New CEO for Swiss MS Society

Lay summary of findings from the MRC Cannabis in MS Trial (CAMS)
Published in the Lancet 7 November 2003

Marinol is now available for MS patients in Denmark (October 2003)

On 21 February, the European Commission published a press release, in which it proposed 1 June 2004 as the launch date of the common European health insurance card :

Medical information is also available on following sites :




Main challenges and priorities of European Health Policy

On 30 October Commissioner David Byrne gave a speech at the European Policy Centre in Brussels, in which he addressed main challenges and priorities of European health policy (cross-border health rights, EU enlargement, pharmaceutical package, future of research, etc.).

The speech is available at the following website:

News Release for Immediate Use – 12 March 2003


A proposal for a European Union law which for the first time prohibits discrimination against disabled people in housing, education, transport and all in all its other forms, will be unveiled at the European Parliament in Strasbourg today (Wednesday 12 March 2003).

The proposal for a new directive, which will be officially unveiled today by the President of the European Disability Forum, Yannis Vardakastanis and the President of the Disability Intergroup of the European Parliament, Richard Howitt MEP, has been drawn up over a two year period by the representatives of the European disability movement and human rights lawyers, currently campaigning to win political support for its agreement during the current European Year of People with Disabilities 2003.

At present, in most EU countries legislation relating to disabled people is contained in provisions that form part of a broader law, for example on transport, education or social security.

The new piece of legislation, the first of its kind at Community level, will radically change the lives of disabled people and their families in the European Union, since it will render discrimination of disabled people illegal in all areas of life. The Disability specific directive will cover, besides equal treatment at the workplace, social protection, healthcare, social advantages, education, access to goods, facilities and services, disability portrayal in the media, design for all products. It will also introduce enforcement procedures to ensure that disabled people can effectively exercise their rights in all EU Member States, including the right to go to court.

“Disabled people want legislation that eliminates existing barriers in society and prevents new ones. The legal base for such a Directive exists. Disabled people and EDF, as the representative voice of 50 million disabled people in Europe, call upon the EU institutions to commit to this draft EU Directive this year, 2003, the European Year of People with Disabilities.”, said Yannis Vardakastanis, President of the European Disability Forum.

Richard Howitt MEP, President of the Disability Intergroup of the European Parliament which has played a crucial role in providing the necessary political support for the promotion of the new directive said: “After the adoption of the EU Race Discrimination Directive in 1997, there was a promise that other forms of discrimination would not be forgotten, and the European Year of Disabled People in 2003 marks the time to honour that promise. Disability legislation in many European countries dates from the time of aid to the veterans of the First World War. It is now time to update the law to reflect a modern human rights approach”.

The press conference will be lead by:

Yannis Vardakastanis, President of the European Disability Forum

Richard Howitt MEP, President of the Disability Intergroup of the European Parliament

March 12th 2003, at 15H30 PM
LOW N -1 Room 201

Should you wish to obtain further information, please contact Mr Richard Howitt MEP, President of the Disability Intergroup- Mobile: + 44 77 68 12 28 88; e-mail: or Helena González-Sancho, EDF Communication Officer – Tel: + 32 2 282 46 04; e-mail:

Human tissue and cell engineering
(Commission’s Consultation paper)


In June 2000, the Council adopted a Directive concerning medical devices incorporating derivatives of human blood and plasma (1). This modifies Directive 93/42. At that time, the Council and Commission agreed that devices incorporating other derivatives of human tissues should be subject to a specific directive.

The field of tissue engineering has evolved significantly since then and as a first step it now seems appropriate to create a regulatory framework for this whole area.

Tissue engineering is a new and rapidly developing technology, the aim of which is to produce viable substitutes that restore, maintain or improve the function of human tissues or organs. It differs from standard therapies because the engineered products become integrated within the patient, affording a potentially permanent and specific cure of the disease, injury or impairment. An interdisciplinary field, it combines the application of principles of biosciences and engineering.

