Pharmaceutical Quality Community

  • 1.  Volunteer opportunity

    Posted 52 minutes ago
    The MHRA has released a draft guidance, The Rare Disease Therapies Regulatory Framework, for public consultation (released 21 May 2026; comments close 30 July 2026, 11:59 PM UK time). The consultation and full draft are here: https://www.gov.uk/government/consultations/draft-rare-disease-therapies-regulatory-framework (also attached) .

    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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