Estimands Framework: Ensuring alignment between the key scientific question of interest, analysis and interpretation during clinical trial design.
The estimands framework, introduced in the ICH E9 (R1) guideline, facilitates discussions during design of clinical trials to ensure alignment between the key scientific question of interest, analysis and interpretation. In the context of oncology solid tumor randomized studies, commonly performed analyses of progression-free survival (PFS) will be reviewed, with a critical eye on which question they address, using the estimands terminology. The estimands framework will not necessarily change common analysis methods within oncology, but will describe in detail how intercurrent events (such as subsequent therapy) will be taken into account and increase transparency on the treatment effect being evaluated.
KEY TAKEAWAYS:
Since the ICH E9 (R1) guideline became effective, more attention has been given to the fact that censoring or not censoring for intercurrent events actually address different clinical questions.
- The different questions of interest will be mapped to the choice of estimands, with examples of wording for practical implementation in protocol.
- The common practice of censoring PFS for intercurrent events will be discussed. In particular, it may be justified in supplemental analyses but should not be used uncritically.
SPEAKERS:
