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

What are the key symptoms reported by the patients that could be used in the case definition of patients? Is the burden of disease less severe in those who develop breakthrough disease after vaccination?

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51-73

Which groups of patients / advocates do they suggest approaching to get the best insights in the most efficient way? We understood that the list of questions is not an exhaustive list. However, it could be beneficial to add a question link to enrollment, adherence or retention of patients in Clinical studies. Indeed, Patient's perspective can also support the design of clinical studies to improve the enrollment, adherence, and retention of patients in the trial (as mentioned line 36) and the current questions don't reflect this opportunity. One important contribution patients and their representatives can have is on the conduct of the research, i.e. the acceptability of the clinical trials constraints, areangements and provisions which repsect ethical considerations etc. What are the patients’ unmet needs? – suggest deleting “potential drug targets”, this should be about understanding patient experience of the disease in its entirety rather than whether there’s an aspect of the disease that’s druggable. New drug targets are important for unmet medical needs, and also for situations where the medical need is met but for a certain time. For example drug resistance can occur, or immune evasion. The drug effect vanishes after several months or years of satisfying response to treatment. Of course it depends on the definityion of an unmet need, which could encompass this situation. However, most often unmet need is used for conditions where no

12

55-63 B

15

55-63 B

To add: - How can the trial design and practical arrangements be improved to ensure high patient retention? What are patients’ unmet needs that suggest potential drug targets?

9

56

15

56

B

What are patients’ unmet needs that suggest potential drug targets? Or needs that are only partially or temporarily covered?

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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