Keywords: estimands, pulmonary-allergy, ORISE Project
In regulatory interactions between the Agency and the sponsor for new drug development, agreement on estimands is crucial because it has direct impact on both drug approval and labeling claims. However, there has been lack of consistency on estimands, possibly due to different opinions on evidence of efficacy or lack of fully clear and agreeable guidance. In 2017, ICH published an addendum to E9 Statistical Principles for Clinical Trials to provide guidance on estimands in clinical trials. However, it gives a general principle on estimands, not specifically tailored to each indication. To discuss with the sponsor more effectively and efficiently, the Agency's clinical and statistical teams are deriving clinically convincing and statistically sound estimands for clinical trials in common disease areas. Estimands are closely tied to trial designs and statistical methods. Considerations related to the design would be disease condition - acute or chronic, or symptomatic or irreversible damage. The main purpose of this roundtable is to share participants’ experience and opinion on estimands in various clinical settings and related statistical issues from both regulatory and drug developer’s perspectives. An FDA clinical and statistical teams’ experience from ORISE Project on estimands in pulmonary-allergy therapeutic area will be shared for discussion. Feedback from the roundtable discussion will be considered in the project outcomes and findings.