1 st Workshop on Advance Therapy Medicinal Products (ATMPs) Scientific Recommendation on the Classification of ATMPs Insert your logo in this area Marie-Helene Pinheiro then delete this text box. EMEA Regulatory Affairs
AGENDA I ntroduction ATMPs Definition 1 . Borderlines’ considerations 2 . Scientific Recom m endation 3 . 3.1 Legal basis 3.2 Procedure 3.3 Role and Responsibilities 3.4 Timetable 3.5 Decision Tree 3.6 Publication of Summaries 3.7 Timing of Request for Scientific Recommendation of Classification Conclusion 2
INTRODUCTION ATMPs LEGI SLATI VE REFERENCES Q & S Human Tissues / Cells donation, procurement, testing, processing, preservation, storage and distribution Blood Dir. 2004/23/EC Other starting Dir. 2002/98/EC materials Medicinal GMP Products Clinical Trials Dir. 2003/94/EC Centralised procedure Dir. 2001/20/EC Reg. (EC) 726/2004 New Variation amended by Reg.1234/2008 Community Code ATMP Dir 2003/63/EC Reg. (EC) 1394/2007 & “New” ‘Annex I MEDICAL DEVICE Dir. 93/42/EEC, as amended “This Regulation lays down specific rules concerning the authorisation, supervision and pharmacovigilance of advanced therapy medicinal products.” 3
1. ATMP DEFINITION ADVANCE THERAPY MEDICINAL PRODUCTS Tissue engineering Gene therapy Somatic cell therapy products medicinal products medicinal products Genetically modified cells www.biomed.brown.edu www.heartandmetabolism.org Nat Biotechnol 2005, 23(7) COMBINED ATMP + MEDICAL DEVICES 4 Source: Dr Christian Schneider, CAT Chairman Source: Dr Christian Schneider, CAT Chairman
1. ATMP DEFINITION: GENE THERAPY MEDICINAL PRODUCT NEW DEFI NI TI ON ANNEX 1 Dir. 2 0 0 1 / 8 3 / EC Gene therapy m edicinal product means a biological medicinal product which has the following characteristics: (a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence; (b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence. Gene therapy medicinal products shall not include vaccines against infectious diseases. 5
1. ATMP DEFINITION: GENE THERAPY MEDICINAL PRODUCT VECTOR, NUCLEI C GENETI CALLY ACI DS, REPLI CATI NG MODI FI ED MI CRO- ORGANI SM HUMAN CELLS ( NOT I NCL. LI VE VACCI NES) cell line cell Direct application: 1) Isolation of the • viral vector target cells • non-viral vector 2) Gene transfer • naked DNA • replicating rec. micro-organism (adenovirus, salmonella) 3) Re-Infusion of the genetically modified cells 6 Source: Prof. K. Cichuteck Source: Prof. K. Cichuteck- - GTWP Chairman GTWP Chairman
1. ATMP DEFINITION: SOMATIC CELL THERAPY MEDICINAL PRODUCT NEW DEFI NI TI ON ANNEX 1 Dir. 2 0 0 1 / 8 3 / EC Somatic cell therapy medicinal product means a biological medicinal product which has the following characteristics: (a) contains or consists of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered, or of cells or tissues that are not intended to be used for the same essential function(s) in the recipient and the donor; (b) is presented as having properties for, or is used in or administered to human beings with a view to treating, preventing or diagnosing a disease through the pharmacological, immunological or metabolic action of its cells or tissues. For the purposes of point (a), the manipulations listed in Annex I to Regulation (EC) No 1394/2007, in particular, shall not be considered as substantial manipulations. 7
1. ATMP DEFINITION: SOMATIC CELL THERAPY MEDICINAL PRODUCT AUTOLOGOUS or XENOGENI C ALLOGENI C CELLS CELLS • ADULT CELLS : AUTOLOGOUS OR ALLOGENEIC • ADULT MULTI POTENT STEM CELLS • I NDUCED PLURI POTENT STEM CELLS ( NEW ) CELL PROCESSI NG and • CORD BLOOD STEM CELLS GRAFT SEEDI NG • EMBRYONI C STEM CELLS GRAFT IMPLANTATION 8
1. ATMP DEFINITION: TISSUE ENGENEERED MEDICINAL PRODUCT NEW DEFI NI TI ON Art. Reg. 1 3 9 4 / 2 0 0 7 � Tissue Engineered products (TEP) Contain/ consist of engineered cells/ tissues � Presented as having properties for, or is � used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue � Examples: Artificial skin (burn wounds) � Neo-organs (corneal, blood vessel, liver, cartillage or � bone tissue engeneereing) 9
