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There has been a recent surge of eHealth programs in cancer and other content areas, but few reviews have focused on the methodologies and designs employed in these studies. We conducted a systematic review of studies on eHealth interventions on cancer prevention and control published between 2001 and 2010 applying the Pragmatic Explanatory Continuum Indicator Summary (PRECIS) criteria and external validity components from the Reach Effectiveness Adoption Implementation Maintenance (RE-AIM) framework. We identified 113 studies that focused on cancer prevention and control of eHealth interventions. Most studies fell midway along the explanatory/pragmatic trial continuum, but few reported on various practical feasibility criteria for translation. Despite vast interest in cancer eHealth and the applied nature of this field, few studies considered key external validity issues. There is a need for use of alternative pragmatic study designs and transparent reporting of external validity components to produce more rapid and generalizable results.

Background: Pharmaceutical pragmatic clinical trials (PCTs) are designed to provide the type of evidence that is desired by patients, clinicians and payers but too often missing from traditional regulatory trials., Purpose: This paper presents framework for designing pragmatic trials incorporating evidence desired by post-regulatory decision makers while remaining within acceptable standards for regulatory approval., Methods: Following a stakeholder meeting convened in May of 2009 to identify gaps in information collected in Phase 3 trials, CMTP staff and the authors drafted recommendations for Pragmatic Phase 3 Pharmaceutical Trials. This draft was circulated first to technical working group members for their comments. After revising the document based on these comments, it was distributed electronically to other select experts and then made available for public comment. The final version of the EGD appears on the CMTP website., Results: The process resulted in a set of 10 recommendations for conducting Phase 3 trials that met regulatory needs while addressing information important to physicians, patients, payers, and policy-makers. These recommendations encompassed three primary areas: generalizability from the trial participants to the clinical population of interest; effectiveness relative to active comparators; and consistently measured relevant outcomes for coverage and treatment decisions., Limitations: While stakeholders were involved throughout the process, not all recommendations will meet the needs of all stakeholders., Conclusions: Pragmatic trial design need not be deferred until a product is in widespread use. Incremental movement toward the more pragmatic design of Phase 3 trials is desirable., (© 2013.)

We describe a conceptual analytic framework for aligning observational and randomized controlled trial (RCT) data. The framework allows one to 1) use observational data to estimate treatment effects comparable to their RCT counterparts, 2) properly include early events that occur soon after treatment initiation in the analysis of observational data, 3) estimate various treatment effects that are of clinical and scientific relevance while appropriately adjusting for time-varying confounders in both the RCT and observational analyses, 4) assess the generalizability of RCT findings in the more diverse populations generally found in the observational data, and 5) combine both types of data to study associations that cannot be addressed by one study or a single dataset. We describe the theoretical application of this framework to the Women's Health Initiative data to examine the relation between postmenopausal hormone therapy and coronary heart disease. The analytic framework can be tailored to specific exposure-outcome associations and data sources, and may be refined as more is learned about its strengths and limitations.

With increased attention to the importance of translating research to clinical practice and policy, recent years have seen a proliferation of particular types of research, including pragmatic trials and dissemination and implementation research. Such research seeks to understand how and why interventions function in real-world settings, as opposed to highly controlled settings involving conditions not likely to be repeated outside the research study. Because understanding the context in which interventions are implemented is imperative for effective pragmatic trials and dissemination and implementation research, the use of mixed methods is critical to understanding trial results and the success or failure of implementation efforts. This article discusses a number of dimensions of mixed methods research, utilizing at least one qualitative method and at least one quantitative method, that may be helpful when designing projects or preparing grant proposals. Although the strengths and emphases of qualitative and quantitative approaches differ substantially, methods may be combined in a variety of ways to achieve a deeper level of understanding than can be achieved by one method alone. However, researchers must understand when and how to integrate the data as well as the appropriate order, priority, and purpose of each method. The ability to demonstrate an understanding of the rationale for and benefits of mixed methods research is increasingly important in today's competitive funding environment, and many funding agencies now expect applicants to include mixed methods in proposals. The increasing demand for mixed methods research necessitates broader methodological training and deepened collaboration between medical, clinical, and social scientists. Although a number of challenges to conducting and disseminating mixed methods research remain, the potential for insight generated by such work is substantial., (Copyright © 2013 Academic Pediatric Association. Published by Elsevier Inc. All rights reserved.)

There is at present some confusion about the relative value of clinical trials performed to investigate efficacy vs. those designed to investigate effectiveness. This is particularly challenging when studies performed as experiments for regulators by companies are used to shape and inform clinical practice, especially if studies conducted under more real life conditions fail to support predicted benefits. We review the field in relation to the new antipsychotics, in particular. Other indications, including mood disorders, which are also briefly touched upon, have so far received less definitive attention, but are likely to encounter the same difficulties. We conclude that, where the results of efficacy trials are positive and an effectiveness trial is negative, one should not necessarily prefer the effectiveness trial - it may simply have failed. Where efficacy trials and effectiveness trials point to similar conclusions, then the findings are mutually supportive.