Brief Report
The Patient-Centered Outcomes Research Institute Research Done Differently
Robin Newhouse ▼ Debra J. Barksdale ▼ Julie Ann Miller
Background: The Patient-Centered Outcomes Research Institute (PCORI) was authorized by the Patient Protection and Affordable Care Act of 2010 to fund comparative clinical effectiveness research to provide reliable evidence to help patients and their healthcare providers make informed decisions. Objective: The aim of this paper is to describe the synergy between nursing research and PCORI funding priorities, discuss unique aspects of PCORI funding, and identify the implications for nurse researchers. Discussion: Goals of nursing research are well aligned with PCORI interests. Given this synergy, many phenomena that nursing scientists study could become topics of a competitive proposal for PCORI funding. Major aspects of PCORI funding with implications for nurse researchers include the nature of the questions asked, funding priorities, engagement of patients and stakeholders, compliance with the PCORI’s methodology standards, and the proposal review criteria and process. Key Words: clinical research comparative effectiveness research nursing research Patient-Centered Outcomes Research Institute PCORI Nursing Research, January/February 2015, Vol 64, No 1, 72–77
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espite impressive advances in science, there is a lag in the translation of research findings into improved care for patients. In many cases, researchers have not effectively addressed the crucial problems that patients and clinicians face, or solutions have not been practical in reallife situations. In other cases, results of exemplary studies fail to shape clinical practice because of clinician biases and divergent financial incentives (Timbie, Fox, Van Busum, & Schneider, 2012). The Patient-Centered Outcomes Research Institute (PCORI) was authorized by the Patient Protection and Affordable Care Act (PPACA) of 2010 to fund research to provide reliable evidence to help patients, caregivers, clinicians, and other healthcare stakeholders make informed decisions. By increasing the quality, quantity, and timeliness of useful information available to support health decisions, PCORI will speed the implementation of patient-centered, evidence-based knowledge. To ensure that the funded projects address questions and outcomes crucial to the healthcare community, all activities at
Robin Newhouse, PhD, RN, NEA-BC, FAAN, is Professor and Chair, Department of Organizational Systems and Adult Health, University of Maryland School of Nursing, Baltimore. Debra J. Barksdale, PhD, FNP-BC, FAANP, FAAN, is Professor and Director, Doctor of Nursing Practice Program, and Robert Wood Johnson Foundation Executive Nurse Fellow Alumnus, School of Nursing, University of North Carolina at Chapel Hill. Julie Ann Miller, PhD, is Senior Editor, Patient-Centered Outcomes Research Institute, Washington, DC. DOI: 10.1097/NNR.0000000000000070
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PCORI and in the research that it funds involve meaningful input and engagement from patients and other healthcare stakeholders. In addition, central components of the PCORI vision include transparency and scientific rigor. In its first 4 years, PCORI has established innovative mechanisms for funding and conducting research that engages patients and other healthcare stakeholders to provide answers to the questions most important to them (Selby & Lipstein, 2014). PCORI funds comparative clinical effectiveness research (CER) designed to provide information that would inform critical decisions that face patients and other stakeholders, including policymakers, payers, and leaders of healthcare systems. Studies of interest assess the benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions, highlighting the comparisons and outcomes (such as function, symptoms, healthrelated quality of life, survival) that matter most to people. The research incorporates a wide variety of settings and participants, taking into account an individual patient’s preferences and needs. It investigates how to optimize those outcomes, while addressing such considerations as the burden to individuals, and resource availability. To facilitate more efficient CER that could significantly increase the reliable information available to healthcare decision makers and the speed at which it is generated, PCORI has invested more than $100 million in the development of PCORnet: the National Patient-Centered Clinical Research Network. This Nursing Research January/February 2015 • Volume 64 • Number 1
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Research Done Differently: PCORI
Nursing Research • January/February 2015 • Volume 64 • No. 1
large, highly representative national network will establish a resource of clinical data gathered in real-world settings. Nurses—with their in-depth experience in working with patients and interdisciplinary healthcare teams—are a natural fit to lead (as principal investigators) and contribute to (as coinvestigators or team members) PCORI-funded projects as well as to suggest research topics and participate in review of applications and other PCORI activities. Currently, PCORI has two nurses in leadership positions: one on the Board of Governors (Dr. Barksdale) and the other on the Methodology Committee (Dr. Newhouse). The purpose of this article is to introduce the synergy between nursing science and PCORI funding priorities and provide nurse researchers with information needed to submit proposals for PCORI-funded projects. To this end, we describe unique aspects of PCORI funding and explore the implications for nurse researchers.