In July 2002 the Commission launched a public consultation about the “Need for a legislative framework for human tissue engineering and tissue-engineered products” so as to complement current rules in relation to medicinal products (2), medical devices and donation and distribution of human tissues and cells (3).


The Commission received fifty-one contributions. Many of them and in particular the ones of the institutional bodies or industrial associations, are the results of wider consultation.

The participants can be subdivided into groups of about the same size but of slightly different opinions: the governmental/institutional officials, the industry and the researchers/experts.

Responses from the governmental/institutional officials to the consultation document were received from nine Member States, one European institution, one intergovernmental organisation and one international regulatory agency.

Industry, in the form of individual companies or industry associations sent eighteen contributions. Ten of the company responses came from what are SMEs. Three were larger companies having already activities in the pharmaceutics area. Other contributions came from: three European Industry Associations, a European association of medical doctors and one national industrial association.

Twenty-one contributions from researchers/experts were received. They came from 12 research institutions, 6 lawyers, 3 from individual professionals (doctors, pharmacist etc.)

All of the contributions received provide valuable background information for the Commission’s further action in this field.

Summary of responses received to the Commission’s consultation paper: “Human tissue and cell engineering products”.

I. Need for specific legislation

Is the suggestion for a new specific legal framework, different from the medical devices and pharmaceutical products regulatory systems the preferred option? Should there be a differentiated approach between different kinds of products?

Responses from Governmental / Institutional officials

Seven MSs and a candidate country were positive about a new specific framework but with the caveat that great care was needed in defining tissue engineering products. Some doubted whether they could be adequately defined (see next question). The suggested fallback for those with reservations was to use the legal framework for medicinal products.

Two Member States declared to favour the use of the existing framework for human medicinal products for tissue engineered products.

The EMEA (CPMP) and the FDA (US) both felt that the existing framework for medicinal products should be used, supplemented as necessary by the framework for medical devices.

Responses from Industry

There is a clear consensus of industry for a new legal framework devoted to tissue engineered products. However there was no consensus on its precise nature. One European Industry Association supports a graded approach depending on the perceived level of risk to the recipient. The same position is reflected in the individual contribution of its participants.
Another European Industries group supports a new specific flexible framework which should depend on the mode of action of the product, and a graded approach to risk assessment /management.
One national association supported a uniform European-wide regulatory process covering all biologics (including plasma-derived medicinal products, vaccines such as cell-based cancer vaccines and cell therapy medicinal products). Individual companies that reacted also hold this view.

There were outlying views from individual companies. One company favours a specific legal framework or its integration into the medical device legislation whereas another favours adapting the medicinal product legislation to cover tissue engineering.

Yet another European Industry Association argues that ‘gene and cell therapy products and vaccines (current and future, such as cancer vaccines and vaccines administered using sophisticated medical devices), including xenogenic cells shall be excluded from any new directive.’

Responses from experts, institutes, consultants, individuals

Most of the experts, institutes, consultants and others that reacted on an individual basis take the view that there is a need for specific legislation for tissue engineered products. It is felt that neither the medicinal products nor the medical device legislation covers this new field adequately. It is agreed that the goal of a new directive should be to assure safety, quality, and efficacy with a risk benefit assessment within a light and adaptable legal framework.
Some respondents recommended using the existing framework to avoid the development of a third framework that diverged from the two existing ones (medical devices, medicinal products) and any resulting confusion.

Respondents recommended a differentiated approach for different kind of products, depending on the perception of risk.

