Although it's an MHRA consultation rather than an FDA docket, the framework has topics across several communities:
- New Approach Methodologies / move away from animal models (§6.4.5, §9.1, §9.4)
- Predictive knowledge - in silico models, MIDD, QSP, digital twins/virtual patients (§6.4.2)
- Real-world evidence, external controls, and natural-history-as-control (§6.4.1, §6.4.4, §6.6)
- Innovative trial designs and endpoint/biomarker validation (§6.4.3)
- Prior knowledge and platform technologies (§6.4.1, §9.2)
- Master files for biological/ATMP starting and raw materials (§9.3)
- CMC and pharmacovigilance for ATMPs, gene/cell therapies, and ASOs, including AI-based signal detection (§6.6)
We are seeking active volunteers to help review the draft framework and assess whether AAPS should provide a response.
Please review the draft guideline and respond to @Subathra Ramamoorthy close of business on Monday, June 1:
1. Does your community feel AAPS should respond? A yes or no response is appreciated.
2. If yes, who would you be interested to represent the Pharmaceutical Quality on the Response Team?
Subathra Ramamoorthy, Vice Chair
Pharmaceutical Quality Community
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Subathra Ramamoorthy
Associate Principal Scientist
Veranova
Westford MA
[email protected]Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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