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📌 Regulatory Guidance Hub

  • 1.  📌 Regulatory Guidance Hub

    Community Leadership
    Posted 09-18-2025 13:57

    NEW REGULATORY GUIDANCE SHARING THREAD

    Use this pinned post to share and discuss the latest regulatory guidance documents, updates, and announcements.

    How to contribute:

    • Share links to new guidance with a brief summary
    • Include the regulatory agency and effective date
    • Tag relevant colleagues who might find it useful
    • Add your key takeaways or implementation insights

    Recent additions: [This section will be updated as new guidance is shared]

    Let's keep our community informed and ahead of regulatory changes. Please reply to this thread with your updates! 👇



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    East Fallowfield PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------



  • 2.  RE: 📌 Regulatory Guidance Hub

    Posted 09-19-2025 09:20

    Hi Allison,

    This is perfect, thank you! I really like the approach we're using to share the documents with reviewers. It's user-friendly and thoughtfully structured, which will definitely help streamline the review process.

    Appreciate your efforts...!

    Regards,
    Vasu



    ------------------------------
    Vasudev Bhupathi M.S., M.B.A.
    Director-Pharmacovigilance
    Moderna Inc
    Littleton MA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 3.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 09-25-2025 08:29

    Busy day for FDA's CDER, 3 draft guidance were released for comment. Two for cell and gene therapies and one for regenerative medicines.


    Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations; Draft Guidance for Industry www.fda.gov/regulatory-information/...


    Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry www.fda.gov/regulatory-information/...


    Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry Expedited Programs Regenerative Medicine Therapies Serious Conditions

    #fda #DraftGuidance



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    East Fallowfield PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 4.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 10-09-2025 19:47

    The FDA posted a table October 8 with "an inventory of drug development contexts for which CDER is open to a streamlined nonclinical program" - that include the use of NAMs.  It includes references to various guidance. A worthwhile reference for those interested in NAMs. 

    Replacing and Reducing Animal Testing at CDER

    #fda #NAM



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    East Fallowfield PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 5.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 01-07-2026 09:19

    Following November 2025 endorsement of the ICH E22 draft Guideline on Patient Preference Studies, the Expert Working Group has released training materials.

    Patient preference studies ask patients what matters most about treatments-effectiveness, side effects, safety, convenience, and other factors-helping developers and regulators understand medical needs from the patient perspective.

    The E22 draft Guideline covers principles, study types, and design recommendations to harmonize approaches globally and support routine inclusion of patient preferences in decision-making.

    The draft guideline, presentation and training materials are available for download here: E22 draft guideline General Considerations for Patient Preference Studies 



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    East Fallowfield PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 6.  RE: 📌 Regulatory Guidance Hub

    Posted 01-12-2026 13:02
      |   view attached

    FDA released today 12Jan26 a new draft guidance on "Use of Bayesian Methodology in Clinical Trials of Drug and Biological Products" and is accepting comments until 13Mar2026. The draft guidance frames the Agency's thought's on the use of Bayesian methods in a variety of clinical situations as outlined in the Introduction:

    "Bayesian methods can be used in various ways in clinical trials. For example, Bayesian calculations can beused to govern the timing and adaptation rules for an interim analysis in an adaptive design, to inform design elements (e.g., dose selection) for subsequent clinical trials, or to support primary inference in a trial. The primary focus of this guidance is on the use of Bayesian methods to support primary inference in clinical trials intended to support the effectiveness and safety of drugs."

    This will obviously be of interest to a wide swath of the scientific community supporting clinical trials and just not the statisticians.

    #fda #Statistics #Bayesian

    https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-bayesian-methodology-clinical-trials-drug-and-biological-products



    ------------------------------
    Mark Arnold Ph.D., FAAPS
    Westampton, NJ
    [email protected]
    Bioanalytical Solution Integration
    LinkedIn: https://www.linkedin.com/in/markearnoldphd/
    Website & Blog: Bioanalysis & Biomarkers <bioanalysisandbiomarkers.blogspot.com>
    ------------------------------

    Attachment(s)

    pdf
    fda-draft-guidance-bayesian-methodology.pdf   451 KB 1 version
    Original Message


  • 7.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 01-16-2026 07:50

    ICH Expert Working Groups released new materials with three technical Guidelines: E2B(R3), M4Q(R2) and E6(R3).

    E2B(R3) - Information Paper and Implementation Guide Package aligning individual case safety reporting with the updated E2D Guideline

    M4Q(R2) - Mapping Document comparing the revised Quality section of the Common Technical Document to the 2002 version

    E6(R3) - Training Module 4 on Informed Consent for Good Clinical Practice

    These updates support harmonized electronic standards, streamlined regulatory submissions, and ethical clinical trial conduct.

    Updated Technical Documents Issued by ICH Expert Working Groups for Three Guidelines


    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    Coatesville PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 8.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 01-22-2026 13:08

    FDA has released ICH's DRAFT "M4Q(R2) The Common Technical Document for the Registration of Pharmaceuticals for Human Use: Quality" for public comment.

    The FDA has taken several months to issue this guidance for comment-ICH endorsed M4Q(R2) back in May 2025. 

