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By Albert W. Wu, Claire Snyder, Carolyn M. Clancy, and Donald M. Steinwachs 10.1377/hlthaff.2010.0660 HEALTH AFFAIRS 29, NO. 10 (2010): 1863–1871 ©2010 Project HOPE— The People-to-People Health Foundation, Inc.
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Adding The Patient Perspective To Comparative Effectiveness Research
Albert W. Wu (
[email protected]) is a professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health, in Baltimore, Maryland.
Comparative effectiveness research generates evidence that helps consumers, clinicians, purchasers, and policy makers make better decisions about health care. Capturing the patient’s perspective is central to this research because it provides a complete picture of treatment impact. This can be done with standardized questionnaires that ask patients to report on their functioning, well-being, symptoms, and satisfaction with care. These data, however, are not collected routinely in either clinical research or practice. Strategies and incentives to link patient-reported outcomes to data from conventional sources—including clinical research, electronic health records, and administrative data—will accelerate the development of useful evidence. ABSTRACT
T
he purpose of comparative effectiveness research is to help consumers, clinicians, purchasers, and policy makers make informed decisions that will improve health care at both the individual and population levels. Various definitions of comparative effectiveness research have been crafted by leading organizations in the field, including the Agency for Healthcare Research and Quality (AHRQ),1 the Institute of Medicine (IOM) Committee on Comparative Effectiveness Research Priorities,2 and the Federal Coordinating Council for Comparative Effectiveness Research.3 Although these definitions differ slightly, they share the direct comparison of existing health care interventions, using a variety of data sources and measurement methods to determine which interventions carry the greatest benefits and harms for which patients. With the creation of the Patient-Centered Outcomes Research Institute in the Patient Protection and Affordable Care Act of 2010, comparative effectiveness research has effectively been renamed “patient-centered outcomes research,” underscoring the goal of personcentered medicine. The term patient-centered out-
Claire Snyder is an assistant professor of medicine, Division of General Internal Medicine, Johns Hopkins School of Medicine. Carolyn M. Clancy is director of the Agency for Healthcare Research and Quality, in Rockville, Maryland.
comes research is consistent with the IOM definition of quality of care.4 In this paper we describe how questionnaires, referred to collectively as patient-reported outcome measures, can be used systematically to capture the experience of patients directly. We explain how this methodology can be linked to the sources of data that are used most commonly in comparative effectiveness research, and we suggest steps that might be taken to promote this kind of research.
Donald M. Steinwachs is a professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health.
The Patient Perspective Some questions about health care can be answered only by patients themselves, such as what is the “right” strategy to treat someone’s elevated blood sugar. A growing number of people aware of the widening array of diagnostic and treatment options expect to be involved in identifying which options are the best fits for them. In short, informed decision making takes into account the individual’s needs as well as his or her view of the likely impacts of treatment. Impact And Effectiveness Comparative effectiveness research poses questions about the impact and relative effectiveness of different O c to b er 2 0 10
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Other Stakeholders treatments. These investigations yield answers that can be used by patients, clinicians, and other stakeholders to help inform their decisions. The results of clinical care have traditionally been evaluated in terms of survival, defined clinical events (such as a stroke), physiological measures (such as blood pressure), and test results (such as laboratory tests, x-ray findings, or pathologic examinations of tissue). These measures are used as the end results in conventional clinical research studies such as clinical trials and longitudinal studies of cohorts of patients. Some of these measures, however, might not have direct relevance to the day-to-day functioning of people with chronic diseases. Some of the most salient information is related to patients’ treatment experiences. In the past three decades, some researchers have extended the range of end results to include measures taken from the societal perspective (for example, costs of care) and from the patient perspective.5 The latter are referred to as “patient-reported outcomes” (Exhibit 1).6 Patients’ reports and evaluations of their own outcomes define effectiveness in a way that can be more meaningful than clinical measures alone. Key Patient-Reported Outcomes Key patient-reported outcomes are shown in Exhibit 2. Health-related quality of life consists of aspects of daily life that can be affected by disease and treatments.7 Another kind of patient-reported outcome is patients’ perceptions of the quality of care that they receive, sometimes referred to as “patient satisfaction.” An additional kind of patient-reported outcome is reports about patients’ health behavior and treatments they receive (for example, adherence to diet or use of a medi-
Exhibit 1 Types Of Patient Outcomes
