6 DEVELOPMENT AND REGULATION OF ADVANCED
THERAPY MEDICAL PRODUCTS
(27059)
15023/05
COM(05)567
+ ADD 1
| Draft Regulation on advanced therapy medicinal products and amending
Directive 2001/83/EC and Regulation (EC) No. 726/2004
Commission staff working document — impact assessment
|
| Legal base | Article 95 EC; co-decision; QMV
|
| Document originated | 16 November 2005
|
| Deposited in Parliament |
1 December 2005 |
| Department | Health |
| Basis of consideration |
EM of 14 December 2005 |
| Previous Committee Report |
None |
| To be discussed in Council
| No date set |
| Committee's assessment | Legally and politically important
|
| Committee's decision | Not cleared; further information and the opinion of the Health Committee requested
|
Background
6.1 Article 14 of the Treaty establishing the European Community
(the EC Treaty) makes provision for measures to establish the
internal market, comprising an area without internal frontiers
and with free movement of goods, people, services and capital.
6.2 Article 95(1) of the EC Treaty requires the
Council to adopt measures for the approximation of the laws of
Member States which have as their objective the establishment
and functioning of the internal market. Article 95(3) requires
that such measures, if concerned with health and safety, should
"take as a base a high level of protection, taking account
in particular of any new development based on scientific fact".
6.3 In 2001, the Council adopted a Directive on medicinal
products for human use.[21]
It contains rules for Member States to follow on the authorisation,
manufacture, importation, labelling and distribution on medicinal
products. An amending Directive was adopted in 2003.[22]
It includes provision on two advanced therapy medicinal products
— gene therapy medicinal products and somatic cell medicinal
products.
6.4 In 2004, the Council adopted the Cells and Tissues
Directive. It sets standards of quality and safety for the donation,
procurement, testing, processing, preservation, storage and distribution
of human tissues and cells.[23]
The Council also adopted a Regulation laying down Community procedures
for the authorisation, supervision and pharmacovigilance of medicinal
products for human use and establishing the European Medicines
Agency.[24]
6.5 A number of gene therapy and somatic cell therapy
products are already being tested for the treatment of cancer,
diabetes, Parkinson's disease and other neurodegenerative diseases.
6.6 Another new biotechnology has emerged: tissue
engineering. It combines aspects of medicine, cell and molecular
biology, materials science and engineering. The aim of tissue
engineering is to develop products to regenerate, repair or replace
human tissues, such as skin, bone and cartilage.
The document
6.7 As noted above, gene therapy medicinal products
and somatic cell medicinal products are already regulated under
EC law. But tissue engineered products (TEPs) are not. The purpose
of the draft Regulation is to make rules for the authorisation,
supervision and pharmacovigilance of all three types of product,
collectively defined as "advanced therapy medicinal products".[25]
6.8 Gene therapy, somatic cell therapy and tissue
engineered products share some features: for example, they are
based on highly innovative manufacturing processes; there is a
scarcity of people with the expertise to evaluate the therapies;
and it is essential to make provision to trace the recipients
of the advanced therapies and to monitor the long-term safety
of the products. Recital 4 of the draft Regulation says that —
because of the novelty, complexity and technical specificity of
advanced therapy medicinal products — specially tailored
rules are needed to ensure their free movement within the Community
and the effective operation of the internal market for biotechnology.
6.9 The objectives of the Commission's proposal are
to:
- protect human health;
- improve the functioning of the internal market
for advanced therapy medicinal products;
- foster the competitiveness of European bio-technology
undertakings; and
- provide legal certainty.
