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

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Not only should the endpoint be robust enough to inform regulatory decision making but also suitable to be included in the labelling to inform patients. To avoid that the ‘endpoint’ prematurely is drawn into the discussion and that the ‘patient perspective’ is lost, consider rewording the 4 th bullet to “What impacts and concepts are most relevant to patients and what endpoints can be constructed to capture these concepts? Can these endpoints be incorporated in clinical trials in a manner that will be robust enough for regulatory decision making?” What endpoint are most relevant to patients, and can these endpoints be incorporated in clinical trials in a manner that will be robust enough for regulatory decision making? • Primary care and Hospital attendances, Quality of life including existing tools and regional reporting system What is a clinically meaningful change in an endpoint from a patient perspective? – it is suggested deleting “clinically” What is a clinically meaningful change in an endpoint from a patient perspective?

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What is a clinically meaningful change in an endpoint from a patient perspective?

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• Number to treat and Outcome measures- absolute risk reduction.

• Transparency with pharmaceutical industry conflict of interest.

Stock availability.

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Traditional clincial trials in cancer care use mostly surrogate markers for evaluating the treatment effect. Many of these are not at all relevant to the patient, especially when advanced/metastatic disease is the target. For example progression free survival (PFS) with varying definitions from trial to another, is totally irrelevant for the patient unless it is associated with relief of symptoms. It should not be used as a surrogate for overall survival; in that it has not been proven a valid measure, except for a few occasions. A

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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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