© Safeguarding public health Meet the Regulators MHRA, London 13 April, 2011 LRMN/ MHRA MEETING

© ATMPs - the regulatory setting Marketing Authorisation centralised licence (MA) (CAT) EMA Commercial Clinical Trials Phase I, II, III Pre- clinical Safety Clinical Trials national licence (CTA) NCA EUTCD or Blood Directive Donation, procurement & testing (processing) Unlicensed (Hospital Exemption / Specials) Cert. of Quality & non-clinical data EMA GP GMP MIA(IMP) GMP MIA GLP Dossier Transplants or Transfusions Processing, preservation, storage, distribution GP Borderline – classification of product: medicinal product? Dossier Advice and guidance NCA / EMA

© Presentation Outline Abbreviation glossary ATMPs – the regulatory setting Useful links

© Abbreviation Glossary ATMP- Advanced Therapy Medicinal Product (GT, SCT, TEP) CAT- Committee for Advanced Therapy EMA- European Medicines Agency EUTCD- EU Tissue and Cells Directive (2004/23/EC) GP- Good Practice (Quality System under Blood & EUTCD) GT- Gene Therapy NCA- National Competent Authority (e.g. MHRA) SCT- Somatic Cell Therapy TEP- Tissue Engineered Products

© Inspectorate contact with ATMP Industry Regulatory advice on manufacturing : -Verbal -Written -Meetings – GMP only / with CTU / with Licensing Division Inspections: -Hospital Exemption - Manufacturer’s Specials -CT - MIA(IMP)- Full - MIA

© Inspectorate contact with ATMP Industry Formal classification advice: -Available from EMA and via MHRA website -MHRA’s centralised process been in place for 3 years: Year No. ·08/09: 8 ·09/10: 24 ·10/11: 13 -System been well received -Advice requests received on a broad range of product types

© Inspectorate contact with ATMP Industry DH initiatives -Stem Cell Tool Kit Commission / ATMP Regulation : -Article 5 (Annex 2) -Article 25 (Report and review)

© Useful links EMA – What’s new in Inspections: - Commission ‘Latest News on Pharmaceuticals’: - GMP Volume 4: -

© Scientific Advice (National) for ATMP present Safeguarding public healthCTMPGTMP (to Mar) 20

© 1. Characterisation and Impurities 2. Mechanism of Action and Potency Testing 3. Demarcation of Drug Substance and Drug Product Recurring issues for CTMP:

© Safeguarding public health Advanced Therapy Medicinal Products: EU procedures LRMN 13 April 2011 Patrick Celis, PhD CAT Secretariat, EMA, London

© CLASSIFICATION EVALUATION CERTIFICATION Tasks of the CAT Tasks of the Committee for Advanced Therapies (CAT) Scientific Advice Support to PDCO Support to CHMP / COMP Interaction with stakeholders Publications, Guidelines

© ATMP Evaluation procedure ATMPs will follow the Centralised procedure (mandatory scope)  single MA (marketing authorisation) for entire EU: 210 Day procedure Evaluation by two independent (Rapp/CoRapp) teams from CAT All scientific discussion and adoption of key documents at CAT ATMP evaluation procedure builds on full transparancy between CAT and CHMP to avoid divergent views. * CHMP = Committee for Human Medicinal products. Responsible for opinion on centralised MAA + post-authorisation follow-up / Pharmacovigilance

© Classification procedure Incentive Open to all applicants Scientific Recommendation from CAT on the Regulatory Classification of their ATMP 60-day procedure (often shorter) Publication of summary information on classification Procedure reviewed (simplified) following experience in 2009

© Classification procedure 44 applications received so far 42 finalised Within 60 days 4 out of 42: 1 month clock stop for clarifications (Status April 2011) GTMP Not ATMP ATMP TEP CTMP

© Certification procedure Only for SMEs Scientific evaluation by CAT of -(early) quality / development data (Module 3) -(early) non-clinical data (Module 4) 90 day procedure Evaluation to the scientific standards of a MAA -The SME applicant will always received the evaluation report (and List of issue for future consideration) -If positive evaluation: Certificate by EMA 1 Certification procedure finalised (May 2010)

© Source: CAT monthly report March 2011

© Thank you for your attention Dr Patrick Celis (CAT Secretariat) Queries: ATMP Microsite: neral/general_content_ jsp&murl=menus/regulations/r egulations.jsp&mid=WC0b01ac e0 neral/general_content_ jsp&murl=menus/regulations/r egulations.jsp&mid=WC0b01ac e0

© CAT workprogramme Useful links Developers CAT MHRA information on ATMPs edicinalproducts/index.htm EMA information on Advanced Therapies ation/general/general_content_ jsp&murl=menus/reg ulations/regulations.jsp&mid=WC0b01ac e0&jsenabl ed=true European Commission’s Directorate for public health and risk assessment therapies/index_en.htm

© Useful links Developers CAT CAT Work programme ork_programme/2010/11/WC pdf CAT Interested Parties and Focus Group Non-Clinical development ation/general/general_content_ jsp&murl=menus/reg ulations/regulations.jsp&mid=WC0b01ac058009a23d&jsenabl ed=true

© Contacts for further information MHRA Dr Gopalan Narayanan - Mr Ian Rees - Dr Elaine Godfrey - EMA CAT Secretariat Dr Lucia D’Apote -