cation). There have been important advances in the development of patient-reported outcome measures, including the use of structured questionnaires or interviews. A broad array of tools can now provide accurate and reliable measures of different dimensions of health from the patient perspective. Some of these are “generic,” which means that they are intended to be used in general populations of people; an example is the SF12 Short-Form Health Survey.8 Others are “specific,” which means that they are intended to be used for patients with particular diseases or problems; an example is the Asthma Quality of Life Questionnaire.9 Most patient-reported outcome tools are easy to administer and can be completed in just a few minutes. In general, specific tools are more sensitive to changes in a specific disease, while generic tools provide information on heterogeneous patient groups. Patient-reported outcomes directly support the primary goal of much of health care: to improve health-related quality of life, particularly for people with chronic illnesses. No one can judge this better than the patient. For example, the main objective of hip replacement surgery is to reduce pain and improve the capacity to get around. The main goal of cataract extraction is to improve visual functioning—that is, the ability to perform activities that require eyesight, such as reading, walking without falls, and working on a computer. In addition, there are often trade-offs between the length and quality of life. Important considerations are the side effects of treatment of HIV disease, the temporary diminution of functioning after coronary bypass surgery, or fatigue resulting from cancer chemotherapy. Even for life-saving treatments, this kind of trade-off can influence a patient’s decision making among alternative courses of care.
Patient outcomes assessment: sources and examples
Main Data Sources Clinician-reported
Physiological
For example: global impression
For example: FEV1
Caregiver-reported
For example: dependency
Patient-reported
For example: global impression, functional status, well-being, symptoms, HRQL
SOURCE Adapted from Note 6 in text. NOTES FEV1 is forced expiratory volume (test of pulmonary function). HRQL is health-related quality of life.
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Comparative effectiveness research often relies on the secondary use of data collected to support other activities such as clinical research, clinical care, and administration. Randomized clinical trials and observational studies provide the strongest evidence for effectiveness of treatments, but they are expensive and lengthy, and they focus on prespecified outcomes, most of them short term. Clinical care data, which record patients’ encounters with health care providers on paper or in electronic health records, are detailed. They do not, however, reflect care that occurs at other locations, and they can be unclear because pro-
Exhibit 2 Types Of Patient-Reported Outcomes Domain
Example
Health-related quality of life
Perceptions about health in general, physical functioning, social and role functioning, psychological well-being
Symptoms Rating of the quality of care/ satisfaction with care
Pain, nausea, itching Evaluation of amount of information given by the physician
Use of health care services Health behavior
Possession of a peak flow meter for asthma Medication adherence, smoking
SOURCE Authors’ analysis.
viders do not apply definitions uniformly or record information consistently. Clinical registries fall between research cohorts and clinical care data, in that they collect specified data elements but generally have broad inclusion criteria and do not dictate the therapy that must be given. Administrative enrollment and claims data, which include billing data from Medicare, Medicaid, private insurers, and pharmacy benefit managers, provide uniformly collected data for very large numbers of patients. They have the potential for complete capture of the use of care across all providers and settings. All of these data sources are limited in the degree to which they supply the kinds of information ideally needed for comparative effectiveness research (Exhibit 3). None of them systematically captures patient-reported outcomes data. All would be enhanced if they could be supplemented by such data. We next describe how this could be accomplished, either by the routine incorporation of questionnaires into data collection procedures or by linkage of patient-reported outcomes data to data from independent sources. Clinical Trials Patient-reported outcomes are sometimes included as endpoints in clinical trials and observational research, but there are gaps. In cancer, for example, the current US and international clinical trials infrastructure is well
developed and positioned to conduct outcomes research within or alongside randomized studies.10 The National Cancer Institute of Canada– Clinical Trials Group has an explicit policy that “there should be a statement about the anticipated impact on quality of life with every proposed phase III clinical trial and whether or not quality-of-life measures will be incorporated in the protocol.”11 Nonetheless, patient-reported outcomes are included unevenly in studies. A survey of twelve US, Canadian, and European cancer collaborative groups found that although health-related quality-of-life measures are usually included in cancer control studies, they are included in only 5–50 percent of cancer treatment trials.12 A similar situation exists for the AIDS Clinical Trials Group of the National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH). With the advent of effective antiviral treatments, HIV in developed countries has largely become a chronic disease.13 Many current trials involve head-to-head comparisons of combined regimens of effective agents in which differences are likely to hinge on variations in tolerability and health-related quality of life. Although the AIDS Clinical Trials Group has developed a suite of patient-reported outcome measures for use in HIV, few large phase III trials include them.
