Overview of comments received on 'ICH reflection paper on proposed ICH guideline work to advance patient focused drug development’

• Reduce impairment or resolve it, and time limited prescription. What would be the most appropriate endpoints to use in clinical trials (and robust enough to inform regulatory decision making)? • NNT (Number Needed to Treat), absolute risk reduction What is a clinically meaningful changes in an endpoint from a patient perspective?

• Resolving or limiting the reason for prescription indication.

• Stop taking the prescription. How to define meaningful change in a patient over time?

• Return to basal status, with limited or no consequences.

Qualitative and quantitative methods to identify disease/treatment impacts important to patients that would be candidate concepts for measurement with patient reported outcome (PRO) measures or other types of COAs or in quantitative assessments of the patient perspective. • The approach to organize and structure the content of the guideline document would undergo further consideration as this work advances under an ICH new topic proposal. One approach would be to develop the main document with an extensive focus on common considerations for all COAs and include annexes with considerations that may only apply to certain COA types such as observer reported (ObsRO), clinician reported (ClinRO), performance based (PerfO) measures, etc. Comments on table (page 2): It should be highlighted that much groundwork is required to identify patient opinion in the initial stage both for disease effects & treatment burdens as well as preferences. This should be done by impartial third parties (i.e.,

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Overview of comments received on 'ICH reflection paper on proposed ICH guideline work to advance patient focused drug development’ (EMA/CHMP/ICH/415588/2020) EMA/194133/2021

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