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

Stakeholder no.

Section No.

Comment and rationale

Proposed change / recommendation

done in collaboration with all the stakeholders. The patients/families’ rights and integrity must be ensured.

It could be helpful to focus on a limited number of (existing or new) methods to translate patient preference into drug development for the sake of harmonization and standardization. It could be considered incorporating simplified binary futility questions, mirrored between patient and treating physician, such as: “has this treatment been useful for me/my patient” to gather better insight into basic correlation/discordance between perspectives. Overall, the content and intent of the reflection paper is welcome and important. We agree with the proposed future topics for ICH guideline development; however, other potential topics should be considered. For example: guidelines that address patient perspectives on unmet needs, input into trial design, protocol development and supporting better enrolment and retention. If guidelines are developed, it may be helpful to include sections on how patient perspective information will be used and what weight will be attached to it versus traditional physician-determined endpoints. Also, it would be helpful to understand the extent to which early patient qualitative data would influence early scientific meetings and the expectations around collecting such data.

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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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