Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Health Science Policy Council recommended and the ISPOR Board of Directors approved the formation of a Task Force to critically examine the major issues related to Quality Improvement in Cost-effectiveness Research (QICER). The Council's primary recommendation for this Task Force was that it should report on the quality of cost-effectiveness research and make recommendations to facilitate the improvement of pharmacoeconomics and health outcomes research and its use in stimulating better health care and policy. Task force members were knowledgeable and experienced in medicine, pharmacy, biostatistics, health policy and health-care decision-making, biomedical knowledge transfer, health economics, and pharmacoeconomics. They were drawn from industry, academia, consulting organizations, and advisors to governments and came from Japan, the Netherlands, Canada and the United States. ⋯ The QICER Task Force recommends that ISPOR implement the following: * With respect to CER guidelines, that ISPOR promote harmonization of guidelines, allowing for differences in application, regional needs and politics; evaluate available instruments or promote development of a new one that will allow standardized quantification of the impact of CER guidelines on the quality of CER studies; report periodically on those countries or regions that have developed guidelines; periodically evaluate the quality of published studies (those journals with CER guidances) or those submitted to decision-making bodies (as public transparency increases). * With respect to methodologies, that ISPOR promote publication of methodological guidelines in more applied journals in more easily understandable format to transfer knowledge to researchers who need to apply more rigorous methods; promote full availability of models in electronic format to combat space restrictions in hardcopy publications; promote consistency of methodological review for all CER studies; promote adoption of explicit best practices guidelines among regulatory and reimbursement authorities; periodically update all ISPOR Task Force reports; periodically review use of ISPOR Task Force guidelines; periodically report on statistical and methodological challenges in HE; evaluate periodically whether ISPOR's methodological guidelines lead to improved quality; and support training and knowledge transfer of rigorous CER methodologies to researchers and health care decision-makers. * With respect to publications, that ISPOR develop standard CER guidances to which journals will be able to refer their authors and their reviewers; lobby to establish these guidances within the International Committee for Medical Journal Editors (ICMJE) Requirements to which most journals refer in their Author Instructions; provide support in terms of additional reviewer expertise to those journals lacking appropriate reviewers; periodically report on journals publishing CER research; periodically report on the quality of CER publications; and support training and knowledge transfer of the use of these guidelines to researchers and reviewers. * With respect to evidence-based health-care decision-making, that ISPOR recognize at its annual meetings those countries/agencies/private companies/researchers using CER well, and those practitioners and researchers supporting good patient use of CER in decision-making; and promote public presentation of case studies of applied use of CER concepts or guidelines.
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The goal of comparative effectiveness analysis is to examine the relationship between two variables, treatment, or exposure and effectiveness or outcome. Unlike data obtained through randomized controlled trials, researchers face greater challenges with causal inference with observational studies. Recognizing these challenges, a task force was formed to develop a guidance document on methodological approaches to addresses these biases. ⋯ Design of nonrandomized studies of comparative effectiveness face several daunting issues, including measurement of exposure and outcome challenged by misclassification and confounding. Use of causal diagrams and restriction are two techniques that can improve the theoretical basis for analyzing treatment effects in study populations of more homogeneity, with reduced loss of generalizability.
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One-year adjuvant trastuzumab therapy increases disease-free and overall survival in the adjuvant treatment of early HER2-positive breast cancer. This study aims to assess the long-term cost-effectiveness of adjuvant trastuzumab treatment in Beijing, Shanghai, and Guangzhou. ⋯ The results suggest that the 1-year adjuvant trastuzumab treatment is cost-effective. Both clinical and economic benefits were superior for the 1-year adjuvant trastuzumab treatment group compared with the standard adjuvant chemotherapy group.
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Pharmacoeconomic analyses typically project the expected cost-effectiveness of a new product for a specific indication. This analysis develops a dynamic life-cycle model to conduct a multi-indication evaluation using the case of trastuzumab licensed in the United States for both early-stage and metastatic (or late-stage) human epidermal growth factor receptor 2 (HER2)-positive breast cancer therapy (early breast cancer [EBC]; metastatic breast cancer [MBC]), approved in 2006 and 1998, respectively. ⋯ Average ICERs for multi-indication compounds can increase or decrease over the product life cycle. In this example, the projected overall life-cycle ICER for trastuzumab was less than one half of that in the initial indication. This dynamic perspective-versus the usual static one-highlights the interdependence of drug development decisions and investment incentives, raising important reimbursement policy issues.
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Comparative Study
How do type 2 diabetes mellitus-related chronic complications impact direct medical cost in four major cities of urban China?
The purpose of this study was to evaluate the direct medical costs of type 2 diabetes mellitus with or without complications, and to determine the economic impact of complications on type 2 diabetic patients. ⋯ The high economic burden raised by diabetes and its complications challenges the Chinese health-care system. It implicates an urgent need of intervention to prevent the development of long-term complications among the diabetic population, especially on the development of complications in high-cost body system.