Nursing and PCORI Research Agenda Much synergy exists between nursing and PCORI research priorities. PCORI has made research awards to more than 15 nurses serving as principal investigators, and more than 50 nurses play major roles in the projects. A nurse also leads the Rare Epilepsy Network, which is one of the components of PCORnet. Nursing research generates knowledge to (a) build the scientific foundation for clinical practice, (b) prevent disease and disability, (c) manage and eliminate symptoms caused by illness, and (d) enhance end-of-life and palliative care (National Institutes of Health, n.d.). Driven by some of the most pressing health needs (HealthyPeople.gov, 2014), nursing research focuses on individual and population health determinants ranging from bench science (e.g., genetic factors and personalized medicine) and physiological and biobehaviorial mechanisms of disease to effectiveness, implementation, and dissemination studies (American Association of Colleges of Nursing, 2006). Table 1 provides a comparison of nursing research goals and PCORI activities. It is also important to note that there are some concepts that are important in patient-centered outcomes research (PCOR) that nurses internationally consider to be central in both care delivery and research. The first is patient-centeredness, and the second is patient engagement. The concept of patient-
centeredness relates to a partnership among the clinicians, patients, and their families, ensuring that decisions respect patient’s wishes, needs, and preferences and that they have the education and support required for decision making and participation in their own care (National Research Council, 2001). The engagement of patients goes one step further, describing “actions individuals must take to obtain the greatest benefit from the healthcare services available to them” (Center for Advancing Health, 2010). Professional nursing organizations have adopted both patient-centeredness and patient engagement as basic principles in nursing practice (Sofaer & Schumann, 2012). Research in the areas of patient engagement and activation in care (Hibbard, Stockard, Mahoney, & Tusler, 2004) has begun to demonstrate improved patient outcomes. Patient engagement in research—not as a patient or subject but as a partner—is a novel intervention to improve the quality, relevance, and transferability of study results. Patient engagement in research is an emerging field where PCORI research is underway to identify the best methods (Domecq et al., 2014).
Unique Attributes of PCOR There are five major aspects of PCORI’s approach that differ from other funders: • • • • •
the nature of the questions asked, how funding priorities are established, engagement of patients and the healthcare community, compliance with the PCORI Methodology Standards, and the proposal review process and criteria.
Each will be described further, with the implications for nurse researchers.
The PCORI Questions Research questions that underlie PCOR are generated through the lens of the people who are the target population of interest. For example (PCORI, 2013): • Given my personal characteristics, conditions, and preferences, what should I expect will happen to me? • What are my options, and what are the potential benefits and harms of those options? • What can I do to improve the outcomes that are most important to me? • How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare?
TABLE 1. Comparison of Nursing Research Goals and Patient-Centered Outcomes Research Institute Activities Nursing researcha Build the scientific foundation for clinical practice Prevent disease and disability Manage and eliminate symptoms caused by illness
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Patient-Centered Outcomes Research Institute Produces dependable information that can inform health decisions Provides evidence that guides decisions about prevention of disease Provides evidence to improve outcomes that patients care about, such as function, symptoms, health-related quality of life, and survival
a
Source: National Institute of Nursing Research (n.d.).
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Research Done Differently: PCORI
Generation of study research questions and hypotheses, while considering the patients’ point of view, is beneficial in two ways. First, it points the study development in the direction of designs and methods to best address patients’ greatest concerns. Second, it forces the research team to articulate clearly the decisions that results will affect. Whether addressing issues related to individuals, aggregates, populations, or healthcare systems, nurses are often on the front line of interactions with patients and caregivers. This gives nurses unique perspectives related to what matters most to the patient and those who care for and about them. Nurses hear the questions that matter most to the people that they care for. Thus, nurses are in a unique position to help PCORI identify the important questions and issues for patients. Nurse researchers partnering with clinicians can submit proposals addressing these patient-centered issues.