II. Definition and Scope
Is the idea to cover “human BioOrgans, tissues and cells, autologous and allogeneic, both nonviable and viable, and including combined tissue/non-tissue type products that have been substantially modified by treatments, and that do not exert their effect through metabolic, pharmacological or immunological means” an acceptable basis for a legislative scheme?
Responses from Governmental / Institutional officials

Only one respondent found the definition acceptable. The other respondents had slightly different reasons for disagreeing. Some noted the difficulties with including the criterion of absence of metabolic, pharmacological or immunological effect, as this may be difficult to discern. In addition there are different views regarding the scope of any new framework. Some noted that the definition given overlaps with the definition of medicinal products. Also the proposed definition would lead to very similar products being handled by separate legislation. There was concern about the ability to define what is a rapidly evolving field. One institution suggested that human tissue engineered products represent a subset of medicinal products.

Responses from Industry

One European Industry Association organisation believes that a tissue-engineered product should be defined by its primary mode of action ie repair, replacement or regeneration of human tissue or function. It was noted that such products might have “metabolic, pharmacological or immunological” effects but that these were not the primary mode of action. In addition, the “degree of manipulation” and not “substantial modification” should be a defining factor.
Another European Industry Association suggests adding “principally/primarily” before the “metabolic, pharmacological or immunological means”.

Responses from experts, institutes, consultants, individuals

The majority proposes that tissue engineering should be clearly defined and subsequently the definitions of medical devices or medicinal products be modified accordingly.
A few propose revising the definitions in existing directives without qualifying precisely how, and a few offer as a definition “all products having a principal intended action on the human body other than metabolic, pharmacological or immunological effects.”
* * *

Would there be a need, in case a specific legal framework has to be set up, to reconsider the scope of existing legal provisions (for Medical devices, Medicinal products or others)?

Responses from Governmental / Institutional officials

Those who want a new framework agree that the scope of existing provisions may need to be changed. The extent of change would depend on the scope of the new framework. Medical devices legislation is considered less likely to need changing than legislation on medicinal products.

Responses from Industry

Most of the answers suggest revisiting the definition of cell therapy medicinal products laid down in Annex I to Directive 2001/83/EC, for one of two reasons:
· (i) these products are close (or even identical) to human tissue engineered products and should be covered by the same new legislation
· or (ii) the definition of cell therapy medicinal products is too broad and overlaps with human tissue engineered products.

Responses from experts, institutes, consultants, individuals

In the event that a new tissue engineered materials legal framework is established, respondents stated that there would be a need to change the scope and use of the medicinal products and medical devices legislation. There was a view that all cell-based and cell-derived therapy should be removed from the medicinal products legislation, irrespective of mode of action. Similarly non-human, non-viable cell materials could be removed from the scope of the Medical Devices Directive where they fulfilled the new definition of tissue engineered products.
This group also wished to modify the definition of gene therapy medicinal products as given in the medicinal product legislation.

* * *

How should borderlines be defined, for instance regarding cell therapy or stem cells? Would the fact that cells have a metabolic pharmacological or immunological effect be the only criteria relevant for legislative purposes?

Responses from Governmental / Institutional officials

There is one view that one cannot differentiate between stem cells and cell therapy and another that one must. There is concern that without care similar products might be handled through different legislation.
Stem cells are recognised/acknowledged to be a very difficult topic and there is a plea to keep ethics out of any new directive. Several used the difficulties in defining borderlines to underline the need to reflect on keeping all within medicinal products legislation. Final engineered construct and therapeutic intent is what should determine definition, not the source of the cells.

Responses from Industry

Some consider that the main purpose of human tissue engineered products is to restore previously existing function without displaying a pharmacological effect. In addition to “primary mode of action” and “degree of manipulation”, some would add two other elements; “integration in the human body” and “physiological/systemic effect” to help define the borderlines with other products such as medicinal products.

Some consider that all new therapy products should be covered under the same umbrella to tackle the inherent diversity. Stem cells, where they are treated or manipulated are regarded as medicinal products (cell therapy) rather than transplantation.

Responses from experts, institutes, consultants, individuals

It is generally accepted that “substantially modified tissue-engineered products that do not exert their principal intended action through metabolic, pharmacological or immunological means” seems in principle sufficient to define the borderline between them on the one hand and medicinal products like somatic cell therapies on the other.
* * *

Should xenogenic organs, tissues and cells be partly covered in the directive, why and how?

