The Advanced Therapies Regulation from the EMEA`s perspective Download

The Advanced Therapy
Medicinal Products
Regulation from the
European Medicines
Agency (EMEA)'s
Nathalie Rampal Olmedo
EMEA – Directorate – Legal Sector
British Institute of International and Comparative Law
25th September 2008
• Medicinal products based on
– genes (gene therapy)
– cells (cell therapy)
– tissues (tissue engineering)
• Revolutionary treatments of a number of diseases or
injuries, such as skin in burn victims, Alzheimer, cancer
or muscular dystrophy
• Huge potential for patients and industry
REGULATION (EC) No 1394/2007
• Centralized marketing authorization (Single scientific evaluation)
• New Committee for Advanced Therapies (CAT)
• Specific rules on authorisation, supervision and pharmacovigilance
• Specific technical requirements (SPC, labelling and package leaflet)
• Combined ATMP evaluated by EMEA
• Post-authorisation requirements (follow-up of efficacy/adverse
reactions, risk management, traceability)
• Special incentives (SMEs)
• Evaluation Procedure
• Incentives
• Application/Implementation Plan
New EMEA Committee (Article 20(1))
A CAT shall be established within the EMEA
Composition (Recital 11 and Article 21)
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 (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
Interaction (Recital 12 and Article 20(3))
EMEA should ensure the coordination between the CAT and its other Committees, advisory groups and
working parties, notably the CHMP, the COMP and the SAWP
Article 8
1. The Committee for Medicinal Products for Human Use shall consult the Committee for
Advanced Therapies on any scientific assessment of advanced therapy medicinal products
necessary to draw 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 reexamination 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
Human 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 with the draft opinion of the Committee for Advanced Therapies, the
Committee for Medicinal Products for Human Use shall annex to its opinion a detailed explanation of
the scientific grounds for the differences.
5. The Agency shall draw up specific procedures for the application of paragraphs 1 to 4.
[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
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
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. 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.
• CHMP shall consult the CAT on any scientific assessment of
ATPM including re-examination of opinions (Article 8(1))
• CAT prepares/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 opinion by 210 days (Article 8(3))
• EMEA to draw up specific procedures (Article 8(5))
• Scientific 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 classification
(Article 17)
• SMEs: Certification of quality and non-clinical data
(Article 18)
• Additional 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
ATMP Regulation will apply from 30/12/2008 (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
Commission has
made public its
priorities for the
implementation of the
ATMP Regulation
The implementation
plan has been
developed and agreed
with the EMEA
• 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))
– 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
Thank you
for your attention
Any questions ?