Exhibit 3 Types Of Information Captured In Different Types Of Data For Comparative Effectiveness Research Type of information captured Health care use Clinical detail Patient perspective
Research data
− ++ −
Clinical care data
Administrative data
+ +++ −
++ + −
SOURCE Authors’ analysis. NOTES A minus sign denotes absence of information of this type. Plus signs indicate the extent of information of this type.
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Other Stakeholders The limited uptake of patient-reported outcome measures may be attributable in part to competing demands placed on research sites, coupled with the relative scarcity of resources. In addition, important differences in patientreported outcomes are not found in all of the studies that include them. However, the failure to collect patient-reported outcome data diminishes our capacity to find those differences. The success of biomedical science in treating many previously fatal diseases and the broad recognition of disease burden attributable to the growing prevalence of chronic illnesses should raise expectations among all stakeholders that patient-reported outcomes be routinely included in clinical research. Observational Studies There has been greater enthusiasm for including patientreported outcomes in major longitudinal cohort studies and large national surveys. For example, the Multicenter AIDS Cohort Study14 and the large HIV Cost and Service Utilization Study15 collect patient-reported outcomes, such as health-related quality of life, symptoms, and medication adherence. Large national surveys such as the Medical Expenditure Panel Survey (MEPS) include multiple patient-reported outcome measures such as the SF-12 Short-Form Health Survey, EuroQol EQ-5D brief health utility measures, and Instrumental Activities of Daily Living Scale.16 The primary care practice–based research networks supported by AHRQ link information on health services with patient-reported outcomes data in community-based clinical care settings for more than thirty-eight million patients in forty-nine states.17 ▸▸ PROMIS INITIATIVE : Begun in 2004, the Patient-Reported Outcomes Measurement Information System (PROMIS) is an NIH-supported initiative that aims to develop more-systematic ways to measure aspects of health-related quality of life and symptoms, such as pain and fatigue, across a wide variety of chronic diseases and conditions.18 Its goal is to become a national resource for the accurate and efficient measurement of patient-reported health outcomes in clinical research and patient care. The initiative plans to develop a set of publicly available “computerized adaptive tests” for the clinical research community. Instead of asking a person a series of questions about an aspect of his or her health, this technology uses the person’s last response to select which question to ask next, making the process simultaneously more precise and less burdensome. ▸▸ PATIENT REGISTRIES : Patient registries have also not yet achieved their potential for collecting patient-reported outcomes. For exam-
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Patient-reported outcomes are included unevenly in studies.
