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

such as process attributes and health-related quality of life)

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Questions related to patient preference—relevant throughout development— could include:

What is the gain in quality of life?

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• What is the length and cost of treatment for patient?

The acceptable trade-off is likely to vary depending on the nature and stage of the disease, particularly for progressive conditions.

acknowledge that patient perspectives on this point, e.g. tolerability/risk acceptance are likely to vary from patient to patient and how this could be addressed, i.e. how to avoid the ‘one-size fits nobody’ outcome that can arise when results/responses are averaged-off

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In cancer care, there are two very distinct treatment entities, ie. treatment to cure and treatment to palliate. Much due to the current end points used in advanced/metastatic cancer (PFS, tumour shrinkage, number of metastases) the focus is in what happens to the disease/tumour, not what happens to the patient. The drugs that are able to shrink the tumour are rendered the best, no matter how much toxicity to the patient they may cause. "T“e treatment was well tolerated" ”s a rather common conclusion in cancer drug trials where in fact 90 % of the patients suffer from adverse events, 30-40 % from serious adverse events, and some die due to treatment related complications. Considering the fact that the shrinkage of the tumour is a poor correlate to the patients overall prognosis, this kind of an approach needs to be changed. Especially if the patient dies due to treatment that is supposed to be palliative, the whole idea of the treatment as palliation has gone totally wrong.

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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