Responses from Governmental / Institutional officials

About half replied yes and half no. Those against thought they should be subject to a separate Directive to avoid adding additional complexity to an already complex subject. Those who were for said that there may need to be some additional requirements for xenogenic cells but that otherwise the same issues (especially with respect to risk) were involved. Others felt they were already covered by the medicinal products legislation.

Responses from Industry

Two European industry associations and several individual companies thought that xenogenic organs, tissues and cells should only be covered by a new framework if used as ancillary elements in the manufacture of human tissue engineered products. They reasoned that xenogenic non-viable tissues are already covered by medical device legislation. However, a number of individual companies (8) felt that there should not separate regulatory frameworks for xenogenic compared with non-xenogenic products.

Responses from experts, institutes, consultants, individuals

There is no convergent opinion. Some respondents felt that we are too far from the reality of xenogenic products to contemplate regulation. Others felt that they should be incorporated within the same regulatory framework. Some pointed out that these are already defined as medicinal products.
* * *

III. Outline for a possible Community legal framework

Is there a role for European standards in support of a future legislative scheme, for instance regarding quality assurance?

Responses from Governmental / Institutional officials

For those who could support a new framework, yes – for both the process and the product. It is recommended that those standards should be concentrated on good manufacturing practise and quality assurance.

Responses from Industry

There is a consensus to have a body of “harmonised essential requirements” and horizontal standards, in the new legislation, based on a risk assessment approach covering quality assurance, manufacturing practices, microbiological/viral safety aspects and ethics. Two European Industry Associations however, emphasise that it is currently premature to define standards prior to the finalisation of the marketing authorisation and the approval structure for these products. Several companies emphasised that some existing standards such as Quality Assurance or Good Manufacturing Practices are not adapted to human tissue engineered products.

Responses from experts, institutes, consultants, individuals

Most of the answers are positive. It is argued that a certain number of harmonised standards relating to the generic issues of quality insurance systems, sterilisation, risk management, labelling, etc. should be developed specifically for human tissue engineered products.

It is noted that any standards should not be agreed in isolation but in co-operation with ISO and other international organisations.

Those against European standards argue that it will provoke bureaucracy and be too static for such a rapidly evolving area.

Detailed rules adopted by Committee procedures were presented as an alternative to standards. This could be a way for vertical specifications for products.

* * *

Would guidance documents in support of a future legislative scheme have to be developed by authorities?

Responses from Governmental / Institutional officials

Unanimously all the Governmental/Institutional officials are in favour of the having supporting documents making it easier to interpret and use the directive. Even the officials in favour of having tissue engineering in the framework of medicinal products (no specific directive) are in favour of the development of those documents.

Responses from Industry

European Industry Associations and their individual members are in favour of guidance on specific aspects: delineation of borderlines; ethics; review process at Member State level, if any; vigilance and traceability. They stress that documents should be developed at a central level (Commission often quoted) and not devolved to Member States.

Responses from experts, institutes, consultants, individuals

All are in favour of guidance document as a way to react rapidly to changes. It is considered to be much quicker than standardisation or amendment of directives or their annexes. It is recommended that those guidances be drafted by a group of experts regrouping research centres, universities, manufacturers and lawyers.
Some propose that the scope of application of those guidance documents cover the areas of quality assurance, risk management, quality, control, microbiological safety of donation, production and processing practices, product performance, including preclinical studies, pre-market clinical studies and post-market evaluation.

* * *

Is there a potential need for complementary binding specifications, adopted by the Commission in co-operation with Member States?

Responses from Governmental / Institutional officials

Most answered yes although certain expressed reservations that the area might develop too quickly for binding specifications but there is a need to balance between rapidity, to do not restrict innovation and to ensure safety.

