Advanced Therapies refers to a newly adopted Regulation (EC) No 1394/2007 on advanced therapy medicinal products that will, for the first time, bring all advanced therapies (gene, cellular and tissue-based) together within a single, integrated European regulatory framework, thereby ensuring consistency across member states.  The Regulation sets out tailor-made technical requirements for these innovative therapies and establishes new standards for clinical trials in the development of advanced medicinal products. A proper legal framework for the market authorisation of these products is also likely to stimulate increased research in this field. The Advanced Therapies Working Group was involved in a campaign working for the adoption of this regulation.  Now that the regulation is in place, the group is working on harmonising implementation strategies.

Following the opinion of the European Parliament on 25 April 2007, the Council of Ministers approved the Regulation on advanced therapies, in first-reading on 31 May 2007. The Regulation was translated into all EU official languages and on 30 October 2007 the Advanced Therapy Regulation was formally adopted by the EU Council.  The Regulation was published in the EU Official Journal on 10 December 2007 and will enter into force on 30 December 2007.   Soon after the publishing of the Regulation, the DG Enterprise and Industry made public its priorities for the implementation of the Advanced Therapies Regulation. The implementation plan has been developed and agreed with the European Medicines Agency (EMEA). 

To view the Regulation click here
http://eur-lex.europa.eu/LexUriServ/site/en/oj/2007/l_324/l_32420071210en01210137.pdf.

The Implementation plan: http://ec.europa.eu/enterprise/pharmaceuticals/
 advtherapies/docs/implementationplan-2007-12-11.pdf

“Europe’s biotechnology sector has promising technologies to offer its citizens and the world, and it is crucial for the future of its life sciences sector that the right legal framework is created to allow these to reach the patients. Europe is already falling behind in this field and we need to act.”

--Wills Hughes-Wilson,
Chair,
EuropaBio’s Advanced Therapies Working Group

EuropaBio's Documents

• EuropaBio's Policy Brief on Advanced Therapies
• Examples of Advanced therapies

• Report by EuropaBio November 2006 Policy Forum on Advanced Therapies

• Report by EuropaBio Industry Hearing on Advanced Therapies – November 2005

• JRC study: Human tissue-engineered products - Today's markets and future prospects

• Report from Dr. Mikolasik, Rapporteur EP Environment Committee
• European Parliament Public Hearing on Advanced Therapies – May 2006
• European Commission Proposal of 16 November 2005
   

Types of Advanced Therapies

Cell and tissues

A person’s own cell and tissue can offer a patient a wide range of health care solutions, from prosthetic and restorative to therapeutic or even cosmetic in nature.
Under normal conditions damaged joint cartilage does not – or only poorly - regenerates in the body. For several years now, cell therapy for restoring defects to knee cartilage has been available by growing a patient's own cartilage cells to repair cartilage defects. Active research, involving human cell-and tissue-based products, is currently being conducted in the regeneration and repair of bones, tendons, nerves and ligaments.

Cell-based cancer immunotherapy like cell-based tumour vaccines to combat cancer are providing compelling news that such therapy may one day provide hope to cancer patients.

EuropaBio fact sheet on human tissue and cells

Stem cells

Research into stem cells may result in important cell-based therapies to treat serious diseases and conditions like Parkinson’s disease, Alzheimer’s disease, spinal cord injuries, as well as diabetes, stroke, burns, skin disorders and heart disease. Researchers work on three types of human stem cells – adult, foetal or embryonic. The use of human embryonic stem cells raises important questions which are currently at the centre of an ethical and societal debate.

Click here for more information on stem cells:
Commission report on human embryonic stem cell research provides basis for discussion on ethics

Gene therapy

Despite the high standard of today's medical treatments, and the number of already available drugs, many of the most debilitating human diseases do not have a cure yet. The molecular basis of many inborn disorders, such as haemophilia, cystic fibrosis or muscular dystrophy, has been exposed by the discovery of affected genes. In many forms of cancer, genetic predisposition plays as important a role as environmental factors in tumour growth, and malignancy. Identifying the gene for such diseases and redirecting its course is one of the most promising ways to cure certain diseases.

Gene therapy has entered a phase of active clinical investigation in many areas of medicine. Human clinical trials have been started for the treatment of severe immunodeficiencies, cystic fibrosis, hypercholesterolemia, haemophilia, muscular dystrophy, many types of tumours (e.g. melanoma, prostate, ovarian and lung cancer), AIDS, and cardiovascular disorders.

EuropaBio Information paper on ethical, social and public awareness issues in gene therapy. (pdf)

Advanced Therapies Working Group

The Working Group is focusing on the proposed new regulation governing advanced therapies, i.e. gene therapy, cell therapy and tissue engineered products.  The legislation is now going through Parliament and Council for decision.