2. BORDERLINE CONSIDERATIONS SOME I NCLUSI ON/ EXCLUSI ON CRI TERI A & PRI NCI PALS ( 1 ) � TEP may contain: � Non viable and viable cells are included � Products which do not contain any viable cells and which do not act principally by metabolic action are excluded � Cell/ device association no longer considered a priori as ‘engineered’ � Aspect/ definition of « substantial manipulation » to be considered 10
2. BORDERLINE CONSIDERATIONS SOME I NCLUSI ON/ EXCLUSI ON CRI TERI A & PRI NCI PALS ( 2 ) � In case ATMP containing both autologus and allogenic cells or tissues it shall be considered to be for allogenic use � In case product falls in definition of TEP and sCT it shall be considered as TEP � In case products falls in definition of GT and TEP or sCT, then GT> TEP> sCT 11
2. BORDERLINE CONSIDERATIONS SOME EXCLUSI ON CRI TERI A AND PRI NCI PALS ( 3 ) ART. 2 8 OF REG. 1 3 9 4 / 2 0 0 7 SO CALLED “HOSPI TAL EXEMPTI ON” � Additional exclusion under very specific conditions e.g.: � Non-routine basis of production � Specific quality standards � Used in same MS in hospital (manufacturing authorised by Comp. Authority of MS) � Custom-made product for individual patient � Under the exclusive professional responsablity of a practitioner National rules on the use of cells on ethical grounds 12
3. SCIENTIFIC RECOMMENDATION 3 .1 LEGAL BASI S: Art. 17 OF Reg.(EC) NO 1394/ 2007 “1. Any applicant developing a product based on genes, cells or tissues m ay request a scientific recommendation of the Agency with a view to determ ining w hether the referred product falls, on scientific grounds, w ithin the definition of an advanced therapy m edicinal product. The Agency shall deliver this recommendation after consultation with the Com m ission and w ithin 6 0 days after receipt of the request. 2. The Agency shall publish sum m aries of the recommendations delivered in accordance with paragraph 1, after deletion of all inform ation of com m ercial confidential nature.” 13
3. SCIENTIFIC RECOMMENDATION Letter Letter of of 3.2 PROCEDURE Intent Intent CAT Request � Company’s Form Report � Company’s Information Information � Product � Product � Product Information: � Product Information: � Scientific Information � Scientific EMEA Information � Summary for Aspect � Summary for Aspect Briefing � Legal/Regul Public Release � Legal/Regul Public Release � Applicant’s Note atory Aspects ITF: � Applicant’s atory Aspects � RA Status& position on RA Appoint � RA Status& position on RA Nomination CAT Current classification of Current classification � EMEA/CAT Medical Use EMEA � EMEA/CAT Medical Use Secretariat CAT � Other Info Comments � Other Info Comments Coordinator � EMEA/CAT � Position of Coordinator � EMEA/CAT � Position of Company on Conclusions Company on Conclusions Product’s Product’s Classification Classification http://www.emea.europa.eu/htms/human/advanced_therapies/intro.htm Applicant 14
3. SCIENTIFIC RECOMMENDATION 3.3 ROLES AND RESPONSIBILITIES � CAT: � responsible for provision of scientific recommendations � CAT Secretariat � coordinates the procedure at the level of the CAT � CAT Coordinator( s) � prepare and finalise the scientific recommendations � identify need to consult NB and/ or WP for agreement at CAT � EMEA Coordinator � Contact point � check adequacy of requests � Support CAT Coordinator � I nnovation Task Force ( I TF) � peer-review including regulatory, legal and scientific 15 aspects
3. SCIENTIFIC RECOMMENDATION 3.4 TIMETABLE (standard) Adoption Checking Step Evaluation Publication Day 60 Day 0 -15 Days -30 Days D 15 D 27 D 30 D50 CAT Submission No need for CAT Co- EMEA Coordinator CAT of letter of additional ordinator Checking: sends draft intent to information supported by adoption EMEA Publication recommendatio CAT CAT Secretariat EMEA of scientific Coordinator of summary n to CAT/ITF Secretariat sends draft finalises the recommendation prepares for comments recommendation to scientific briefing within 7 days EC for comments recommenda note within 10 days tion. CAT Coordinator supported by EMEA CAT scientific coordinator Clock start recommendation finalises scientific sent to Applicant recommendation EC: European Commission 16
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