Challenges to Implementing Patient-Centered Research
Authors: Michael Pignone
Journal: Annals of Internal Medicine (2012)

Editorials18 September 2012Challenges to Implementing Patient-Centered ResearchFREEMichael Pignone, MD, MPHMichael Pignone, MD, MPHFrom Sheps Center for Health Services Research, University of North Carolina, Chapel Hill, NC 27599.Search for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-157-6-201209180-00521 SectionsAboutVisual AbstractPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail In this issue, Krumholz and Selby (1) present a compelling vision for the newly formed Patient-Centered Outcomes Research Institute (PCORI). Funded through the Patient Protection and Affordable Care Act (ACA), PCORI will substantially increase funding for research focused on the needs of “patients, their caregivers, and clinicians” and will also help inform health policymakers as they seek to promote effective and efficient services. Perhaps on a more fundamental level, PCORI also seeks to change the way in which health researchers interact with their intended “audiences” by amplifying the patient's role in the research process.The vision that Krumholz and Selby articulate on behalf of PCORI is transformative and, if successful, could potentially improve the health research landscape not only for PCORI but also for other federal and nonfederal research-funding bodies. However, several potential obstacles must be overcome to optimize PCORI's success.The first potential and perhaps most vexing obstacle is the possibility of PCORI's work falling victim to partisan political activity. At the most basic level, patient-centeredness, whether in health care or health care research, should not be a partisan issue: Democrats and Republicans alike benefit when care is better directed to the needs of patients and their caregivers, and our current research base is clearly insufficient to guide providers, patients, and policymakers in determining the best course of action in many important clinical scenarios.That said, the current political environment offers several potential pitfalls, even after the Supreme Court's ruling confirming the ACA's constitutionality. First, political efforts to repeal the entire ACA may continue without regard to the merit of its specific components, including PCORI. Second, even in the absence of full repeal, opponents of the ACA have vowed to block funding or implementation of components of the law as a means of hobbling its effectiveness. Even if these efforts are unsuccessful, failure of bipartisan support for the mission of PCORI may leave its long-term future in doubt. This would keep PCORI from realizing its potential for supporting longer-term studies and for providing a “home” for researchers to build their careers, as has been the case for the National Institutes of Health.A second obstacle to PCORI's success relates to the potential mismatch between funding opportunities and the type of study designs that will be required to definitively answer many of the research questions that have been asked. For example, a key theme that Krumholz and Selby articulate is the need for personalized information for informing prognosis (“Given my personal characteristics, what should I expect will happen to me?”) and promoting informed decision making. However, providing accurate information for such questions is challenging, particularly when comparing 2 effective interventions or tests.The proposed size of initial awards for the first round of PCORI grants, although substantial, may not be sufficient to answer some of the most important questions facing patients and providers. It is more critical that the 3-year time frame for these awards will preclude answering questions that require longer durations, including comparisons of screening tests for and treatments of chronic conditions when the outcome of interest is actual clinical events rather than intermediate measures. In order to examine whether the effectiveness of tests or treatments differ by sex, ethnic background, or socioeconomic status, investigators must recruit and retain large sample sizes for randomized, clinical trials; therefore, developing mechanisms to support such studies will be an important challenge for PCORI going forward.In some cases, other types of designs, including time series or observational studies, may be more efficient, assuming that the risks of bias and confounding can be effectively controlled. Before launching large-scale trials of therapeutic interventions or prognostic strategies, investigators should consider first performing modeling studies or value-of-information analyses to gain a sense of whether large investments of time and resources can be justified (2, 3). In the spirit of