Responses from Industry

One European Industry Association and two of its members that participated individually in this consultation are strongly opposed to any fixed technical specifications.
The other European Industry Association and some of its affiliates favour adoption by the Commission of ancillary binding texts easily adaptable to take account of technical progress. Some members dissented from this view.

Responses from experts, institutes, consultants, individuals

Legally binding specifications adopted by the Commission in co-operation with the Member States are generally not supported, at least at this stage of development.

* * *

Are these instruments mutually exclusive, or can one envisage them being applied to different and distinct aspects?

Responses from Governmental / Institutional officials

All said that the instruments are not mutually exclusive

Responses from Industry

One European Industry Association and some but not all its affiliates think they should be mutually exclusive for reasons of clarity for the industries. The other European Industry Association believes that they are not necessarily mutually exclusive because the functions they perform are very different.

Responses from experts, institutes, consultants, individuals

Only a few respondents expressed an opinion on this point. Their view was that these instruments are not mutually exclusive; they can be applied to different and distinct aspects. One or another instrument could be applicable depending on various factors, such as the level of risk in the production process.

* * *

Are the provisions on clinical tests for new biological medicinal products (approval to start the clinical trials) appropriate?

Responses from Governmental / Institutional officials

Reactions covers the full range from yes (four MSs officials) to yes with revisions, to non-committal response (two), to yes something is needed but not 2001/20/EC (under revision).

Responses from Industry

The answers are divided in two groups:
The first group including one European Industry Association believes that Directive 2001/20/EC could be extended to human tissue engineered products provided that a distinction from cell therapy medicinal products is clearly established.
The second group, with an other European industry association, emphasised that the Directive 2001/20/EC applies to clinical trials on medicinal products for humans and therefore many of the requirements of the directive can not be applied to human tissue engineered products. Nevertheless several principles of this Directive could be incorporated into the requirements for clinical trials for human tissue engineered products, e.g. via a European Standard.

Responses from experts, institutes, consultants, individuals

The need to obtain an approval before starting clinical trials is generally supported. Some participants recommend avoiding this for autologous products as they feel the risk related to these products is minimal. (However, most of the European existing or planned products are autologous.)

IV. Authorisation and market access

Under this heading, the Commission invited comments on whether an approval scheme could be based on :
– a two-stage approval system, distinguishing between the licensing of the production plant, and market approval of the product, or
– an integrated quality system, in which the market access of products would be based on the company’s quality system in relation to specific products and a specific assessment of that particular product
Are the two approaches mutually exclusive?
Which one is to be preferred from the safety and policy point of view?

Responses from Governmental / Institutional officials

There are diverging opinions but a majority prefer a two-stage approach. Two stated the integrated approach to be better for the patient safety; six gave the two-stage approach as the preferred option for this safety.

Responses from Industry

No consensus: One European Industry Association and most of its affiliate is rather in favour of a two-step procedure (not mutually exclusive).
The other group of European Industry consider them to be mutually exclusive.

Views expressed included safety concerns about a company placing on the market a range of similar products under a single Quality Assurance certification, the need for a quality system applied both to the entire facility and the specific product, the preference for licensing of the production plant plus market approval (as for medicinal products) if a therapeutic claim is made with a second option (quality system plus specific assessment of the product) if there is no therapeutic claim.

Responses from experts, institutes, consultants, individuals

The “two stage approval system” and the “integrated quality system” are considered to be mutually exclusive to avoid uncertainties.

There is no consensus about the safety (3 for two steps, 4 in favour of integrated system, 2 regard the systems as equivalent, others have no opinion)
The group favouring the two step approach argue that it ensured sufficient safety and is a practical solution that could be based on the experience made with existing legislation. They argue that the rules for an integrated approach will have to be developed from scratch and that this will take too long.
The group favouring the integrated procedure argue that it offers a higher degree of safety for the patient. Some consider that this higher safety of the “integrated approach” comes from the evaluation of the whole “company quality system” in contrast with the “licensing of the production plant separately”.