Funding Areas One of the first responsibilities of PCORI was to develop National Priorities for Research to guide the funding of CER. These priorities were to be cross-cutting areas where additional health research is needed to give patients and those who care for them more information to support decision making. A workgroup set up by the Board of Governors deliberated, made presentations, invited comment from stakeholder groups and the public, and refined its recommendations. Eventually, five priorities were identified and accepted by the Board of Governors (see Figure 1). Within any priority area, studies may focus on specific diseases, conditions, and interventions—or they may examine issues that apply across multiple diseases and conditions. In addition to funding research proposed by investigators within these areas, PCORI generates and prioritizes research topics related to questions contributed by patients and other stakeholders through its web site, workshops, and roundtables and similar efforts undertaken by others. These questions are
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reviewed to ensure they address an important research gap, assessed and ranked according to PCORI criteria (see Figure 2), and submitted to the Board of Governors for consideration as the subject of targeted (topic-specific) funding announcements. This process is guided by special workgroups and advisory panels selected through public nominations. There are advisory panels on six topics (Assessment of Prevention, Diagnosis, and Treatment Options; Improving Healthcare Systems; Addressing Disparities; Patient Engagement; Clinical Trials; and Rare Disease). There are currently 11 nurses on PCORI advisory panels.
Patient and Stakeholder Engagement Engagement of patients and stakeholders is a basic tenet for PCOR. This approach recognizes that, when researchers partner with healthcare stakeholders, they are best positioned to identify the compelling questions that underlie a truly patient-centered research agenda. PCORI-funded studies have patients and other stakeholders involved in the development of the research and its conduct, governance, oversight, and dissemination strategy. Funded projects engage patients and community members at all stages—from generating the initial ideas to sharing study results. In some cases, clinical researchers enlist patients from their practices, local community groups, or advocacy organizations. On the basis of PCORI’s experience with patient engagement in research, an engagement rubric has been developed (PCORI, 2014a). Sample engagement plans have also been posted on the web site to provide examples for investigators (http://www.pcori.org/assets/2013/11/PCORISample-Engagement-Plans.pdf). There are also other models for patient engagement approaches that may be helpful (Mullins, Abdulhalim, & Lavallee, 2012). Finding potential partners is sometimes a challenge to researchers who have not engaged patients and other healthcare
FIGURE 1. National priorities for PCORI-sponsored research. Used with the permission of the Patient-Centered Outcomes Research Institute. 2012–2014, Patient-Centered Outcomes Institute. More information at www.pcori.org.
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FIGURE 2. PCORI criteria for evaluating research topics. Used with the permission of the Patient-Centered Outcomes Research Institute. 2012–2014, Patient-Centered Outcomes Institute. More information at www.pcori.org.
stakeholders in past studies. PCORI has sponsored contests in which developers create matchmaking applications (PCORI, 2014b). PCORI has developed competitive engagement awards to assist potential applicants in developing their engagement partnerships and strategies prior to submitting for research funding (PCORI, 2014c). PCORI recently launched its Ambassador Program to help patients and other stakeholders, as well as organizations, share PCORI’s vision of PCOR with their communities, participate as full partners in research, and encourage the use of information generated from PCORI-funded projects. Four individual nurses and two nursing organizations (the Association of Perioperative Registered Nurses and the National Association of Nurse Practitioners in Women’s Health) are currently PCORI ambassadors.
Methodology Standards The PPACA established the PCORI Methodology Committee to develop and improve CER methods. As new methodological approaches emerge over time, minimal standards, in specific areas, are helpful in enhancing the quality of study design and methods. Higher-quality studies produce more reliable and trustworthy results that are more likely to be adopted into clinical practice. Within the first 18 months of its creation, the Methodology Committee contracted with teams of national experts through a competitive request for proposals to develop recommendations for the first set of standards. A set of 47 standards (see Figure 3) were refined and endorsed by the Board of Governors to guide PCORI-funded studies (PCORI Methodology Committee, 2013).The standards are organized into two categories: those relevant to all PCOR and those that apply to specific study designs or methods, such as data registries and networks, systematic reviews, and specific types of trials. All proposals for PCORI funding are required to address how the work will apply the methodology standards. PCORI
expects to add new standards in additional areas and revise the current ones—both as research methods evolve and in response to advice from the advisory panels, patients, other stakeholders, and the research community.
Proposal Review Two major aspects of PCORI’s review of proposals differ from other extramural funding processes. Some of PCORI’s criteria are unique, and PCORI includes patients and other stakeholders on the review team. Research proposals submitted to PCORI are reviewed using five criteria (those that are unique are designated with asterisks): • impact of the condition to be studied on the health of individuals and populations (for CER applications) or impact on the field of PCOR methods (for methodology applications), including whether the condition or disease is associated with a significant burden across the U.S. population or within a subgroup; • potential for the study to improve healthcare and outcomes (for CER applications) or PCOR methods (for methodology applications), including whether the proposed project addresses a critical gap in current knowledge that is important to patients, caregivers, or clinicians; • technical merit, including the qualifications of the team and the rigor of the research plan; • patient-centeredness, including whether the research measures outcomes important to patients; and • patient and stakeholder engagement, including evidence of patent and stakeholder engagement in the development of the proposal and proposed roles.