Chairman: Mrs. Wills HUGHES WILSON (Genzyme)
Secretary: Ms. Stefanie PINGITZER (EuropaBio)

Press Releases

May 2007

31/05/2007
European framework adopted today on “Advanced Therapies” will allow European-wide access to a new generation of treatments
> documents : Word /  Pdf

April 2007

25/04/2007
EuropaBio congratulates European Parliament on big step towards Advanced Therapies Regulation
 >  documents : Word / Pdf
 
10/04/2007
Europe loses as trialogue negotiations on proposed Advanced Therapies Regulation fail
> documents : Word /  Pdf

3/04/2007
EU Regulation on Advanced Therapies - Negotiations break down- Patients react - Eurordis Press Release
> documents : Pdf

March 2007

14/03/2007
New studies show the benefits of biotech healthcare innovation for patients and society
> documents : Word /  Pdf

12/03/2007
EuropaBio Announces the Launch of the BioImpact.org Website and Findings
> documents : Word/  Pdf
Visit BioImpact.org

28/02/2007
EU Regulation Urgently Needed! European Patients are Waiting for the Next Generation of Medical Treatments - EPPOSI Press Release
> documents : Word /  Pdf

27/02/2007 Press Conference on Advanced Therapies

January 2007

24/01/2007
EuropaBio supports patients, science and industry's call to value healthcare innovation
> statement: Word  /  Pdf

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Press coverage: articles with “ EuropaBio & Advanced therapies” 11/04/2007 – 26/04/2007

Advanced Therapies FAQ (February 2007)

• What are “advanced therapies”?
• Why does there need to be a European Regulation?
• How will the proposed Regulation help?
• What is the industry position?
• Where’s the controversy?
• What is the timeline of the draft Regulation?

What are “advanced therapies”?

“Advanced therapies” is an umbrella term that covers the three new techniques that are revolutionising modern medicine: cell therapy, gene therapy and tissue engineering. These techniques are already producing new treatments, and in the future are likely to produce many more.

In cell therapy, new cells are introduced into tissue to treat a disease – either from the patient’s own body (autologous), from another person (allogenic) or from an animal (xenogenic). One current example is the use of human adult stem cells in treating leukaemia. Around the world, researchers have been making progress towards using stem cells to treat conditions ranging from heart disease, spinal cord injury and brain damage to muscle damage, low blood supply and even deafness and baldness.

In gene therapy, genes are introduced to replace, repair or switch off defective ones. It has been used, so far with varying degrees of success, in severe combined immunodeficiency (SCID), cystic fibrosis, muscular dystrophy and sickle-cell anaemia, among others.

Tissue engineering is aimed at replacing or repairing defective tissue, and is generally seen as relating to bone, cartilage and blood vessels such as veins and arteries. This expanding field covers the techniques of bioengineering and cell biology (and often cell therapy itself). It is already being used to repair or replace burnt skin, for example.

Why does there need to be a European Regulation?

Patients – and companies seeking cures for them – in the different countries of the European Union face a bewildering patchwork of guidelines, regulations and procedures relating to advanced therapies. Some countries have no specific framework at all.

This diversity is failing patients in terms of safety and efficacy, quality, and availability.
Safety and efficacy. These new products are all at the leading edge of medical discovery, and there are many new challenges in judging whether they are safe to use, and whether they work as claimed – so standards need to be set and expertise needs to be highly specific. That requires bringing together the best experts from across Europe.

Quality. These new treatments are targeted at very specific processes inside and between cells in the body, and will require highly specialised manufacturing procedures to ensure quality. Again, standards need to be set and expertise needs to be highly specific.

Availability. With so many different national systems, companies making the new treatments face enormous problems in bringing them onto each national market. This leads an unfair situation where patients in some countries miss out on treatments that could save their lives. It also puts the European advanced therapy industry at a competitive disadvantage vis-à-vis the United States, whose manufacturers have a single home market to support them.

How will the proposed Regulation help?

One procedure. The proposed Regulation will provide one single centralised procedure, run by the European Medicines Agency (EMEA), for the whole of the European Union for assessing the safety, efficacy and quality of advanced therapies and giving them marketing authorisation. While a single procedure under the EMEA already exists for products of cell and gene therapy, the Regulation will now bring tissue engineering into the system for the first time.

Specialist knowledge. The Regulation will set up a Committee for Advanced Therapies (CAT) within the EMEA to ensure the most thorough scientific evaluation possible. The CAT will be multidisciplinary, and include representatives of patients and doctors.

Monitoring. Normal medicines disappear from the body relatively quickly, but the cells, genes and tissues of advanced therapies are intended to last for many years. The Regulation will set up a “traceability” system that will track all patients treated with these advanced therapies, as well as the products themselves and their starting materials (for example, original cell lines). This system will allow regulators to see what is happening to patients once treated, even decades after the original treatment.

