Health Outcomes Research in Medicine

The Rise of Patient-Centered Outcomes Research

Rachael Fleurence — 2012-01-24

The newly formed US Patient-Centered Outcomes Research Institute (PCORI) has been tasked with “helping people make informed health care decisions and improving health care delivery and outcomes by producing and promoting high integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community.” PCORI is currently embarking on funding its first set of pilot research projects, on which it will spend close to $13 million in 2012. In this issue of Health Outcomes Research in Medicine, we are indeed reminded of the rising importance of patient-centered outcomes research. Patient-centered outcomes research is diverse and has to address a large range of issues varying from stakeholder involvement to health care quality, efficiency, and delivery, and our current issue certainly reflects this mixture. With a variety of contributions, we address such diverse topics as patient satisfaction with different types of providers, health-related quality of life (HRQoL) in head and neck cancer, the importance of appropriate outcome measures both in economic evaluations and in safety database studies, and real-world treatment patterns in patients with anxiety disorders.

Who Will Take Care of Us? Exploring Differences in Respondents’ Satisfaction with Primary Care vs Specialty Care Physicians

Michael Adolph, Jun Wu, Steven R. Feldman, Rajesh Balkrishnan — 2011-11-07

Abstract: Objective: Our study explored perceived patient satisfaction with either primary care or specialist physicians to identify factors accounting for the differences.Study Design: The data were collected from an Internet-based survey, DrScore.com, for measuring patient satisfaction with physicians. Participants found their doctors through the DrScore search engine and rated their physicians with anonymity. A total satisfaction score was the sum of scores based on 9 physician rating items and then was scaled to the range of 0-100. Logistic regressions were used to analyze associations between patient satisfaction (score ≥70) and various factors.Results: The mean satisfaction score was 79.4 for primary care (n = 11,558) and 75.5 for specialty care (n = 11,068) (P > .05). Nearly 50% of primary care patients waited for 0-2 days to get an appointment, while more than 50% of specialty care patients waited for more than 6 days. As waiting days became longer than 2 weeks, patient ratings of specialty care were lower than those of primary care. Patients (≥45 years) were 24% less likely to be satisfied with primary care (P < .01) but 40% more likely with specialty care (P < .01) than patients (<25 years).Conclusions: Although differences in overall patient satisfaction with primary and specialty care were not observed, more specialists obtained extremely low satisfaction scores than primary care providers did. Age and factors related to waiting time for the visit or time spent with a doctor were associated with patient satisfaction with physicians.

The Quality-of-Life Impact of Head and Neck Cancer: Preference Values from the Canadian General Public

2011-12-19

Abstract: Objectives: To elicit preferences for standardized head and neck cancer (HNC) health states describing the health-related quality of life (HRQoL) impact of cancer type or stage, progression, or treatment-related toxicities; and to measure the association between an individual’s locus of control (LOC) and mean preference values for HNC health states.Study Design: We elicited preferences from a sample of 106 members of the Canadian general public, using the standard gamble method. Eight health states representing HNC characteristics, and 10 describing treatment-related toxicities, were developed based on literature review, data analysis, and clinician interview. All participants valuated these, anchored against full health and dead, and completed the Multidimensional Health LOC scale. A mixed-regression model was used to calculate adjusted preference decrements for all states compared with a reference state (locoregional preprogression nonlaryngeal HNC).Results: Mean participant age was 47 years, and 48% were male. All health states were associated with substantially decreased preferences compared with full health. Mean preferences ranged from 0.62 (locoregional laryngeal HNC) to 0.33 (hospitalization for severe toxicity). After adjusting for age and sex, mean preference decrements were: −0.28 (postprogression), −0.11 (metastases), and −0.05 (recurrent disease). There was suggestive evidence that LOC was associated with preferences overall (P=.079); those with stronger beliefs in Chance rated health states lower (P=.012).Conclusions: Health state preferences elicited here demonstrate that members of the Canadian general public rate HNC to have a large negative impact on HRQoL. The greatest impact was for postprogression and metastatic health states. These values are useful for quantifying the devastating impact of HNC on HRQoL, and for economic modeling.

