Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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Develop and validate a health-related quality-of-life (measure for patients with acute and chronic leukemia. ⋯ The FACT-Leu scale is a valid, reliable, and efficient measure of leukemia-specific health-related quality of life for acute and chronic disease.
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1) To compare the Food and Drug Administration's (FDA's) Risk Evaluation and Mitigation Strategies (REMS) and European Medicines Agency's (EMA's) Risk Management Plan (RMP) guidances and 2) to compare REMS and RMPs for specific chemical entities and biological products. ⋯ Both REMS and RMPs provide positive guidance for identification, monitoring, and minimization of risk to patient safety. Currently, neither agency provides specific guidance on how risk should be related to benefit either qualitatively or quantitatively.
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Comparative Study
Comparison of generic, condition-specific, and mapped health state utility values for multiple myeloma cancer.
Resource allocation informed by cost-utility analysis requires that the benefits be comparable across patient groups and interventions. One option is to recommend the use of one generic utility measure, but this raises the issue of comparability when the preferred measure is inappropriate or unavailable. Many cancer trials do not include generic measures such as the EuroQol five-dimensional (EQ-5D) questionnaire and instead include condition-specific measures and use these to generate utility estimates. We analyze the comparability of generic, condition-specific, and mapped utility values for a multiple myeloma cancer patient data set. ⋯ Our findings suggest that condition-specific EORTC-8D or mapped EQ-5D utility estimates are broadly comparable to directly obtained EQ-5D utilities for a multiple myeloma patient data set. However, EORTC-8D estimates captured changes in quality of life for patients in mild health states that were not captured by the EQ-5D, but estimated lower utility gains than did the use of the EQ-5D directly.
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Personalized medicine technologies can improve individual health by delivering the right dose of the right drug to the right patient at the right time but create challenges in deciding which technologies offer sufficient value to justify widespread diffusion. Personalized medicine technologies, however, do not neatly fit into existing health technology assessment and reimbursement processes. ⋯ Key gaps in health economics and outcomes research best practices, decision standards, and value assessment processes are also discussed, along with next steps for evolving health economics and outcomes research practices in personalized medicine.
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To investigate Food and Drug Administration (FDA) regulatory actions against drug companies' health economic promotions from 2002 through 2011 to understand how frequently and in what circumstances the agency has considered such promotions false or misleading. ⋯ The FDA has cited inappropriate health economic promotions in roughly 12% of the letters issued by the DDMAC. The letters highlight drug companies' interest in promoting the value of their products and the FDA's concerns in certain cases about the lack of supporting evidence.