Lori Frank

The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda.

THE PATIENT PROTECTION AND AFFORDABLE CARE ACT of 2010 created the Patient-Centered Outcomes Research Institute (PCORI) to fund and promote comparative clinical effectiveness research (CER) that will “assist patients, clinicians, purchasers, and policymakers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis.” CER is not a new concept, but appreciation of its potential for providing patients and their clinicians with uniquely valuable information on what works, tailored to the clinical situation and to patient priorities, has increased rapidly in recent years. The research institute founded by this legislation was named to emphasize the critical importance of a patientcentered perspective in conducting this research. The PCORI Board of Governors determined early on that taking this name seriously, placing patients at the center of CER and actively engaging clinicians and other stakeholders was an ideal strategy to ensure that the PCORI research agenda stays focused on practical questions, relevant outcomes and study populations, and the possibility that treatment effects may differ across patient populations. The institute developed a definition of patient-centered outcomes research that emphasizes the voice of the patient in assessing health care options. The PCORI mission statement commits to producing and promoting high-integrity research that is “guided by patients, caregivers, and the broader healthcare community.” The institute’s first funding announcement solicited projects focused on methods for engaging patients and other stakeholders in all aspects of the research process. Merit review of more than 800 responses to this announcement is being conducted by the Center for Scientific Review of the National Institutes of Health (NIH). PCORI has modified the process in 2 ways. First, stakeholders, including patients, caregivers, and clinicians, were invited via the PCORI website to sign up if they were interested in participating in the merit reviews. A total of 48 individuals were subsequently recruited, trained by the NIH, and included as voting members of the 16 study sections (3 per section). Second, proposed scoring criteria included an additional criterion—extent of patient engagement. The founding legislation required PCORI to develop national priorities for research and a research agenda, and to post both for a 45to 60-day public comment period before major funding for research could begin. The development process extended from July to December 2011. It included examination of other recent prioritization efforts and consideration of 9 criteria cited in the statute (BOX), of the PCORI definition of patient-centered outcomes research, and of input received through discussions with stakeholder groups, including patients and their caregivers, clinicians, hospitals and health care systems, payers, the life sciences industry, and the research community. The process produced 5 national priorities (Box) and the research agenda, which are available on the PCORI website. The national priorities encompass many research areas cited by earlier priority-setting groups, but add a patientcentered perspective. Helping patients make informed health care choices will not be achieved solely by producing more CER evidence on prevention, diagnostic, or treatment strategies. New evidence is unlikely to be rapidly adopted unless the systems in which patients and clinicians make decisions are improved, removing current barriers to acting on the new information. Better methods for making CER results available and for communication about those results between patients and their clinicians are needed. Similar to studies evaluating health disparities, CER seeks evidence on possible differences in the effectiveness of treatments, in preferences for various outcomes, and in information needs across various populations. CER evidence can be further purposed to reduce or eliminate health disparities among vulnerable populations. Another priority addresses the need to build and sustain a national research infrastructure that facilitates learning from clinical experience. It calls for building clinical research databases, improving analytic methods for conducting this research, and train-

The PCORI Perspective on Patient-Centered Outcomes Research

The Patient-Centered Outcomes Research Institute (PCORI) was established as part of the US Patient Protection and Affordable Care Act of 2010 to fund patient-centered comparative clinical effectiveness research, extending the concept of patient-centeredness from health care delivery to health care research. In the United States, patient-centered outcomes research is new and not defined in the legislation, and the rationale is unclear to many. In this Viewpoint, we address 2 related questions: What does patientcenteredness in research mean? Why conduct patientcentered outcomes research? The essence of the PCORI definition of patientcentered outcomes research is the evaluation of questions and outcomes meaningful and important to patients and caregivers. The definition rests on the axiom that patients have unique perspectives that can change and improve the pursuit of clinical questions. Relevant to both the definition and rationale is the hypothesis that including the perspectives of end users of the research, which include patients, physicians, and other health care stakeholders, will enhance the relevance of research to actual health decisions these end users face. In turn, increased relevance is hypothesized to improve uptake of the evidence and improve the likelihood that patients will achieve the health outcomes they desire. Although this proposed causal chain is readily understood in relation to questions of direct interest to pa

Health-related quality of life associated with irritable bowel syndrome: Comparison with other chronic diseases

