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

evaluate therapies aiming to prolong patient life and it seems likely that, in this setting, any demonstrable effect on life prolongation will trump other factors such as patient experience. However, the transition from early phase, in which drugs may be used at maximum dose in order to not jeopardize efficacy signals, to later phase clinical research in non-end-of-life settings often does not take the changing scenario into account and leads to frequent licensing of drugs and their combinations at doses and posologies that are not optimal for patient experience. Both this and post-approval research with the aim of label changes seem to be areas where patients’ perspectives should be much more strongly incorporated. Very significant progress for patient wellbeing has been made in post-approval academic clinical research; e.g., demonstration of lower toxicity of low-dose dexamethasone or significantly reduced rate of peripheral neuropathy with subcutaneous and weekly bortezomib in multiple myeloma. This research was partly patient-driven and has likely spared thousands of patients unnecessary side effects. However, as it was never a regulatory requirement and/or submitted to regulators, these improved ways of drug delivery have not found their way into drug regulatory labels. Still to date, this allows for new research to be conducted using sub-par comparator arms based on clinically outdated regulatory labels to patients’ detriment. Tolerability and patient preference research should be made mandatory post- approval by regulators. Frameworks should be developed in particular for flagging drugs that showed disproportionate risk/benefit scores in patient evaluation in early stages of development, and for committing stakeholders to post-licensing research that should feed directly into the license label again if improved ways of administering drugs are identified. This could be enforced via a new conditional approval mechanism that takes patient experience gathered in early research into account, whilst acknowledging that early drug development is particularly complex and often has to focus on efficacy, above all.

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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