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

Patient preferences informing drug development, benefit-risk assessment, and other decisions: among other decisions, the decision made by the patients to ake the medicine, once authorised. For this, different methods to explain the benefit/risks should be tested: textual, tabular, graphic methods... (e.g. as a follow-up of IMI PROTECT project).

New ICH guideline addressing methods for elicitation/ collection, analysis, reporting and application of qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among the alternatives, and also methods for the explanation of benefits and risks understandable by patients What would be the best way to measure these disease or treatment burdens/effects in a clinical trial? And are the methods appropriate for the patients?

8

77-78 Table

In ‘Drug Development Process Informed by Patient Perspective’, it would be useful to emphasise the importance of methodology to encourage patient compliance in the second bullet.

7

77-78 C – Table Comment to "potential ICH guideline topic" for the Discovery/development process: The development of COAs should require mutual understanding and

appreciation among the stakeholders involved. Clinical Outcomes should be not only meaningful for patients but also concord with Primary Endpoints set by the Regulator. It should be specified that COAs can be used to demonstrate clinical efficacy and/or value.

10

82-98 1

Extremely well formulated; we undersing everything presented here.

6

82-109 C

It should be considered that, apart from social or cultural patient sub-groups, there are also biological patient sub-groups. If we aim to include patients in the development of relevant COA etc, we should not forget that, at least for many incurable cancers, we will have an over-representation of ‘long-term surviving’ patients in working groups. Depending on the cancer, patients with aggressive sub-types of disease often will struggle to get involved into discussions around drug development and may have a different perspective

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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