The role of patients and other stakeholders on the PCORI application review team is unique. The nonscientists participate fully and have an impact equal to that of the scientists. To achieve this, PCORI trains all the reviewers to understand its review criteria and to interact productively with the full range of reviewers, and it also provides mentors who give additional support to the nonscientists. There are currently
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FIGURE 3. Cross-cutting standards for patient-centered outcomes research established by PCORI. Used with the permission of the Patient-Centered Outcomes Research Institute. 2012–2014, Patient-Centered Outcomes Institute. More information at www.pcori.org.
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more than 60 nurses on PCORI Merit Review panels, and two other nurses are Merit Review mentors.
Goals and priorities for nursing research are highly aligned with those of PCORI. PCORI is interested in examining whether patient and other shareholder contributions to the review process lead to different decisions. To begin this exploration, PCORI staff examined outcomes of the first round of the reviews for research funding. They analyzed the scores that different types of reviewers—scientist, patients, other stakeholders— had assigned to proposals before and after face-to-face discussion. The evaluation concluded that patient and stakeholder reviewers add different perspectives to the review process, and in-person discussion led to closer agreement (Fleurence et al., 2014). Of the 25 proposals selected for award by the full review process, only 13 had ranked in the top 25 of a preliminary review by scientists alone.
Conclusions Goals and priorities for nursing research guiding high-quality clinical practice, preventing disease and disability, and managing and eliminating symptoms caused by illness are highly aligned with those of PCORI. Given this synergy, many phenomena that nurses study could be developed into a competitive proposal for funding. Development of proposals will require attention to the unique aspects of PCORI funding, which include the nature of the questions asked, funding priorities, engagement of patients and stakeholders, compliance with the PCORI methodology standards, and proposal review criteria. Nurses are central to guiding patients as they make some of their most difficult decisions. Nurse researchers are in a unique position to focus on what evidence is needed to produce the outcomes important to their patients.
Accepted for publication October 2, 2014. The authors acknowledge Robin Newhouse was appointed to the Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee, Debra Barksdale was appointed to the PCORI Board of Governors, and Julie Miller is employed by PCORI. Corresponding author: Robin Newhouse, PhD, RN, NEA-BC, FAAN, University of Maryland School of Nursing, 655 West Lombard Street, Suite 316, Baltimore, MD 21201 (e-mail: [email protected]).
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REFERENCES American Association of Colleges of Nursing. (2006). AACN position statement on nursing research. Retrieved from http://www .aacn.nche.edu/publications/position/NsgResearch.pdf Center for Advancing Health. (2010). A new definition of patient engagement: What is engagement and why is it important. Washington, DC: Author.org. http://www.cfah.org/file/CFAH_ Engagement_Behavior_Framework_current.pdf Domecq, J. P., Prutsky, G., Elraiyah, T., Wang, Z., Nabhan, M., Shippee, N., . . . Murad, M. H. (2014). Patient engagement in research: A systematic research. BMC Health Services Research, 14, 89. doi:10.1186/1472-6963-14-89 Fleurence, R. L., Forsythe, L. P., Lauer, M., Rotter, J., Ioannidis, J. P. A., Beal, A., . . . Selby, J. V. (2014). Engaging patients and stakeholders in research proposal review: The Patient-Centered Outcomes Research Institute. Annals of Internal Medicine, 161, 122–130. doi:10.7326/M13-2412 HealthyPeople.gov. (2014). 2020 topics & objectives—Objectives A–Z. Retrieved from http://www.healthypeople.gov/2020/topicsobjectives2020/ default.aspx Hibbard, J. H., Stockard, J., Mahoney, E. R., & Tusler, M. (2004). Development of the Patient Activation Measure (PAM): Conceptualizing and measuring activation in patients and consumers. Health Services Research, 39, 1005–1026. doi:10.1111/j.1475-6773.2004. 00269.x Mullins, C. D., Abdulhalim, A. M., & Lavallee, D. C. (2012). Continuous patient engagement in comparative effectiveness research. JAMA, 307, 1587–1588. doi:10.1001/jama.2012.442 National Institutes of Health. (n.d.). National Institute of Nursing Research. Retrieved from http://www.ninr.nih.gov National Research Council. (2001). Envisioning the national health care quality report. Washington, DC: National Academies Press. Retrieved from http://www.nap.edu/openbook.php?record_id=10073 Patient-Centered Outcomes Research Institute. (2014). Patient-centered outcomes research (PCOR). Retrieved from http://pcori.org/researchwe-support/pcor/ Patient-Centered Outcomes Research Institute. (2014a). PCORI patient and family engagement rubric. Retrieved from http://www.pcori .org/assets/2014/02/PCORI-Patient-and-Family-Engagement-Rubric.pdf Patient-Centered Outcomes Research Institute. (2014b). The PCORI matchmaking app challenge. Retrieved from http://www.pcori .org/funding-opportunities/challenge/ Patient-Centered Outcomes Research Institute. (2014c). Eugene Washington PCORI Engagement Awards. Retrieved from http://www .pcori.org/content/eugene-washington-pcori-engagement-awards Patient Protection and Affordable Care, 42 U.S.C. } 18001 (2010). PCORI Methodology Committee. (2013). The PCORI methodology report. Retrieved from http://www.pcori.org/assets/2013/11/PCORIMethodology-Report.pdf Selby, J. V., & Lipstein, S. H. (2014). PCORI at 3 years—Progress, lessons, and plans. New England Journal of Medicine, 370, 592–595. doi:10.1056/NEJMp1313061 Sofaer, S., & Schumann, M. J. (2012). Guiding principle for patient engagement. Washington, DC: Nursing Alliance for Quality Care. Timbie, J. W., Fox, D. S., Van Busum, K., & Schneider, E. C. (2012). Five reasons that many comparative effectiveness studies fail to change patient care and clinical practice. Health Affairs, 31, 2168–2175. doi:10.1377/hlthaff.2012.0150
