3 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
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| Legal base | Article 95 EC; co-decision; QMV
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| Department | Health |
| Basis of consideration | Opinion of the Health Committee
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| Previous Committee Report | HC 34-xiv (2005-06), para 6 (11 January 2006)
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| To be discussed in Council | No date set
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| Committee's assessment | Legally and politically important
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| Committee's decision | Not cleared; further information awaited
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Background
3.1 In 2001, the Council adopted a Directive on medicinal products
for human use.[5] It contains
rules for Member States to follow on the authorisation, manufacture,
importation, labelling and distribution of medicinal products.
An amending Directive was adopted in 2003.[6]
It includes provision on two advanced therapy medicinal products
— gene therapy medicinal products and somatic cell medicinal
products.
3.2 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.[7]
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.[8]
3.3 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.
Previous scrutiny of the draft Regulation
3.4 Gene therapy medicinal products and somatic cell
medicinal products are already regulated under EC law. But tissue
engineered products 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".[9]
3.5 When we considered the draft Regulation in January,
the then Minister of State for Quality and Patient Safety at the
Department of Health (Jane Kennedy) told us that the Government
was still considering whether Article 95 of the EC Treaty was
an appropriate legal base for the proposal. Moreover, the document
was being considered by the Medicines and Healthcare Products
Regulatory Agency and the Department of Trade and Industry to
see if it met the Government's objectives. The Government was
seeking the views of the bio-technology and healthcare industries.
3.6 We noted that the Government's and the Council's
consideration of the draft Regulation was at a very early stage.
We asked the Minister for further information. Moreover, in accordance
with paragraph (11) of our Standing Order No. 143, we asked the
Health Committee for its opinion on the draft Regulation.
3.7 On 18 May, we received the Health Committee's
opinion.
The written opinion of the Health Committee
3.8 In forming its opinion, the Health Committee
took account of the views of the Medicines and Healthcare Products
Regulatory Authority and the BioIndustry Association (the trade
association for innovative bioscience enterprises in the UK).
The Association has argued, for example, for greater clarity in
the definition of tissue-engineered products. The Health Committee
tells us that the BioIndustry Association has also suggested that
it would be unrealistic to expect companies currently developing
products to implement the draft Regulation fully because it would
extend the development time and, consequently, increase the costs
of developing the products.
3.9 The Health Committee concludes:
"While the Committee acknowledges the concerns
expressed by the BioIndustry Association and others, it welcomes
the draft regulation as a sensible method of bringing together
different forms of advanced therapy products under the same regulatory
framework. A single framework will allow greater clarity and transparency
of regulation, as well as bringing under supervision an important
area of medical technology which was not previously monitored
by EC regulations. It will also allow producers across the bioscience
sector to compete on the same terms and receive the same financial
treatment and incentives."
3.10 The full text of the opinion is reproduced below.
Conclusion
3.11 We are grateful to the Health Committee for
its clear and cogent opinion on the draft Regulation. We commend
it to the attention of the House. We shall take the opinion into
account in reaching our own conclusions on the proposal, after
we receive the Minister's reply to the questions we put to her
in January.
ANNEX
OPINION FOR THE EUROPEAN SCRUTINY COMMITTEE FROM
THE HEALTH COMMITTEE ON THE DRAFT REGULATION ON ADVANCED THERAPY
MEDICINAL PRODUCTS
INTRODUCTION
The Health Select Committee has been asked by the
Clerk of the European Scrutiny Committee for its opinion on the
draft EC regulation concerning advanced therapy medicinal products.
The Committee's opinion is expressed herein. In forming its opinion,
the Committee received a briefing from the Parliamentary Office
of Science and Technology, and, through POST, heard the opinions
of the Medicines and Healthcare products Regulatory Authority
(MHRA) and the BioIndustry Association (BIA), the trade association
for innovative enterprises in the UK bioscience sector.
DEFINITIONS
Advanced therapy medicinal products cover three areas
of medicine: gene therapy; somatic cell therapy; and tissue engineering.
Gene therapy involves
the introduction of genetic material into a cell in order to treat
a disease, especially genetic diseases such as cystic fibrosis,
and cancer. The majority of gene therapy products are at the clinical
research stage.
Somatic cell therapy is
the use of somatic (that is, non-reproductive) cells, apart from
transfusable blood products, for therapeutic, diagnostic or preventative
purposes; for example, the implantation of cells as an ongoing
source of a therapeutic agent, such as an enzyme or coagulation
factor. Somatic cell therapy is also at the clinical research
stage.
Tissue engineering involves
the production of therapeutic products which use a combination
of a delivery system and viable human cells or bioactive molecules
derived from human cells, usually with the aim of restoring, replacing
or regenerating human tissue. This process has already led to
the development of products used to treat burns and ulcers, and
more complex products, such as heart valves, are in development.