BACKGROUND Despite the rapidly growing body of literature on health-related quality of life (HRQoL). placing the results in a context that is meaningful to clinicians and patients is often overlooked. OBJECTIVE This study sought to quantify the impact of irritable bowel syndrome (IBS) on HRQoL by comparing the Medical Outcomes Study 36-item Short-Form Health Survey (SF-36) scores of IBS patients with normative US data and with the scores of patients having other chronic gastrointestinal (GI) and non-GI disorders. METHODS Two IBS reference groups were identified from the published literature: a largely untreated community sample of health maintenance organization (HMO) members (N = 92) and a sample of patients with IBS recruited through clinics and in the community (N = 140). SF-36 scores for these groups were compared with published US population norms (N = 2474) and with published scores for 3 other IBS samples (N = 464); a sample with other chronic GI disorders (dyspepsia [N = 126], gastroesophageal reflux disease [GERD] [N = 516]); and samples with other chronic episodic disorders (asthma [N = 375], migraine [N = 303], panic disorder [N = 73], rheumatoid arthritis [N = 693]). RESULTS The scores of patients in both IBS reference groups were significantly lower on several SF-36 domains than those of the US normative population (P < 0.003). Scores on several SF-36 scales were also significantly lower in the IBS reference groups compared with the GERD, asthma, and migraine samples (P < 0.003). Depending on the IBS sample used, scores did not differ or were higher compared with those in the sample with dyspepsia. Relative to the samples with panic disorder and rheumatoid arthritis, the IBS groups had significantly higher scores on most SF-36 domains (P < 0.003). Scores for the HMO reference group were generally higher than those for the clinic/community reference group. CONCLUSIONS Based on the results of this analysis, IBS is associated with impairment of HRQoL relative to US population norms and to populations with GERD, asthma, or migraine. HRQoL appears to be greater in patients with IBS than in those with panic disorder or rheumatoid arthritis, although the relative symptom severity in these samples was not known.

Pharmacoeconomic Evaluation in the Real World: Effectiveness Versus Efficacy Studies

Pharmacoeconomic data may be obtained within the context of randomised clinical trials (RCTs) and from effectiveness studies in the ‘real world’. The differences between the 2 types of study design have implications for the types of data that can be obtained and the interpretation of the resulting findings. Because RCTs are designed to assess the safety and efficacy of pharmaceuticals, and because the study design of RCTs emphasises internal validity over generalisability, the pharmacoeconomic data collected from them are limited. The data may not be applicable to the more heterogeneous patients encountered in actual clinical practice, and cost estimates may be inaccurate because of protocol requirements. Effectiveness studies, in which treatments are studied under real-world conditions, remedy some of these limitations. Generalisability to actual users is generally enhanced in effectiveness designs, but data may be biased in other ways.This brief review compares the 2 study designs as they relate to pharmacoeconomic evaluations in terms of the research questions they address, design differences and their implications for study bias, data collection and data analysis and the generalisability of their results.

Improving dementia care: The role of screening and detection of cognitive impairment

The value of screening for cognitive impairment, including dementia and Alzheimers disease, has been debated for decades. Recent research on causes of and treatments for cognitive impairment has converged to challenge previous thinking about screening for cognitive impairment. Consequently, changes have occurred in health care policies and priorities, including the establishment of the annual wellness visit, which requires detection of any cognitive impairment for Medicare enrollees. In response to these changes, the Alzheimers Foundation of America and the Alzheimers Drug Discovery Foundation convened a workgroup to review evidence for screening implementation and to evaluate the implications of routine dementia detection for health care redesign. The primary domains reviewed were consideration of the benefits, harms, and impact of cognitive screening on health care quality. In conference, the workgroup developed 10 recommendations for realizing the national policy goals of early detection as the first step in improving clinical care and ensuring proactive, patient‐centered management of dementia.

Upper Gastrointestinal Symptoms in North America

The aim of this study was to determine the prevalence of upper gastrointestinal symptoms (UGIS) in a general population and quantify the relationship of those symptoms to health-care utilization and quality of life. In-person interviews were conducted with 2056 United States and Canadian residents selected at random. Subjects reported frequency and severity for 11 symptoms, prescription and over-the-counter medication use, primary care and specialty physician visits in prior three months, and completed the Psychological General Well-Being Scale. For analyses, subjects were classified into four mutually exclusive symptom groups: gastroesophageal reflux disease (GERD) -like, GERD plus motility-like (GERD+), ulcerlike, and motility-like. Of the total sample, 51.4% reported the occurrence of at least one UGIS in the prior three months. Subjects in the GERD+ and ulcer groups used more prescription medications and were more likely to see a physician about the symptoms (P < 0.001). Subjects with symptoms demonstrated poorer quality of life compared to subjects with no symptoms. The prevalence of UGIS in the general population is high and symptoms are associated with significant health-care utilization and poorer quality of life.

