draft guidance in 2006, the FDA approved nearly a quarter of all new molecular and biologic license applications incorporating PRO evidence, along with other evidence sources for labeling claims during the period of 2006–2010.¹⁵ More recently, in May 2015 the FDA published draft guidance on the inclusion of patient preference information to support product labeling for diagnostics and therapeutic devices, further underscoring the agency’s recognition of the importance of understanding what benefits and risks are most important from a patient’s perspective.¹⁶

To improve the availability of valid and reliable PRO measures, the U.S. National Institutes of Health (NIH) has funded the Patient Reported Outcomes Measurement Information System (PROMIS) initiative. Initially developed in 2004 and expanded in 2010, the PROMIS initiative develops, maintains, and validates its own unique set of PRO instruments to measure physical, mental, and social health outcomes in adult and pediatric populations with chronic disease.¹⁷ Importantly, all the instruments have been designed for self-administration by adults, children, or caregivers, reflecting the growing emphasis on PROs as important health measures.

Pharmaceutical companies are also beginning to actively engage patients to enhance the relevance of outcomes studied during drug development as well as the breadth of treatment options evaluated. Pharmaceutical companies are undertaking a variety of different approaches to increase patient engagement. For instance, Sanofi hired a Chief Patient Officer, whose role is to “infuse the patient perspective into all of our activities, ranging from early stage R&D [research and development] to product launches.”¹⁸ Others, like Pfizer, are reaching out to patients through online crowdsourcing platforms and still others, like Genzyme, have formed internal patient advocacy groups.^19,20 Finally, at least one pharmaceutical company, Roche, has developed a set of good practice guidelines to inform their patient engagement efforts.²¹

Lastly, it is important to note the critical role played by patient advocacy groups. An increasing number of well-organized, vocal patient advocacy groups are actively contributing to the research landscape in a variety of ways. For instance, some patient advocacy groups are driving research agendas through funding grants and facilitating collaborations across academic sites; serving as valuable clinical trial recruitment partners, particularly for rare conditions, while leveraging innovative outreach strategies to diversify enrollment in trials; and enhancing capacity to develop biomarkers or other clinical screening and monitoring tests for therapeutic products.²² Patient-advocacy groups also actively participate in and lead conversations on the need for legislation and continued meaningful participation of patients in all aspects of health care and policy.

In summary, patients are actively participating in and shaping the way that health care, research, and regulatory decisions are made in many ways. All of these efforts share a common goal of improving the ability of patients, their caregivers, and clinicians to make informed health care decisions.

Ability of Registries To Meet the Needs of Patient-Centered Research

With some specific exceptions, such as pregnancy registries, registries are usually designed to address multiple questions of interest, often over long periods of time. As such, registries are able to incorporate both questions that patients prioritize as being of central interest to the understanding of their condition and their treatment options as well as questions that may address concerns of other stakeholders. Registries are often designed to examine changing patterns of treatment and the outcomes of treatment over time, as well as other new questions that may arise over the span of the registry. Patients are also important and reliable sources of information for data known only to them, such as time and dose of medications used as needed, e.g., migraine medication, and personal habits that may affect drug effectiveness or safety.²³

PROs are frequently incorporated into registries to capture unfiltered measures of general and health-related quality of life, clinical, and functional measures directly from patients. Indeed, a key area in which patients may provide important recommendations is in the selection of the most relevant and appropriate measures to address a specific research question. Therefore, patient input is sought prior to and/or during the development of the registry protocol.

Key Features of Registries Relevant To Patient-Centeredness

As noted above and described within the User’s Guide, patient registries may be designed to fulfill many purposes, and not all registries are designed to be patient-centered. However, several features of registry design and implementation may further strengthen the extent to which the registry is patient-centered. A full review of these and other features of registry design and best practices for their implementation may be found in the third edition of the AHRQ Registries Guide.¹ A brief summary of these key features is presented in Table 1-2 below.

Table 1-2

Features of registries that enhance patient-centeredness.

