Advanced Therapy RegulationHearing, Brussels May 11, 2006

Dr.Andreas ReimannCEO, Mukoviszidose e.V. (German Cystic Fibrosis Association)

Vice-Chairman, ACHSE e.V. (German Alliance for Rare Diseases)Member of the Board of Directors, European Organisation for Rare Diseases

(EURORDIS)

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Statement on potential Conflicts of Interest• Mukoviszidose e.V. (Bonn)

- Financed up to 4% in total by funds provided by approx. 25 pharmaceutical companies

- Mainly financed by private donors not related to health industry

• ACHSE e.V. (Berlin)- Financed mainly (approx.90%) by funds provided by

institutional or corporate donors not related to the health industry

• EURORDIS (Paris)- Financed up to 16% by funds provided by the members

of the EURORDIS Round Table of Companies and 37% provided as an unrestricted grant from the French Muscular Dystrophy (AFM) association. Remainder mainly financed through EC-project grants (31 %) and members (3 %).

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Disclaimer• Unless mentioned specifically, the views

expressed in that presentation represent the author‘s personal opinion and may or may not represent the views of Mukoviszidose e.V., ACHSE or EURORDIS.

• EURORDIS has commented on a previous draft in 2005 and this contribution is included in that presentation, where applicable.

• Neither of the organisations has issued a consensus document on the use of human embryonic stem cells.

• This disclaimer should, however, not be understood in a way that the views expressed in that presentation are reflecting an isolated position only.

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Why patients with rare diseases hope for Advanced Therapies (AT)

• 30 mio citizens of Europe affected by one of the 5-6000 rare diseases

• 80% are of genetic background, most affect children

• For the majority of rare diseases, no cure is available today

• Patients put therefore considerable hope on new therapies including but not limited to ATs

• We realize that nobody can for sure say when and whether these therapies will be helpful for specific diseases

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General positive comments

• Because patients put considerable hope on Advanced Therapies, the scope and content of the EC proposal is very much welcomed

• Central Evaluation and composition of CAT (patient representation, expertise) is welcomed because of the need to evaluate and to monitor both safety and efficacy of ATs

• Incentives for applicants are appropriate• The need for „sufficient flexibility“ is well

recognized

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Critical issues - general

• No support for the commercialisation of unmodified human tissues and cells, therefore donation of tissues and cells must (rather than „should“) be unpaid and voluntary

• It is fine to exclude tailor-made products for single patients from need for registration, however, this must not become an easy way to evade the central registration

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Critical issues – role of CAT

• Who provides the scientific advice to the applicants, the CAT or the CHMP/SAWP or both? (re: art.17.1)

• Can the CAT provide scientific advice to the applicant on its own right or only by request of CHMP?

• What happens if CAT scientific advice on development is not in accordance with CHMP?

• What happens if CAT and CHMP are in disagreement with regard to MA? Is the CAT a scientific body only or will it have the opportunity to appeal to the EC? (re: art.9.4)

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Critical issues – traceability and post-authorisation follow-up• Long-term follow up of both safety and efficacy

must be mandatory to establish a „learning system“. Provisions needs to be in place to ensure so.

• Therefore, relevant data (clinical trials, observational studies, registries, spontaneous reporting) on PM-surveillance (safety and efficacy) must be reported systematically to the EMEA, where a public risk management system needs to be established.

• Traceability to the end-user must be established independent of the MA-holder.

• Art.16 should therefore be amended accordingly

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• 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.

• 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.

Central Data Base

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Points to consider

• Concept of rec.6 and art.28 is welcome, i.e. central evaluation but „opt-out“ option for MS if there exists a conflict with their national legislation or policy in using certain types of cells (e.g. human embryonic stem cells)

• That principle must be legally „water-tight“

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Points to consider (cont‘)

• However, there is no need to include animal cells in that exceptional provision for ethical reasons

• The regulation should specifically exclude the evaluation and marketing-authorisation of techniques using genetic manipulation of germ-cells, reproductive cloning or human/animal chimaeras

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German Cystic Fibrosis Associationwww.muko.info

European Organisation for Rare Diseaseswww.eurordis.org

German Rare Disease Alliance:Allianz Chronischer Seltener Erkrankungenwww.achse-online.de