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

indications, populations do not lend themselves to typical efficacy or safety endpoints, or to randomized trials etc. Therefore, it’s important that any guidelines are flexible in their application and aid the identification of appropriate (and helpful) patient-perspective measures. In the context of preference-based methodologies, discreate choice experiments (DCEs) have been commonly used to (1) estimate difference in utilities between health states; and (2) help to prospectively identify which outcome of a trial is more relevant to the patient (Curran et al 2021.)

We would propose to focus on developing the recommendations on:

Criteria and Best practice to determine the optimal attributes and level within preference-based studies Propose methodological recommendations to address the

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potential sources of bias in preference-based studies.

Best practices for quantitative analyses of the outcomes from preference-based studies

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76-78 Table

What disease effects and treatment burdens matter most to patients that might be addressed by a medical therapy? (How) does this vary by subpopulation?

(Box left on top)

Cardiovascular effects

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Risk for Infectious diseases

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Cancerous effects

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Mental health effects

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• Neurodegenerative effects What would be the best way to measure these disease or treatment burdens/effects in a clinical trial?

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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