ADVANCED THERAPY MEDICINAL PRODUCTS (15023/05)
Letter from Andy Burnham MP, Minister
of State, Department of Health to the Chairman
I am writing to update you on the negotiations
that are taking place in the Council on the European Commission's
proposals for a Regulation on advanced therapy medicinal products.
The committee will be aware that negotiations
in the Council commenced in January 2006 under the Austrian Presidency
and have continued under the Finnish Presidency. There has been
some progress in negotiations on a range of issues. These include,
for example, the transitional period. There is broad consensus
among Member States that a longer period of around four years
would be preferable to the two year period proposed by the Commission.
However, there are key issues which are fundamental to the Regulation
which have yet to be resolved and we expect to be progressed during
the Germany Presidency in the first half of 2007. The two substantive
issues which have featured in the negotiations and which are yet
to be resolved are the definition of the products to be covered
by the proposals including the overlap with the review of the
medical devices legislation which is currently under negotiation
in the Council and the proposed hospital exemption.
One of the key issues which remains unresolved
relates to the definition of products covered by the proposals
and the overlap with medical devices legislation. The committee
will be aware that the MHRA is leading for the UK on both sets
of legislative proposals and the objective remains to achieve
a coherent regulatory framework. I have written to you separately
about the Medical Devices Directives negotiations. In negotiations,
it was proposed that all products containing viable human or animal
tissues or cells should be regulated as medicines. Our position
is 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 on the basis of its principal
mode of action and intended purpose. This could allow for the
possibility of some products being regulated as devices. The position
of some Member States is fluid on this issue and there is some
support for the UK's position. There is a fairly strong degree
of support in the Council for opening up the medical devices regime
to products containing non viable, human tissues or cells. However,
there is less support in relation to viable tissues or cells.
We continue to pursue our negotiations across the two separate
working groups.
The other key issue which remains unresolved
relates to the proposed hospital exemption. This has raised a
number of complex issues in negotiations. Under the proposal,
as drafted, any advanced therapy medicinal product which is both
prepared in full and used in a hospital, in accordance with a
medical prescription for an individual patient would be exempt
from the new regulatory requirements. The committee will be aware
that the Government's position is that some form of exemption
is required. However, as drafted, we do not think the exemption
is satisfactory. Where the exemption as currently drafted would
apply, it is very wide in effect. On the other hand, linking the
exemption to a single hospital appears to us overly restrictive
given that hospitals are likely to co-operate in this highly specialised
area. Ideally, we would like to see an exemption that, while including
greater safeguards for patients, was not linked to institutional
arrangements but rather to the characteristics of the activity.
So far, we have not managed to garner significant support for
taking a non institutional approach. Overall, the views of Member
States are divergent. Some Member States want to widen the exemption
to cover situations where different hospitals work in collaboration.
Others want to limit the exemption in the interests of patient
safety and of establishing a level playing field between hospitals
and industry. There has also been a suggestion that a specific
exemption is not needed given that there are provisions in existing
European medicines legislation that could have the effect of exempting
some activity carried out in hospitals from aspects of medicines
regulation. No further discussions on the hospital exemption will
take place during the Finnish Presidency.
Given the fundamental issues that have yet to
be resolved in relation to the definitions and scope of the proposals
we are not yet in a position to revise the initial regulatory
impact assessment. The MHRA has continued to meet with interested
parties, including industry, hospital and academic research interests,
during the negotiations to brief them on progress and to seek
their views on emerging issues.
The committee asked about the Government's position
on the proposed legal base for the Regulation and will be aware
that our preliminary view is that we are now unlikely to have
any convincing arguments on legal base and may have to accept
that article 95 is appropriate in the case. We will confirm our
formal position on this in due course.
The committee may also be interested to know
that the European Parliament's Environment and Public Health (ENVI)
Committee voted on the rapporteur's draft report in September
2006. The committee voted against adopting the report (33 votes
against, 24 in favour and 1 abstention). Our understanding is
that there was resistance to the strong position on ethical issues
taken by the rapporteur. The rapporteur has drafted a new report
which will be considered by the ENVI committee in January 2007.
