9 Rare diseases
(a) (30171) 15775/08 COM(08) 679
+ ADDs 1 & 2
(b) (30172) 15776/08 COM(08) 726
+ ADDs 1 & 2
| Commission Communication —Rare Diseases: Europe's challenges
Commission staff working documents: impact assessment and summary of assessment
Draft Council Recommendation on European action in the field of rare diseases
Commission staff working documents: impact assessment and summary of assessment
|
| Legal base | (a) —
(b) Article 152(4) EC; co-decision; QMV
|
| Document originated | (Both) 11 November 2008
|
| Deposited in Parliament | (Both) 18 November 2008
|
| Department | Health |
| Basis of consideration | EM of 25 November 2008
|
| Previous Committee Report | None
|
| To be discussed in Council | No date set
|
| Committee's assessment | (Both) Legally and politically important
|
| Committee's decision | (Both) Not cleared; further information requested
|
Background
9.1 Article 152 of the EC Treaty requires a high level of protection
of human health to be ensured in the formulation and implementation
of all Community policies and activities. It also requires that
Community action on public health should complement national policies.
Moreover, the Community should encourage cooperation between Member
States; and Member States should coordinate their public health
policies and programmes. Article 152(4) authorises the Council
to adopt recommendations for the purposes of the Article. Article
152(5) expressly provides that Community action must fully respect
the responsibilities of the Member States for the organisation
and delivery of health services and medical care.
9.2 In 1999, the Council adopted a Decision to set
up an expenditure programme to support Community action on rare
diseases.[31] The third
Recital of the Decision defined a rare disease as one affecting
less than 5 people in 10,000. The programme ran from the beginning
of 1999 to the end of 2003 and had a budget of €6.5 million.
Grants from the budget were made to the development of a European
information network on rare diseases; to training in the detection,
identification, treatment and prevention of rare diseases; and
to the development of monitoring and early warning systems.
9.3 Support for work on rare diseases has been included
in all the EC's public health programmes since 2003, and successive
EC Framework Programmes for R&D have included provision for
research on rare diseases.
9.4 According to the Commission there are between
5,000 and 8,000 rare diseases and about 6% of the people in the
EU are affected by them at some point in their lives. Rare diseases
include, for example, disorders of the auto-immune system, genetic
disorders, rare cancers and some infectious illnesses.
Document (a)
9.5 By the nature of rare diseases, it is impossible
for any Member States to guarantee patients quick and easy access
to medical services which have the necessary knowledge, expertise
and equipment to diagnose and treat all rare diseases. Similarly,
specialised research facilities and scientists are scarce and
widely dispersed. The majority of Member States do not have even
a partial national plan on rare disease. The impact assessment
by the Commission's staff says that:
"There is probably no other area in public health
in which 27 national approaches could be considered as inefficient
and ineffective as with rare diseases. The reduced number of patients
[with] these diseases and the need to mobilise resources means
that the scale and nature of effective action requires action
at European level in support of the Member States … ."[32]
9.6 Accordingly, the Commission proposes document
(a), a Communication which sets out a Community strategy to help
Member States ensure that that rare diseases are efficiently and
effectively identified, prevented (if possible), diagnosed, and
treated. The Commission says that this will require:
- improvement of the identification
and classification of rare diseases and the dissemination of information
about them;
- a common approach by Member States to work on
rare diseases (helped by document (b), a draft Recommendation);
and
- European cooperation to improve access to high
quality care for rare diseases.
9.7 The Communication sets out the action the Commission
will take under each of those three headings. For example, under
the first heading, the Communication says that the Commission:
"will lead work with regard to rare diseases
within the process of revising the existing ICD (International
Classification of Diseases) in order to ensure a better codification
and classification of rare diseases."[33]
9.8 The Commission intends to create an EU Advisory
Committee on Rare Diseases to advise on the implementation of
the strategy; and will make a progress report to the Council and
European Parliament on implementation after five years.