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The Patient-Centered Outcomes Research Institute Research Done Differently
Robin Newhouse ▼ Debra J. Barksdale ▼ Julie Ann Miller
Background: The Patient-Centered Outcomes Research Institute (PCORI) was authorized by the Patient Protection and Affordable Care Act of 2010 to fund comparative clinical effectiveness research to provide reliable evidence to help patients and their healthcare providers make informed decisions. Objective: The aim of this paper is to describe the synergy between nursing research and PCORI funding priorities, discuss unique aspects of PCORI funding, and identify the implications for nurse researchers. Discussion: Goals of nursing research are well aligned with PCORI interests. Given this synergy, many phenomena that nursing scientists study could become topics of a competitive proposal for PCORI funding. Major aspects of PCORI funding with implications for nurse researchers include the nature of the questions asked, funding priorities, engagement of patients and stakeholders, compliance with the PCORI’s methodology standards, and the proposal review criteria and process. Key Words: clinical research comparative effectiveness research nursing research Patient-Centered Outcomes Research Institute PCORI Nursing Research, January/February 2015, Vol 64, No 1, 72–77
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espite impressive advances in science, there is a lag in the translation of research findings into improved care for patients. In many cases, researchers have not effectively addressed the crucial problems that patients and clinicians face, or solutions have not been practical in reallife situations. In other cases, results of exemplary studies fail to shape clinical practice because of clinician biases and divergent financial incentives (Timbie, Fox, Van Busum, & Schneider, 2012). The Patient-Centered Outcomes Research Institute (PCORI) was authorized by the Patient Protection and Affordable Care Act (PPACA) of 2010 to fund research to provide reliable evidence to help patients, caregivers, clinicians, and other healthcare stakeholders make informed decisions. By increasing the quality, quantity, and timeliness of useful information available to support health decisions, PCORI will speed the implementation of patient-centered, evidence-based knowledge. To ensure that the funded projects address questions and outcomes crucial to the healthcare community, all activities at
Robin Newhouse, PhD, RN, NEA-BC, FAAN, is Professor and Chair, Department of Organizational Systems and Adult Health, University of Maryland School of Nursing, Baltimore. Debra J. Barksdale, PhD, FNP-BC, FAANP, FAAN, is Professor and Director, Doctor of Nursing Practice Program, and Robert Wood Johnson Foundation Executive Nurse Fellow Alumnus, School of Nursing, University of North Carolina at Chapel Hill. Julie Ann Miller, PhD, is Senior Editor, Patient-Centered Outcomes Research Institute, Washington, DC. DOI: 10.1097/NNR.0000000000000070
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PCORI and in the research that it funds involve meaningful input and engagement from patients and other healthcare stakeholders. In addition, central components of the PCORI vision include transparency and scientific rigor. In its first 4 years, PCORI has established innovative mechanisms for funding and conducting research that engages patients and other healthcare stakeholders to provide answers to the questions most important to them (Selby & Lipstein, 2014). PCORI funds comparative clinical effectiveness research (CER) designed to provide information that would inform critical decisions that face patients and other stakeholders, including policymakers, payers, and leaders of healthcare systems. Studies of interest assess the benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions, highlighting the comparisons and outcomes (such as function, symptoms, healthrelated quality of life, survival) that matter most to people. The research incorporates a wide variety of settings and participants, taking into account an individual patient’s preferences and needs. It investigates how to optimize those outcomes, while addressing such considerations as the burden to individuals, and resource availability. To facilitate more efficient CER that could significantly increase the reliable information available to healthcare decision makers and the speed at which it is generated, PCORI has invested more than $100 million in the development of PCORnet: the National Patient-Centered Clinical Research Network. This Nursing Research January/February 2015 • Volume 64 • Number 1
Copyright © 2014 Wolters Kluwer Health | Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited.
