ADVANCED THERAPY MEDICINAL

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ADVANCED THERAPY MEDICINAL

• Draft for Regulation COM(2005) 567 final

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Advanced Therapies

• Gene therapy
• Somatic cell therapy
• Tissue engineered product
• Modification of directive 2001/83/EC on
  medidicinal regulation 726/2004 on centralised
  procedure for marketing autorisation of
  medicines on role of European Medicines Agency

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Authorisation

• tissue engineered products are submitted to the
  centralised european market autorisation for
  medicines
• EMEA have to be consulted
• Committee for advanced therapies to be set up
• MD advanced therapy product EMEA consultation
  for single scientific evaluation of quality,
  safety, efficacy

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Definition product from tissue engineering

• contains or consists of engineered cells or
  tissues and
• is presented as having properties for, or is
  used in or administered to human beings with a
  view to regenerating,repairing or replacing a
  human tissue
• may contains cells or tissues of human or
  animal origin or both , viable or not, cellular
  products, bio-molécules, biomaterials , chemical
  substances,scaffolds , matrices

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Clinical Trials

• directive 2001/20/EC is applicable

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DM Advanced Therapy

• Submitted to essential requirements laid down in
  annex I of directives 93/42/EEC or 90/385/EEC
• Market Autorisation application
• Describing physical characteristics
• functioning
• design methods
• Consultation of Committee for advanced therapies
• EMEA take into account the assessment of NBs

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Summary of product characteristics

• Name , composition
• pharmaceutical form
• clinical information therapeutic
  indications,instructions, contra-indications,
  interaction , undesirable effects, overdose,
  pregnancy, lactation, ability to drive
• pharmacodynamic and kynetic properties
• Incompatibilities (preclinical data)
• Informations conservation, excipients,incompatib
  ilities, stability, stockage, container and
  delivery device,handling and elimination
• Holder, MA N, 1st MA date, revision date

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Other requirements

• Labelling (externe ou primaire, patient
  identification for autologous)
• Pharmacovigilance / risk management after MA
• traceabilityraw and starting materials,substances
  into contact, from sourcing gt use (including
  manufacturing,packaging, transport,delivery)
• Data kept 30 years(transfered to the agency in
  case of bankrupt)
• Reduced fees for SME
• Transitory period 2 years