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

Comment: As patients’ perspectives are considered in drug development, it is essential to qualify that the impact is on average. Lines 13-14 refer to a patient’s perspective i.e., the individual patient, rather than to patients’ perspectives. This background section sets up an unrealistic expectation that drug development will evolve to a unique, singular patient. The text throughout the document should be made clear that any input from patients (plural) would be implemented in a clinical trial or in a drug development program, on average. That as we identify what exactly can be measured, there will be a mean and a standard deviation that will influence rather than a separate solution for individual patient preferences. Patient reported outcomes (PROs) have been collected in clinical trials and also in routine clinical practice for decades; thus we are not dealing with a new thing. They have been collected eg. With different questionnaires first in paper format, later as electronic. Quality of life questionnaires have also been there earlier. In a way the patient’s perspective has been there to some extent. Patient’s perspective might be considered even earlier during research phase/pre-clinical studies One of the major problems with the earlier approach has been the vast heterogeneity of the ways of collecting PROs and including QoL data. See above. In addition to identifying, collecting, and analysing what is meaningful to patients, also the utilization of the data has not been optimal, or not at all there. Maybe to add a definition on patient centricity, patient focused research? ISPOR published this one, with the contribution of patients: “The active, meaningful, and collaborative interaction between patients and

patients’ perspective, beginning early in drug development

10

13-14 1

9

14

It is important for health authorities and for drug developers to incorporate the patient’s perspective, at research or in beginning early development phase

10

18-19 1

10

20-21 1

15

20-24 A

In many instances patient focus is already considered in traditional development plans, and patient input, when needed, is already

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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