Availability. The centralised procedure will make it dramatically simpler and faster for companies producing advanced therapies to make their products available to patients across the European Union. There are also incentives built into Regulation to encourage small and medium-sized enterprises (SMEs) to invest in the new treatments.

What is the industry position?

In general, the biotechnology industry welcomes the proposed Regulation. It believes that the centralised procedure through the EMEA is the best way to ensure that patients have access to high-quality, safe and effective products, and that it will help companies by providing them with universal access to the European market.

Realistically, though, this new procedure will require a transition period of five years, rather than the two years in the current proposal. And advanced therapies already authorised should continue to be available to patients during this transition period while the products are being evaluated under the procedure.

Industry welcomes the incentives for SMEs, but believes they should be extended to all innovative companies, of whatever size, to encourage investment in advanced therapies generally.

While supporting the idea of the CAT, industry is concerned that the Regulation proposes that it should only advise the EMEA’s Committee for Medicinal Products for Human Use on the scientific evaluation of new advanced therapies. It thinks there is no point having two committees doing basically the same thing, and that the CAT should decide on scientific issues, rather than merely give an opinion.

Industry is also keen to ensure that the EMEA, and specifically the CAT, is adequately funded to carry out its new duties.

Where’s the controversy?

There are four possible flashpoints around the proposed regulation. One is neatly sidestepped. The other three could create short- or long-term issues.

Ethics…and embryonic stem cells. Some advanced therapies are and will be based on embryonic stem cell lines – and for ethical reasons some countries will not want to have products based on these available in their health systems. The Regulation basically sidesteps this issue with the “subsidiarity” principle: whatever the EMEA evaluation, it will be up to individual countries to decide whether these particular products should be placed on their own markets. But that may not satisfy some diehard critics of embryonic stem cell technology.

Combined products. Medical devices have always been covered by separate and generally simpler procedures than medicines. But the Regulation is suggesting that any products that combine devices and advanced therapies should come under the new centralised procedure. This is a potential concern for the medical devices industry, which might find its authorisation procedures more costly, complicated and time-consuming.

Hospitals. Products that are both prepared in full and used in a single hospital, in accordance with a medical prescription for an individual patient, are excluded from the scope of the proposal. There is some concern in industry, however, that this might allow an unlevel playing field to develop – especially as it might relate to large hospitals providing specialist care to large numbers of patients.

The grey areas. Some products will very clearly fall inside the category of advanced therapies, some very clearly outside – and there are bound to be number of grey areas. The Commission acknowledges this, and in response proposes a “case law” approach where companies unsure about where their products might lie can approach the CAT for a recommendation. But that approach lacks the legal certainty companies need, and could leave them in a research and legal limbo, not knowing whether proposed products fall within the Regulation, and hence not knowing what data sets will be required, for example. The solution, says industry, is to have clearer and more detailed definitions upfront of what constitutes an advanced therapy.

November 2005 - the European Commission published its proposed text for a Regulation;

13 September 2006 - the ENVI Committee rejected the first Report by the European Parliament Rapporteur Miroslav Mikolasik because it contained a large number of so-called “ethical amendments”, which would have banned a range of products and technologies;

30 January 2007 - the Rapporteur’s second draft was adopted by the ENVI Committee with a convincing majority, and with none of the “ethical amendments” included;

Early 2007 - “Trialogue” negotiations started (discussions between the European Commission, the Council and the European Parliament) by the beginning of the year 2007 to explore the possibility of an early adoption;

The European Parliament‘s Plenary vote was postponed from 13 March to the end of April to give the trialogue negotiations enough time to explore an eventual agreement;

April 2007 - The trialogue negotiations failed and the plenary vote will take place in the week of the 23 April in Strasbourg.

25 April 2007 - European Parliament approves Advanced Therapies Regulation

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Events

November  2006 - The EuropaBio Policy Forum on Advanced Therapies (part of the Jacob Fleming Conference)

November 2005 - EuropaBio Industry Hearing on Tissue Engineering  

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BIO 2006 Session on Advanced Therapies

“How Europe and the US regulate emerging advanced therapies?”
Presentations by:

Mrs. Georgette LALIS Director, European Commission DG Enterprise & Industry
Genes, Cells and Tissues: Commission proposal on advanced therapies

Mr. Ashok BATRA MD FACS, Division Director, Division of Clinical Evaluation and Pharmacology Toxicology
Regulation of Biologic Therapies (not yet available)

Mr. John PURVES, European Medicines Agency (EMEA)
Advanced Therapies Regulation in European Union: Role of EMEA

Mr. Gil BEYEN, CEO, TiGenix N.V.
Regulation of Advanced Therapies - An SME Perspective

Human cell and tissue based products fact sheet (23/10/2003 | pdf - 381.2kb)

Cell and Tissues Directive documentation (DG Sanco Legislation)