Recommendations to address respondent burden associated with patient-reported outcome assessment.

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making. We therefore conducted an initial literature review to generate a list of candidate recommendations aimed at reducing respondent burden. This was followed by a two-stage Delphi survey by an international multi-stakeholder group. A consensus meeting was held to finalize the recommendations. The final consensus statement includes 19 recommendations to address PRO respondent burden in healthcare research and clinical practice. If implemented, these recommendations may reduce PRO respondent burden.

How to conduct cost and value analyses in health professions education: AMEE Guide No. 139.

Growing demand for accountability, transparency, and efficiency in health professions education is expected to drive increased demand for, and use of, cost and value analyses. In this AMEE Guide, we introduce key concepts, methods, and literature that will enable novices in economics to conduct simple cost and value analyses, hold informed discussions with economic specialists, and undertake further learning on more advanced economic topics. The practical structure for conducting analyses provided in this guide will enable researchers to produce robust results that are meaningful and useful for improving educational practice. Key steps include defining the economic research question, identifying an appropriate economic study design, carefully identifying cost ingredients, quantifying, and pricing the ingredients consumed, and conducting sensitivity analyses to explore uncertainties in the results.

Cost evaluations in health professions education: a systematic review of methods and reporting quality.

CONTEXT: High-quality research into education costs can inform better decision making. Improvements to cost research can be guided by information about the research questions, methods and reporting of studies evaluating costs in health professions education (HPE). Our objective was to appraise the overall state of the field and evaluate temporal trends in the methods and reporting quality of cost evaluations in HPE research. METHODS: We searched the MEDLINE, CINAHL (Cumulative Index to Nursing and Allied Health Literature), EMBASE, Business Source Complete and ERIC (Education Resources Information Centre) databases on 31 July 2017. To evaluate trends over time, we sampled research reports at 5-year intervals (2001, 2006, 2011 and 2016). All original research studies in HPE that reported a cost outcome were included. The Medical Education Research Study Quality Instrument (MERSQI) and the BMJ economic checklist were used to appraise methodological and reporting quality, respectively. Trends in quality over time were analysed. RESULTS: A total of 78 studies were included, of which 16 were published in 2001, 15 in 2006, 20 in 2011 and 27 in 2016. The region most commonly represented was the USA (n = 43). The profession most commonly referred to was that of the physician (n = 46). The mean ± standard deviation (SD) MERSQI score was 10.9 ± 2.6 out of 18, with no significant change over time (p = 0.55). The mean ± SD BMJ score was 13.5 ± 7.1 out of 35, with no significant change over time (p = 0.39). A total of 49 (63%) studies stated a cost-related research question, 23 (29%) stated the type of cost evaluation used, and 31 (40%) described the method of estimating resource quantities and unit costs. A total of 16 studies compared two or more interventions and reported both cost and learning outcomes. CONCLUSIONS: The absolute number of cost evaluations in HPE is increasing. However, there are shortcomings in the quality of methodology and reporting, and these are not improving over time.

Responding to a small-scale bioterrorist anthrax attack: cost-effectiveness analysis comparing preattack vaccination with postattack antibiotic treatment and vaccination.

BACKGROUND: In 2001, a small-scale bioterrorism-related anthrax attack was perpetrated via the US mail. The optimal future response may require strategies different from those required in a large-scale attack. METHODS: We conducted a cost-effectiveness analysis using Monte Carlo simulation during a 10-year time frame from a societal perspective to determine the optimal response strategy for a small-scale anthrax attack perpetrated against US Postal Service distribution centers in a large metropolitan area. Three strategies were compared: preattack vaccination of all US distribution center postal workers, postattack antibiotic therapy followed by vaccination of exposed personnel, and postattack antibiotic therapy without vaccination of exposed personnel. Outcome measures were costs, quality-adjusted life-years, and incremental cost-effectiveness. The probabilities for anthrax exposure and infection; vaccine and antibiotic benefits, risks, and costs; and associated clinical outcomes were derived from the medical literature and from bioterrorism experts. RESULTS: Postattack antibiotic therapy and vaccination of exposed postal workers is the most cost-effective response compared with other strategies. The incremental cost-effectiveness is $59 558 per quality-adjusted life-year compared with postattack antibiotic therapy alone. Preattack vaccination of all distribution center workers is less effective and more costly than the other 2 strategies. Assuming complete adherence to preattack vaccination, the incremental cost-effectiveness compared with postattack antibiotic therapy alone is almost $2.6 million per quality-adjusted life-year. CONCLUSION: Despite uncertainties about a future anthrax attack and exposure risk, postattack antibiotic therapy and vaccination of exposed personnel seems to be the optimal response to an attack perpetrated through the US Postal Service.

Cost-effectiveness of dilation and evacuation versus the induction of labor for second-trimester pregnancy termination.

OBJECTIVE: The purpose of this study was to compare the cost-effectiveness of dilation and evacuation versus misoprostol induction of labor for second-trimester termination. STUDY DESIGN: Using decision analysis, we compared the cost-effectiveness of dilation and evacuation and misoprostol induction of labor for second-trimester termination. Complications for dilation and evacuation and induction of labor included repeat dilation and curettage, cervical laceration repair, hospital admission, laparotomy, hysterectomy, and maternal death. Induction of labor complications also included failed induction of labor. The primary outcome was cost per quality-adjusted life year. Sensitivity analyses were performed for all relevant variables. RESULTS: Dilation and evacuation was less costly and more effective than misoprostol induction of labor for second-trimester termination with baseline estimates. In 1-way sensitivity analysis, the model was robust to all variation in probabilities and costs. In Monte Carlo simulation with 1000 trials and a cost-effectiveness threshold of $50,000/quality-adjusted life year, dilation and evacuation was the preferred approach in 97.9% of trials. CONCLUSION: Dilation and evacuation is less expensive and more effective than misoprostol induction of labor for second-trimester termination.