The Ends Justify the Mean: Outcome Measures for Estimating the Value of New Cancer Therapies

2012-01-16

Abstract: Objective: Overall survival is a commonly reported end point in clinical trial publications and a key determinant of therapies’ cost-effectiveness. Patients’ survival times have skewed distributions. Outcomes are typically presented in clinical trials as the difference in median survival times; we compare median survival gain with the measure required for economic evaluation, the mean difference.Study Design: We summarize the relationships between median and mean survival in 4 parametric survival distributions and the relationship of the differences in these measures between trial arms and parameterized treatment effects. Parametric estimates of mean survival were compared with median survival in a case study of a recent trial in metastatic melanoma.Results: In a trial of alternative therapies in unresectable metastatic melanoma, median overall survival with ipilimumab alone was 10.1 months versus 6.4 months with gp100-alone (hazard ratio 0.66; P = 0.003). A log-normal parametric survivor function fitted the gp100 Kaplan-Meier function and a time ratio of 1.90 applied only after 90 days gave a suitable fit to the Kaplan-Meier function for ipilimumab, with mean survival difference of 7 months, compared with an estimate of 5.7 months employing a Weibull distribution, and with a 3.7-months median difference.Conclusion: Parametric assessment of mean survival gain in clinical trials may indicate potential benefits to patients that observed medians may greatly underestimate.

Health Outcomes of Interest in Observational Data: Issues in Identifying Definitions in the Literature

2011-11-28

Abstract: Objectives: To describe the literature search strategies and results to inform development of health outcomes of interest (HOI) definitions studied within the Observational Medical Outcomes Partnership (OMOP) research program. These HOIs represent the safety and benefit endpoints that may be associated with the use of particular medications.Methods: Two organizations implemented search strategies to identify articles that described the validation of various HOI definition algorithms and produced systematic reviews of the published literature for the OMOP HOIs; reviews of 5 HOIs were available from each organization. Search results were summarized descriptively.Results: Based on the differences in publications identified by each organization, neither search strategy captured all the relevant literature across 5 HOIs. A composite search strategy developed by the OMOP research team failed to effectively capture all relevant publications as well. Among publications in observational databases, a large proportion did not report actual codes used to identify HOIs in administrative claims databases, nor did they provide substantive detail of any validation studies.Conclusion: No single search strategy or literature database captured all relevant articles efficiently. The lack of specific search terms hampered identification of relevant articles. Due to limited details about positive predictive value of various HOI definitions, information culled from available literature was insufficient to identify one best definition for each HOI. Researchers seeking to capture and understand prior published work that defined relevant outcomes of interest need to be aware of the shortcomings of this approach and the lack of detail of validation studies available in published literature.

Health Care Utilization and Costs in Patients with Generalized Anxiety Disorder Initiating Add-on Therapy with Benzodiazepines

2011-11-14

Abstract: Objectives: To examine patterns of health care utilization and costs in patients with generalized anxiety disorder (GAD) who begin treatment with benzodiazepine anxiolytics as add-on therapy.Study Design: In a large US health insurance database, we identified all patients with evidence of GAD (International Classification of Diseases, 9th Revision, Clinical Modification diagnosis code 300.02) who received ≥90 days of therapy with a selective serotonin reuptake inhibitor or venlafaxine between January 1, 2003 and December 31, 2007. Among these patients, we selected those who initiated a course of benzodiazepine add-on therapy. Designating the date of initial receipt of a benzodiazepine as the “index date,” we examined health care utilization and costs over the 6-month period preceding this date (“pre-index”) and the 12-month period following it (“follow-up”).Results: A total of 2131 patients met all study inclusion criteria. Patients averaged 32 days of therapy with benzodiazepines (median [interquartile range] = 20 [10-30]); 13% of patients received >90 days of therapy, however. In general, levels of health care utilization during the first 6 months of follow-up were higher than those during the pre-index period; between months 7 and 12 of follow-up, however, they were somewhat lower than pre-index levels. Mean (SD) total health care costs were $5148 ($10,658), $6325 ($15,741), and $5373 ($11,230) during pre-index, months 1-6 of follow-up, and months 7-12 of follow-up, respectively.Conclusions: Levels of health care utilization and costs increase following initiation of add-on therapy with a benzodiazepine in patients with GAD receiving selective serotonin reuptake inhibitors or venlafaxine. Although duration of add-on therapy is typically brief, some patients are treated for >90 days, raising potential concerns about risks of dependency and sedation.