Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
-
Comparative Study
The Long-Term Effectiveness and Cost Effectiveness of Organized versus Opportunistic Screening for Breast Cancer in Austria.
In 2014, Austrian health authorities implemented an organized breast cancer screening program. Until then, there has been a long-standing tradition of opportunistic screening. ⋯ The decision to adopt organized screening is likely an efficient use of limited health care resources in Austria.
-
To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR. ⋯ HB is associated with high economic burden, primarily because of clotting factor costs. Nevertheless, prophylaxis treatment leads to clinical benefits and may reduce other nonfactor costs.
-
Randomized Controlled Trial
Less Is More: Cross-Validation Testing of Simplified Nonlinear Regression Model Specifications for EQ-5D-5L Health State Values.
The conventional method for modeling of the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) health state values in national valuation studies is an additive 20-parameter main-effects regression model. Statistical models with many parameters are at increased risk of overfitting-fitting to noise and measurement error, rather than the underlying relationship. ⋯ Simplified nonlinear regression models look promising and should be investigated further using other EQ-5D-5L data sets. To reduce the risk of overfitting, cross-validation is recommended to inform model selection in future EQ-5D valuation studies.
-
Pragmatic trials evaluate the comparative benefits, risks, and burdens of health care interventions in real-world conditions. Such studies are now recognized as valuable to the perimarketing stage of drug development and evaluation, with early pragmatic trials (EPTs) being explored as a means to generate real-world evidence at the time of regulatory market approval. In this article, we present an analysis of the ethical issues involved in informed consent for EPTs, in light of the generally recognized concern that traditional ethical rules governing randomized clinical trials, such as lengthy informed consent procedures, could threaten the "real world" nature of such trials. Specifically, we examine to what extent modifications (waivers or alterations) to regulatory consent for EPTs would be ethical. ⋯ The analysis shows that neither waivers nor alterations of regulatory consent for premarket EPTs will be ethically permissible. For postmarket EPTs with newly approved interventions, waivers of consent will be ethically problematic, but some studies might be conducted in an ethical manner with alterations to regulatory consent.
-
To analyze how value is determined within the scope of the German Pharmaceutical Restructuring Act, which came into effect in 2011. ⋯ Price premiums, or "value," are driven by health gain, the share of patients benefiting from a pharmaceutical, European price levels, and whether comparators are generic. No statement can be made, however, about the appropriateness of the level of price premiums.