The Patient-Centered Outcomes Research Institute

The Patient-Centered Outcomes Research Institute (PCORI) is a not-for-profit organization established in the Affordable Care Act (ACA). It was created to help patients, clinicians, purchasers, and policymakers make more informed health care decisions “by advancing the quality and relevance of evidence” on how to prevent, diagnose, treat, monitor, and manage diseases, disorders, and other conditions.¹ PCORI’s objective is to better ensure that patients, health care professionals, and other stakeholders have the evidence they need to make informed decisions when comparing treatment options through research guided by patients, caregivers, and the broader health care community.²

This column discusses PCORI’s focus on patient-centered care, comparative effectiveness research (CER), research funding, and innovative projects. It also provides information on how surgeons can get involved.

How will PCORI meet the ACA mandate?

To meet the ACA mandate of enhancing the quality and relevance of patient-centered outcomes research, PCORI has adopted the following strategic goals:

• Increase the quality, quantity, and timeliness of usable, trustworthy information to support health care decisions
• Advance the implementation and use of research evidence
• Influence research funded by others so it is more patient-centered³

To meet these goals, PCORI specifically focuses on clinical comparative research to investigate which currently available health care products and treatments work best for a given patient, caregiver, or other stakeholder. To achieve this aim, PCORI works from the premise that research might be enhanced if investigators routinely interact with patients to develop research questions. PCORI-funded research must demonstrate that it answers questions and addresses gaps identified by stakeholders to determine the solutions that work best for them. To gain the information needed to address a broad range of health care decisions, PCORI funds research based on five stakeholder-identified national priorities (see table).^3,4

PCORI funds research related to five national priorities

National priorities	Examples of funded projects
Assessment of Prevention, Diagnosis, and Treatment Options	Improving Patient Decisions about Bariatric Surgery
	Patient-Centered, Risk-Stratified Surveillance after Curative Resection of Colorectal Cancer
Improving Healthcare Systems	A Comparative Effectiveness Trial of Optimal Patient-Centered Care for U.S. Trauma Care Systems
	Building a Multidisciplinary Bridge Across the Quality Chasm in Thoracic Oncology
Communication and Dissemination Research	Improving Communication in the Pediatric Intensive Care Unit for Patients with Life-Changing Decisions
	[University of California-San Francisco Computed Tomography] Radiation Dose Registry to Ensure a Patient-Centered Approach for Imaging
Addressing Disparities	Eliminating Patient-Identified Socio-legal Barriers to Cancer Care
	Impact of Patient Navigators on Health Education and Quality of Life in Formerly Incarcerated Patients
Accelerating Patient-Centered Outcomes Research and Methodological Research	Understanding Treatment Effect Estimates When Treatment Effects Are Heterogenous for More Than One Outcome
	Improving the Use of Patient Registries for Comparative Effectiveness
Access all upcoming and previous funding opportunities.

In what innovative ways is PCORI advancing national research on PCOR?

In March, PCORI launched the National Patient-Centered Clinical Research Network (PCORnet), a national, interoperable multicenter research network specifically designed to support observational and randomized PCOR studies.⁵ PCORnet is a health data initiative which will work to conduct clinical research within patient communities and health care systems across a wide geographic area.

PCORI has awarded more than $93 million to 11 Clinical Data Research Networks (CDRNs) and 18 Patient-Powered Research Networks (PPRNs), which fall under the PCORnet umbrella. The purpose of CDRNs and PPRNs is to stimulate patient-driven research. The figure illustrates how many sites affiliated with one or more of PCORnet’s 29 partner networks are expected to be established in each state.⁶ CDRNs are health system-based clinical data networks covering large populations that collect information in electronic health records (EHRs) in the course of routine patient care. The CDRNs are charged with building a large nationwide patient cohort with longitudinal electronic clinical data, developing policy for the standardization of data, and focusing on data security to improve patients’ ability to participate in multi-network, randomized clinical trials and observational studies.⁷