6.10 The draft Regulation includes provision on:
- the application to advanced
therapy medicinal products of the provisions of the Cells and
Tissues Directive on donation, procurement and testing;
- clinical trials, including a requirement for
the Commission to issue detailed guidance on good clinical practice
specific to advanced therapy medicinal products;
- the publication by the Commission of good manufacturing
practice for advanced therapy medicinal products;
- specific technical requirements for TEPs;
- the evaluation procedure for advanced therapy
medicinal products;
- information to be displayed on the packaging
of the product and in the leaflet inside the package;
- post-authorisation risk management;
- the traceability of products;
- a 90% reduction in the European Medicines Agency's
fees for advice to the applicants for or holders of a marketing
authorisation of an advanced therapy medicinal product;
- assistance to small and medium-sized enterprises
(SMEs) which are developing advance therapy medicinal products;
and
- the creation of a Committee for Advanced Therapies
within the European Medicines Agency.
6.11 The draft Regulation also contains a requirement
for products which are on the market when the Regulation comes
into effect to be made compliant with the Regulation within two
years. Applicants for the authorisation of such products would
be exempt from the European Medicines Agency's normal fee for
authorisation.
The Government's view
6.12 The Minister of State for Quality and Patient
Safety at the Department of Health (Jane Kennedy) tells us that
the Government's view had been that Article 95 of the EC Treaty
was not an appropriate legal base for measures to establish a
centralised EC procedure or body. She says that the Government
is reviewing its position in the light of the judgement of the
European Court of Justice on 6 December 2005 in the "Smoke
Flavourings" case.[26]
6.13 The Minister tells us that, in 2002, the Medical
Devices Agency (now the Medicines and Healthcare Products Regulatory
Agency) issued a code of practice for the production of human-derived
therapeutic products. Compliance with the code is voluntary.
6.14 The proposed Regulation is currently being considered
by the Medicines and Healthcare Products Regulatory Agency and
the Department of Trade and Industry to see if it meets the Government's
objectives. The Government is also seeking the views of the bio-technology
and healthcare industries.
6.15 The Minister has sent us her Department's initial
Regulatory Impact Assessment of the proposal. It concludes as
follows:
"The tissue engineering sector is currently
small and is expected to develop as a result of scientific and
technological progress in the coming years. The impact of the
proposed regulatory framework is, therefore, difficult to measure.
An analysis of the potential costs and benefits associated with
this proposal will be undertaken in consultation with stakeholders
who are currently involved in tissue engineering activity in order
to better gauge the impact of the proposal for the UK. Given the
fragmented and developing nature of the sector it may prove difficult
to establish detailed costs."
Conclusion
6.16 The Government's and the Council's consideration
of this draft Regulation is at a very early stage. We cannot reach
a view on it without further information. We ask the Minister
to tell us:
- the Government's view of
the appropriateness of Article 95 of the EC Treaty as the legal
base for the Regulation;
- the outcome of the Government's consultations
with the bio-technology and healthcare industries; and
- the Government's views on the proposal in
the light of its own and the Medicines and Healthcare Products
Regulatory Agency's detailed examination of the draft Regulation;
and progress reports on the negotiations in the Council.
In due course, we shall also need to see the completed
Regulatory Impact Assessment.
6.17 Moreover, in accordance with paragraph (11)
of our Standing Order No. 143, we request the opinion of the Health
Committee on the draft Regulation by 18 April 2006.
6.18 Pending receipt of this information, we shall
keep the document under scrutiny.
21 Directive 2001/83/EC: OJ No. L 311, 28.11.2001,
p.67. Back
22
Directive 2003/63/EC: OJ No. L 159, 27.6.2003, p.46. Back
23
Directive 2004/23/EC: OJ No. L 102, 7.4.2004, p. 48. Back
24
Regulation (EC) No. 726/2004: OJ No. L 136, 30.4.2004, p.1. "Pharmacovigilance"
is the monitoring of the quality, safety and efficacy of marketed
medicines. Back
25
The Regulation would apply to products to be placed on the market
and either prepared industrially or manufactured by a method involving
an industrial process. But the Regulation would not apply to products
wholly prepared and used in a hospital in accordance with a prescription
for an individual patient. Back
26
Case C-66/04, UK v European Parliament and the Council of the
Union. Back
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