V. Procedural aspects of the evaluation process

Under this heading, the Commission invited comments as to whether
– An application for market access would be submitted to a national authority. The national authority would prepare a draft, to be submitted to an advisory committee made up of representatives of Member States. The opinion of this committee would be sufficiently authoritative for the national authority to which the application was introduced to accept it as a basis for its decision providing access to the Community market.
– The application would be submitted to a central Body/authority responsible for both evaluation and approval. This body would be composed of representatives of Member States

Is one of the options set out to be preferred?

Responses from Governmental / Institutional officials

The officials of 5 States preferred the centralised approach with an agency developing the needed expertise. One other stated he could conceive to have centralised and decentralised procedure for different products. One is opposed to a centralised system. The officials of two Member States presented diverging opinions and two preferred not to comment at this time.

Responses from Industry

Again the division in two groups stays valid:
· One declines to choose between the two systems. Some of its individual affiliates, however, prefer the centralised procedure.
· By contrast, the other group prefers a formal European centralised approval system. Most of the supporters of the centralised procedure support their position citing the absence of trust in a mutual recognition system and the need to harmonise requirements, practices and post authorisation controls (vigilance, inspections).

Responses from experts, institutes, consultants, individuals

A majority is in favour of a more centralised system for reasons of open access throughout the whole Community and the need for a level playing field. Authorisation by a “central body” should be rapid, unbureacratic and should not favour larger companies in comparison of smaller ones.
Those preferring a system of national authorisations give as reasons simplicity, transparency and the possibility of direct contact while the system is developing.

If an agency becomes involved, should a separate agency be created, or could the competencies of EMEA be extended?

Responses from Governmental / Institutional officials

One preferred not to comment. All the others said if an agency becomes involved this could be the EMEA. Two of them felt that this could be within the EMEA or a separate agency.

Responses from Industry

The majority supported the concept of a central agency. Whether this should be the EMEA appeared to depend on experience or familiarity with the EMEA and its way of working. Thus one European association supported the EMEA. Whereas, the other did not support a role for the EMEA, although it accepted the idea of a European Agency.
All noted that it would be important for the central agency to have access to the appropriate expertise. Some small companies are wary of a centralised agency, in particular of the EMEA, and prefer a Notified Body as a centralised agency.

Responses from experts, institutes, consultants, individuals

Not all respondents answered this point. Of those in favour of a new legal framework, most favoured a centralised body. Of those favouring a centralised body half favoured expanding the competencies of the EMEA, and several did not express a preference between the EMEA on a new separate body. Only one respondent who favoured a centralised body felt that this should be separate from the EMEA.

* *

The Commission will continue its efforts to define the outline of a Community strategy taking into consideration the results of this web-consultation and the opinion of the Scientific Committee for Medicinal Products and Medical Devices. It will make the results known in due time.

* * *
Footnotes :

1. Council Directive 93/42/EEC of the 14 of June 1993 Concerning medical devices.

2. Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use

3. Proposal for a Directive of the European parliament and the Council setting standards of quality and safety for the collection, testing, processing, storage, and distribution of human blood and blood components and amending Council Directive 89/381/EEC. Com(2000)816 final
Proposal for a Directive of the European parliament and the Council setting standards of quality and safety for the donation, procurement, testing, processing, storage, and distribution of human tissues and cells COM(2002)319 final.

The prestigious Descartes Prize of the EU has been awarded on 5 December to two research projects in the fields of medicine and astrophysics. One project, led by Danish Researcher Prof. Lars Fugger, greatly advanced our understanding of Multiple Sclerosis and is offering leads for new drugs. The other project has discovered the origins of Gamma Ray Bursts and is providing insights into star and planet formation.

The €1 million prize rewards outstanding scientific research through transnational collaboration.

For more information :