Patient Perspectives on Registry Participation

Recently, several studies were conducted with different patient focus groups to understand their views on patient registries; the methods and results of these studies are published in more detail elsewhere.²⁴ The opinions of three targeted patient groups were solicited: those already enrolled in a chronic disease registry, those with chronic illnesses not enrolled in a registry, and a similar group with chronic arthritis who spoke only Spanish and had never been approached about enrolling in a registry. In total, 23 patients from the three groups described above participated in 60-minute facilitated discussion sessions. The following themes, presented in italics, emerged from these discussions.

Convenience: In all three focus groups, patients emphasized the need for the logistics of participation to be convenient. However, there was wide variation in what was considered convenient. Some patients preferred that study visits coincide with clinical appointments, while others preferred the opportunity to participate on a more flexible schedule with mailed surveys. Patients also expressed a range of views regarding the acceptable frequency of participation, as well as the desire to interact with research staff. Spanish-speaking patients indicated that translated research materials would facilitate their involvement, a consideration that would extend beyond Spanish-speaking patients in the United States, to the overall desirability of having research materials available in the native language for any registry participants. Improving the convenience of registry participation may play an important role in patient responsiveness and retention, as well as increase the likelihood of achieving the registry objectives.

Motivation(s) to participate: Motivations such as altruism and the desire to help others through research were discussed, and have similarly been documented in other studies.^25–27

Another motivation of focus group patients to participate in registries was in using registries as an opportunity to connect with other patients, and creating or joining a patient community. Focus group participants who were current members of a registry described specific features that the registry provided, including educational sessions and live feedback opportunities that allowed interaction with investigators and other patients and supported a sense of community. Social support may increase motivation and engagement among registry participants through sharing of lived experiences with their shared condition or treatment.

Focus group participants also wished to receive longitudinal information on their own disease/treatment activity compared with other patients in the aggregate cohort. This information could be provided by the registry directly or through a patient’s health care provider. However, considerations of how to present such information – graphically or in tables – and when it should be presented – at clinical visits, registry visits, or in the mail – must be weighed carefully. Additionally, disseminating this information should be weighed against the possibility of modifying patient or physician behavior as a result of registry participation, and what type of support is appropriate in interpreting the information shared with patients.

Perceived relevance to their experience: Questionnaires that are perceived as relevant by patients may increase their interest in enrolling in and staying involved with a registry. Focus group participants suggested that patients want to communicate their “lived experience” with their condition, and that researchers should ask about the non-physical aspects of disease, such as mental and emotional health. In that context, they described the experience of answering certain types of questions as having “therapeutic” benefit. Focus group patients also indicated that questions of unclear relevance were an annoyance and might detract from their motivation to participate. Patients wished to see questions and issues of concern to them reflected in registry data collection, and indicated that in the case of poorly understood questions or assessments, linking these to the registry’s objectives would be helpful. Essentially, patients wanted to know that the information they provide is useful in addressing registry objectives and is being interpreted accurately by researchers; this finding in itself was an endorsement of the need for patient engagement in registry planning, implementation, and dissemination of results.

Privacy and trust: Multiple patients shared the concern that participation in a registry could pose a risk to privacy and confidentiality of their health information. This concern has been shared by other patient groups from previous research as well.²⁶ Feelings of distrust were especially pronounced among the Spanish-speaking patients, who were afraid they would be treated like “guinea pigs.” This was consistent with prior studies suggesting a general distrust towards research in minority populations.^28–30

Challenges to Gathering Patient Perspectives During Registry Design

Stakeholder engagement is vital when new registries are being developed or existing registries are being repurposed. Such engagement can provide a path through common challenges, described below.

• Maintaining a reasonable, patient-centric focus: It is important that the registry’s mission and objectives are clearly defined. This allows the registry to maintain a reasonable scope of data collection while ensuring that the data collected can answer questions of importance to stakeholders.
• Developing clear and ethical operating procedures and processes: When establishing a registry (or in the early stages of launching), it is important to develop formal processes that prompt the inclusion of patient and caregiver perspectives. This systematic, integrated approach helps encourage long-term, meaningful participation by patients and caregivers and mitigate against the pitfall of “endorsement engagement” (i.e., patients invited only to validate a pre-determined concept). In addition, patients, caregivers, clinicians, and other stakeholders may have diverse priorities for how their data are used by the registry holder, preferences about types and number of research contacts they receive, and preferences about when and with whom their data is shared.
• Creating a positive user experience: Data collection efforts and platforms must also be user-oriented and provide some incentive for data providers to engage and remain engaged over time. For longitudinal registries, it is especially imperative to build in education, support, and/or other community resources.

