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Role of the Patient-Centered Outcomes Research Institute in Addressing Disparities and Engaging Patients in Clinical Research

  • Romana Hasnain-Wynia
    Correspondence
    Address correspondence to: Romana Hasnain-Wynia, PhD, Addressing Disparities Program, Patient-Centered Outcomes Research Institute, 1828 L Street NW Floor 9, Washington, DC 20036
    Affiliations
    Director, Addressing Disparities Program Patient-Centered Outcomes Research Institute Washington, DC 20036
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  • Anne C. Beal
    Affiliations
    Deputy Director and Chief Officer for Engagement Patient-Centered Outcomes Research Institute Washington, DC 20036
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      The performance of US health care is lagging, with unacceptable variations and disparities in care. These disparities lead to poor health outcomes in patients of racial and ethnic minorities; in those living in rural communities; in patients with disabilities and/or low income; and in lesbian, gay, bisexual, and transgender populations. Although achieving health equity for populations has been an explicit US policy goal for years, success in reducing disparities has been elusive. Still, after years of documenting the existence of disparities in health and health care, the research enterprise has begun to move beyond documentation with a focus on improving health outcomes for underserved populations.
      Simultaneously, in an effort to improve patient outcomes generally, there is an important movement underway with a focus on integrating patients into all aspects of health care. Examples of patient engagement include shared decision-making in clinical care, involvement in patient safety and quality improvement initiatives, and being a key player in patient-centered outcomes research (PCOR).
      In drug development, patients are intensely committed to playing a more active role in the assessment of the efficacy and tolerability of new drugs. Although there has been some experience with patient partnerships and engagement in clinical activities, the inclusion of patients in health care research—in ways other than as research subjects—has been rare, which presents challenges but also opportunities for meaningful innovation. Some of the advantages and challenges of engaging patients in research are amplified when the research in question is addressing disparities. Community-based participatory research (CBPR) approaches have provided an excellent foundation on which to build future patient-engagement efforts. CBPR models that integrate community, academic, and industry partners in a range of activities from protocol design through dissemination of study findings have also shown potential for increasing recruitment of underserved populations into biomedical research and for making the outcomes of the research itself more meaningful.
      • Greiner K.A.
      • Friedman D.B.
      • Adams S.A.
      • et al.
      Effective recruitment strategies and community-based participatory research: community networks program centers’ recruitment in cancer prevention studies.

      Why Engage Patients in Research?

      Active efforts are underway to engage patients, families, and caregivers in setting research agendas, determining which research to fund, conducting research, and disseminating research results.
      • Fleurence R.
      • Selby J.V.
      • Odom-Walker K.
      • et al.
      How the Patient-Centered Outcomes Research Institute is engaging patients and others in shaping its research agenda.
      The basic goal of these engagement efforts is to ensure that health care research goes beyond what has been its traditional model of seeking answers to questions important only to researchers and funders. Although health research has generally been conducted with a view toward benefiting patients, the old model was driven by investigators who conceived, designed, implemented, and even disseminated research findings largely without direct or explicit input from patients and those who care for them.
      This reflected a model of health care delivery that also often did not seek to engage patients as partners in their care. But there has been a growing awareness of the need, and right, of patients to be directly involved in medical decision making. Many patients and advocates have adopted the view that, in health care, there must be “nothing about us without us.”
      • McLure K.
      • Kosky M.
      Nothing about us without us.
      In particular, among groups that experience health disparities, there is reason to believe that improved patient engagement might help to alleviate these disparities.
      Similarly, the involvement of patients in research might be of particular value among underserved populations who experience poor health outcomes because of language, cultural, socioeconomic, and other barriers. Individuals from these groups have unique experiences, perspectives, and understanding of the myriad barriers they may face in achieving optimal health outcomes. Reviews of research paradigms that engage patient communities that experience disparities have shown that the research results coupled with engagement can lead to policies and practices that promote health equity.
      • Minkler M.
      Linking science and policy through community-based participatory research to study and address health disparities.
      Just as with CBPR and its growing use, patient engagement raises important ethical issues: securing informed consent (at the individual and patient-community level) and demonstrating respect for individual (and community) autonomy. This is especially important because patient engagement may be embedded within the broader context of community engagement.

      The PCORI Mandate

      The Patient-Centered Outcomes Research Institute (PCORI) is helping to drive new thinking about engaging patients in every phase of research. PCORI was authorized as part of the Affordable Care Act, section 6301, to fund research that will assist patients, caregivers, clinicians, payers, and policymakers in making informed health decisions.
      A key objective of PCORI is to help people to make informed health care decisions by supporting research that is “done differently” and integrates patients, families, caregivers, and other important stakeholders throughout the continuum of the research process. To have the greatest societal and health care impact, PCORI prioritizes and funds research that meets several key criteria, including whether the results will likely change practice and whether the research addresses an important question from the perspective of patients, their caregivers, and their clinicians.
      • Selby J.V.
      • Beal A.C.
      • Frank L.
      The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda.