ple, the National Cancer Institute’s Surveillance Epidemiology and End Results (SEER) Program is one of the oldest and best-established registries in the United States, covering approximately 26 percent of the US population. However, although the program’s registries track the vital status of each patient, they do not routinely collect data on patient-reported outcomes.19 To address this limitation, the program has supported studies to augment the registry data with data from patient interviews.20 One special study, which tracked 3,500 men for five years after their diagnosis of prostate cancer, found that those treated with radical prostatectomy for localized prostate cancer experienced worse urinary incontinence, but similar sexual functioning, compared with those treated with external beam radiotherapy.21 Clinical Data In recent years, there has been increased interest in using patient-reported outcomes as clinical care tools to aid in patient management. There are various ways that patientreported data can be used in clinical practice. ▸▸ TIME HORIZONS AND CARE TEAMS : For example, questionnaires can be used at single points in time to screen patients for conditions (such as depression) or followed longitudinally to monitor patients’ functional status over time (for example, to assess response to treatment). Another application of patient-reported outcomes in clinical practice involves multidisciplinary care teams, using patients’ reports to help assess their health and formulate appropriate treatment strategies (such as in the rehabilitation setting). ▸▸ AGGREGATING DATA : Patient-reported outcomes data can also be aggregated and put to “secondary use” to help evaluate the quality of health care delivery and the effectiveness of treatments. An example of patient-reported outcomes’ being used as part of quality evaluations is the Patient-Reported Outcomes Measures initiative by the National Health Service (NHS) in England and Wales. As of April 1, 2009, the NHS became the first system in the world to collect patient-reported outcomes measures routinely.22 Patients having hip or knee replacements, varicose vein surgery, or groin hernia
surgery are asked to complete preoperative and postoperative questionnaires. The main goals for using the data include comparing providers and thus helping patients and general practitioners choose among providers for these procedures. There has been broad participation in the program: 99,740 preoperative questionnaires were collected in the first nine months of the program, and the participation rate has been as high as 61.7 percent for knee replacements.23 Besides being used to compare the results obtained by different surgeons, the data could be used to evaluate the efficiency and cost-effectiveness of different types of artificial joints, surgical approaches, or rehabilitation regimens. Data for the Patient-Reported Outcomes Measures initiative are being collected primarily to evaluate the quality of care. However, incorporating these data into routine clinical care for individual patient monitoring provides an alternative mechanism for creating a data resource that includes patients’ reports of their outcomes. To be most useful, patients’ reported outcomes data should be linked with their other clinical data in an electronic medical record. ▸▸ PATIENT PORTALS : Recent technological innovations make such linkages increasingly feasible. For example, so-called patient portals are an option frequently offered by electronic health record vendors. Functions commonly include secure communication with the clinician, appointment scheduling, prescription refills, and the opportunity to complete intake and other forms that used to be completed on paper in the office. A logical extension would be to use patient portals as a mechanism for making patient-reported outcomes data available within the electronic health record. Our multidisciplinary team is currently developing a website specifically to collect patientreported outcomes and link them with the electronic health record.24 The website, Patient Viewpoint.org, has functions allowing care providers to order questionnaires for the patient to fill in and e-mail reminders to patients; patients to complete the questionnaires; and patients and clinicians to view results graphically over time. In addition, the website can provide clinicians with suggestions to address issues identified through the questionnaires. PatientViewpoint .org is linked with the Johns Hopkins electronic health record. It is expected that the database’s back end could link with other institutions’ electronic health records, depending on the specific institution’s requirements. In one possible scenario, a woman with breast cancer is being followed by an oncologist who would like to know how she is doing on the
chemotherapy regimen she is receiving. The oncologist logs on to PatientViewpoint.org, enters the patient’s number, and orders the BR-23 Breast Cancer–Specific Quality of Life Questionnaire for her to complete online before her next visit. The patient receives an e-mail notification to do this, logs on to PatientViewpoint.org, and completes the survey. The patient’s results are automatically calculated and are made available both on the website and within the hospital’s electronic health record alongside all of her other laboratory test results. At the visit, the oncologist pulls up the results and asks the patient about an increase in her depression scores. It would also be possible to aggregate all of the patient’s questionnaire results with those of other patients receiving chemotherapy for similar breast cancer cases and to use these data to help compare the effectiveness of different regimens. Administrative Data It could also be useful to tie administrative claims data, such as those generated by Medicare, private insurers, and pharmacy benefits managers, to patient-reported data on outcomes and behavior. There are several strategies that could be used to achieve this. ▸▸ ROUTINE COLLECTION : One strategy is to build the