Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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To compare US Food and Drug Administration (FDA) and European Medicines Agency (EMA) labeling for evidence based on patient-reported outcomes (PROs) of new oncology treatments approved by both agencies. ⋯ During this time period, the FDA and the EMA used different evidentiary standards to assess PRO data from oncology studies, with the EMA more likely to accept data from open-label studies and broad concepts such as health-related quality of life. An understanding of the key differences between the agencies may guide sponsor PRO strategy when pursuing labeling. Patient-focused proximal concepts are more likely than distal concepts to receive positive reviews.
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The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is the preferred measure of health outcome in clinical trials in motor neuron disease (MND). It, however, does not provide a preference-based health utility score required for estimating quality-adjusted life-years in economic evaluations for health technology assessments. ⋯ This is the first study to present mapping algorithms to crosswalk between ALSFRS-R and EQ-5D-5L. This analysis demonstrates that the ALSFRS-R can be used to estimate EQ-5D-5L utilities when they have not been collected directly within a trial.
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Predictors of Drug Shortages and Association with Generic Drug Prices: A Retrospective Cohort Study.
Prescription drug shortages can disrupt essential patient care and drive up drug prices. ⋯ Study findings may not be generalizable to drugs that became generic after 2008 or those commonly used in an inpatient setting. The lowest priced drugs are at a substantially elevated risk of experiencing a drug shortage. Periods of drug shortages were associated with modest increases in drug prices.
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Valuations of health states were affected by the wording of the two instruments (EQ-5D-3L and EQ-5D-Y) and by the perspective taken (child or adult). ⋯ The results identified an interaction effect between wording of the instrument and perspective on elicited values, suggesting that current EQ-5D-3L value sets should not be employed to assign values to EQ-5D-Y health states.
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Studies have shown that methods based on mixture models work well when mapping clinical to preference-based methods. ⋯ Beta-based mixture models marginally outperformed adjusted limited dependent variable mixture models with the same number of components in this data set. Nevertheless, they require a larger number of parameters and longer estimation time. Both mixture model types closely fit both EQ-5D-5L and HUI data. Standard mapping approaches typically lead to biased estimates of health gain. The mixture model approaches exhibit no such bias. Both can be used with confidence in applied cost-effectiveness studies. Future mapping studies in other disease areas should consider similar methods.