Research Done Differently: PCORI
Nursing Research • January/February 2015 • Volume 64 • No. 1
large, highly representative national network will establish a resource of clinical data gathered in real-world settings. Nurses—with their in-depth experience in working with patients and interdisciplinary healthcare teams—are a natural fit to lead (as principal investigators) and contribute to (as coinvestigators or team members) PCORI-funded projects as well as to suggest research topics and participate in review of applications and other PCORI activities. Currently, PCORI has two nurses in leadership positions: one on the Board of Governors (Dr. Barksdale) and the other on the Methodology Committee (Dr. Newhouse). The purpose of this article is to introduce the synergy between nursing science and PCORI funding priorities and provide nurse researchers with information needed to submit proposals for PCORI-funded projects. To this end, we describe unique aspects of PCORI funding and explore the implications for nurse researchers.
Nursing and PCORI Research Agenda Much synergy exists between nursing and PCORI research priorities. PCORI has made research awards to more than 15 nurses serving as principal investigators, and more than 50 nurses play major roles in the projects. A nurse also leads the Rare Epilepsy Network, which is one of the components of PCORnet. Nursing research generates knowledge to (a) build the scientific foundation for clinical practice, (b) prevent disease and disability, (c) manage and eliminate symptoms caused by illness, and (d) enhance end-of-life and palliative care (National Institutes of Health, n.d.). Driven by some of the most pressing health needs (HealthyPeople.gov, 2014), nursing research focuses on individual and population health determinants ranging from bench science (e.g., genetic factors and personalized medicine) and physiological and biobehaviorial mechanisms of disease to effectiveness, implementation, and dissemination studies (American Association of Colleges of Nursing, 2006). Table 1 provides a comparison of nursing research goals and PCORI activities. It is also important to note that there are some concepts that are important in patient-centered outcomes research (PCOR) that nurses internationally consider to be central in both care delivery and research. The first is patient-centeredness, and the second is patient engagement. The concept of patient-
centeredness relates to a partnership among the clinicians, patients, and their families, ensuring that decisions respect patient’s wishes, needs, and preferences and that they have the education and support required for decision making and participation in their own care (National Research Council, 2001). The engagement of patients goes one step further, describing “actions individuals must take to obtain the greatest benefit from the healthcare services available to them” (Center for Advancing Health, 2010). Professional nursing organizations have adopted both patient-centeredness and patient engagement as basic principles in nursing practice (Sofaer & Schumann, 2012). Research in the areas of patient engagement and activation in care (Hibbard, Stockard, Mahoney, & Tusler, 2004) has begun to demonstrate improved patient outcomes. Patient engagement in research—not as a patient or subject but as a partner—is a novel intervention to improve the quality, relevance, and transferability of study results. Patient engagement in research is an emerging field where PCORI research is underway to identify the best methods (Domecq et al., 2014).
Unique Attributes of PCOR There are five major aspects of PCORI’s approach that differ from other funders: • • • • •
the nature of the questions asked, how funding priorities are established, engagement of patients and the healthcare community, compliance with the PCORI Methodology Standards, and the proposal review process and criteria.
Each will be described further, with the implications for nurse researchers.
The PCORI Questions Research questions that underlie PCOR are generated through the lens of the people who are the target population of interest. For example (PCORI, 2013): • Given my personal characteristics, conditions, and preferences, what should I expect will happen to me? • What are my options, and what are the potential benefits and harms of those options? • What can I do to improve the outcomes that are most important to me? • How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare?