Document (b)
9.9 Document (b) is the Commission's draft of a Recommendation
to Member States to:
- draw up national plans to ensure
that all patients with rare diseases have access to high quality
diagnosis, treatment and care;
- support national or regional databases, registries
and networks of information about rare diseases;
- identify research needs and research priorities
and foster participation by national scientists and laboratories
in EC-funded research projects on rare diseases;
- identify existing national centres of excellence
on rare diseases (also known as centres of expertise or reference
centres) and create new centres where necessary;
- foster the participation of national centres
of excellence in European networks;
- "ensure that national or regional centres
of expertise are based on a multidisciplinary approach to care
when addressing complex and diverse conditions such as rare diseases;
and promote the integration of medical and social levels within
the centres";[34]
- make national expertise available to help develop
EU guidelines and protocols on, for example, diagnostic tools
or screening the population for rare diseases;
- support the activities of patients' organisations
and ensure that patients and their representatives are fully involved
in the formulation of policies on rare diseases, including the
establishment and management of centres of excellence; and
- provide enough money to ensure that research
facilities, databanks, centres of excellence and other aids to
research and the treatment of rare diseases can be sustained in
the long term.
9.10 The draft Recommendation also invites the Commission
to make a report on the implementation of the Recommendation after
five years.
The Government's views on documents (a) and (b)
9.11 In her Explanatory Memorandum of 25 November
2008, the Minister for Public Health at the Department of Health
(Dawn Primarolo) tells us that, while the Government recognises
the value of sharing information and experiences, decision-making
needs to remain with the individual Member States. This is because
decisions need to be tailored to the particular circumstances
of the Member State's health system and, in the UK, should reflect
the priorities for health of the devolved administrations.
9.12 The Minister questions whether some of the provisions
of the draft Recommendation are consistent with the express requirement
in Article 152(5) EC that Community action should fully respect
the responsibilities of Member States for the organisation and
delivery of health services and medical care.
9.13 The Government does not object to the proposal
that Member States should have national plans on rare diseases.
But it would resist the inclusion in the Recommendation of provision
to harmonise rights of access to therapy in Member States because
it would unacceptably affect the autonomy of the devolved administrations
and Primary Care Trusts in England to decide local funding policies
and priorities.
9.14 The Government strongly supports the improvement
of the coding and classification of rare diseases and considers
that it will best be achieved through the next revision of the
World Health Organisation's International Classification of Diseases.
9.15 In the Government's view, the definition of
a rare disease as one prevalent in 5 out of 10,000 people is not
helpful. The Minister says that it might be sensible to lobby
for an additional category of "very rare" conditions,
prevalent in 0.1 in 10,000 people.
9.16 The Minister notes that the draft Recommendation
calls for the creation and maintenance of disease-specific databases
and registries. She comments that registries are expensive to
set up and run; so expectations of what Member States might do
should be modest and best use should be made of existing collections.
9.17 The Minister also tells us that the research
community considers that increases in research into rare diseases
should be financed through the EC's 7th R&D Framework
Programme, rather than through the proposed national plans.
9.18 The Government will be seeking detailed amendments
to some of the other provisions of the draft Recommendation.
Conclusion
9.19 We welcome the Minister's careful attention
to the question whether the proposed Recommendation complies with
the terms of Article 152(5) EC. We share her doubts. For example,
paragraph 4.4 of the draft Recommendation calls on Member States
to ensure that national or regional centres of excellence are
based on a multidisciplinary approach to care. To our mind, that
provision raises two questions. First, is it necessary or desirable
to make such detailed recommendations to Member States? And, second,
does paragraph 4.4 comply with the requirement in Article 152(5)
EC that Community action should respect the responsibilities of
Member States for the organisation and delivery of health services
and medical care?
9.20 We note the UK's other reservations about
the draft Recommendation and Communication.
9.21 We ask the Minister to tell us if other Member
States and the Council's Legal Service share her doubts about
the legal base for the draft Recommendation. We also ask her for
progress reports on the negotiations. Meanwhile, we shall keep
both documents under scrutiny.
31 Decision No. 1295/1999/EC: OJ No. L 155, 22.6.99,
p.1. Back
32
ADD 1, page 13, section 4.4. Back
33
Commission Communication, page 5, second sentence of section 4.2. Back
34
Draft Recommendation, page 6, paragraph 4(4). Back
| 