      Engaging Patients in Setting the Research Agenda

      The PCORI board identified 5 national priorities for research in 2012
      • Selby J.V.
      • Beal A.C.
      • Frank L.
      The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda.
      : (1) assessing options for prevention, diagnosis, and treatment; (2) improving health care systems; (3) addressing disparities; (4) communicating and disseminating research; and (5) improving PCOR methods and infrastructure. Each program at PCORI has a multi-stakeholder advisory panel of up to 21 members representing a range of perspectives. The Advisory Panel for the Addressing Disparities program helps to guide the program’s missions, which are to reduce disparities in health care outcomes and to advance equity in health and health care, while its guiding principle is to fund studies that identify the best options for eliminating disparities rather than just describing them. The Engagement Team works with the Advisory Panel to ensure that PCORI’s research is guided by the perspectives of patients, caregivers, clinicians, and other health care stakeholders.

      Engaging Patients in Conducting Research

      PCORI’s work addresses both a national and global focus on patient engagement. The health care sector is facing emerging challenges in translating biomedical advances into efficient, safe, and affordable therapies for patients worldwide, and patients are paying attention to playing a more active role in the assessment and tolerability of new drugs and other therapies.
      • Goldman M.
      The Innovative Medicines Initiative: a European response to the innovations challenge.
      There is a strong consensus among an array of stakeholders on the need for establishing innovative means to enhance collaboration among patients, families, clinicians, industry, academia, and pharmaceutical and biotech companies to improve outcomes.
      • Goldman M.
      The Innovative Medicines Initiative: a European response to the innovations challenge.
      Key considerations propel the need for engagement models that fully integrate patient participation in the research enterprise. For example, an article by Selby et al
      • Selby J.V.
      • Fleurence R.
      • Lauer M.
      • Scheneeweiss S.
      Reviewing hypothetical migraine studies using funding criteria from The Patient-Centered Outcomes Research Institute.
      used a hypothetical case to underscore the importance of patient and other stakeholder collaboration in clinical trials, especially when considering diverse populations, heterogeneity of treatment effects, and disparities. In this hypothetical case, after approval from the US Food and Drug Administration and marketing of 1 or more pharmaceutical agents, observational comparative effectiveness studies using data from routine clinical settings produced results not found in randomized efficacy trials conducted before the drug was approved. Selby et al
      • Selby J.V.
      • Fleurence R.
      • Lauer M.
      • Scheneeweiss S.
      Reviewing hypothetical migraine studies using funding criteria from The Patient-Centered Outcomes Research Institute.
      posited that the randomized efficacy trial did not take into consideration the differential effectiveness of the drug in various population subgroups. As they suggested, these types of findings warrant communication with patients and family members, caregivers, and clinicians. Had there been involvement of subpopulations in the trials from the start, this discrepancy could have been prevented. Communicating these results to patients also requires effective engagement to reach the target populations with tailored messaging to enhance understanding.

      Engaging Patients in New Discovery

      The integration of patients into the research enterprise is being adopted by the US Food and Drug Administration (FDA), which has launched different initiatives to promote patient engagement. This is happening at a time when drug developers are grappling with the impact of personalized medicine, and appropriate companion diagnostic strategies, on their portfolios.
      • Agarwal A.
      Do companion diagnostics make economic sense for drug developers?.
      Because there remain many unanswered questions in this area, the need to engage patients in this dialogue now is crucial.

      American Medical Association. Policy perspective on personalized medicine. http://www.ama-assn.org/resources/doc/washington/policy-perspective-on-personalized-medicine.pdf. Accessed March 30, 2014.

      The Patient-Focused Drug Development initiative is working to gather patients’ perspectives on their conditions and the therapies to treat those conditions. The FDA is holding a series of public meetings related to different conditions to gain patient insights that may help with drug development.

      US Dept of Health and Human Services, Food and Drug Administration. Patient-focused drug development: disease area meetings planned for fiscal years 2013–2015. http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm. Accessed April 16, 2014.

      Other efforts for eliciting patient input at the FDA include a public workshop titled The Patient Preference Initiative: Incorporating Patient Preference Information into the Medical Device Regulatory Processes, which was developed to inform the Center for Devices and Radiological Health regulatory decision making by including patient preferences on the benefit–risk tradeoffs of medical devices.

      US Dept of Health and Human Services, Food and Drug Administration. Public workshop—the Patient Preference Initiative: incorporating patient preference information into the medical device regulatory processes, September 18–19, 2013. http://www.fda.gov/medicaldevices/newsevents/workshopsconferences/ucm361864.htm. Accessed April 16, 2014.

      We applaud this work and believe that giving patients a voice in shaping the agenda for new drug discoveries will promote responses of those new therapies to patient needs. However, from the perspective of disparities, this initiative provides an ideal opportunity for a call for action to ensure the participation of underserved populations to meet their needs.

