EuropaBio Industry Hearing on Tissue Engineering and Advanced Therapies

1
EuropaBio Industry Hearing on Tissue Engineering
and Advanced Therapies

• Comments from Patients
• Fabrizia BIGNAMI
• Eurordis Therapeutic Development Officer
• Brussels
• 9 November 2005

2
Eurordis and the new EC proposal on advanced
therapies

• As the organisation representing 30 million EU
  patients affected by rare diseases, we have
  always placed considerable hope on the
  advancement of genomic research and on advanced
  therapies. (5 years after the OMP Regulation,
  only less than 10 OMP over the 337 OMP
  designations are advance therapy products)
• EURORDIS and its members have welcomed the
  Commissions proposal as an appreciated
  recognition of the status of advanced therapies.
  (Eurordis written contribution sent to the EC on
  June 2005)
• Few well-identified patient groups in the EU are
  at the forefront of innovation in this field (the
  AFM -French Muscular Dystrophy Association and
  the Italian Telethon, for ex.).

3
EURORDIS supports most of the proposed provisions
of the future regulatory framework for advanced
therapies and in particular

• The compulsory centralised Community marketing
  authorisation, with the intervention of a
  specialised Committee for Advanced Therapies
  (CAT),
• The recognised need for sufficient flexibility,
  
• The recognition of the principles underlying the
  donation of human tissue and cells.
• .But we insist that the nature of this donation,
  must be - rather than should be- voluntary
  and unpaid.

4
Some reservations

• We totally agree that appropriate and long-term
  patient follow-up and post-authorisation
  monitoring are crucial aspects of these products,
• However we do not agree that the sponsor should
  be directly in charge of these aspects.
• We do agree that the data collected during any
  clinical study performed after the marketing
  authorisation have to be reported systematically
  by the sponsor, but out of the context of any
  clinical study, we would prefer a Centralised and
  Public Risk Management System to perform these
  tasks.

5
Some reservations

• Why should the applicant be given the role of
  managing a system allowing complete traceability
  of the patient? We consider this as being
• A threat to patients privacy
• An interference with the role of health care
  professionals
• And a harm for the complete independence of the
  data collection
• It is a general Public Health Issue which should
  be treated at the MS and EU level and not left to
  the single sponsors initiative.

6
Some reservations

• Proposal for a EU centralised and highly
  specialised structure, (or network) for Risk
  Management. Already some examples of national
  initiatives- France (RNPV), the Netherlands.
• Because of its skills, and of its harmonised
  procedures, this structure would allow for a
  better follow-up of these products and the
  constitution of a precious public registry on
  safety and efficacy of advanced therapies
  products.
• Any new compulsory Risk Management system,
  associated to a MA, while financed by the sponsor
  - at least until the 5-year MA renewal could be
  later continued through public funds and managed
  at European level. (Amgens G-CSF for ex.)
• This long term follow-up and data collection
  would certainly benefit the development of the
  whole sector and would enhance public support and
  confidence towards advanced therapies.