Biomarker-guided personalized therapies offer great promise to improve drug development and improve patient care, but also pose difficult challenges in designing clinical trials for the development and validation of these therapies. We first give a review of the existing approaches, briefly for clinical trials in new drug development and in more detail for comparative effectiveness trials invol...

Comparative effectiveness research (CER) has assumed an increasing role in drug coverage and, in some cases, pricing decisions in Europe, as decision-makers seek to obtain better value for money. This issue brief comparatively examines the use of CER across six countries--Denmark, England, France, Germany, the Netherlands, and Sweden. With CER gaining traction in the United States, these intern...

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Comparative effectiveness research (CER) can provide valuable information for patients, providers and payers. These stakeholders differ in their incentives to invest in CER. To maximize benefits from public investments in CER, it is important to understand the value of CER from the perspectives of these stakeholders and how that affects their incentives to invest in CER. This article provides a...

The ultimate goal of comparative effectiveness research (CER) is to develop and disseminate evidence-based information about which interventions are most effective for which patients under what circumstances. To achieve this goal it is crucial that researchers in methodology development find appropriate methods for detecting the presence and sources of heterogeneity in treatment effect (HTE). C...

Not all patient-reported outcomes (PROs) are patient centered, and not all patient-centered outcomes are patient reported. The essential characteristic of a patient-centered approach to outcome measurement is that it assesses concepts (i.e., healthrelated phenomena) that are considered most important by members of a given target population, based on direct input from representatives of that pop...

CONTEXT Comparative effectiveness research is increasingly being recognized as a method to link research with the information needs of decision makers. As the United States begins to invest in comparative effectiveness, it would be wise to look at other functioning research networks to understand the infrastructure and funding required to support them. METHODS This case study looks at the com...

Currently, there is wide-spread recognition that much of health care is insufficiently grounded in evidence as to what works for whom under what circumstances. An important reason for this is that research efforts often do not compare alternative options of health care nor are they sufficiently focused on outcomes that matter to patients. In addition, the methods to conduct research designed to...

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In comparative effectiveness research, we are often interested in the estimation of an average causal effect from large observational data (the main study). Often this data does not measure all the necessary confounders. In many occasions, an extensive set of additional covariates is measured for a smaller and non-representative population (the validation study). In this setting, standard appro...