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Efficacy vs Effectiveness — SCE Medical Oncology MCQ

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HardClinical Trials & StatisticsEfficacy vs EffectivenessSCE Medical Oncology

A phase III trial asks what 6-month symptom score would have been observed had protocol-prohibited rescue radiotherapy not been available. Which ICH E9(R1) strategy is being used for this intercurrent event?

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Correct answer: DHypothetical strategy

Explanation lettering: C = shown as B · B = shown as C · E = shown as D · D = shown as E

E is correct because the target outcome is defined in a world without rescue radiotherapy. A would use outcomes regardless of rescue; B would make rescue part of the endpoint; C would use response only before rescue; D would target a subgroup defined by potential rescue use.

Reference: ICH E9(R1) estimands training material (Current ICH training material): https://database.ich.org/sites/default/files/E9%28R1%29%20Training%20Material%20-%20PDF_0.pdf