patient-centeredness, making modeling and value of information available and accessible to patients, caregivers, and providers as part of prioritization may be an effective way to determine research agendas and efficiently allocate the $3 billion that PCORI will disburse in the next 7 years.A final potential challenge for PCORI is determining effective and representative means for soliciting the patient's perspective in research. It seeks patient and caregiver involvement in all phases of research: determination of research questions, funding decisions, study design, interpretation of findings, and dissemination of results. However, how these goals are implemented in actual studies remains to be determined.Investigators may choose to partner with patient advocacy organizations, for example, to gain access to a large, motivated, and potentially easy-to-mobilize constituency. In some cases, this choice may be optimal; in others, the perspective of members of advocacy groups may differ from those of other potential stakeholders.As an example, survivors of prostate cancer may be particularly well-suited to provide input on the content of a decision-support intervention for men deciding about treatment of prostate cancer (4). On the other hand, these same cancer survivors alone may not provide a representative sample for informing an intervention to assist men in deciding whether to undergo prostate cancer screening. Overcoming the challenges of sampling a diverse range of potential patients and caregivers (and hence drawing into research the voices of patients who may never have contemplated participating in “research”) will be one of the most exciting methodological challenges faced by PCORI-funded investigators.Assuming that the key obstacles and challenges can be overcome or mitigated sufficiently, PCORI offers a novel path for the future of health research. Many of the innovations proposed by PCORI, if successful, can hopefully be incorporated into the research programs of the National Institutes of Health, the Agency for Healthcare Research and Quality, and other federal and nonfederal research organizations so that patient-centered research becomes the norm rather than the exception.Michael Pignone, MD, MPHSheps Center for Health Services Research, University of North CarolinaChapel Hill, NC 27599References1. Krumholz HM, Selby JV; Patient-Centered Outcomes Research Institute. Seeing through the eyes of patients: the Patient-Centered Outcomes Research Institute funding announcements. Ann Intern Med. 2012;157:446-7. AbstractGoogle Scholar2. Pletcher MJ, Pignone M. Evaluating the clinical utility of a biomarker: a review of methods for estimating health impact. Circulation. 2011;123:1116-24. [PMID: 21403122] CrossrefMedlineGoogle Scholar3. Claxton K, Neumann PJ, Araki S, Weinstein MC. Bayesian value-of-information analysis. An application to a policy model of Alzheimer's disease. Int J Technol Assess Health Care. 2001;17:38-55. [PMID: 11329844] CrossrefMedlineGoogle Scholar4. Feldman-Stewart D, Capirci C, Brennenstuhl S, Tong C, Abacioglu U, Gawkowska-Suwinska M, et al. Information needs of early-stage prostate cancer patients: a comparison of nine countries. Radiother Oncol. 2010;94:328-33. [PMID: 20116122] CrossrefMedlineGoogle Scholar Comments0 CommentsSign In to Submit A Comment Author, Article, and Disclosure InformationAffiliations: From Sheps Center for Health Services Research, University of North Carolina, Chapel Hill, NC 27599.Disclosures: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M12-1722.Corresponding Author: Michael Pignone, MD, MPH, Division of General Internal Medicine, CB #7110 University of North Carolina Hospitals, 5045 Old Clinic Building, Chapel Hill, NC 27599-7110; e-mail, [email protected].This article was published at www.annals.org on 31 July 2012. PreviousarticleNextarticle Advertisement FiguresReferencesRelatedDetailsSee AlsoSeeing Through the Eyes of Patients: The Patient-Centered Outcomes Research Institute Funding Announcements Harlan M. Krumholz , Joe V. Selby , and Metrics Cited ByClinicians’ perspectives on and interest in participating in a clinical data research network across the Southeastern United StatesDevelopment and validation of the Person-Centeredness of Research ScaleEffectiveness of a decision aid for patients with depression: A randomized controlled trialUnresolved tensions in consumer engagement in CER: a US research perspective 18 September 2012Volume 157, Issue 6Page: 450-451KeywordsCancer screeningCaregiversForecastingHealth services researchPatient advocacyProstate cancerResearch fundingVision ePublished: 18 September 2012 Issue Published: 18 September 2012 CopyrightCopyright © 2012 by American College of Physicians. All Rights Reserved.PDF DownloadLoading ...