There are no amendments related to ethical issues in the revised
draft report we have seen. The European Parliament is expected
to vote in plenary on the proposals in February/March 2007.
A progress report on the discussions that have
taken place to date was presented to the Health Council on 30
November. The report highlighted the state of play in negotiations
on the key issues. I would be pleased to keep the committee informed
of significant developments in negotiations during the German
Presidency.
19 December 2006
Letter from the Chairman to Andy Burnham
MP
Thank you for your letter of 19 December 2006
which explained the latest position on negotiations relating to
the proposed Regulation. This was considered by Sub-Committee
G on 18 January.
In view of the implications for patient safety
and the public interest generally, we have a particular concern
about the suggested form of the exemption from the Regulation.
We find persuasive your arguments that an exemption should depend
upon the characteristics of a tissue engineering activity, rather
than on the specific institution where the activity is carried
out. We encourage you to pursue this issue in future negotiations.
We note your point that, because a number of
fundamental issues with the proposed Regulation have yet to be
resolved, it has not been possible to produce a revised regulatory
impact assessment (RIA).
You suggest that you may have to accept that
Article 95 is an appropriate legal base for the Regulation, but
that you will confirm the Government's position on this.
We look forward to hearing from you on the outcome
of current negotiations, to receiving a revised RIA when this
can be produced, and to your confirmation of your position on
the legal base. In the meantime we will retain the proposed Regulation
under scrutiny.
19 January 2007
Letter from Lord Hunt of Kings Heath,
Minister of State, Department of Health to the Chairman
I am writing to update you on recent progress
in the negotiations that are taking place in the Council on the
European Commission's proposals for a Regulation on advanced therapy
medicinal products.
Progress was made under the Finnish Presidency
in the second half of 2006 on a range of issues. The current German
Presidency has given priority to this dossier and there has been
a significant increase in momentum in recent weeks. In Andy Burnham's
letter of 19 December, he mentioned that there were two substantive
issues which had yet to be resolved; the proposed hospital exemption
and the scope of the Regulation/overlap with the scope of the
medical devices legislation (also currently under negotiation
in the Council). In recent weeks, the current Presidency has brought
forward proposals which seek to bring those two contentious issues
to a head.
The committee may also be interested to know
that the European Parliament's Environment and Public Health (ENVI)
Committee voted on the rapporteur's draft report on 30 January
2007. There were no amendments related to ethical issues voted
through by the ENVI Committee. The European Parliament is expected
to vote in plenary on the proposals on 13-14 March 2007. However,
it is possible that the plenary vote may be postponed depending
on the level of progress made in discussions between the Council,
the European Parliament and the European Commission.
The proposed hospital exemption has raised a
number of complex issues in the negotiations. Under the Commission's
original proposal, any advanced therapy medicinal product which
is both prepared in full and used in the same hospital, in accordance
with a medical prescription for an individual patient would be
exempt from the new regulatory requirements. The committee will
be aware that the Government had serious concerns about the original
text. Our fundamental concern was about linking the exemption
to a single hospital. This approach seemed overly restrictive
given the evidence that hospitals may wish to collaborate in this
highly specialised area. In negotiations we have made the case
for an exemption which would include greater safeguards for patients
but was not linked to specific institutional arrangements. Under
the latest proposal put forward by the Presidency (this was based
on one of the amendments put forward in the European Parliament),
the exemption would relate more to the nature of the activity
carried out as opposed to the institution where the product was
made. There would be quality requirements, yet to be specified
but likely to include compliance with good manufacturing practice
(GMP). It is not yet clear whether the majority of Member States
will support the current proposal given the views of Member States
have hitherto been divergent on this issue. From our perspective,
this approach would provide a positive basis from which to move
forward though the text would need to be modified.