routine collection of these data into standard operating procedures. An example is the development of a standardized survey instrument for the routine collection of patients’ perspectives on their hospital care: the Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) survey by the Centers for Medicare and Medicaid Services (CMS).25 Bolstered by a matching data-collection methodology and requirements for compliance with data quality assurance guidelines, HCAHPS provides a national standard that can be used to compare different hospitals. ▸▸ REQUIRED PARTICIPATION : A second example is the required participation of all Medicare managed care plans with Medicare Advantage contracts in the Medicare Health Outcomes Survey.26 This involves administering the Veterans RAND 12-Item Health Survey (VR-12)27 to a sample of patients (see Appendix Exhibit 128 for sample questions). This survey is a close cousin to the widely used SF-12.8 ▸▸ REIMBURSING PROVIDERS : A third example is a CMS demonstration that reimbursed medical oncologists for collecting three symptom-related outcome measures (pain, nausea, and fatigue) on their Medicare enrollees undergoing chemotherapy.29 The success of this effort was limited by variability in how the data were collected and submitted, but it shows how the collection of patient-reported outcomes could be ordered and administered in ways similar to other diagO c tob e r 2 0 1 0
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Other Stakeholders nostic tests. ▸▸ USING EXISTING DATA : Because large-scale routine data collection involves substantial costs, a another strategy is to link to patientreported data after the administrative data have already been collected. A recent development (Andy Paris, Vigilytics, personal communication with Albert Wu, June 9, 2010) permits the merging of administrative health care claims data with other databases. This method allows respondents from online survey panels to allow their survey responses to be augmented with administrative data, such as from a health insurer. These methods create separate deidentified research databases that comply with the Health Insurance Portability and Accountability Act (HIPAA) and that can be used to validate selfreported information, add economic variables, or allow more-comprehensive compliance research to be conducted. Existing registries or clinical trial databases can be linked to prescription histories, which would allow analysis of preexisting conditions and the impact of treatment compliance on patient outcomes. In the examples given above, the creation of linked data sets that include both administrative and patient-reported data sources would complement comparative effectiveness research. Administrative data could provide large, generalizable samples, while patient-reported data could provide the patient perspective and be used to confirm diagnoses, treatments, adherence, and care use.
Policy Implications A century ago, Sir William Osler counseled physicians: “Listen to the patient: He is telling you the diagnosis.” A century later, modern medicine relies primarily on tests and procedures for diagnosis and assessments of disease (health) outcomes. However, assessing some outcomes requires directly asking patients about their physical, mental, and social functioning, as well as symptoms. Fortunately, as the measurement of biological status has advanced, so has the measurement of patient-reported health status and symptoms.We are now at a point where there exist standardized and valid methods for asking patients their status, which makes it possible to track patient outcomes over time and compare patients to others. Just as we record the results of all the biologic tests, we can also record patient-reported status in the medical record. These data can be reported on flowcharts, while clinically significant outcomes can be flagged to ensure follow-up similar to what is done with laboratory findings. The routine collection of patient-reported 1868
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It is apparent that there is growing demand for the collection of patientreported outcome measures.
health information is not a novel idea; it was suggested by Paul Ellwood in his 1988 Shattuck lecture on outcomes management.30 However, there has not been a rush to adopt the practice across American medicine. In addition to measuring value in clinical care and guiding patientcentered care, patient-reported outcomes play an important role in comparative effectiveness research. The rationale for data collection may also be strengthened by the strong interest among purchasers and the public in using outcomes—rather than processes—to assess quality. Several steps could be taken to permit it. Payment It is apparent that there is increasing interest in and growing demand for the collection of patient-reported outcome measures. Indicators of this interest include proposals that CMS pay for episodes of care, not just individual services, and take patient outcomes into account. An episode of care might be defined by a surgical procedure and functional status ninety days after discharge. Providers with the best riskadjusted patient outcomes could receive additional reimbursement or might be identified as “preferred” to encourage patients to select their services. In keeping with the concepts of using bundled payments for hospital and physician services under Medicaid, payers might pay for the collection of patient-reported outcomes data in the same way as they pay for laboratory tests. Technology Assessment Another approach is a policy mechanism that links coverage by payers with a requirement that patients participate in relevant research such as registries or clinical trials. The CMS strategy has been termed “coverage with evidence development.” It has been used in several high-profile coverage decisions.31 This mechanism was designed to be applied when promising findings suggested that patients might benefit from a new technology, but additional evidence was needed to determine this with confidence.
