12 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. 736/2004
Commission staff working document — impact assessment |
| Legal base |
Article 95 EC; co-decision; QMV |
| Department |
Health |
| Basis of consideration |
Minister's letters of 12 March and 14 May 2007
|
| Previous Committee Report |
HC 34-xiv (2005-06), para 6 (11 January 2006); HC 34-xxx (2005-06), para 3 (24 May 2006); and HC 34-xxxi (2005-06), para 5 (14 June 2006) |
| To be discussed in Council |
30-31 May 2007 |
| Committee's assessment |
Legally and politically important |
| Committee's decision |
Cleared |
Background
12.1 In 2001, the Council adopted a Directive on medicinal products
for human use.[29] 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.[30]
It includes provision on two advanced therapy medicinal products
— gene therapy products and somatic cell products.
12.2 In 2004, the Council adopted the Cells and Tissues Directive.[31]
It sets standards of quality and safety for the donation, procurement,
testing, processing, preservation, storage and distribution of
human tissues and cells. The Council also adopted a Regulation
laying down Community procedures for the authorisation, supervision
and pharmacovigilance of medicinal products for human use.[32]
12.3 Tissue engineering 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
12.4 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".
12.5 When we considered the draft Regulation in January, May and
June 2006, we identified three key issues:
● The
proposed exemption of products from the requirements of the Regulation
where the product is both prepared in full and used in a hospital
in accordance with a medical prescription for an individual patient.
The Government thought it might be better to link the proposed
exemption to the characteristics of the activity rather than to
specific institutional arrangements (we refer to this as "the
exemption").
● There
are some products which contain human and animal tissues and are
used for medicinal purposes but which fall into the gap between
the existing regulatory regimes and would not be adequately regulated
by the proposed Regulation (we refer to this as "the gap").
● Whether
Article 95 of the EC Treaty would provide an appropriate legal
base for the proposed Regulation (we refer to this as "the
legal base").
The Minister's letter of 12 March 2007
12.6 In his letter of 12 March, the Minister of State
at the Department of Health (Lord Hunt of Kings Heath) told us
that, having reflected on the rulings of the European Court of
Justice in the Smoke Flavourings case[33]
and the ENISA case,[34]
the Government had now accepted that Article 95 provides an appropriate
legal base for the Regulation.
The Minister's letter of 14 May 2007
THE EXEMPTION
12.7 The Minister tells us that the Government's
main concern had been about the proposal to link the exemption
to a single hospital. He says:
"This approach seemed overly restrictive given
the evidence that hospitals may wish to collaborate in this highly
specialised area. Under the [revised] version of the exemption,
production would not need to take place in the hospital where
the product was used. The exemption would apply where advanced
therapy medicinal products were prepared on a non routine basis
and used within the same Member State in a hospital under the
exclusive professional responsibility of a medical practitioner
in order to comply with an individual medical prescription for
a custom made product. Member States would be responsible for
developing specific quality and pharmacovigilance requirements
that would apply under the exemption and for authorising manufacturing.
We believe that this outcome would provide a useful degree of
flexibility at national level in helping to manage regulatory
impact where hospitals are engaging in the crucial early developmental
stages of tissue engineering, while ensuring necessary public
health protection."
THE
GAP
12.8
The Minster says that:
"The
Government's preferred position was that where a product containing
tissues or cells is at the borderline of the medicines and devices
regime it should be regulated with appropriate health safeguards,
either as a medicine or as a device, on the basis of its principal
mode of action in accordance with existing regulatory arrangements
for determining the classification of medicine/medical device
borderline products. However, the majority of other Member States
took the view that where a product meeting the definition of a
medicinal product contains viable tissues or cells it should always
be regulated under the advanced therapy medicinal products Regulation.
This majority position was consistent with that taken by the European
Parliament."
THE
EUROPEAN PARLIAMENT
12.9
The Minister says that the European Parliament considered the
draft Regulation on 25 April. It adopted amendments which reflected
prior discussions with the German Presidency and the Commission.
The amendments are also acceptable to the UK Government. It appears,
therefore, that a first reading agreement can be reached between
the Council, the Commission and the European Parliament.
REGULATORY
IMPACT ASSESSMENT
12.10
The Minister encloses with his letter an initial Regulatory Impact
Assessment. It concludes that the main effect of the Regulation
would be to put in place a regulatory framework which will be
filled out by subsequent technical legislation and guidelines.
It is not yet possible, therefore, to quantify the costs to and
benefits for businesses. Moreover, the tissue engineering sector
is currently small but expected to grow as a result of scientific
and technological progress. The Minister says that:
"It
will therefore take a number of years for new, innovative products
to be developed and to come on to the market. As a result, it
will take some time before we will be in a position to make a
robust assessment of the economic impact."
FURTHER
CONSULTATIONS
12.11
The Minister also says that the main industry trade associations
and a range of organisations which represent patients support
the package of amendments for which the European Parliament voted
on 25 April. They "have expressed a strong view that the
opportunity should be taken to secure a first reading agreement
thereby ending the current regulatory uncertainty that is holding
back development".
COUNCIL
MEETING
12.12
Finally, the Minister says that the Government intends to support
the approval of the draft Regulation as amended by the European
Parliament when it is discussed by the Health Council on 30/31
May.
Conclusion
12.13 We are grateful to the Minister for his
comprehensive letters of 12 March and 14 May. He and his Ministerial
colleagues at the Department of Health have been assiduous in
giving us progress reports on the negotiations.
12.14 In the light of the Minister's explanations,
we can understand why the Government is willing to accept the
compromises that have been negotiated on "the exemption"
and "the gap". We note the Government's conclusion,
in the light of the ECJ's rulings, that Article 95 is an appropriate
legal base for the measure. We are, therefore, now content to
clear the draft Regulation from scrutiny.
29 Directive 2001/83/EC: OJ No. L 311, 28.11.01, p.67. Back
30
Commission Directive 2003/63/EC: OJ No. L 159, 27.6.03, p.46. Back
31
Directive 2004/23/EC: OJ No. L 102, 7.4.04, p. 48. Back
32
Regulation (EC) No. 726/2004: OJ No. L 136, 30.4.04, p.1. "Pharmacovigilance"
is the monitoring of the quality, safety and efficacy of marketed
medicines. Back
33
C-66/04. Back
34
C-217/04. Back
| 
