All Times EDT
Keywords: estimands, estimators, intercurrent events, ICH E9(R1), missing data, sensitivity analysis
This will be a panel discussion in which panelists will have specific roles and provide responses pertinent to their function and experience. This panel will include confirmed participants in the following actual roles: Industry Statistician, Industry Clinician (EU), FDA Statistical Division Head, FDA Clinical Team Leader, ICH E9(R1) Addendum Expert, and former European Medicines Agency Statistician. In addition a panelist will respond from the perspective of prescribers and patients. A sampling of questions are, as follows: • What are relevant questions for different stakeholders (health authorities, prescribers, payers, sponsors, etc.) in a trial? Any examples? • Are estimand proposals driving innovation in terms of using more focused clinical questions and proposing novel ways to handle intercurrent events? Are we only seeing analytic approaches similar to the existing pre-estimand approaches? • How would estimand choices relate to labeling language? • Now that sponsors are submitting estimand descriptions in their protocols/SAPs, is anything changing in how the primary efficacy endpoint is analyzed? Or has the estimand framework been generally used to describe/support the standard analyses that have been performed? • Treatment policy strategy is widely recommended by health authorities. It can be implemented only if data can be retrieved after the intercurrent events handled by this strategy. For example, how likely are patients who have discontinued treatment be followed up at the targeted clinical visit? How much data needs to be retrieved for a meaningful analysis? • How are treatment discontinuations handled? Does the reason for discontinuation make a difference in how these are handled for the analysis? • Are there differences in preference for estimand strategies between different health authorities?
The audience will have the opportunity to ask additional questions and interact with the panel.