An example was research on the safety and effectiveness of bariatric surgery in patients age sixty-five and older. A logical extension of this might be a requirement that the evaluation include reports directly from the patients who receive the treatment. Electronic Health Records Another policy focus is the implementation of electronic health records under the American Recovery and Reinvestment Act. Incentive payments will be provided through Medicare and Medicaid for the adoption and so-called meaningful use of electronic health records, as defined in regulations.32 Meaningful use includes using the electronic health record to collect, analyze, and report quality indicators. For example, CMS provides incentives to report quality indicators through its Physician Quality Reporting Initiative.33 That initiative is expanded each year and could include patient-reported outcome measures in the future. Increasing Value Health policy is increasingly being driven by the value proposition, with a focus on the ratio of outcomes to cost. Higher value is achieved by improving outcomes, reducing costs, or both. Today we frequently use process-of-care quality indicators as proxies for outcomes. Although many process measures are linked to outcomes in clinical research, there is a clear need and strong interest in assessing outcomes directly. Many of the relevant outcomes, and certainly many that patients value, require patient-reported information.
Conclusion In coming years, new treatments, diagnostic modalities, and systems of care will continue to be developed. Payment systems will continue
to evolve, along with increasing pressure to align financial incentives with quality and outcomes. Stakeholders—including patients, institutional purchasers, and governmental payers—will continue to pursue value-based purchasing, and individual providers and organizations will continue to be held accountable for the results that they deliver. All of these forces will accelerate the demand for more and better evidence to inform decision making. At this moment, no single data source or mechanism for data collection is adequate to support the growing demand for comparative effectiveness research. Accelerating the process will depend on augmenting existing sources of research, clinical, and administrative data with patient-reported outcomes. We have suggested several mechanisms and policy opportunities to support research that incorporates patientreported outcomes that are otherwise unavailable in any health care data or records. In particular, we have described methods and structures to collect, combine, or link existing data sources with patient-reported outcomes. These could include inclusion of patientreported outcomes more uniformly in clinical trials and large cohort studies, integration into a high-quality system of disease and treatment registries, inclusion in electronic health records in a way that would support secondary use, and incorporation into administrative data via both standardized collection and targeted projects. Patient-reported outcomes are not a panacea, and they will not make a difference in all comparative effectiveness studies. However, understanding treatment and disease impact from the patient perspective will make it possible for comparative effectiveness research to support delivery of high-quality, patient-centered care. ▪
The authors acknowledge valuable collaborations with members of the International Society for Quality of Life Research (ISOQOL), which contributed to this work.
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Other Stakeholders NOTES 1 Agency for Healthcare Research and Quality. What is comparative effectiveness research? [Internet]. Rockville (MD): AHRQ; [cited 2010 Sep 13]. Available from: http://www .effectivehealthcare.ahrq.gov/ index.cfm/what-is-comparativeeffectiveness-research1/ 2 Sox HC, Greenfield S. Comparative effectiveness research: a report from the Institute of Medicine. Ann Intern Med. 2009;151(3):203–5. 3 US Department Health and Human Services, Federal Coordinating Council for Comparative Effectiveness Research. Report to the president and the Congress [Internet]. Washington (DC): HHS; 2009 Jun 30 [cited 2010 Sep 13]. Available from: http://www.hhs.gov/ recovery/programs/cer/ cerannualrpt.pdf 4 Institute of Medicine. Crossing the quality chasm: a new health system for the 21st century. Washington (DC): National Academies Press; 2001. 5 Clancy CM, Eisenberg JM. Outcomes research: measuring the end results of health care. Science. 1998; 282(5387):245–6. 6 Acquadro C, Berzon R, Dubois D, Leidy NK, Marquis P, Revicki D, et al. Incorporating the patient’s perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. Value Health. 