Beyond “Intent‐to‐treat” and “Per protocol”: Improving assessment of treatment effects in clinical trials through the specification of an estimand

DOI Web Site Web Site Web Site 1 Citations
  • Frank Pétavy
    European Medicines Agency Domenico Scarlattilaan 6 Amsterdam HS 1083 the Netherlands
  • Lorenzo Guizzaro
    European Medicines Agency Domenico Scarlattilaan 6 Amsterdam HS 1083 the Netherlands
  • Inês Antunes dos Reis
    Medicines and Healthcare products Regulatory Agency 10 South Colonnade London E14 4PU UK
  • Steven Teerenstra
    Dutch Medicines Evaluation Board College ter Beoordeling van Geneesmiddelen Graadt van Roggenweg 500 Utrecht AH 3531 the Netherlands
  • Kit C.B. Roes
    Dutch Medicines Evaluation Board College ter Beoordeling van Geneesmiddelen Graadt van Roggenweg 500 Utrecht AH 3531 the Netherlands

Search this article

Description

<jats:p>There is a key problem in randomised clinical trials as outcomes can be distorted due to informative post‐randomisation events. This is inadequately addressed by the use of traditional intention‐to‐treat or per protocol analysis sets and often either ignored or wrongly labelled as missing data. As a consequence, the treatment effects of interest in a clinical trial are not well defined and their estimates might be misinterpreted.</jats:p><jats:p>The estimand framework should help all those planning, conducting and analysing clinical trials as well as those interpreting the results to better define, estimate and understand the treatment effects of interest.</jats:p><jats:p>This framework is described in the addendum to ICH E9 and addresses precisely this problem. It is relevant for regulatory drug trials and academic‐run trials, as well as for trials of nonpharmacological interventions.</jats:p>

Journal

Citations (1)*help

See more

Report a problem

Back to top