Cruise ship care: a proposed alternative to assisted living facilities.

Options for elderly patients who can no longer remain independent are limited. Most choices involve assisted living facilities, 24-hour caregivers, or nursing homes. State and federal assistance for payment for individual care is limited, and seniors usually pay for most costs out of pocket. For those patients who have the means to afford assisted living centers or nursing homes, "cruise ship care" is proposed. Traveling alongside traditional tourists, groups of seniors would live on cruise ships for extended periods of time. Cruise ships are similar to assisted living centers in the amenities provided, costs per month, and many other areas. This article begins with an examination of the needs of seniors in assisted living facilities and then explores the feasibility of cruise ship care in answering those needs. Similarities between cruise ship travel and assisted living care, as well as the monetary costs of both options, are defined. A decision tree with selections for non-independent care for seniors was created including cruise ship care as an alternative. Using a Markov model over 20 years, a representative cost-effectiveness analysis was performed that showed that cruises were priced similarly to assisted living centers and were more efficacious. Proposed ways that cruise ship companies could further accommodate the needs of seniors interested in this option are also suggested. Implementation for cruise ship care on the individual basis is also presented. Ultimately, it is wished to introduce a feasible and possibly more desirable option to seniors who can no longer remain independent.

Cost minimization study of image-guided core biopsy versus surgical excisional biopsy for women with abnormal mammograms.

PURPOSE: To describe the clinical and economic consequences of image-guided core biopsy versus surgical excisional biopsy of mammographically identified breast lesions. PATIENTS AND METHODS: Clinical and economic data were collected for 1121 patients undergoing core biopsies and 501 patients undergoing surgical biopsies between 1996 and 1998. Lesions were classified according to mammographic degree of suspicion and type of radiographic abnormality. Costs were measured from the societal perspective. A decision analytic model was constructed, with probabilistic sensitivity analysis. RESULTS: Lesions diagnosed via core versus surgical biopsy were less likely to be masses (39% v 55%), less likely to be classified as high cancer suspicion (17% v 26%), and less likely to be treated with a single procedure (74% v 81%; P <.001 for each). Cancers diagnosed by a surgical biopsy were less likely to have had a single operative procedure (33% v 84%) and were associated with higher total costs whether mastectomy (US dollars 2775 v US dollars 1849) or lumpectomy (US dollars 2112 v US dollars 1365) was used. Sensitivity analysis showed core biopsy optimal in 95.4% of trials. Core biopsy was favored for low-suspicion lesions, calcifications, and masses, and overall for patients who underwent lumpectomy alone. CONCLUSION: Image-guided core biopsy can be cost-saving compared with surgical biopsy, particularly when the mammographic abnormality is classified as low suspicion or consists of calcifications or masses. Moving to a policy in which core biopsy is the preferred approach in these settings has the potential to result in significant cost savings.

Cost-effectiveness considerations in the treatment of essential thrombocythemia.

Factors that influence the choice of anagrelide, hydroxyurea, or interferon-alfa (IFN-alpha) for treatment of essential thrombocythemia include efficacy, toxicity, and cost. Anagrelide has the US Food and Drug Administration's approval to be used for treating patients with thrombocythemia secondary to chronic myeloproliferative disorders. In contrast, the use of IFN-alpha and hydroxyurea are considered "off-label." We performed an incremental cost-effectiveness analysis to compare anagrelide, hydroxyurea, and IFN-alpha for treating essential thrombocythemia, in terms of estimated impact on life expectancy. The case used for this analysis was of a 40-year-old man with essential thrombocythemia. Clinical assumptions were based on information obtained from nonrandomized clinical trials, and the economic assumptions were derived from information abstracted from observational studies. Lifelong treatment use of anagrelide versus hydroxyurea would cost approximately $72,000 per additional year of life gained, while the use of IFN-alpha was found to be both more costly and less effective than anagrelide. The results were very sensitive to the risk of leukemia caused by hydroxyurea, with an incremental cost-effectiveness of anagrelide compared with hydroxyurea of $156,969 per additional year of life gained if the lifetime leukemia risk drops from a baseline of .08 to.05. Given that many commonly used medical interventions cost in the range of $50,000 to $100,000 per year of life gained, and the generally poor outcome associated with treatment-related leukemia that can result from hydroxyurea, anagrelide could be considered a therapeutic alternative that is clinically effective at an acceptable cost.

Pilot study of a utilities-based treatment decision intervention for prostate cancer patients.

This pilot study evaluates a shared decision-making approach to individual decision making in localized prostate cancer care. The approach is based on a decision analytic model that incorporates patient utilities, ie, patient preferences among possible health states that might occur with prostate cancer treatments. Data on comorbidities, histologic grade of the biopsy, and age were obtained for 13 patients with newly diagnosed localized prostate cancer who received care in a Veterans Administration medical center. Using a standard gamble technique, interviewers obtained patient utilities for 5 distinct health states related to prostate cancer treatment. Utilities and patient clinical and pathologic characteristics were incorporated into the decision analytic model, and the derived quality-adjusted life expectancies were shared with the treating urologist before the first patient-physician discussion about treatment options. The results of the pilot study raised 2 major concerns. First, 4 patients had utility scores of 1.0 for all of the possible health states, and 7 patients had inconsistent utilities in which they rated both impotence and incontinence as a better health state than having just one of these problems. Second, the model recommended radiation therapy to individuals with a broad range of clinical characteristics, pathologic findings, and utility scores. Many of the patients who were recommended radiation therapy by the model received discordant recommendations from the treating urologist. Future refinements of both the utility assessment exercise and decision analytic model may be needed before the feasibility of the model in the clinical setting can be determined.