Expected State Sites for PCORnet’s Partner Networks

PPRNs are operated and governed by groups of patients, caregivers, or families connected by a shared health condition and who are interested in participating in CER. PPRNs are required to collect data on 80 percent of the population they represent.⁷ Half of existing PPRNs represent patients with rare diseases and are therefore critical for advancing research on these conditions. PPRNs organized around a specific disease will represent a group of motivated individuals ready to participate indefinitely in studies related to that disease. Typically, research grants have supported collaboration across a consortium of academic medical centers to study a rare disease, but the researchers and study group have often dispersed when the specific project is completed. PPRNs will not disperse, and the PPRN rare disease cohort will remain available to participate in future studies.

The initial phase of development for the CDRNs and PPRNs is 18 months, after which it is anticipated that PCORnet will have available information on several million diverse patients. This will greatly increase the statistical power of patient-centered CER due to larger samples. PCORnet works to advance the speed and efficiency of research by building a health care community that will produce meaningful information for end users. PCORnet has the potential to seamlessly include health research as a part of routine patient visits without interrupting care, thereby creating a national resource for health research.

How does PCORI research involve patients and other stakeholders in the application and review process?

The specific focus on providing useful and practical comparative data for patients and caregivers is what distinguishes PCOR. PCORI’s funding criteria specifically address patient-centeredness, engagement, and likelihood of changing practice.³ To receive funding, applicants must involve patients in clinical research design and decision-making processes to ensure that research questions are relevant to patients and other stakeholders.⁴

Examples of questions that PCOR might address include:

• “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 health care?”2

Another way that PCORI engages patients and stakeholders is through the research application and review process. PCORI is the first large funding agency in the U.S. to require non-scientist reviewers to be involved in the review of all applications for its funding.⁸ The purpose of their involvement is to improve the relevance of PCORI-funded research for the stakeholders who take action based on the findings.Each funding application submitted to PCORI funding is reviewed by four merit reviewers—two clinical scientists, one patient or patient advocate, and one health care stakeholder, such as a clinician, purchaser, or representative from the health care industry. Through this process, end users have the ability to provide input on which research proposals PCORI should fund.

PCORI has developed resources to help stakeholders communicate and work with patients, because those researchers seeking funding for PCOR have identified patient engagement as one of their biggest challenges. View a webinar that provides examples of meaningful engagement.⁹

How can surgeons get involved in PCORI?

Surgeons can get involved in PCORI in several ways. PCORI has approved $549 million to more than 300 research projects and initiatives since 2012, and as much as $1.5 billion will be committed to research projects in the upcoming three years—most of which will focus on targeted funding announcements and large pragmatic studies.³ A few engagement opportunities include:

• Eugene Washington PCORI Engagement Awards: These awards provide funding for smaller projects to develop knowledge concerning how consumers of health care information view and use PCOR, to build capacity for community engagement in PCOR, and to support channels for the dissemination and implementation of PCOR. Applications for funding are reviewed on an ongoing basis. For more information, visit the Eugene Washington PCORI Engagement Awards.
• Large pragmatic studies: In 2014, PCORI launched a new initiative to fund large pragmatic clinical trials, large simple trials, or large-scale observational studies. This initiative provides an opportunity for clinicians to partner with diverse stakeholders, including patients, purchasers and/or payors, and professional organizations. These studies will involve broadly represented patient populations to ensure a large enough sample to evaluate differences in treatment effectiveness in patient subgroups. The studies aim to address prevention, diagnosis, treatment, or management of a disease and/or symptom improvement in health care system performance, and elimination of disparities in health care, and must be conducted in a typical clinical care and community setting.¹⁰
• Other PCORI funding opportunities: PCORI also issues regular funding announcements under its five broad national priorities for research and occasional calls for proposals on specific, high-impact topics. All funding opportunities are listed in PCORI’s funding center.
• PCORI funding reviews: PCORI seeks guidance from clinicians and other stakeholders on the evaluation of proposals for its funding and to formulate research questions. Clinicians are critical stakeholders in the PCORI merit review process. Learn about the experience of past reviewers.
• PCORnet: Once PCORnet is operational in late 2015, surgeons involved in research may be able to link external data to PCORnet to supplement their current study population. Linking clinical data registries to PCORnet may be especially effective in creating a large, diverse study cohort. Surgeons may also have the opportunity to help leverage EHRs to help recruit eligible patients, monitor patient safety and study conduct, and collect clinical outcomes.

