The Advanced Therapy Medicinal Products Regulation from the European Medicines

Agency (EMEA)'s perspective

Nathalie Rampal OlmedoEMEA – Directorate – Legal Sector

British Institute of International and Comparative Law25th September 2008

ADVANCED THERAPY MEDICINAL ADVANCED THERAPY MEDICINAL PRODUCTS (ATMP)PRODUCTS (ATMP)

• Medicinal productsMedicinal products based on – genesgenes (gene therapy)– cellscells (cell therapy)– tissuestissues (tissue engineering)

• Revolutionary treatmentsRevolutionary treatments of a number of diseases or injuries, such as skin in burn victims, Alzheimer, cancer or muscular dystrophy

• Huge potential potential for patients and industry

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REGULATION (EC) No 1394/2007REGULATION (EC) No 1394/2007

KEY ELEMENTSKEY ELEMENTS

• Centralized marketing authorizationCentralized marketing authorization (Single scientific evaluation)

• New Committee for Advanced Therapies (CAT)Committee for Advanced Therapies (CAT)

• Specific rulesSpecific rules on authorisation, supervision and pharmacovigilance

• Specific technical requirements Specific technical requirements (SPC, labelling and package leaflet)

• Combined ATMP Combined ATMP evaluated by EMEA

• Post-authorisationPost-authorisation requirements (follow-up of efficacy/adverse reactions, risk management, traceability)

• Special incentivesincentives (SMEs)

• CATCAT

• EvaluationEvaluation ProcedureProcedure • IncentivesIncentives

• Application/ImplementationApplication/Implementation PlanPlan

• New EMEA CommitteeNew EMEA Committee (Article 20(1))

• A CAT shall shall be established within the EMEA

• Composition Composition (Recital 11 and Article 21)

• EU best available expertise on ATMP EU best available expertise on ATMP (gene therapy, cell therapy, tissue engineering, medical devices, pharmacovigilance, ethics)

• 5 CHMP members (co-opted or not), 1/MS and alternates from same MS or, in the case of co-opted CHMP members, identified by CHMP + 22 from MS not represented through CHMP and alternates + 2 representing clinicians and alternates+2 representing patients and alternates

• Tasks Tasks (Article 23)

• Draft opinion on the Q/S/E of each ATMP for final approval by the CHMP

• Advice on ATMP classification

• Advice on any question related to ATMP

• Assist scientifically in the elaboration of any documents related to the objectives of ATMP Regulation

• Scientific expertise and advice for any Community IMI and therapies

• Scientific advice procedures

• InteractionInteraction (Recital 12 and Article 20(3))

• EMEAEMEA should ensure the coordination between the CAT and its other Committees, advisory groups and coordination between the CAT and its other Committees, advisory groups and working parties, working parties, notably the CHMP, the COMP and the SAWP

COMMITTEE FOR ADVANCED THERAPIES COMMITTEE FOR ADVANCED THERAPIES (CAT)(CAT)

Article 8Article 81. The Committee for Medicinal Products for Human Use shall consult the Committee for shall consult the Committee for Advanced TherapiesAdvanced Therapies on any scientific assessment of advanced therapy medicinal products on any scientific assessment of advanced therapy medicinal products necessary to draw up the scientific opinionsdraw up the scientific opinions referred to in Article 5(2) and (3) of Regulation (EC) No 726/2004[1]. The Committee for Advanced Therapies shall also be consulted in the event of re-re-examination examination of the opinion pursuant to Article 9(2) of Regulation (EC) No 726/2004[2].2. When preparing a draft opinion for final approval by the Committee for Medicinal Products for preparing a draft opinion for final approval by the Committee for Medicinal Products for Human UseHuman Use,, the Committee for Advanced Therapies shall endeavour to reach a scientific consensus. If such consensus cannot be reached, the Committee for Advanced Therapies shall adopt the position of the majority of its members. The draft opinion shall mention the divergent positions and the grounds on which they are based.3. The draft opinion given by the Committee for Advanced Therapies under paragraph 1 shall be sent to the Chairman of the Committee for Medicinal Products for Human Use in a timely manner so as to ensure that the deadline laid down in Article 6(3)[3] or Article 9(2) of Regulation (EC) No 726/2004 can be met.4. Where the scientific opinion on an advanced therapy medicinal product drawn up by the Committee for Medicinal Products for Human Use under Article 5(2) and (3) of Regulation (EC) No 726/2004 is not in accordance not in accordance with the draft opinion of the Committee for Advanced Therapies, the the Committee for Medicinal Products for Human Use shall annex to its opinion a detailed explanation of Committee for Medicinal Products for Human Use shall annex to its opinion a detailed explanation of the scientific grounds for the differencesthe scientific grounds for the differences.5. The Agency shall draw up specific proceduresAgency shall draw up specific procedures for the application of paragraphs 1 to 4.