2003;6(5):522–31. 7 Patrick DL, Bergner M. Measurement of health status in the 1990s. Annu Rev Public Health. 1990;11: 165–83. 8 Ware J Jr., Kosinski M, Keller SD. A 12-item Short-Form Health Survey: construction of scales and preliminary tests of reliability and validity. Med Care. 1996;34(3):220–33. 9 Juniper EF, Guyatt GH, Epstein RS, Ferrie PJ, Jaeschke R, Hiller TK. Evaluation of impairment of health related quality of life in asthma: development of a questionnaire for use in clinical trials. Thorax. 1992;47(2): 76–83. 10 Gotay C, Hinds PS, Kornblith AB, Movsas B, Sloan J, Wenzel L, et al. Issues and challenges with integrating patient-reported outcomes in clinical trials supported by the National Cancer Institute–sponsored clinical trials networks. J Clin Oncol. 2007;25(32):5051–7. 11 Brundage M, Osoba D, Bezjak A, Tu D, Palmer M, Pater J, et al. Lessons learned in the assessment of healthrelated quality of life: selected examples from the National Cancer Institute of Canada Clinical Trials Group. J Clin Oncol. 2007;25(32):
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5078–81. 12 Bruner DW, Bryan CJ, Aaronson N, Blackmore CC, Brundage M, Cella D, et al. Issues and challenges with integrating patient-reported outcomes in clinical trials supported by the National Cancer Institute–sponsored clinical trials networks. J Clin Oncol. 2007;25(32):5051–7. 13 Wu AW. Quality of life assessment comes of age in the era of highly active antiretroviral therapy. AIDS. 2000;14(10):1449–51. 14 Bing EG, Hays RD, Jacobson LP, Chen B, Gange SJ, Kass NE, et al. Health-related quality of life among people with HIV disease: results from the Multicenter AIDS Cohort Study. Qual Life Res. 2000;9(1): 55–63. 15 Hays RD, Cunningham WE, Sherbourne CD, Wilson IB, Wu AW, Cleary PD, et al. Health-related quality of life in patients with human immunodeficiency virus infection in the United States: results from the HIV Cost and Services Utilization Study. Am J Med. 2000;108(9): 714–22. 16 Agency for Healthcare Research and Quality. Medical Expenditure Panel Survey (MEPS) [home page on the Internet]. Rockville (MD): AHRQ; [cited 2010 Sep 13]. Available from: http://meps.ahrq.gov 17 Agency for Healthcare Research and Quality. Practice based research networks (PBRNs) [home page on the Internet]. Rockville (MD): AHRQ; [cited 2010 Sep 13]. Available from: http://pbrn.ahrq.gov 18 National Institutes of Health. PROMIS [home page on the Internet]. Bethesda (MD): NIH; [cited 2010 Sep 13]. Available from: http:// www.nihpromis.org 19 National Cancer Institute. Surveillance Epidemiology and End Results (SEER) [home page on the Internet]. Bethesda (MD): NIH; [cited 2010 Sep 13]. Available from: http://seer .cancer.gov/ 20 National Cancer Institute. Rapid Response Surveillance Studies: evaluating cancer treatment and outcomes [home page on the Internet]. Bethesda (MD): NIH; [cited 2010 Sep 13]. Available from: http:// seer.cancer.gov/rapidresponse/ outcomes/ 21 Potosky AL, Davis WW, Hoffman RM, Stanford JL, Stephenson RA, Penson DF, et al. Five-year outcomes after prostatectomy or radiotherapy for prostate cancer outcomes study. J Natl Cancer Inst. 2004;96(18): 1358–67. 22 National Health Service. Patient Reported Outcome Measures (PROMS) [home page on the Internet]. London: NHS; [cited 2010 Sep 13]. Available from: http://www
.ic.nhs.uk/proms 23 National Health Service. Provisional monthly Patient Reported Outcome Measures (PROMs) in England, April to November 2009 [Internet]. London: NHS Information Centre; 2010 [cited 2010 Sep 13]. Available from: http://www.ic.nhs.uk/ statistics-and-data-collections/ hospital-care/patient-reportedoutcome-measures-proms/ provisional-monthly-patientreported-outcome-measures-promsin-england–april-2009-to-april2010-pre-and-post-operative-dataexperimental-statistics 24 Snyder CF, Jensen R, Courtin SO, Wu AW. PatientViewpoint: a website for patient-reported outcomes assessment. Qual Life Res. 2009;18: 793–800. 25 Centers for Medicare and Medicaid Services. HCAHPS: CAHPS hospital survey [home page on the Internet]. Baltimore (MD): CMS; [cited 2010 Sep 13]. Available from: http://www .hcahpsonline.org 26 Safran DG. Measuring, monitoring, and reporting functional health outcomes: opportunities and challenges in a bold national initiative. Int J Qual Health Care. 2001;13(1): 7–8. 27 Kazis LE, Selim A, Rogers W, Ren XS, Lee A, Miller DR. Dissemination of methods and results from the veterans health study: final comments and implications for future monitoring strategies within and outside the veterans healthcare system. J Ambul Care Manage. 