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

o PRO-MID (https://promid.mcmaster.ca/) Of special note, the PROTEUS initiative aims to promote systematic use of methodologic tools developed to optimize the design, analysis, reporting, and

interpretation of PROs in clinical trials. (https://more.bham.ac.uk/proteus/)

2. We appreciate the inclusion of these core assumptions in the document. We agree that measures that will be used to assess the patient perspective should be developed with the idea that this will be used to assess the same disease in multiple regions of the world. Ensuring that these measures are developed simultaneously in multiple countries (guaranteeing translatability) should be considered best practice. The EORTC has standardized guidelines on how to develop PRO measures for various cancer diseases that takes into account the patient perspective from various countries and cultures. This can be used as a reference in the development of such measures for other diseases. 3. Additional questions that are relevant in the discovery and development phase should include a. What is the patient reported experience regarding their disease and treatment? How can this information be incorporated in the benefit/risk assessment in clinical trials?

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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