Genes, Cells and Tissues:

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European Commission
Enterprise and IndustryDirectorate-General
BIO 2006 April 9-12, 2006
Genes, Cells and Tissues Commission
proposalon Advanced Therapies
Georgette LALISDirector, European CommissionDG
Enterprise Industrygeorgette.lalis_at_cec.eu.int
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Agenda

• Background and Objectives
• Overview of the proposal
• Next steps, Timing and Conclusions

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Agenda

• Background and Objectives
• Overview of the proposal
• Next steps, Timing and Conclusions

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Advanced therapy products What do we mean?

• Products based on
• Genes gene therapy
• Cells cell therapy
• Tissues tissue engineering

Advanced Therapies
Chemicals(e.g. aspirin)
Biotech(e.g. insulin)
Gene Therapy
Cell Therapy
Tissue Engineering
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Benefits for patients

• Promising new treatment opportunities
• (Tissue engineering)
• Skin replacement in burn victims
• Cartilage defects
• Reconstruction of blood vessels, cardiac tissue
• Long term organ regeneration?
• (Cell Therapy)
• Cancer
• Parkinson
• Alzheimer
• (Gene Therapy)
• Repair of genetic disorders

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Example tissue engineering in Europe
(80 are SMEs)
Ref. Joint Research Centre, European
Commission, 2003 (see also impact assessment of
the proposal)
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The current regulatory gap
Legislation
Medical Devices 93/42/EEC
Medicinal Products Directive 2001/83/EC

?
Advanced Therapies
Medical Devices
Chemicals
Biotech
Gene Therapy
Cell Therapy
Tissue Engineering
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Todays picture
autologous products allogeneic
products
Ref. Joint Research Centre, European
Commission, 2003 (see also impact assessment of
the proposal)
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Why do we need legislation?

• Different approaches across Europe
• hinder patients access to treatments
• lead to market segmentation
• undermine industry development
• Public consultations (2002, 2004, 2005) All
  stakeholders want clear EU rules
• gtNeed for a specific, harmonised and coherent EU
  regulatory framework

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Policy objectives

• Proposal adopted on 16.11.2005
• Guarantee a high level of health protection
• Harmonise and facilitate market access
• Foster competitiveness
• Provide overall legal certainty

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Agenda

• Background and Objectives
• Overview of the proposal
• Next steps, Timing and Conclusions

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The proposed approach 3 levels
Existing Legislation on Tissues Cells,Medical
Devices and Medicines
Co-decisionCommissionEuropean ParliamentCouncil
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Proposal for aRegulation on Advanced Therapies
gt Do not reinvent the wheel
Technical Requirements
gt Focus on specificities
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Comitology Commission
Guidelines
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GuidanceCommission/EMEA
gt Provide flexibility
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Overview of the proposal 6 pillars

• Definitions and Scope
• Key principles of the proposal
• Evaluation procedure and Committee
• Technical requirements
• Competitiveness aspects
• Final provisions

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Definitions and Scope

• Advanced therapy medicinal products
• Gene therapy
• Somatic cell therapy
• Tissue engineered products
• Combined product incorporate medical device(s)
• The definition of tissue engineering does not
  cover non-substantially manipulated cells/tissues
  for homologous transplantation(e.g. skin to
  replace skin)

Ref. Article 2 and Annex I of the proposal
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Ethical aspects

• The proposal does not affect the application of
  national legislation prohibiting or restricting
  the use of any specific type of human or animal
  cells, or the sale, supply or use of medicinal
  products containing, consisting of or derived
  from these cells.

Ref. Article 28 of the proposal and Art. 2(1)
of Dir. 2001/83/EC
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Key principles of the proposal

• For products within the scope
• No marketing without prior authorisation
• Demonstration of Quality, Safety Efficacy
• Post-authorisation vigilance
• Centralised procedure for marketing
  authorisation
• pooling of Community expertise
• uniform and direct access to market
• harmonised requirements evaluation

Ref. Articles 27-28 of the proposal
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EMEA Committees
COMP
HMPC
PC
CAT
OrphanMedicinal Products
HerbalMedicinal Products
Medicinal Productsfor PaediatricUse
Committee for Advanced Therapies
CHMP
Medicinal Products for Human Use
EMEAopinion
Ref. Articles 20-23 of the proposal
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Technical Requirements

• Pre-authorisation requirements
• Specific pre-clinical and clinical requirements
• Compliance with Essential Requirements for
  products incorporating medical devices
• Specific guidelines on
• GMP (Good Manufacturing Practice)
• GCP (Good Clinical Practice)
• Specific rules for labelling/packaging
• Post-authorisation requirements
• Risk management
• Traceability

Ref. Articles 3-7, 11-16 of the proposal
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Competitiveness AspectsGeneral provisions

• Stemming from existing legislation
• Direct access to Community market
• Harmonised data protection
• Orphan status linked to market exclusivity
• Accelerated (fast-track) assessment
• Conditional marketing authorisation
• New in the proposal
• Scientific Advice (90 fee reduction)
• Scientific recommendation on advanced therapy
  classification

Ref. Articles 17-18 of the proposal
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Competitiveness AspectsSpecific provisions for
SMEs

• Existing legislation Reg. (EC) No 2049/2005
• Fee reductions and deferrals
• Handling of translations
• General administrative assistance(EMEA SME
  Office)
• Workshops/Training sessions
• New in the proposal Certification of quality
  and non-clinical data

Ref. Article 19 of the proposal
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Agenda

• Background and Objectives
• Overview of the proposal
• Next steps, Timing and Conclusions

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Timing Next steps

• Regulation to be adopted by Council/European
  Parliament
• Currently in discussion adoption by 2007 ?
• No transposition delay
• Technical requirements should be ready shortly
  after adoption of the Regulation
• More information http//pharmacos.eudra.org/F2/ad
  vtherapies/index.htm

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