    The draft begins with a brief scope statement followed by extensive commentary before the definitions, glossary, and references. From the Introduction:

    "The M4Q(R2) guideline establishes the location and structure of quality information for registration applications of all medicinal products for human use. It supports various submission types, including those referring to or consisting of master files, and applies to both initial marketing authorisation and post-approval submissions. This guideline is structured to be flexible to accommodate all types of medicinal products and their components."


    https://lnkd.in/eSYJKwjD



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    Coatesville PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 9.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 01-22-2026 16:24

    Thanks Allison! I'm curiously on the changes as I am a frequent consumer of module 3 and module 2.3 in the context of immunogenicity risk assessments and integrated summary of immunogenicity. Adding to my list of things to read, but probably not positioned to comment directly.



    ------------------------------
    Joleen White Ph.D.
    AAPS 2026 National Biotechnology Conference Track Chair
    Bioanalytical 101 Course Development
    Senior Advisor
    BioData Solutions LLC
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 10.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 01-30-2026 11:33
    The British Pharmacopoeia released two draft guidance documents for public consultation on characterization of capsid particle populations in rAAV products (attached):
    1. Determination of Vector Genome Identity, Integrity and Encapsidated DNA Impurities
    2. Capsid Protein Characterisation

    Consultation for Characterisation of the Capsid Particle Population in rAAV Products: Determination of Vector Genome Identity, Integrity and Encapsidated DNA Impurities + Capsid Protein Characterisation

    Comments are due March 27, 2026. If any communities would like to respond to this guidance, please contact me by February 6.



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    Coatesville PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 11.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 29 days ago
    The FDA released a new draft guidance: "Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause."

    This aligns with the Agency's recent announcement accepting a single pivotal trial for "targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations."

    The Introduction lays out what is needed for a 'plausible mechanism framework' to achieve approvals:

    "The plausible mechanism framework outlines recommendations to help developers generate sufficient clinical safety and efficacy data to demonstrate that a drug or biological product is safe and effective for the intended use, and that the product can be manufactured to regulatory quality standards. This includes evaluation of nonclinical and clinical data and chemistry, manufacturing, and controls (CMC) data to support product quality. Application of the plausible mechanism framework involves:

    • Identifying a specific genetic, cellular, or molecular abnormality with a clear connection between specific alteration and disease indication

    • Developing a therapy that targets the underlying or proximate pathogenic biological alterations

    • Relying on a well-characterized natural history of the disease in an untreated population

    • Confirming that the target was successfully drugged or edited or both

    • Demonstrating improvement in clinical outcomes or course"

    Considerations for the use of the Plausible Mechanism Framework



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    Coatesville PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 12.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 29 days ago

    This one I would love to help comment on. 



    ------------------------------
    Joleen White Ph.D.
    AAPS 2026 National Biotechnology Conference Track Chair
    Bioanalytical 101 Course Development
    Senior Advisor
    BioData Solutions LLC
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 13.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 19 days ago

    The FDA Published Draft Guidance on 3-Year New Clinical Investigation Exclusivity

    FDA has released a new draft guidance for industry, "New Clinical Investigation Exclusivity (3-Year Exclusivity) for Drug Products: Questions and Answers," aimed at clarifying the pathway for applicants seeking 3-year exclusivity for NDAs and NDA supplements.

    The guidance addresses the statutory and regulatory eligibility criteria and clarifies the processes for requesting exclusivity, how FDA makes eligibility determinations, and how those determinations are communicated through the Orange Book. To qualify, an application must be an NDA or NDA supplement for a drug with a previously approved active moiety under a 505(b) application, supported by new clinical investigations (other than bioavailability studies) that are essential to approval and conducted or sponsored by the applicant.

    Issued as part of FDA's Drug Competition Action Plan, this guidance supports a more efficient and transparent generic drug development and approval process. FDA has indicated it intends to update the document with additional Q&A content over time.

    Comments are due by May 4, 2026.

    New Clinical Investigation Exclusivity (3-Year Exclusivity) for Drug Products: Questions and Answers



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    Coatesville PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 14.  RE: 📌 Regulatory Guidance Hub

    Posted 12 days ago

    Hi Allison,

    Thank you very much for initiating this thread. Sharing timely regulatory updates in one place will be extremely valuable for the scientific community and will help many of us stay aligned with evolving guidance while continuously improving how we implement these practices in our work. I look forward to learning from the contributions shared here.

    Best Regards,

    Susana



    ------------------------------
    Susana Catacora-Lopez
    Immunoassays Manager
    Integrated Biotherapeutics
    Rockville MD
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message


  • 15.  RE: 📌 Regulatory Guidance Hub

    Community Leadership
    Posted 6 days ago

    Today, the FDA released "General Considerations for the Use of New Approach Methodologies in Drug Development" for public comment [https://www.fda.gov/regulatory-information/search-fda-guidance-documents/general-considerations-use-new-approach-methodologies-drug-development]. 

    While animal studies have long been a key tool for identifying safety risks, CDER is prioritizing the development of reliable alternatives that improve human relevance and reduce reliance on animal testing.



    ------------------------------
    Allison Radwick Ph.D.
    Sr Regulatory & Policy Comms Mgr
    USP
    Coatesville PA
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
    ------------------------------

    Original Message