BioPhorum CGT Regulatory Strategy workstream response to revised chapter 5.14 leading to EDQM Pharmeuropa monograph 34.3 gene therapy products for human use, chapter 5.34 and chapter 5.2.12. This content is available to BioPhorum members only.
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This paper is the BioPhorum response to the recent ICH Q14 and Q2(R2) consultation. Overall, the first-draft guidelines seen by the team need to be less detailed in the main body of the documents but more specific to analytical methods. The documents must also be consistent throughout (across Q14 and Q2 and between the main body texts and examples). Further, detailed examples are needed for modalities other than small molecules (e.g., biologics, cell and gene therapies, etc.) as the small molecule examples are not relevant in some key respects. In the view of the BioPhorum team, the current documents will not drive a consistent understanding of regulatory expectations and the provision of information by industry to regulators.
A cross sub-team team of 18 members from 14 companies submitted feedback on WHO Approach towards the development of a global regulatory framework for cell and gene therapy products (document WHO/BS/2022.2424)
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BioPhorum CGT Regulatory Strategy workstream provided feedback on FDA draft guidance on human gene therapy products incorporating human genome editing .
BioPhorum response to USP <1083> draft monograph: Supplier qualification and guidance for associated risk assessments
BioPhorum member only content. This is detailed consultation response from BioPhorum Supply Partner to USP on draft monograph <1083>.
Industry feedback on ICH harmonized guideline: Continuous manufacturing of drug substances and drug products Q13 draft version
A comprehensive response to ICH Continuous manufacturing of drug substances and drug products Q13 draft version. The BioPhorum team overall think the draft reads well and believes the concepts and ideas are aligned with BioPhorum thinking on continuous manufacturing for biologic drug substance and drug product.
This BioPhorum paper contains feedback on the USP bioreactivity monographs to plastics used for manufacturing, and the use of resources. It also comments on the potential in vitro tests that should be included, the removal of the implantation test and safety, the removal of Class I to VI, and the topics for omission or inclusion into the <1031> chapter outline. The expert feedback provided in these documents aims to support the USP and, ultimately, produce documents that improve patient safety – but with adequate use of finite analytical resources, which would not be the case with divergent expectations.
Feedback on the FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early phase clinical trial
This member document summarizes consolidated feedback and recommendations from a collaboration of BioPhorum members on the FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early phase clinical trial consultation.
Feedback on FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial
BioPhorum members provide feedback on FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial.