Use of Bayesian techniques in randomized clinical trials a CMS case study

The use of Bayesian statistical approaches has gained broader acceptance within the clinical trial community. The impact of these methods on CMS decisional contexts and policy-level decisionmaking however was uncertain. Our analyses explore the main proclaimed advantages of Bayesian statistics (name...

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Bibliographic Details
Main Authors: Sanders, Gillian D., Inoue, Lurdes Y. T. (Author), Samsa, Gregory Paul (Author), Kulasingam, Shalini L. (Author)
Corporate Authors: Duke University Evidence-based Practice Center, Technology Assessment Program (Agency for Healthcare Research and Quality)
Format: eBook
Language:English
Published: Rockville, Maryland AHRQ, Technology Assessment Program September 18, 2009, 2009
Series:Technology assessment report
Subjects:
Online Access:
Collection: National Center for Biotechnology Information - Collection details see MPG.ReNa
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100 1 |a Sanders, Gillian D. 
245 0 0 |a Use of Bayesian techniques in randomized clinical trials  |h Elektronische Ressource  |b a CMS case study  |c Duke Evidence-based Practice Center; Gillian D. Sanders, Lurdes Inoue, Gregory Samsa, Shalini Kulasingam, David Matchar ; prepared for Agency for Healthcare Research and Quality 
260 |a Rockville, Maryland  |b AHRQ, Technology Assessment Program  |c September 18, 2009, 2009 
300 |a 1 PDF file (184 pages)  |b illustrations 
505 0 |a Includes bibliographical references 
653 |a Death, Sudden, Cardiac / prevention & control 
653 |a Defibrillators, Implantable 
653 |a Randomized Controlled Trials as Topic 
653 |a Bayes Theorem 
653 |a United States Government Agencies 
700 1 |a Inoue, Lurdes Y. T.  |e [author] 
700 1 |a Samsa, Gregory Paul  |e [author] 
700 1 |a Kulasingam, Shalini L.  |e [author] 
710 2 |a Duke University Evidence-based Practice Center 
710 2 |a Technology Assessment Program (Agency for Healthcare Research and Quality) 
041 0 7 |a eng  |2 ISO 639-2 
989 |b NCBI  |a National Center for Biotechnology Information 
490 0 |a Technology assessment report 
500 |a Title from PDF title page. - "Project ID: STAB0508." 
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082 0 |a 580 
520 |a The use of Bayesian statistical approaches has gained broader acceptance within the clinical trial community. The impact of these methods on CMS decisional contexts and policy-level decisionmaking however was uncertain. Our analyses explore the main proclaimed advantages of Bayesian statistics (namely, the use of prior information, sample size determination, borrowing strength from different trials, and sequential monitoring of trials) and provide examples of decisionmaking situations where the findings reached using these approaches both agree with and differ from findings reached using frequentist statistical techniques. Our findings confirm that, like classical techniques, Bayesian approaches are affected by the problems of model specification (i.e., the relationship between various factors - patient, provider, intervention, and other contextual features - and the outcome of interest). In addition, Bayesian approaches can be substantially affected by the "Bayesian prior" - the representation of beliefs about the quantity of interest (e.g., relative risk of events when a new device is used vs. a conventional device). Thus, when considering using or interpreting Bayesian analyses, the focus of attention and thoughtful ex ante agreement are the specification of the model and specification of the Bayesian prior. The case study of the use of ICD therapy in the prevention of sudden cardiac death demonstrates the application of these techniques and highlights some of the practical challenges. The use of Bayesian statistical approaches, and incorporation of our findings concerning their strengths and limitations into the CMS decisionmaking process will enable policymakers to harness the power of the available sources of clinical evidence, explore subgroup effects within a trial and across trials in a methodologically rigorous manner, assess the uncertainty in clinical trial findings, and - ideally - improve health outcomes for Medicare beneficiaries