Advanced Therapies Regulation in European Union: Role of EMEA

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Advanced Therapies Regulation in European
UnionRole of EMEA

• BIO 2006 Chicago
• 11th April 2006
• John Purves
• European Medicines Agency (EMEA)

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Content

• Requirements for authorisation of cell therapy
  medicinal products in Europe
• Advanced Therapy Regulation Role of EMEA
• Assistance to applicants sponsors

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Content

• Requirements for authorisation of cell therapy
  medicinal products in Europe
• Advanced Therapy Regulation Role of EMEA
• Assistance to applicants sponsors

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Requirements for cell therapy medicinal products

• A proposal for a Regulation on Advanced Therapies
  is currently under discussion
• Gene and Cell Therapy medicinal product
• Tissue Engineered Products
• Gene and cell therapy medicinal products already
  covered by pharmaceutical legislation
• Annex I (Part IV) to Directive 2001/83/EC
  describes technical requirements

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Requirements for cell therapy medicinal products

• Guidance is already in place
• Note for Guidance on the Quality, Preclinical and
  Clinical Aspects of Gene Transfer Medicinal
  Products
• Point to Consider on Human Somatic Cell Therapy
  (Quality only)
• Point to Consider on Xenogeneic Cell Therapy
  Medicinal Products (Quality, Non-clinical and
  clinical aspects)
• All documents available on EMEA Website
  http//www.emea.eu.int

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Content

• Requirements for authorisation of cell therapy
  medicinal products in Europe
• Advanced Therapy Regulation Role of EMEA
• Assistance to applicants sponsors

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Advanced Therapies Regulation Role of EMEA
Nov 2005 Adv. Therapy Proposal
Mid 2007 Adoption Adv. Therapy Regulation
2006
2007
2008
Development of guidelines
EMEA activities
Technical requirements
Setting up CAT
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Advanced Therapies Regulation Role of EMEA

• Development of new guidelines for cell-based
  products
• For cell therapy medicinal products and TEP
• Quality, non-clinical and clinical aspects and
  Pharmacovigilance (traceability)
• Concept paper published in January
  2006http//www.emea.eu.int/pdfs/human/cpwp/323774
  05en.pdf
• Proposed timeframe for releasing guideline for
  consultation end 2006

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Guidance on cell based products
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Advanced Therapies Regulation Role of EMEA

• Development of technical requirements
• Quality, non-clinical and clinical requirement
  for tissue engineered products
• Requirements for cells / tissues
• Requirements for devices (combination products)
• Cell-device interactions
• Will become part of Annex I of Directive
  2001/83/EC (pharma legislation)

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Advanced Therapies Regulation Role of EMEA

• Setting up the Committee for Advanced Therapies
  (CAT)
• Identify experts with additional expertise (e.g.
  device / matrix development, cell-matrix
  interactions)
• Develop mandate, work-plan, procedures e.g.
  interaction CAT-CHMP
• Appointment of CAT members
• Once establish EMEA provide scientific
  secretariat to CAT

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Content

• Requirements for authorisation of cell therapy
  medicinal products in Europe
• Advanced Therapy Regulation Role of EMEA
• Assistance to applicants sponsors

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Assistance to applicants and sponsors

• Scientific advice
• EU-wide advice
• Available for all medicinal products (including,
  in the future, for TEPs)
• On development quality aspect, non-clinical
  testing, clinical trials and post-marketing
  issues
• Written procedure (normally 40 days, extended to
  70 days if hearing)

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Assistance to applicants and sponsors

• Orphan drug designation
• For rare disease (less than 5/10,000)
• Specific legislation (Regulation (EC) 141/2000)
  and separate scientific Committee COMP
• Incentives to stimulate Orphan drug development
• Market exclusivity, protocol assistance, direct
  access to centralised procedure and fee
  reductions

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Assistance to applicants and sponsors

• Regulatory advice
• Briefing meetings with EMEA Innovation Task Force
• Forum for early dialogue with applicants
• Advice on the classification of medicinal
  products prior to submission for scientific
  advice, orphan medicinal product designation or
  marketing authorisation procedures
• Formal regulatory advice

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Assistance to applicants and sponsors

• Initiatives for Small Medium-sized enterprises
  (SME)
• Commission Regulation (EC) No 2049/2005
  promoting innovation and the development of new
  medicinal products by SMEs
• SME Office established at EMEA
• Incentives
• Administrative and procedural assistance
• Fee reductions, fee deferral (of the fee payable
  for a MA application)
• Assistance with translations

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How to contact EMEA

• General queries, Request for briefing meetings or
  Request for regulatory Classification
• http//www.emea.eu.int/htms/human/itf/itfintro.ht
  m
• SME Office
• http//www.emea.eu.int/SME/SMEoverview.htm
• EMEA Scientific advice procedure
• http//www.emea.eu.int/htms/human/sciadvice/Scien
  tific.htm
• EMEA Orphan drug designation
• http//www.emea.eu.int/htms/human/comp/orphapp.ht
  m

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Thank you for your attention !

• For your information
• EMEA Website http//www.emea.eu.int
• John.Purves_at_emea.eu.int
• Direct line 44 (0) 20 7418 8402