The Advanced Therapy Medicinal Products Regulation from the European Medicines Agency (EMEA)'s perspective

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The Advanced Therapy Medicinal Products
Regulation from the European Medicines Agency
(EMEA)'s perspective

• Nathalie Rampal Olmedo
• EMEA Directorate Legal Sector
• British Institute of International and
  Comparative Law
• 25th September 2008

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ADVANCED THERAPY MEDICINAL PRODUCTS (ATMP)

• 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

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REGULATION (EC) No 1394/2007
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KEY ELEMENTS

• 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)

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• CAT
• Evaluation Procedure
• Incentives
• Application/Implementation Plan

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COMMITTEE FOR ADVANCED THERAPIES (CAT)

• 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
  alternates2 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

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EVALUATION PROCEDURE
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 726/20041. The
Committee for Advanced Therapies shall also be
consulted in the event of re-examination of the
opinion pursuant to Article 9(2) of Regulation
(EC) No 726/20042. 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.
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EVALUATION PROCEDURE (2)
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 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. 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.
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EVALUATION PROCEDURE (3)

• 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))

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INCENTIVES

• 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

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APPLICATION

• 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) gt no later than 30/12/2011
• 4 years (TEP) gt no later than 30/12/2012

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30th December 2008 !
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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/pha
rmaceuticals/advtherapies/index.htm http//www.em
ea.europa.eu/htms/human/mes/advancedtherapies.htm
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IMPLEMENTATION PLAN

• 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
• Specific procedures for evaluation (Article 8)
• Guidelines on Post-authorisation follow up/Risk
  management (Article 14)
• Establishment of CAT (Articles 20 and 21)
• CATs Rules of Procedure (Article 61(8)
  Regulation (EC) No 726/2004)

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Thank you for your attention
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