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Advanced Therapies

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The proposed EU regulation from the viewpoint of a ... Organogenesis. formed. 1995. 1998. 2000. 2003. VSU-001. DUS-001. FDA. Approval. Venous Leg. Ulcers ... – PowerPoint PPT presentation

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Title: Advanced Therapies


1
Advanced Therapies
  • The proposed EU regulation from the viewpoint of
    a Pharmaceutical company with experience in the
    field of Tissue Engineering
  • Priv.Doz.Dr,med.Detlef Niese
  • Novartis Pharma AG, Basel CH
  • European Biopharmaceutical Enterprises (EBE)
  • Member of EuropaBio Advanced Therapies Working
    Group

2
About EBE
  • EBE European Biopharmaceutical Enterprises is a
    specialised group of EFPIA created in 2000
  • EBE has 65 corporate members engaged in research
    in Europe involving the application of emerging
    bioscience technologies with the aim of launching
    new healthcare products

3
Advanced Therapy Products and Pharmaceutical
Companies
  • Academic institutions, small biotech companies
    and some large pharmaceutical companies have been
    involved and are still involved in research and
    development of cell therapy, tissue engineering
    and gene therapy products.
  • Regulatory uncertainty and development risks
    associated with these products prove to be
    significant hurdles
  • As a pharmaceutical company, Novartis has
    gathered development experience in all sectors of
    advanced therapies

4
A Real Life Case Apligraf

What it looks like
Histology Apligraf
Normal Skin
5
Apligraf A Long Clinical Regulatory Path
VSU-001
Organogenesis formed
DUS-001
1979
1985
1995
1998
2000
2003
2004
1992
1996
E. Bell M.I.T.
FDA Approval Diabetic Foot Ulcers
FDA Designated as device class III interactive
wound dressing
FDA Approval Venous Leg Ulcers
I.D.E. submitted
6
A Diverse Regulatory Environment
Eventual Regulatory Classification
Initial Regulatory Classifications
Apligraf
Regulatory
Apligraf
Other
Reg
Device
Medicinal
Unregulated
Device
Medicinal
Unregulated
Germany
Germany
France (Cell
UK
Canada
France (Cell
UK
Canada
Therapy)
Therapy)
Sweden
Italy
Sweden
Italy
USA
USA
Belgium(Tissue
Belgium(Tissue
Denmark
Ireland
Denmark
Ireland
Finland
Finland
Bank)
Bank)
Norway
Austria
Norway
Austria
Greece
Greece
Spain
Spain
Netherlands
Netherlands
Portugal
Switzerland
Portugal
Switzerland
(Transplant)
(Transplant)
7
Lessons Learned
  • A harmonized regulatory framework for all cell
    based products in Europe is urgently needed to
    address
  • Quality assurance
  • Development and Marketing authorisation
  • Address unique safety efficacy issues
  • Include efficacy assessments based on trial
    designs that address ethical and practical
    aspects of surgical research
  • Benefits of harmonised regulations
  • Protection of patient safety
  • Encourage investment by industry and successful
    global commercialisation through level playing
    field
  • Provide patients access to innovative valuable
    therapies

8
How We See the Proposed Regulation What We
Welcome
  • A single, harmonised regulatory framework for all
    advanced therapy products
  • Use of the Centralised Procedure through EMEA
    ensuring harmonised efficacy, quality and safety
    requirements for all products
  • The creation of a Committee for Advanced
    Therapies consisting of experts in the field
  • The use of subsidiarity for ethical aspects such
    as embryonic stem cells

9
How We See the Proposed Regulation Our Concerns
  • To ensure safety and effectiveness of AT products
    the regulation should apply to all products
    produced following a standardised process (no
    exemptions)
  • Instead of exemptions from the regulation,
    consider providing administrative support or
    regulatory expertise as an additional incentive
    for all manufacturers who may require it

10
How We See the Proposed Regulation Our Concerns
  • The formation of a specialised committee (CAT) at
    EMEA is welcomed but
  • Expertise of members should reflect the broad
    spectrum of products and technologies
  • Expertise of members should prevail over national
    representation (which is provided by CHMP)
  • Existing legislation needs adaptation to (e.g.
    2001/20, GMP)

11
Conclusion
  • The new regulation is principally welcome
  • Assurance of safety and efficacy through review
    by regulatory authorities supported by an
    expertise based CAT should drive development of
    high quality products and support access for
    patients
  • Instead of excluding specific products from the
    regulation, administrative hurdles for hospital
    based products or SMEs should be minimised and
    may require additional incentives
  • Careful adaptation of all affected existing
    legislation (like Directive 2001/20 EC) is a
    crucial success factor
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