Systematically soliciting preferences in these areas comprises key initial stakeholder engagement and addresses common challenges of data collection and data access. The following overlapping strategies, presented in italics, may be effective ways to incorporate patient perspectives into registry formation and operations.

Establish a multi-stakeholder steering/leadership committee: Formation and maintenance of a governance body, such as a steering committee, board, or workgroup, is the most fundamental way to enable stakeholder feedback to inform the work of the registry. These committees may be composed of a mixed set of stakeholders, or of all-patients/caregivers. There are benefits and challenges to each approach.

For example, the “Arthritis Patient Partnership with Comparative Effectiveness Researchers” Registry (AR-PoWER PPRN) established a governance structure that includes an Executive Board, a Patient Governor Group (PGG), a Research Advisory Board (RAB), and a Corporate Advisory Board (CAB). The hierarchy of these governing bodies is shown below in Figure 1-1. The governance structure was developed in collaboration between investigators and patient community members. The PGG is the patient steering committee and is composed of 11 patients from a variety of professional, geographic, and demographic backgrounds with conditions relevant to the research mission of the registry. The PGG receives input from the wider patient community via online surveys and through a “Send Feedback” function on the registry’s Web site and mobile application.

Figure 1-1

Governance structure of the ARthritis Partnership with Comparative Effectiveness Researchers Registry (AR-PoWER PPRN). The Executive Board includes the Principal Investigator (PI) and one Co-PI, and the Executive Director and a Board Member of the Global (more...)

Early in the development of the registry, six highly engaged patients (CreakyJoints advocates and/or bloggers) helped define the role of the Patient Governors (PGs). In the first year, PGs participated in an in-person kick-off “Summit” and conference calls approximately once per month for registry updates and discussions in which they were asked to provide input and make decisions about the registry including initial feedback related to software development. As part of their training during the start-up period, PGs completed the Collaborative Institutional Training Initiative Human Subjects Protection training and the Cochrane Collaboration’s Evidence-Based Healthcare modules.

Define criteria for Patient Governor Selection (AR-PoWER):

• Experience advocating on behalf of rheumatologic conditions or for own diagnosis and/or treatment
• Facilitative and collaborative approach to group discussion and decision making
• Commitment to fostering representativeness across demographic factors (i.e., supportive of the need for the PGG & the patient-centric registry to reflect gender, age, race/ethnicity, regional diversity)
• Willingness to be a public face of rheumatology research
• Existing relationship and involvement with the patient advocacy organization (CreakyJoints)
• Skills necessary for patient-powered research (e.g., outreach/recruitment, public speaking, social media, information technology (IT), research design/analysis/dissemination)
• Dependable, committed and responsive

The DuchenneConnect PPRN uses a slightly different model. The highest level of oversight comes from the advocacy organization Parent Project Muscular Dystrophy (PPMD), the owner of the DuchenneConnect registry, through the PPMD Board of Directors and leadership. Thus, DuchenneConnect’s activities must be responsive to the mission and vision of PPMD. The DuchenneConnect registry was founded with direction from a large, multi-stakeholder advisory committee comprised of patients, caregivers, clinicians, translational scientists, and biopharmaceutical company scientists. This committee remains active in reviewing policies and providing scientific oversight.

Guidance in day-to-day operations comes from the smaller Leadership Committee made up of parents and individuals with Duchenne/Becker muscular dystrophy. Leadership Committee members were identified through a nominations process where individuals could self-nominate or identify a candidate. PPMD leadership and DuchenneConnect’s project staff selected the committee members to represent diverse experiences and skill sets. As the registry has grown and matured, the leadership committee has taken oversight over consent, data access, research prioritization, and engagement activities They meet in person at least once a year and participate in monthly conference calls. They engage in trainings to empower their participation in the network, while also providing reciprocal training to project staff based on their lived experience. Project staff are a diverse group of researchers, health care providers, patients, relatives, and advocates who meet at least weekly to manage the Registry and conduct community engagement. Since the project staff is involved in data collection, all staff complete human subjects research protections trainings. The DuchenneConnect project staff and Leadership Committee receive regular input from the Duchenne community from surveys, social media outreach, focus groups, and PPMD’s annual conference.

