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

diseases and in paediatric, elderly and cognitively impaired populations. Supporting the proposal that the guideline covers methods around the definition and interpretation of clinically meaningful within-patient score changes, we believe that emerging novel methodologies should be acknowledged as valuable complements to the legacy anchor-based and distribution-based methods, such as qualitative methods (see J Patient Rep Outcomes 2019 Mar 4;3(1):16 for example). We suggest including in the finalised guideline clear references to modern test theory approaches, which add value in generating genuine patient-centric measurement (see references as examples). Comments on proposed Guidance on Patient Preferences: We appreciate the inclusion of guidance on the methods and approaches that can be used to measure the benefit- risk trade-offs from the patient perspective. Beyond benefit-risk trade-offs, patient preference information can also be used to provide valuable patient-centred insights along the drug development pathway. Therefore, we strongly propose the guidance document present a clear position on the situations where patient preference information can add value to regulatory decision making. Further, we would value specific guidance on when patient preference data, that is collected outside clinical

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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