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Methodologies for pragmatic and efficient assessment of benefits and harms: Application to the SOCRATES trial.

Scott R EvansMikael KnutssonPierre AmarencoGregory W AlbersPhilip M BathHans DenisonPer LadenvallJenny JonassonJ Donald EastonKazuo MinematsuCarlos A MolinaYongjun WangKs Lawrence WongS Claiborne Johnston
Published in: Clinical trials (London, England) (2020)
Ordinal outcomes can improve efficiency through required pre-specification, careful construction, and analyses. Greater pragmatism can be obtained by composing outcomes within patients. Desirability of outcome ranking provides a global assessment of the benefits and harms that more closely reflect the experience of patients. The desirability of outcome ranking probability, the proportion in favor of treatment, the win ratio, and partial credit can more optimally inform patient treatment, enhance the understanding of the totality of intervention effects on patients, and potentially provide efficiencies over standard analyses. The methods provide the infrastructure for incorporating patient values and estimating personalized effects.
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