Other areas of engagement include opportunities to provide input on study design for integration with physician clinic or office operations, disseminate study findings, or contribute to the evaluation of PCORI-funded projects.¹¹

For additional questions and concerns regarding PCORI, contact the American College of Surgeons Division of Advocacy and Health Policy at ahp@facs.org or 202-337-2701.

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References

1. Patient-Centered Outcomes Research Institute. Patient Protection and Affordable Care Act. 42 U.S.C. § 1320e. 2010. Available at: http://www.pcori.org/assets/PCORI-Authorizing-Legislation-032310.pdf. Accessed September 16, 2014.
2. Patient-Centered Outcomes Research Institute. Available at: http://www.pcori.org/. Accessed July 16, 2014.
3. Selby J, Lipstein S. PCORI at 3 years—progress, lessons, and plans. N Engl J Med. 370(7):592-595. Available at: http://www.nejm.org/doi/full/10.1056/NEJMp1313061. Accessed June 18, 2014.
4. Patient-Centered Outcomes Research Institute. PCORI funding awards: Cycle III and inaugural improving methods for conducting PCOR. September 10, 2013. Available at: http://www.pcori.org/assets/2013/09/PCORI-Funding-Awards-Cycle-III-and-Inaugrual-Improving-Methods-for-Conducting-PCOR.pdf. Accessed September 16, 2014.
5. Daugherty S, Wahba S, Fleurence R, PCORnet. PPRN Consortium. Patient powered research networks: Building capacity for conducting patient-centered clinical outcomes research. J Am Med Inform Assoc. 2014;21(4):583-586. Available at: http://jamia.bmj.com/content/21/4/583.long. Accessed June 16, 2014.
6. Patient-Centered Outcomes Research Institute. PCORnet coverage map. Available at: http://www.pcori.org/assets/2013/12/PCORI-CDRN-Award-Map.jpg. Accessed June 16, 2014.
7. Fleurence R, Curtis L, Califf R, Platt R, Selby J, Brown J. Launching PCORnet, a national patient-centered clinical research network. J Am Med Inform Assoc. 2014;21(4):578-582. Available at: http://jamia.bmj.com/content/early/2014/05/12/amiajnl-2014-002747.full. Accessed June 11, 2014.
8. Fleurence RL, Forsythe LP, Lauer M, et al. Engaging patients and stakeholders in research proposal review: The Patient-Centered Outcomes Research Institute. Ann Intern Med. 2014;161(2):122-130. Available at: http://annals.org/article.aspx?articleid=1887029. Accessed August 13, 2014.
9. Patient-Centered Outcomes Research Institute. Promising practices of meaningful engagement in the conduct of research. September 19, 2013. Available at: http://www.pcori.org/events/2013/promising-practices-meaningful-engagementconduct-research. Accessed September 16, 2014.
10. Patient-Centered Outcomes Research Institute. Pre-announcement: Pragmatic clinical studies and large simple trials to evaluate patient-centered outcomes. Available at: http://www.pcori.org/funding-opportunities/fundingannouncements/pre-announcement-pragmatic-studies-to-evaluatecomparativeclinical-effectiveness/. Accessed June 16, 2014.
11. Patient-Centered Outcomes Research Institute. Recent PCORI research initiatives: Highlighting opportunities for clinicians. Available at: http://encore.meetingbridge.com/MB005418/140228/. Accessed June 16, 2014.