For both the AR-PoWER and DuchenneConnect PPRNs, leadership/advisory committee activities include a diverse set of activities such as establishment of a group charter and leadership rules, participation in research prioritization activities, data access and research collaboration guidance, selection of relevant PRO domains (i.e., symptoms such as fatigue) to be routinely captured for all registry participants, informed consent processes, guidance on disseminating research findings back to participants, and assistance in the development of documents to facilitate queries from patient members, research partners and external collaborators.

Challenges To Incorporating Patient Perspectives Over the Registry Life Cycle

Patient-centered registries require engagement that extends beyond the development phase. Common challenges across the registry life cycle are briefly described below.

Sustainable model for engagement of many registrants: Patient/caregiver-oriented committees offer one means of gathering regular input from patient representatives. However, it is also important to hear from a wider representation of patients, caregivers, or other stakeholders both anecdotally and systematically, via in-person or through online focus groups, one-on-one interviews, surveys, Webinars, social media communities, chat forums, or conferences. These approaches can also address another potential pitfall of expecting a small number of patients to speak for “all” others.

As previously noted in the section “Ability of Registries To Meet the Needs of Patient-Centered Research,” patients have requested that research networks offer opportunities for patients to connect with other patients within the registry. Participants may find support and benefit in sharing their experiences with others who are experiencing similar challenges to their health and economic, social, and emotional well-being. Supporting these relationships may help build a foundation for long-term registry engagement.

Patient advocacy groups can play additional roles in providing support opportunities and building a sense of community around registry data collection and research studies. Some methods include providing a forum for engagement about research priorities; social media interactions sharing information about the study through the enrollment, implementation, and dissemination phases; in-person and virtual social support; virtual “research club” interactions to review and discuss relevant peer-reviewed publications; authoring and distributing literature on care and management and the patient experience; and regional support groups.

Patient-responsive infrastructure: A serious challenge for many registries is developing an approach that is geared towards long-term participant involvement while reducing participant burden and ‘survey fatigue’. Asking patients to contribute data repeatedly over time is facilitated when registries offer something immediate and concrete to patient registrants in return. Additionally, in the longer-term, providing a report of how data were used also discourages survey fatigue. For AR-PoWER PPRN, a Web site and mobile application were developed that provide access to education and news, as well as a longitudinal personal health tracker (of symptoms or other PRO data) to facilitate health care decision-making. New features, such as a symptom journal, were later added to the app in response to input from patients. The DuchenneConnect registry offers a comparison of each registrant to aggregated data from the registry community, modular surveys with a smart-phone friendly interface, educational content related to the topic of the surveys, access to clinical experts and those in therapeutic development, summaries of all active clinical trials in Duchenne, and lay reports of results.

Challenges Associated With Representation of Minorities Among Patient Stakeholders

Minority participation is important for all registries and their associated research, because this representation touches on issues of equality and the elimination of health care and research disparities, which are core values for improving the health care of all individuals. It is critical to bear in mind historical precedents across the entire spectrum of clinical research when considering the participation of disenfranchised groups; the literature on minorities’ distrust, poorer access to health care, and other barriers to research study participation needs to be weighed.³² While achieving diverse representation is difficult in all registries, there are different types of challenges and approaches in rare and common disorder communities. We have found it useful to explore motivations and preferences for registry and research participation across different groups to understand how to be most inclusive. Moreover, inclusion of diverse patient groups will contribute to better understanding about treatment heterogeneity, which will benefit patients, health care providers and health systems.

Challenges in Responsiveness to Multiple Stakeholders’ Needs

Most registries address the interests of multiple stakeholders, and this should be conceptualized and explored as an opportunity rather than perceived as a challenge. Though participant engagement is vital in developing meaningful and acceptable research protocols, it remains the responsibility of scientific partners (e.g., academic, government, or industry researchers) to maintain the research rigor of a patient-centric registry, since the ultimate purpose of a registry is to conduct meaningful, reliable research that meets scientific standards. If appropriate methods are not used, it threatens the integrity of the research findings, which then cannot meet the needs of the patient community. At the same time, registries should provide clinically meaningful results that are important to the lives of patients and caregivers. To empower patients and caregivers to articulate their research priorities, scientific partners should strive to activate and build the capacity of multiple stakeholders to participate in research activities as discussed, in italics, below.