2006; 29(4):310–9. 28 To access the Appendix, click on the Appendix link in the box to the right of the article online. 29 Lipscomb J, Gotay CC, Snyder CF. Patient-reported outcomes in cancer: a review of recent research and policy initiatives. CA Cancer J Clin. 2007;57(5):278–300. 30 Ellwood PM. Shattuck lecture—outcomes management: a technology of patient experience. N Engl J Med. 1988;318(23):1549–56. 31 Pearson SD, Miller FG, Emanuel EJ. Medicare’s requirement for research participation as a condition of coverage: is it ethical? JAMA. 2006; 296(8):988–91. 32 Centers for Medicare and Medicaid Services. Final rule CMS-1390-F, changes to the hospital inpatient prospective payment systems and fiscal year 2009 rates. Fed Regist. 2008 Aug 19 [updated 2010 Apr 9]. 33 Centers for Medicare and Medicaid Services. Physician Quality Reporting Initiative (PQRI) [home page on the Internet]. Baltimore (MD): CMS; [cited 2010 Sep 13]. Available from: https://www.cms.gov/pqri/
ABOUT THE AUTHORS: ALBERT W. WU, CLAIRE SNYDER, CAROLYN M. CLANCY & DONALD M. STEINWACHS
Albert W. Wu is a professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health.
Albert Wu and coauthors discuss the importance of capturing the patient perspective in comparative effectiveness research, as an essential element in understanding the full impact of a given treatment. “I think it’s still not routine” to incorporate a patient’s views on, say, pain, in assessing the pros and cons of a health care intervention, says Wu, a professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health and of internal medicine at the Johns Hopkins School of Medicine. Coauthor Carolyn Clancy, director of the federal Agency for Healthcare Research and Quality (AHRQ), agrees. She points out that understanding so-called patient-reported outcomes is critical to assessing life-extending treatments in particular. “Knowing if you’re doing more harm than good has to come from the individual patients,” Clancy says. Wu directs a Hopkins center for comparative effectiveness research funded by AHRQ. Since 1987 he has focused on the adoption and use of patient-reported outcomes to take account of the patient’s experience in measuring treatment efficacy. An expert in the care of people with HIV infection, Wu was among the first to develop tools to measure patient experience in clinical trials of HIV/AIDS treatments.
Wu is a past president of the International Society for Quality of Life. Since 1988, he has had a research interest in the handling of medical errors. He received his medical degree from Cornell University and a master of public health degree from the University of California, Berkeley.
AHRQ’s Center for Outcomes and Effectiveness Research. A general internist, Clancy is clinical associate professor of medicine at the George Washington University School of Medicine and a senior associate editor of Health Services Research. She is a member of the Institute of Medicine. Clancy’s major research interests are patient safety, health care quality, and reducing health disparities. She earned her medical degree from the University of Massachusetts Medical School.
Claire Snyder is an assistant professor at Johns Hopkins.
Claire Snyder is an assistant professor in the Division of General Internal Medicine at the Johns Hopkins School of Medicine, with joint appointments in oncology at the medical school and in health policy and management at the Bloomberg School of Public Health. Her research interests include quality of life for cancer patients undergoing treatment and the coordination of care for cancer survivors. She earned her doctorate in health policy at the Bloomberg School.
Carolyn M. Clancy is director of the Agency for Healthcare Research and Quality.
Carolyn Clancy was appointed director of AHRQ in February 2003. Previously, she directed
Donald M. Steinwachs is a professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health.
Donald Steinwachs is director of the Health Services Research and Development Center at the Bloomberg School. He holds joint appointments in the Johns Hopkins schools of medicine, nursing, and arts and sciences, and served for eleven years as chair of Bloomberg’s Department of Health Policy and Management. A member of the Institute of Medicine since 1993, Steinwachs has studied medical effectiveness and outcomes for people with medical, surgical, and psychiatric needs. He currently focuses on health care quality. Steinwachs earned his doctorate in operations research from what is now the Johns Hopkins Whiting School of Engineering.
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