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

What is the scope for differentiation in how a pharma company interacts with patients? What are some of the questions that development organizations can start proactively answering as they go through regulatory approval? It may be worth to add (e.g. in appendix) the definition of patient’s perspective and patient preference, so that the text can be better understood. Definition of patient preference (CDER, 2016) is provided in the table (page 3) but it does not clearly appear as a definition. We welcome the opportunity to submit comments on the ICH reflection paper to advance patient-focused drug development. Comments on proposed Guidance on COAs: We endorse the proposal of not restricting guideline scope to patient-reported outcome instruments, in favour of a guideline covering all types of clinical outcome assessments (COAs). We strongly value the reference to ‘concepts’ (line 44), as it is time to move away from an ‘instrument-led’ approach to a ‘concept-led’ approach to best capture what matters most to patients. Likewise, we strongly support the reference to ‘qualitative and quantitative methods’, since mixed method research is best suited to generate holistic and patient-centred evidence. Qualitative methods are of particular relevance in rare

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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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