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Statistical Decision Properties of Imprecise Trials Assessing COVID-19 Drugs

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  • Charles F. Manski
  • Aleksey Tetenov

Abstract

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are imprecise. Seeing imprecision, clinicians reading research articles may find it difficult to decide when to treat patients with experimental drugs. Whatever decision criterion one uses, there is always some probability that random variation in trial outcomes will lead to prescribing sub-optimal treatments. A conventional practice when comparing standard care and an innovation is to choose the innovation only if the estimated treatment effect is positive and statistically significant. This practice defers to standard care as the status quo. To evaluate decision criteria, we use the concept of near optimality, which jointly considers the probability and magnitude of decision errors. An appealing decision criterion from this perspective is the empirical success rule, which chooses the treatment with the highest observed average patient outcome in the trial. Considering the design of recent and ongoing COVID-19 trials, we show that the empirical success rule yields treatment results that are much closer to optimal than those generated by prevailing decision criteria based on hypothesis tests.

Suggested Citation

  • Charles F. Manski & Aleksey Tetenov, 2020. "Statistical Decision Properties of Imprecise Trials Assessing COVID-19 Drugs," NBER Working Papers 27293, National Bureau of Economic Research, Inc.
    • Handle: RePEc:nbr:nberwo:27293
    Note: EH TWP
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    References listed on IDEAS

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    1. Stoye, Jörg, 2009. "Minimax regret treatment choice with finite samples," Journal of Econometrics, Elsevier, vol. 151(1), pages 70-81, July.
    2. Charles F. Manski, 2004. "Statistical Treatment Rules for Heterogeneous Populations," Econometrica, Econometric Society, vol. 72(4), pages 1221-1246, July.
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    Cited by:

    1. John Mullahy, 2020. "Discovering Treatment Effectiveness via Median Treatment Effects—Applications to COVID-19 Clinical Trials," NBER Working Papers 27895, National Bureau of Economic Research, Inc.
    2. Domenico Depalo, 2021. "True COVID-19 mortality rates from administrative data," Journal of Population Economics, Springer;European Society for Population Economics, vol. 34(1), pages 253-274, January.
    3. John Mullahy, 2021. "Discovering treatment effectiveness via median treatment effects—Applications to COVID‐19 clinical trials," Health Economics, John Wiley & Sons, Ltd., vol. 30(5), pages 1050-1069, May.

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    More about this item

    JEL classification:

    • C44 - Mathematical and Quantitative Methods - - Econometric and Statistical Methods: Special Topics - - - Operations Research; Statistical Decision Theory
    • I10 - Health, Education, and Welfare - - Health - - - General

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