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Safeguarding public health

Meet the Regulators MHRA, London

13 April, 2011

LRMN/ MHRA MEETING

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ATMPs - the regulatory setting

Marketing Authorisationcentralised licence (MA)

(CAT) EMA

CommercialClinical TrialsPhase I, II, III

Pre-clinical

Safety

Clinical Trialsnational licence (CTA)

NCA

EUTCDor

Blood Directive

Donation, procurement

& testing (processing)

Unlicensed (Hospital Exemption / Specials)

Cert. of Quality & non-clinical data

EMA

GP GMPMIA(IMP)

GMP

GMPMIA

GLP

DossierDossier

Transplants or TransfusionsProcessing, preservation, storage, distribution

GP

Borderline – classification of product: medicinal product?

Dossier

Advice and guidance NCA / EMA

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Presentation Outline

• Abbreviation glossary

• ATMPs – the regulatory setting

• Useful links

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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

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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

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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

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Inspectorate contact with ATMP Industry

• DH initiatives

- Stem Cell Tool Kit

• Commission / ATMP Regulation :

- Article 5 (Annex 2)

- Article 25 (Report and review)

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Useful links

• EMA – What’s new in Inspections:- http://www.emea.europa.eu/Inspections/WhatsNew.html

• Commission ‘Latest News on Pharmaceuticals’:

- http://ec.europa.eu/health/documents/new_en.htm

• GMP Volume 4:- http://ec.europa.eu/health/documents/eudralex/index_en.htm

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Scientific Advice (National) for ATMP Scientific Advice (National) for ATMP 2009 - present2009 - present

Safeguarding public health

CTMPCTMP GTMPGTMP

20092009 5 3

20102010 5 1

20112011(to Mar)(to Mar)

2 0

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1. Characterisation and Impurities

2. Mechanism of Action and Potency Testing

3. Demarcation of Drug Substance and Drug Product

Recurring issues for CTMP:Recurring issues for CTMP:

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Safeguarding public health

Advanced Therapy Medicinal Products: EU procedures

LRMN 13 April 2011

Patrick Celis, PhDCAT Secretariat, EMA, London

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CLASSIFICATION

EVALUATION

CERTIFICATION

Tasks of the CATTasks of the Committee for Advanced Therapies (CAT)

Scientific Advice

Support to PDCO

Support to CHMP / COMP

Interaction with

stakeholders

Publications, Guidelines

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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

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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

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Classification procedure

44 applications received so far42 finalised• Within 60 days • 4 out of 42: 1 month clock stop

for clarifications

(Status April 2011)

Outcome

10

15

13

1 3

GTMP CTMP TEP

ATMP Not ATMP

GTMP

Not ATMPATMP

TEP

CTMP

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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)

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Source: CAT monthly report March 2011

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Thank you for your attention

Dr Patrick Celis (CAT Secretariat)Patrick.celis@ema.europa.eu+44 207 418 8656

Queries: AdvancedTherapies@ema.europa.euATMP Microsite: www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000294.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac05800241e0

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CAT workprogramme 2010-201519

Useful links

Developers

CAT

MHRA information on ATMPshttp://www.mhra.gov.uk/Howweregulate/Advancedtherapymedicinalproducts/index.htm

EMA information on Advanced Therapieshttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000294.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac05800241e0&jsenabled=true

European Commission’s Directorate for public health and risk assessmenthttp://ec.europa.eu/health/human-use/advanced-therapies/index_en.htm

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Useful links

Developers

CAT

CAT Work programme 2010-2011http://www.ema.europa.eu/docs/en_GB/document_library/Work_programme/2010/11/WC500099029.pdf

CAT Interested Parties and Focus Group Non-Clinical developmenthttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000309.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac058009a23d&jsenabled=true

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Contacts for further information

MHRADr Gopalan Narayanan - gopalan.narayanan@mhra.gsi.gov.ukMr Ian Rees - ian.rees@mhra.gsi.gov.ukDr Elaine Godfrey - elaine.godfrey@mhra.gsi.gov.uk EMA CAT Secretariat Dr Lucia D’Apote - lucia.dapote@ema.europa.eu