Literature DB >> 20018711

Bandit solutions provide unified ethical models for randomized clinical trials and comparative effectiveness research.

William H Press1.   

Abstract

As electronic medical records enable increasingly ambitious studies of treatment outcomes, ethical issues previously important only to limited clinical trials become relevant to unlimited whole populations. For randomized clinical trials, adaptive assignment strategies are known to expose substantially fewer patients to avoidable treatment failures than strategies with fixed assignments (e.g., equal sample sizes). An idealized adaptive case--the two-armed Bernoulli bandit problem--can be exactly optimized for a variety of ethically motivated cost functions that embody principles of duty-to-patient, but the solutions have been thought computationally infeasible when the numbers of patients in the study (the "horizon") is large. We report numerical experiments that yield a heuristic approximation that applies even to very large horizons, and we propose a near-optimal strategy that remains valid even when the horizon is unknown or unbounded, thus applicable to comparative effectiveness studies on large populations or to standard-of-care recommendations. For the case in which the economic cost of treatment is a parameter, we give a heuristic, near-optimal strategy for determining the superior treatment (whether more or less costly) while minimizing resources wasted on any inferior, more expensive, treatment. Key features of our heuristics can be generalized to more complicated protocols.

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Year:  2009        PMID: 20018711      PMCID: PMC2793317          DOI: 10.1073/pnas.0912378106

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN: 0027-8424            Impact factor:   11.205


  6 in total

1.  Ethics and statistics in randomized clinical trials.

Authors:  Richard M Royall; Robert H Bartlett; Richard G Cornell; David P Byar; William D Dupont; Robert J Levine; Foster Lindley; R John Simes; M Zelen
Journal:  Stat Sci       Date:  1991       Impact factor: 2.901

2.  Stopping clinical trials early.

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Journal:  BMJ       Date:  2004-09-04

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Authors:  D A Berry; S G Eick
Journal:  Stat Med       Date:  1995-02-15       Impact factor: 2.373

Review 4.  The early termination of clinical trials: causes, consequences, and control. With special reference to trials in the field of arrhythmias and sudden death. Task Force of the Working Group on Arrhythmias of the European Society of Cardiology.

Authors: 
Journal:  Circulation       Date:  1994-06       Impact factor: 29.690

Review 5.  Review: use of electronic medical records for health outcomes research: a literature review.

Authors:  Bonnie B Dean; Jessica Lam; Jaime L Natoli; Qiana Butler; Daniel Aguilar; Robert J Nordyke
Journal:  Med Care Res Rev       Date:  2009-03-11       Impact factor: 3.929

Review 6.  Bayesian clinical trials.

Authors:  Donald A Berry
Journal:  Nat Rev Drug Discov       Date:  2006-01       Impact factor: 84.694

  6 in total
  6 in total

Review 1.  Statistics and ethics: models for strengthening protection of human subjects in clinical research.

Authors:  Christine K Cassel
Journal:  Proc Natl Acad Sci U S A       Date:  2009-12-22       Impact factor: 11.205

Review 2.  The immune system's role in sepsis progression, resolution, and long-term outcome.

Authors:  Matthew J Delano; Peter A Ward
Journal:  Immunol Rev       Date:  2016-11       Impact factor: 12.988

3.  Adaptive treatment allocation and selection in multi-arm clinical trials: a Bayesian perspective.

Authors:  Elja Arjas; Dario Gasbarra
Journal:  BMC Med Res Methodol       Date:  2022-02-20       Impact factor: 4.615

4.  Some performance considerations when using multi-armed bandit algorithms in the presence of missing data.

Authors:  Xijin Chen; Kim May Lee; Sofia S Villar; David S Robertson
Journal:  PLoS One       Date:  2022-09-12       Impact factor: 3.752

5.  Response-adaptive randomization for multi-arm clinical trials using the forward looking Gittins index rule.

Authors:  Sofía S Villar; James Wason; Jack Bowden
Journal:  Biometrics       Date:  2015-06-22       Impact factor: 2.571

6.  In silico study of medical decision-making for rare diseases: heterogeneity of decision-makers in a population improves overall benefit.

Authors:  Juan Wang; Ryo Yamada
Journal:  PeerJ       Date:  2018-09-25       Impact factor: 2.984

  6 in total

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