Activating a registry: Patients can play a fundamental role in research engagement and advocacy. Patients should be empowered and encouraged to identify their unmet needs, the health care decisions they make, and the information that is (or is not) available for them to make these decisions. Patients do not have to be able to articulate their needs and experiences as research questions, since the research team can assist in the translation. The research questions derived from such discussions can be prioritized by stakeholders and then voiced to decision makers.

Building capacity: The training and development of patient stakeholders to be full participants in research is critical. Different stakeholders have different needs, interests, and capabilities. Whereas many patient and caregiver partners have little or no familiarity with research, most providers have at least some experience conducting health research. Patient partners may need education about the scientific method, research design, and unfamiliar language or acronyms. Other stakeholders, such as providers or regulatory bodies, may also need training in how best to work in partnership with patient groups or representatives. Patient advocacy groups may be a valuable resource to assist providers with this training and to increase understanding of the common goals shared by all stakeholders. Within the registry community, all patients and caregivers benefit from higher-level education to understand general research principles, approaches, common benefits and risks of research participation, and questions to consider.

Challenges in Disseminating Registry Findings to Patients in a Useful Manner

Transparency about data use and research findings combats the perception of “helicopter research,” where participants perceive that researchers obtain data and then are never heard from again. Patient-centered registries have the opportunity to engage in a different approach where both registrants and their data are valued and respected. For example, the DuchenneConnect registry provides registrants with a report of when their data is used, and by whom and for what general purpose.

Once research is complete it is important to compose a research summary document that is easily accessible and understandable to the general public to prepare for dissemination of findings. The summary document may include an overview of key findings and a fact sheet in non-scientific terms. This summary document can be sent to all research participants along with a letter thanking them for their participation. Once finalized, findings can be further disseminated through at least two broad channels:

1. Patient organizations provide many opportunities for dissemination of results to the patient communities. Dissemination activities may include presenting study results to patients at face-to-face and online patient education events and Webinars, organization newsletter articles, blogs, press releases, and social media posts and/or conversations on Facebook™, Instagram™, Snapchat™, Periscope™, Twitter™ and YouTube™. These traditional and social media streams permit results to be disseminated repeatedly in a way that can enhance penetration into the patient community. Findings can also be shared via legislative advocacy and patient/provider mobilization activities.
2. Scientific meetings and conferences provide opportunities to present results via project-related issue briefs, slide presentation sets, and printed materials.

Data holders should prioritize dissemination in their data use agreements. DuchenneConnect, for example, requires in their data use agreements a lay report of findings, requests that a high-level summary be released prior to publication, provides a Webinar forum for results to be provided to the lay community, and is able to publish findings independently if researchers do not publish in a reasonable timeframe.

Ethical Challenges in Patient-Centered Registries

Though registry data collection is typically considered to be moderate or low risk, there are important ethical considerations. First, valid informed consent is essential. In an attempt to make participation in a research registry attractive to patient participants, the registry may be touted as a “health tracker app” or other tool. It is important to remind participants that their participation constitutes research and to give patients ways of opting out of participation to ensure that participation remains voluntary. Participants must understand why the data are being collected and the ways the data may be used. Transparency regarding access to the data, including whether patient reported data will be shared with their health care provider or health care insurer or if there is a mechanism for sharing of data with external researchers who apply for access should be provided. Also, part of registry planning should include consideration of what will happen to the data and any ongoing research when the registry ends, due to loss of funding or at a pre-planned time point. These policies should be clearly stated in the informed consent.

It is vital to engage patients, caregivers, and other stakeholders in developing a consent process that is robust and meaningful, yet flexible enough for participants so that they may participate to a large degree but opt out of some response options or data linkage or data sharing options that may otherwise discourage participation.

