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Generating Evidence for Comparative Effectiveness Research Using More Pragmatic Randomized Controlled Trials

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Author Info

  • C. Daniel. Mullins

    (University of Maryland School of Pharmacy, Baltimore, Maryland, USA; Center for Medical Technology Policy, Baltimore, Maryland, USA)

  • Danielle Whicher

    (Center for Medical Technology Policy, Baltimore, Maryland, USA)

  • Emily S. Reese

    (University of Maryland School of Pharmacy, Baltimore, Maryland, USA)

  • Sean Tunis

    (Center for Medical Technology Policy, Baltimore, Maryland, USA)

Registered author(s):

    Abstract

    Comparative effectiveness research (CER), or research design to meet the needs of post-regulatory decision makers, has been brought into the spotlight with the introduction of the American Recovery and Reinvestment Act, which provided $US1.1 billion over 2 years to support CER. In the short run, the majority of this money will be invested in observational studies and building of infrastructure; however, in the long run, we will likely see an increase in the number of randomized controlled trials (RCTs), as this method is arguably the most unbiased approach for establishing causal effect between treatments and health outcomes. RCTs are an integral component of CER for generating credible evidence on the relative value of alternative interventions in order to meet the needs of post-regulatory decision makers (patients, physicians, payers and policy makers). Explanatory phase III RCTs are fit for purpose; researchers make use of guidance documents produced by the US FDA to inform the design of these clinical trials. Historically, without explicit FDA guidance, broad patient populations, including women and minorities, often were not considered in trial design. In addition, attempts to minimize cost and maximize efficiency have led to smaller sample sizes, as is clear from the increase in 'creeping phase II-ism'. To demonstrate effectiveness, RCTs must be reflective of how an intervention will be used in the healthcare market. The concept of pragmatic clinical trials has emerged to describe those trials that are designed explicitly with this need in mind. Use of pragmatic trials will be most impactful if post-regulatory decision makers are engaged in the development of recommendations for trial design features, such as indicating outcomes measures and articulating patient populations of interest, which clearly express their evidence needs.

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    Bibliographic Info

    Article provided by Springer Healthcare | Adis in its journal PharmacoEconomics.

    Volume (Year): 28 (2010)
    Issue (Month): 10 ()
    Pages: 969-976
    Download reference. The following formats are available: HTML (with abstract), plain text (with abstract), BibTeX, RIS (EndNote, RefMan, ProCite), ReDIF
    Handle: RePEc:wkh:phecon:v:28:y:2010:i:10:p:969-976

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    Web page: http://pharmacoeconomics.adisonline.com/

    For corrections or technical questions regarding this item, or to correct its listing, contact: (Dave Dustin).

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    Keywords: clinical-trial-design; randomised-controlled-trials; treatment-outcome;

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