EXISTING EC LAW
Gene therapy and somatic cell therapy products are
currently classified and regulated as biological medicinal products
by EC law under Directive 2001/83/EC, as amended by Directive
2003/63/EC. Those which are created by a biotechnology process
are required to be approved by the European Medicines Agency (EMEA),
while other products are approved under the "mutual recognition"
procedure, whereby the approval of a product application by the
competent authority in one member state (for example, the UK's
MHRA) allows that product to be marketed across the EU. However,
many tissue engineered products currently fall outside this framework.
In the UK, for products without the existing regulations, the
MHRA exercises a Code of Practice for the Production of Human-Derived
Therapeutic Products, but this is not legally binding. Some
other member states, such as Austria, Finland and Sweden, have
also implemented national regulations.
THE PROPOSAL FOR A NEW REGULATION
The principal argument which the European Commission
has advanced in favour of the new regulation, grouping together
gene therapy products, somatic cell therapy products and tissue
engineered products under one regulatory framework, is that they
are all cell-based therapies, constituting "a coherent ensemble
insofar as they share several key scientific, regulatory and economic
features".[10] The
Commission also argues that the pooling of regulatory and scientific
expertise from all member states in the evaluation of such therapies
will improve the levels of scrutiny, as this expertise is otherwise
scarce. In pursuance of this goal, it intends to create a Committee
for Advanced Therapies within EMEA.
Because advanced therapy products, by their very
nature, can remain in the human body over long periods of time,
the Commission intends to ensure traceability from donor to patient,
and long-term patient follow-up. Under the draft regulation, the
market authorisation holder for any given product will be responsible
for setting up and maintaining systems which ensure that the therapy
product can be traced from its source, through manufacturing,
packaging and transport to its delivery at the institution where
it is to be used. That institution will then be responsible for
establishing a protocol which records a link between the product
and the patient who receives it. (Similar protocols already exist
in hospitals for blood products.)
The products which will fall under the draft regulation
are typically developed by small or medium-sized enterprises;
for example, spin-off companies which have grown out of university
research, hospitals or tissue banks. The draft regulation will
bring advanced therapy products within the same broad regulatory
framework as other medicinal products, and will thereby entitle
them to a number of existing incentives - a harmonised data protection
period, the opportunity to apply for a 10-year exclusivity period
under orphan medicinal product status, options for conditional
marketing authorisations and financial incentives and administrative
assistance for small and medium-sized enterprises. The draft regulation
would also give manufacturers of advanced therapy products a 90%
reduction in fees for scientific advice, and a system of early
evaluation and certification of producers' quality and non-clinical
safety data, both from EMEA.
THE VIEWS OF THE MHRA AND BIA
1. The BioIndustry Association has argued for greater
clarity in the definition of tissue-engineered products. It has
recommended that the Medical Devices Directive 93/42/EEC (which
is currently being revised) include an amendment to permit the
regulation of medical devices containing or utilising human tissue
and tissue-engineered products in circumstances where such combination
products do not meet the definition of a medicinal product, in
order to enhance conformity assessment of such products.
2. There have also been concerns raised by the BIA
about the exclusion from the ambit of the draft regulation of
the treatment on prescription of individual patients with advanced
therapy products in an academic or hospital environment. The BIA
has argued for appropriate regulatory controls introduced to guarantee
patient safety in these situations. The MHRA has noted the proposal
for the exclusion and is considering the implications of it.
3. There are a number of advanced therapy products
currently in various stages of clinical development. The biotechnology
industry has suggested to us that it would be unrealistic to expect
companies currently developing products fully to implement the
draft regulation, on the grounds that it would extend the development
time and therefore the costs of development of the products in
question. Therefore, the BIA suggests, there should be interim
arrangements to allow eventual patient access to products currently
in development.
THE COMMITTEE'S CONCLUSION
While the Committee acknowledges the concerns expressed
by the BioIndustry Association and others, it welcomes the draft
regulation as a sensible method of bringing different forms of
advanced therapy products under the same regulatory framework.
A single framework will allow greater clarity and transparency
of regulation, as well as bringing under supervision an important
area of medical technology which was not previously monitored
by EC regulations. It will also allow producers across the bioscience
sector to compete on the same terms and receive the same financial
treatment and incentives.
5 Directive 2001/83/EC: OJ No. L 311, 28.11.2001, p.67. Back
6
Directive 2003/63/EC: OJ No. L 159, 27.6.2003, p.46. Back
7
Directive 2004/23/EC: OJ No. L 102, 7.4.2004, p. 48. Back
8
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
9
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
10
Explanatory Memorandum to draft Regulation 15023/05 (27059), p.
2. Back
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