Finally, to support a culture change where patient and caregiver engagement becomes the norm, it is vital to engage patients and caregivers in developing systematic evaluation approaches to quantify and qualify outcomes of engagement. As in the development of any other research question, stakeholders should help identify meaningful outcomes associated with engagement activities and work with research teams to determine approaches to measurement. The results of the research should be disseminated through both lay and peer-reviewed venues.

PCORI Methodology Report and Related Guidelines

As mentioned in the section “Definitions of Patient-Centered Care and Patient-Centered Clinical Research,” PCORI led the development of a set of minimum standards for PCOR, summarized in the PCORI Methodology Report, which includes 47 standards that PCORI-funded research must adhere to, and also serves more broadly as guidance to other researchers.³³ Standards cover areas including formulating research questions, patient-centeredness and engagement, data integrity, statistical methods, and the design of registries and data networks. Specifically, for the design and features of registries, the PCORI Methodology Report provides standards for the following: the type, extent, and length of patient followup; transparency of data use agreements, informed consent agreements, and data security documentation for institutional review boards; data quality assurance via structured training of data abstractors, use of data quality checks, and procedures for data review and verification; documentation and explanation of any protocol modifications; consistent data collection with clear, operational definitions of data elements; systematic, unbiased patient enrollment; monitored and minimized loss to followup; and collection of data to address confounding.³³

AHRQ Registry User’s Guide Handbook

The User’s Guide, developed under a contract with the AHRQ in collaboration with the Centers for Medicare & Medicaid Services (CMS) through the Developing Evidence to Inform Decisions about Effectiveness (DEcIDE) Network of AHRQ’s Effective Health Care (EHC) Program, was first published in 2007 as a reference for “establishing, maintaining, and evaluating the success of registries created to collect data about patient outcomes.” Since then, the User’s Guide has been subsequently updated with additional information and new chapters on emerging and expanding areas of science impacting registries. Ongoing updates ensure a comprehensive reference source for researchers and other stakeholders involved in registry planning, design and implementation. As noted earlier, this document is an eBook supplement to the third edition of the Registry User’s Guide.¹

Inventory of Registry Networks and the Registry of Patient Registries (RoPR)

PCORI maintains a comprehensive inventory of research networks, including the PPRNs and Clinical Data Research Networks (CDRNs), which are part of PCORnet.³⁴ The report summarizes key findings about patient engagement for each type of network, creates a taxonomy for the two types of collaborative networks, and an inventory of all identified networks and registries.

The RoPR was designed and funded by AHRQ for the purpose of providing a searchable, central listing of registries, linked to ClinicalTrials.gov. Information including the purpose, design, disease area, outcomes is available for each participating registry on a voluntary basis, and the RoPR is intended to be of use to researchers seeking to develop a registry in a specific area, as well as to patients and other stakeholders seeking this information.

Patient-Advocate Organizations or Voluntary Health Agencies and Nonprofits

While there is not a single comprehensive source listing all active patient-advocate organizations in specific disease areas, the National Health Council does maintain a list of numerous patient-advocate organizations or “Voluntary Health Agencies” across a range of diseases and conditions.³⁵ Professional organizations and foundations dedicated to research and support of patients with specific conditions are additional sources of links to active patient advocacy groups.

The White Dress Project is an example of a nonprofit patient advocacy organization dedicated to raising the awareness of uterine fibroids and raising funds to support research on this condition. Its members, women living with uterine fibroids, find support and benefit in sharing their experiences with symptomatic uterine fibroids and the impacts fibroids have on their economic, social, and emotional well-being. These impacts include the financial burden of treatment, the daily emotional toll of symptoms, and frustration from lack of access to care, lack of broader education about the condition, lack of definitive research regarding comparative effectiveness of treatment options, and dissatisfaction with the available treatment options. The White Dress Project advocates to address the previously described challenges of “helicopter research,” as it is felt that very little research on fibroids is disseminated effectively and clearly to patients. Members of The White Dress Project attend conferences, join forums and make efforts to directly connect with researchers, scientists and physicians to serve as a vehicle for sharing the latest information on the condition and treatment options with the patient community. Additionally, the White Dress Project has advocated for legislation in multiple states, to declare July as Fibroids Awareness Month and works to communicate the importance of continued education and awareness to legislators through legislative study committees and forums.³⁶