In an ideal world, appropriate pharmacoeconomic evidence would be collected to meet the needs of the local reimbursement environment. Unfortunately, due to clinical research limitations and time constraints, we are often faced with a lack of the necessary data. OBJECTIVE: Using an anti‐infective as our case study, we present an approach that we developed to overcome the challenge of creating an economic argument for Canadian hospital setting in the absence of country‐specific data. METHODS: Our methodology involved a multi‐step approach: (i) hospital formulary committee members were consulted to identify the submission requirements; (ii) a panel of experts were surveyed to understand local treatment patterns; (iii) the survey results were used in a case modeling exercise to determine the applicability of the randomized clinical trial (RCT) protocol to the Canadian environment; and (iv) a chart review was undertaken to validate results of the case modeling exercise. RESULTS: Hospital formulary committees preferred Canadian‐specific evidence of a new product's economic value. Committee members were generally unfamiliar with pharmacoeconomic concepts and preferred that a complicated economic model not be used. Results of the expert survey indicated that there are important differences between the RCT protocol and Canadian treatment patterns. These differences include restrictive inclusion/exclusion criteria, an excess of protocol‐driven tests and procedures, and treatment strategies that are not necessarily reflective of real‐life. The case modeling exercise allowed for a structured method to hypothesize on the real‐world impact of the product. An ongoing chart review will be used to confirm these hypotheses. CONCLUSIONS: It is anticipated that issues surrounding the absence of country‐specific data will continue to plague researchers for the foreseeable future. Use of this multi‐step approach provides a rigorous method for making conclusions in the absence of locally acquired evidence.
The Health Utilities Index is designed to describe and to quantify the health status and the health‐related quality‐of‐life (HRQOL) of subjects. This validated and reliable instrument has been employed by over 100 research groups worldwide and exists in three formats (face‐to‐face interview, telephone interview and self‐administration). Translations into several languages are also available. As a generic preference‐weighted system, the Health Utilities Index not only measures and describes health status, but also provides a quantitative measure of HRQOL as a single summary score based on community preferences for health status. The score, founded on utility theory and anchored to the conventional 0–1 (dead–healthy) scale, is appropriate as a quality‐weight in the calculation of quality‐adjusted life‐years (QALY's). Given that QALYs have been recognized as a universal measure for use in cost‐effectiveness/cost‐utility analyses and are recommended in the reporting and monitoring of population health, this instrument has wide ranging applications in outcomes and health economic research. This workshop will provide an introduction to the Health Utilities Index and focus on its practical application. We will describe our experiences as they relate to issues such as frequency and mode of administration, patient recall, compliance, sample size considerations, data analysis and interpretation. Citing previous studies, this workshop will demonstrate how the Health Utilities Index can be utilized in prospective clinical research, patient surveys and as a means for developing utility estimates in retrospective health economic models. Researchers and analysts involved in quality‐of‐life and health economic evaluations will gain a working knowledge of the Health Utilities Index and an appreciation of its diversified use as a generic HRQOL instruments.
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