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

early stage of disease may differ from late stage. This is alluded to at line 105 but could be made more explicit here. The sentence “…reflect concepts (e.g., pain, fatigue, physical function, etc.) that matter…” can be modified to emphasize that the patient’s perspective addresses both effect and safety issues Collection of patient information should occur in a way that is transparent and understood by patients

6

44

A

“…reflect concepts (e.g., pain, fatigue, physical function, adverse events, etc.) that matter…” We would recommend to add one point to the bullet points list after line 46: - ensure that the information, including Patient-Reported Outcomes, is collected and processed in a manner that is transparent and clearly communicated to patients

7

40-46 A

11

41

A

There is a need to clarify “planning” and “decision-making” Does “planning” refer to the development of the product and the related planned clinical studies ? While “decision making” would refer to regulatory for Regulators, does “decision making” include that of sponsors’ with regards to a drug development program ? This suggests a completely parallel research line (methods and measures), parallel to what is happening already regularly. That is in fact a waste of means and personnel. The proposed research should be replacing – possibly invalid - research proposed and initiated by the industry to prove the effectiveness of the drug, to finetune the promotion and marketing. In vaccine development, PROs could support the assessment of symptom severity in breakthrough cases after vaccination and also help to develop case definitions in case of symptoms based definitions. Does heterogeneity include stage of disease ?

11

46

A

16

47-50

Pinpoint where the new research is going to make a difference.

8

51-63

Inclusions of two additional questions:

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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