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

Patients and caregivers have direct experience in living with a disease.

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10

Patients have direct experience however, it is recommended to include “caregivers”, especially for mentally impaired patients as caregiver’s perspective is crucial. “Patients have direct experience in living with a disease”: the wording suggests that this pertains only to chronic diseases. It would be beneficial to provide additional information about rationale for greater use of patients’ perspective throughout the drug development process. Following references could strengthen the message: EPIC study – Systematic Evaluation of Patient-Reported Outcome Protocol Content and Reporting in Cancer Trials – https://academic.oup.com/jnci/article/111/11/1170/5430934 Patients experience – Defining Patient Experience – https://pxjournal.org/journal/vol1/iss1/3/ Research waste and outcomes to patients that matters – Maximising the impact of patient reported outcome assessment for patients and society – https://www.bmj.com/content/364/bmj.k5267 FDA Patient-Focused Drug Development Guidance Series – https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient- focused-drug-development-guidance-series-enhancing-incorporation-patients- voice-medical PROs in RWE development – Harnessing the patient voice in real-world

Patients have direct experience in living with a disease (chronic or temporary).

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10-11 A

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10-14 A

evidence: the essential role of patient-reported outcomes – https://www.nature.com/articles/d41573-019-00088-7

PROs in the regulatory decision-making process – Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada – https://www.sciencedirect.com/science/article/pii/S1470204518300974?via% 3Dihub

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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