PharmacoEconomics
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In the era of the Information Age and personalized medicine, healthcare delivery systems need to be efficient and patient-centred. The health system must be responsive to individual patient choices and preferences about their care, while considering the system consequences. While dynamic simulation modelling (DSM) and big data share characteristics, they present distinct and complementary value in healthcare. ⋯ Big data can inform patient-centred care with its high velocity, volume, and variety (the three Vs) over traditional data analytics; however, big data are not sufficient to extract meaningful insights to inform approaches to improve healthcare delivery. DSM can serve as a natural bridge between the wealth of evidence offered by big data and informed decision making as a means of faster, deeper, more consistent learning from that evidence. We discuss the synergies between big data and DSM, practical considerations and challenges, and how integrating big data and DSM can be useful to decision makers to address complex, systemic health economics and outcomes questions and to transform healthcare delivery.
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Review
Ipilimumab for Previously Untreated Unresectable Malignant Melanoma: A Critique of the Evidence.
The National Institute for Health and Care Excellence (NICE) invited the manufacturer of ipilimumab (Bristol-Myers Squibb Pharmaceuticals Limited) to submit clinical and cost-effectiveness evidence for previously untreated advanced (unresectable or metastatic) melanoma as part of the Institute's Single Technology Appraisal process. The Centre for Reviews and Dissemination and Centre for Health Economics at the University of York were commissioned to act as the independent Evidence Review Group (ERG). This article presents a summary of the manufacturer's submission of ipilimumab, the ERG review and the resulting NICE guidance TA319, issued in July 2014. ⋯ The manufacturer's response was based on concerns relating to uncertainty surrounding the relative efficacy of ipilimumab 3 mg/kg monotherapy compared with DTIC and vemurafenib, comparability of the patient populations at first and second line, and the effects of concomitant DTIC. These additional analyses indicated that the incremental cost-effectiveness ratio (ICER) was £ 47,900 per quality-adjusted life-year (QALY) gained for ipilimumab compared with DTIC and £ 28,600 per QALY gained for ipilimumab compared with vemurafenib. Following consideration of the additional evidence and the responses from a large number of consultees and commentators, the committee recommended ipilimumab as an option for adults with previously untreated advanced melanoma.
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The EQ-5D is one of the most frequently used, generic, preference-based instruments for measuring the health utilities of patients in economic evaluations. It is recommended for health technology assessment by the National Institute for Health and Clinical Excellence. Because the EQ-5D plays such an important role in economic evaluations, useful information on its responsiveness to detect meaningful change in health status is required. ⋯ The EQ-5D is an appropriate measure for economic evaluation and health technology assessment in conditions where it has demonstrated evidence of responsiveness. In conditions with mixed evidence of responsiveness, researchers should consider using the EQ-5D with other condition-specific measures to ensure appropriate estimates of effectiveness. These conditions should be a main focus for future research using the new EQ-5D version with five response levels.
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Adjuvant chemotherapy is a key component of advanced ovarian cancer treatment, when surgery alone is not sufficient. Recurrence is common in ovarian cancer patients and most women require prolonged second-line and higher-line chemotherapy. With newer targeted therapies, modest improvements in survival and quality of life may be attained at substantial cost, but the relative economic efficiency of these newer agents remains unknown. ⋯ Despite varying methodological approaches and multiple sources for cost and effectiveness inputs, this systematic review demonstrated that standard platinum-taxane combination chemotherapy for first-line treatment was most cost-effective. There was unanimous agreement that bevacizumab was not a cost-effective front-line therapy compared with platinum-taxane combination for the overall ovarian cancer population, though its use in the high-use population may yield better value. For second-line treatment, platinum-based chemotherapy remained cost-effective among patients with recurrent platinum-sensitive disease, while there was limited evidence to conclude the most valuable treatment alternative among patients with recurrent platinum-resistant disease. Future research incorporating real-world data is essential to corroborate findings from trial-based economic evaluations. In addition, for improving consistency in reporting and quality of studies, incorporating QALYs in this population is important, especially since chemotherapy is administered for lengthy periods of time.
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The challenges of comparative effectiveness to support health technology assessment (HTA) agencies are important considerations in the choices of antipsychotic medications for the treatment of schizophrenia. ⋯ This review of comparative effectiveness studies of second-generation antipsychotic agents for schizophrenic patients revealed a wide range of study types, study methodologies, and outcomes. For traditional efficacy outcomes and select safety outcomes, there is strong evidence from many well-conducted studies; however, there are fewer studies of types preferred by HTA with limited head-to-head comparisons and a higher risk of bias in the execution of these studies.