Conceptual and practical foundations of patient engagement in research at the patient-centered outcomes research institute

PurposeTo provide an overview of PCORI’s approach to engagement in research.MethodsThe Patient-Centered Outcomes Research Institute (PCORI) was established in 2010 to fund patient-centered comparative effectiveness research. Requirements for research funding from PCORI include meaningful engagement of patients and other stakeholders in the research. PCORI’s approach to engagement in research is guided by a conceptual model of patient-centered outcomes research (PCOR), that provides a structure for understanding engagement in research.ResultsTo understand and improve engagement in research PCORI is learning from awardees and other stakeholders. Those efforts are described along with PCORI’s capacity building and guidance to awardees via the Engagement Rubric. PCORI’s unique model of engaging patients and other stakeholders in merit review of funding applications is also described. Additional support for learning about engagement in research is provided through specific research funding and through PCORI’s major infrastructure initiative, PCORnet.ConclusionPCORI requires engagement of stakeholders in the research it funds. In addition PCORI engages stakeholders in activities including review of funding applications and establishment of CER research infrastructure through PCORnet. The comprehensive approach to engagement is being evaluated to help guide the field toward promising practices in research engagement.

Should older adults be screened for dementia

The question of whether to screen for dementia and Alzheimers disease (AD) has been discussed in many forums throughout the world. Generally, medical advisory groups and policy‐making groups have recognized the importance of early diagnosis but have uniformly avoided making recommendations to screen at‐risk populations. This presentation reflects the support for reconsidering the importance of screening individuals at risk or above a certain age. In this statement, the majority of the authors support the consideration of dementia risk factors in individuals at age 50, with routine yearly screening after 75. Other authors remain concerned that the benefits of treatments of early disease do not yet support a general screening recommendation. These statements are made to encourage progress toward the development of a consensus regarding the widespread institution of screening policy. Accordingly, members of the worldwide scientific community are invited to add their perspective by contributing short commentaries (1500 words) on this subject.

Should older adults be screened for dementia? It is important to screen for evidence of dementia!

Multiple arguments for considering routine dementia screening have been presented. Furthermore, dementia diagnoses are widely unrecognized. As a result, persons with dementia are missing important clinical care and treatment interventions. By distinction, the problems of defining, diagnosing, and treating mild cognitive impairment (MCI) are not yet resolved, and MCI is not ready for a screening recommendation. Dementia screening approaches, including cognitive testing and functional assessment, must be evaluated on their scientific merits, including sensitivity and specificity for recognizing affected individuals in at‐risk populations. Screening tests must be “cost‐worthy”, with the benefits of true‐positive test results justifying the costs of testing and resolving false‐positive cases, with due consideration for proper diagnostic evaluation and potential harms. With the tremendous number of new cases projected in the near future and the expected emergence of beneficial therapies, considerably more research is needed to develop more efficient screening systems.

Symptom and Health-Related Quality-of-Life Measures for Use in Selected Gastrointestinal Disease Studies: A Review and Synthesis of the Literature

AbstractPatient-rated symptom and health-related quality-of-life (HR-QOL) outcomes are important end-points for clinical trials of medical treatments for gastrointestinal (GI) disorders. Based on this review, patient outcomes research is focused on gastroesophageal reflux disease and dyspepsia, with a growing interest in irritable bowel syndrome but little research in gastroparesis. State-of-the-art for patient-rated symptom scales is rudimentary with an abundance of scales and little attention to systematic instrument development or comprehensive psychometric evaluation. Generally, disease-specific HR-QOL measures have been more systematically developed and evaluated psychometrically, but few have been incorporated into clinical trials. More comprehensive outcome assessments are needed to determine the effectiveness of new medical treatments for functional GI disorders. Future clinical trials of GI disorders should combine clinician assessments of outcomes and symptoms with patient-rated symptom and HR-QOL end-points.