The other key substantive issue which remains
unresolved relates to the scope of the proposed Regulation and
the overlap with medical devices legislation. The committee will
be aware that we have advocated in negotiations 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 on the basis of its principal mode of action. This
approach could allow for the possibility of some products being
regulated as devices. Although there has been some support for
our position, the majority of Member States and the European Commission
have all taken the view that where products contain viable tissues
or cells, they should be regulated as advanced therapy medicinal
products. The ENVI Committee recently voted through amendments
consistent with this approach. On the other hand, there is, however,
a fairly strong degree of support in the Council for opening up
the medical devices regime to products containing non viable human
tissues and cells, in which case such products would be classified
either as medicines or as devices according to the principal mode
of action. This would bridge a significant regulatory gap. We
will press for this to be addressed in the negotiations. It now
seems likely that this approach may form the basis for reaching
a compromise on this important issue.
Given the fundamental issues that have yet to
be resolved in relation to the definitions and scope of the proposals
it has not yet been possible to revise the initial regulatory
impact assessment. Moreover, the current scale of activity in
this area is relatively small and developmental in nature so it
has proved difficult to date to gauge the likely regulatory impact
on those involved in tissue engineering. The proposed Regulation
would not change the definition of a medicinal product and so
the main effect of the Regulation for products affected would
be to modify the normal requirements of the medicines regulatory
regime to put in place more tailored requirements reflecting the
nature of the product. This also makes any quantification problematic.
The MHRA has continued to meet with interested
parties, including industry, hospital and academic research interests,
during the negotiations to brief them on progress and to seek
their views on emerging issues. We know from our discussions with
stakeholders that some operators total output of products/procedures
on an annual basis does not exceed double figures. Under the proposals,
the Commission would publish a general report on the application
of the Regulation within five years of its entry into force. This
would include comprehensive information about the different types
of advanced therapy products authorised under the Regulation.
The report would also assess the impact of technical progress
on the application of the Regulation.
The committee asked about the Government's position
on the proposed legal base. In relation to the proposed legal
base for the Regulation, the Government has now reached a formal
position. We accept that the proposals for a centralized procedure
relating to advanced therapy products and for the establishment
of a new Committee for Advanced Therapies will improve the conditions
for the functioning of the internal market and lead to further
harmonization in the field of medicinal products. Having regard
to the judgments of the European Court of Justice in Case C-66/04
UK v Parliament and the Council ("the smoke flavourings case")
and Case C-217/04 UK v Parliament and the Council ("the ENISA
case"), therefore, we now accept that Article 95 is the appropriate
legal base for this measure.
The current Presidency has indicated that a
first reading agreement is a possibility on this dossier. I will
therefore continue to keep the committee informed of significant
developments in negotiations as it is possible that we may need
to move quickly if a compromise package emerges.
12 March 2007
Letter from the Chairman to Lord Hunt
of Kings Heath
Thank you for your letter of 12 March providing
an update on negotiations relating to this document during the
German Presidency. This was discussed by Sub-Committee G (Social
Policy and Consumer Affairs) at their meeting on 29 March.
As I noted in my letter of 19 January, we have
sympathy with the Government's view that any exemption from the
Regulation would much more appropriately be based upon the nature
of the activity in question rather than upon the institution where
it was carried. We therefore welcome the news that the European
Parliament suggested an amendment to the proposed Regulation to
introduce this approach, and that the Presidency has put this
forward in the latest text of the draft Regulation.
We note that the Government now accepts that
Treaty Article 95 does provide an appropriate legal base for the
proposed Regulation. We note also that further issues need to
be clarified before a revised Regulatory Impact Assessment (RIA)
can be produced. However, although it may be difficult to produce
this for the reasons you advance, we do emphasise that to do so
is an essential piece of work. Even if it is possible to make
only inexact estimates of impact, these should be compiled systematically
in an RIA together with information about the limitations of the
analysis. Please would you send us a copy of the revised RIA when
this has been completed.
We are encouraged that the German Presidency
has indicated that a first reading agreement is now possible on
this dossier and we ask you to continue to keep us informed about
progress. In the meantime we clear this document from scrutiny.
29 March 2007
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