TABLE 1. Comparison of Nursing Research Goals and Patient-Centered Outcomes Research Institute Activities Nursing researcha Build the scientific foundation for clinical practice Prevent disease and disability Manage and eliminate symptoms caused by illness
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Patient-Centered Outcomes Research Institute Produces dependable information that can inform health decisions Provides evidence that guides decisions about prevention of disease Provides evidence to improve outcomes that patients care about, such as function, symptoms, health-related quality of life, and survival
a
Source: National Institute of Nursing Research (n.d.).
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Generation of study research questions and hypotheses, while considering the patients’ point of view, is beneficial in two ways. First, it points the study development in the direction of designs and methods to best address patients’ greatest concerns. Second, it forces the research team to articulate clearly the decisions that results will affect. Whether addressing issues related to individuals, aggregates, populations, or healthcare systems, nurses are often on the front line of interactions with patients and caregivers. This gives nurses unique perspectives related to what matters most to the patient and those who care for and about them. Nurses hear the questions that matter most to the people that they care for. Thus, nurses are in a unique position to help PCORI identify the important questions and issues for patients. Nurse researchers partnering with clinicians can submit proposals addressing these patient-centered issues.
Funding Areas One of the first responsibilities of PCORI was to develop National Priorities for Research to guide the funding of CER. These priorities were to be cross-cutting areas where additional health research is needed to give patients and those who care for them more information to support decision making. A workgroup set up by the Board of Governors deliberated, made presentations, invited comment from stakeholder groups and the public, and refined its recommendations. Eventually, five priorities were identified and accepted by the Board of Governors (see Figure 1). Within any priority area, studies may focus on specific diseases, conditions, and interventions—or they may examine issues that apply across multiple diseases and conditions. In addition to funding research proposed by investigators within these areas, PCORI generates and prioritizes research topics related to questions contributed by patients and other stakeholders through its web site, workshops, and roundtables and similar efforts undertaken by others. These questions are
www.nursingresearchonline.com
reviewed to ensure they address an important research gap, assessed and ranked according to PCORI criteria (see Figure 2), and submitted to the Board of Governors for consideration as the subject of targeted (topic-specific) funding announcements. This process is guided by special workgroups and advisory panels selected through public nominations. There are advisory panels on six topics (Assessment of Prevention, Diagnosis, and Treatment Options; Improving Healthcare Systems; Addressing Disparities; Patient Engagement; Clinical Trials; and Rare Disease). There are currently 11 nurses on PCORI advisory panels.
Patient and Stakeholder Engagement Engagement of patients and stakeholders is a basic tenet for PCOR. This approach recognizes that, when researchers partner with healthcare stakeholders, they are best positioned to identify the compelling questions that underlie a truly patient-centered research agenda. PCORI-funded studies have patients and other stakeholders involved in the development of the research and its conduct, governance, oversight, and dissemination strategy. Funded projects engage patients and community members at all stages—from generating the initial ideas to sharing study results. In some cases, clinical researchers enlist patients from their practices, local community groups, or advocacy organizations. On the basis of PCORI’s experience with patient engagement in research, an engagement rubric has been developed (PCORI, 2014a). Sample engagement plans have also been posted on the web site to provide examples for investigators (http://www.pcori.org/assets/2013/11/PCORISample-Engagement-Plans.pdf). There are also other models for patient engagement approaches that may be helpful (Mullins, Abdulhalim, & Lavallee, 2012). Finding potential partners is sometimes a challenge to researchers who have not engaged patients and other healthcare
FIGURE 1. National priorities for PCORI-sponsored research. Used with the permission of the Patient-Centered Outcomes Research Institute. 2012–2014, Patient-Centered Outcomes Institute. More information at www.pcori.org.
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FIGURE 2. PCORI criteria for evaluating research topics. Used with the permission of the Patient-Centered Outcomes Research Institute. 2012–2014, Patient-Centered Outcomes Institute. More information at www.pcori.org.
stakeholders in past studies. PCORI has sponsored contests in which developers create matchmaking applications (PCORI, 2014b). PCORI has developed competitive engagement awards to assist potential applicants in developing their engagement partnerships and strategies prior to submitting for research funding (PCORI, 2014c). PCORI recently launched its Ambassador Program to help patients and other stakeholders, as well as organizations, share PCORI’s vision of PCOR with their communities, participate as full partners in research, and encourage the use of information generated from PCORI-funded projects. Four individual nurses and two nursing organizations (the Association of Perioperative Registered Nurses and the National Association of Nurse Practitioners in Women’s Health) are currently PCORI ambassadors.