      Research Networks for All: Pcornet

      One of the biggest challenges in designing clinical trials is engaging underserved populations. PCORI is actively investing in efforts to facilitate engagement at all levels of the research enterprise through its advisory panels, workshops, roundtables, merit review processes, and integration of patients and other important stakeholders throughout the conduct of the research itself, with particular attention to ensuring the inclusion of underserved patient populations in those efforts. Furthermore, PCORI maintains a focus on including heterogeneous patient populations in data collection to ensure that the research they support is able to include subpopulation analyses, whether it targets the elderly or racial and ethnic minorities. As an example, in December 2013, PCORI approved $93.5 million to support 29 clinical research data networks that together will form an ambitious new resource for research known as PCORnet (National Patient-Centered Outcomes Research Network).

      Patient-Centered Outcomes Research Institute. PCORnet: The National Patient-Centered Clinical Research Network. http://www.pcori.org/funding-opportunities/pcornet-national-patient-centered-clinical-research-network/. Accessed April 16, 2014.

      By integrating data available in the 29 individual networks, PCORnet aims to provide access to a large amount of diverse, nationally representative health information that can support a range of study designs. PCORnet will integrate data from 11 clinical data research networks (networks that include at least 1 million patients and originate in health care systems such as hospitals, health plans, and practice-based networks and that securely collect real-time, “real-world” health information during the routine course of patient care) and 18 patient-powered research networks (those operated and governed by groups of patients and their partners who are focused on a particular condition and interested in sharing health information and participating in research). Because one of the national priorities for PCORI’s research is Addressing Disparities, PCORI wants to ensure that the networks has a specific focus on addressing disparities. To this end, below are descriptions of networks with a disparities focus.
      The Louisiana Public Health Institute, which will work with several New Orleans health systems and academic institutions to address health disparities specifically in the areas of obesity, diabetes, sickle cell disease, and a selection of 10 rare cancers. Their work stemmed, in part, from the need to rebuild the destroyed safety net health care system after Hurricane Katrina.
      The Oregon Community Health Information Network is partnering with Health Choice Network, so collectively, they will have the ability to combine data and potentially conduct research with nearly 100 safety net health systems (representing 744 clinics). They are focusing on co-occurring HIV and hepatitis C virus, diabetes, and obesity.
      Harvard’s clinical data research network is bringing in Boston Health Net (a safety net provider) as well as Columbia University Medical Center, Morehouse/Grady School of Medicine, and the University of Mississippi Medical Center. Extending Harvard’s mature and robust technical capabilities to these sites is intended to speed and enable research in sites that care for predominantly underserved populations.
      Chicago’s application (led by the Chicago Community Trust) has a focus on asthma and discusses in their application the disproportionate impact of asthma in the African-American communities that will be served by some of their participating health systems.
      Similarly, the consortium in New York includes the Clinical Directors Network, another practice-based research network for clinicians providing care in low-income, minority, and other underserved communities.
      The era of patient engagement, partnership, and empowerment in health care has finally come. Whether we are partnering with patients to ensure their safety in health care settings or to develop research agendas that respond to their needs, the inclusion of patients in these discussions can only help to build a health care system that is more responsive to their needs. However, as initiatives roll out to include patients as partners, we need to keep in mind that not all patients have been able to secure a seat at the table. Historically, underserved patient populations have been challenged to gain access to health care and to achieve optimal health outcomes. We need to make sure we do not perpetuate this exclusion by not ensuring targeted outreach and inclusion of patients in engagement activities who have not been previously well-represented. Furthermore, robust engagement of underserved patient populations should include recognizing that all types of patients experience a range of issues, and we need to include a range of perspectives. Lastly, the issue of disparities continues to block us from achieving levels of high performance in the health care system. We can collectively work to improve care and research, but we should always maintain a specific focus on eliminating disparities within that work. The ethos of “nothing about us without us” is crucial for engagement of patients in the general population, but even more so for patients who have not been previously well-served or engaged as partners in health care.

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      1. American Medical Association. Policy perspective on personalized medicine. http://www.ama-assn.org/resources/doc/washington/policy-perspective-on-personalized-medicine.pdf. Accessed March 30, 2014.

      2. US Dept of Health and Human Services, Food and Drug Administration. Patient-focused drug development: disease area meetings planned for fiscal years 2013–2015. http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm. Accessed April 16, 2014.

      3. US Dept of Health and Human Services, Food and Drug Administration. Public workshop—the Patient Preference Initiative: incorporating patient preference information into the medical device regulatory processes, September 18–19, 2013. http://www.fda.gov/medicaldevices/newsevents/workshopsconferences/ucm361864.htm. Accessed April 16, 2014.

      4. Patient-Centered Outcomes Research Institute. PCORnet: The National Patient-Centered Clinical Research Network. http://www.pcori.org/funding-opportunities/pcornet-national-patient-centered-clinical-research-network/. Accessed April 16, 2014.