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

2. Specific comments on text

Stakehol der no.

Line no.

Section no.

Comment and rationale

Proposed changes / recommendation

“inform regulatory, and health technology assessment (HTA) decision making” (lines 5 and 61)

13

5-61

Whilst we appreciate the proposed ICH guideline stems from and is aimed at regulatory authorities, we believe that the future guidance should adopt a more holistic perspective and also aim to meet the evidence requirements from other healthcare decision-makers, such as Health Technology Assessment bodies. The latter, too, are increasingly endorsing the value of patient perspective into their own decision-making processes. Any divergence across healthcare decision-makers on the definition of acceptable, reliable, valid and representative patient experience data may result in conflicting decision-making outputs, which may ultimately negatively impact timely access for patients to novel therapies. Patient satisfaction with new processes put in place for patient-focused drug development is essential. This should be reflected in the wording of the guideline.

We would propose to amend the sentence as follows:

7

6-8

It also presents opportunities for development of new ICH guidelines to provide a globally harmonized approach to inclusion of the patients’ perspective in a way that is satisfactory for the patients as well as methodologically sound and sustainable for both regulated industry and regulatory authorities

6

9-50

A

General comments on section A: Most early development programs in cancer, including hematological cancer, take place in incurable cancers with highly limited treatment options; they

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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