EVALUATION PROCEDUREEVALUATION PROCEDURE

[1] Article 5(2) and (3) of Regulation (EC) No 726/2004:2. Without prejudice to Article 56 or to other tasks which Community law may confer on it, the Committee for Medicinal Products for Human Use shall be responsible for drawing up the opinion of the Agency on any matter concerning the admissibility of the files submitted in accordance with the any matter concerning the admissibility of the files submitted in accordance with the centralised procedure, the granting, variation, suspension or revocation of an authorisation to place a centralised procedure, the granting, variation, suspension or revocation of an authorisation to place a medicinal product for human use on the market in accordance with the provisions of this Title, and medicinal product for human use on the market in accordance with the provisions of this Title, and pharmacovigilance.pharmacovigilance. 3. At the request of the Executive Director of the Agency or the Commission representative, the Committee for Medicinal Products for Human Use shall also draw up an opinion on any scientific matter concerning the evaluation of medicinal products for human use. The Committee shall take due account of any requests by Member States for an opinion. The Committee shall also formulate an opinion whenever there is disagreement in the evaluation of medicinal products through the mutual recognition procedure. The opinion of the Committee shall be made publicly accessible.

[2] Article 9(2) of Regulation (EC) No 726/2004: Within 15 days after receipt of the opinion referred to in paragraph 1, the applicant may give written notice to the Agency that he wishes to request a re-examination of the opinion.request a re-examination of the opinion. In that case, the applicant shall forward to the Agency the detailed grounds for the request within 60 days after receipt of the opinion.Within 60 days following receipt of the grounds for the request, the said Committee shall re-examine its opinion in accordance with the conditions laid down in the fourth subparagraph of Article 62(1). The reasons for the conclusion reached shall be annexed to the final opinion.

EVALUATION PROCEDUREEVALUATION PROCEDURE (2)

EVALUATION PROCEDURE (3)EVALUATION PROCEDURE (3)

• CHMP shallshall consult the CAT on any scientific assessment of ATPM including re-examination of opinions (Article 8(1))

• CAT prepares/is responsible forprepares/is responsible for a draft opinion on the Q/S/E of each ATMP for final adoption by the CHMP (Recital 10 and Article 8(2))

• CHMP adopts the final opinionfinal opinion by 210 days (Article 8(3))

• EMEA to draw up specific procedures procedures (Article 8(5))

INCENTIVES• Scientific AdviceScientific Advice on the design and conduct of

pharmacovigilance and risk management (Article 16)

– 90% fee reduction for SMEs, 65% for others

• Scientific Recommendation on ATMP classificationATMP classification (Article 17)

• SMEs: CertificationCertification of quality and non-clinical data (Article 18)

• Additional fee reduction for SME or hospitalAdditional fee reduction for SME or hospital proving there is a particular public health interest in the Community (Article 19)

– 50% reduction on Marketing Authorisation fees

– 50% reduction on post-authorisation activities during 1 year

– Applies during transitional period referred to in Article 29

APPLICATIONAPPLICATION

ATMP Regulation will apply from 30/12/200830/12/2008 (Article 30)

– Transitional period for ATMP «legally on the Community market» (Article 29)

• 3 years (SCT and GT) => no later than 30/12/2011

• 4 years (TEP) => no later than 30/12/2012

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Commission has made public its priorities for the

implementation of the ATMP Regulation

The implementation plan has been

developed and agreed with the EMEA

http://ec.europa.eu/enterprise/pharmaceuticals/advtherapies/index.

htm

http://www.emea.europa.eu/htms/human/mes/advancedtherapies.htm

IMPLEMENTATION PLAN • Commission:Commission:

– GCP guidelines (Article 4)– GMP guidelines (Article 5)– Amend Annex I to Directive 2001/83/EC (Article 7)– Traceability guidelines (Article 15(7))– Implementing provisions on certification of quality/non-clinical data

(Article 18)– Appoint CAT patients/clinicians representatives (Article 21(c)(d))

• EMEA:EMEA:– Specific procedures for evaluation (Article 8)– Guidelines on Post-authorisation follow up/Risk management

(Article 14)– Establishment of CAT (Articles 20 and 21)– CAT’s Rules of Procedure (Article 61(8) Regulation (EC) No

726/2004)

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Thank youfor your attention

Any questions ?