Methodology Standards The PPACA established the PCORI Methodology Committee to develop and improve CER methods. As new methodological approaches emerge over time, minimal standards, in specific areas, are helpful in enhancing the quality of study design and methods. Higher-quality studies produce more reliable and trustworthy results that are more likely to be adopted into clinical practice. Within the first 18 months of its creation, the Methodology Committee contracted with teams of national experts through a competitive request for proposals to develop recommendations for the first set of standards. A set of 47 standards (see Figure 3) were refined and endorsed by the Board of Governors to guide PCORI-funded studies (PCORI Methodology Committee, 2013).The standards are organized into two categories: those relevant to all PCOR and those that apply to specific study designs or methods, such as data registries and networks, systematic reviews, and specific types of trials. All proposals for PCORI funding are required to address how the work will apply the methodology standards. PCORI
expects to add new standards in additional areas and revise the current ones—both as research methods evolve and in response to advice from the advisory panels, patients, other stakeholders, and the research community.
Proposal Review Two major aspects of PCORI’s review of proposals differ from other extramural funding processes. Some of PCORI’s criteria are unique, and PCORI includes patients and other stakeholders on the review team. Research proposals submitted to PCORI are reviewed using five criteria (those that are unique are designated with asterisks): • impact of the condition to be studied on the health of individuals and populations (for CER applications) or impact on the field of PCOR methods (for methodology applications), including whether the condition or disease is associated with a significant burden across the U.S. population or within a subgroup; • potential for the study to improve healthcare and outcomes (for CER applications) or PCOR methods (for methodology applications), including whether the proposed project addresses a critical gap in current knowledge that is important to patients, caregivers, or clinicians; • technical merit, including the qualifications of the team and the rigor of the research plan; • patient-centeredness, including whether the research measures outcomes important to patients; and • patient and stakeholder engagement, including evidence of patent and stakeholder engagement in the development of the proposal and proposed roles.
The role of patients and other stakeholders on the PCORI application review team is unique. The nonscientists participate fully and have an impact equal to that of the scientists. To achieve this, PCORI trains all the reviewers to understand its review criteria and to interact productively with the full range of reviewers, and it also provides mentors who give additional support to the nonscientists. There are currently
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Research Done Differently: PCORI
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FIGURE 3. Cross-cutting standards for patient-centered outcomes research established by PCORI. Used with the permission of the Patient-Centered Outcomes Research Institute. 2012–2014, Patient-Centered Outcomes Institute. More information at www.pcori.org.
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Research Done Differently: PCORI
Nursing Research • January/February 2015 • Volume 64 • No. 1
more than 60 nurses on PCORI Merit Review panels, and two other nurses are Merit Review mentors.
Goals and priorities for nursing research are highly aligned with those of PCORI. PCORI is interested in examining whether patient and other shareholder contributions to the review process lead to different decisions. To begin this exploration, PCORI staff examined outcomes of the first round of the reviews for research funding. They analyzed the scores that different types of reviewers—scientist, patients, other stakeholders— had assigned to proposals before and after face-to-face discussion. The evaluation concluded that patient and stakeholder reviewers add different perspectives to the review process, and in-person discussion led to closer agreement (Fleurence et al., 2014). Of the 25 proposals selected for award by the full review process, only 13 had ranked in the top 25 of a preliminary review by scientists alone.
Conclusions Goals and priorities for nursing research guiding high-quality clinical practice, preventing disease and disability, and managing and eliminating symptoms caused by illness are highly aligned with those of PCORI. Given this synergy, many phenomena that nurses study could be developed into a competitive proposal for funding. Development of proposals will require attention to the unique aspects of PCORI funding, which include the nature of the questions asked, funding priorities, engagement of patients and stakeholders, compliance with the PCORI methodology standards, and proposal review criteria. Nurses are central to guiding patients as they make some of their most difficult decisions. Nurse researchers are in a unique position to focus on what evidence is needed to produce the outcomes important to their patients.
Accepted for publication October 2, 2014. The authors acknowledge Robin Newhouse was appointed to the Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee, Debra Barksdale was appointed to the PCORI Board of Governors, and Julie Miller is employed by PCORI. Corresponding author: Robin Newhouse, PhD, RN, NEA-BC, FAAN, University of Maryland School of Nursing, 655 West Lombard Street, Suite 316, Baltimore, MD 21201 (e-mail: [email protected]).
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