proposal for a commission communication to the european ... · the 4th european conference on rare...

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Proposal for a Commission Communication to the European Parliament, the Council, the Economic and

Social Committee and the Committee of the Regions on a European action on rare diseases

Paris, 13-14th October 2008

Antoni MontserratEuropean Commission

DG SANCO Health Information Unit

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Legal basis for the developments of the EU Public Health Policy

Based on Article 152 of the EU Treaty

A Community action programme on RD, including genetic diseases, was adopted for the period of 1 January 1999 to 31 December 2003 with the aim of ensuring a high

level of health protection in relation to RD. As the first EU effort in this area, specific attention was given to improving knowledge and facilitating access to information

about these diseases.

For the period 2008-2013 the Commission has adopted the White Paper COM(2007) 630 final “Together for Health: A Strategic Approach for the EU 2008-2013” of 23

October 2007 developing the EU Health Strategy. Actions under Objective 1 of this EU Strategy cover a Communication on European Action in the Field of Rare Diseases and

in point 4.1 of this EU Strategy it is suggested to put forward EC-level structured cooperation mechanisms to advise the Commission and to promote cooperation

between the Member States.

As a consequence Rare diseases are now one of the priorities in the EU Health Programme 2008-2013. According to the DG SANCO Work Plans for the

implementation of the Public Health Programme, the two main lines of action are the exchange of information via existing European information networks on rare diseases, and the development of strategies and mechanisms for information exchange and co-

ordination at EU level to encourage continuity of work and trans-national cooperation.

http://eur-lex.europa.eu/pri/en/oj/dat/2002/l_271/l_27120021009en00010011.pdf

http://eur-lex.europa.eu/pri/en/oj/dat/2002/l_271/l_27120021009en00010011.pdf

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EU Projects creating networks of action for rare diseases

1. A priority for action is to guarantee the exchange of information via existing European information networks on rare diseases,

2. Registries and databases constitute a strong priority to guarantee patient's information of quality and a solid information basis permitting an efficient monitoring, research and knowledge management of all rare diseases.

3. To promote better classification, 4. To develop strategies and mechanisms for exchanging

information between people affected by a rare disease, volunteers and professionals,

5. To define relevant health indicators 6. To develop comparable epidemiological data at EU level, 7. To support an exchange of best practise and develop measures

for patient groups.

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EU Projects creating networks of action for rare diseases

A solid benchmarking could be established with successful EU Public Health Programme ongoing projects, having World relevance in the area [ORPHANET, EUROCAT (Surveillance of congenital anomalies in Europe), ENERCA (European Network for Rare Congenital Anaemias, or EAIS (European Autism Information System)] with

EU FP6 ongoing projects [EUROWILSON, RBDD (Rare Bleeding Disorders Database) or EUROSCA for ataxias].

These project outputs should also supports specific international consensus conferences such as the Consensus Conference on Primary Immunodeficiency in 2006 or the European Haemophilia Consortium Conferences.

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DG SANCO priorities on rare diseasesPilot reference networks (centres of reference) for rare diseases

DG SANCO has established the High Level Group on Health Services and Medical Care as a means of taking forward the recommendations made by the reflection process on patient mobility. One of the Working Groups of this High Level Group refers to reference networks (centres of reference).

In 2006 the Rare Diseases Task Force Working Group on centres of reference has submitted a report ‘Contribution to policy shaping: For a European collaboration on health services and medical rare in the field of rare diseases’ updating the information about Centres of Reference in Europe. The report details the use of the concept of centres of reference in Europe as well as the respective functions.

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Pilot reference networks (centres of reference) for rare diseases

The suggested criteria to be fulfilled by the European reference networks are:

Sufficient activity and capacity to provide relevant services and maintain quality of the services providedCapacity to provide expert advice, diagnosis or confirmation of diagnosis, to produce and adhere to good practice guidelines and to implement outcome measures and quality controlDemonstration of a multi-disciplinary approach;High level of expertise and experience documented through publications, grants or honorific positions, teaching and training activitiesStrong contribution to researchInvolvement in epidemiological surveillance, such as registries Close links and collaboration with other expert centres at national and international level and capacity to network Close links and collaboration with patients associations where they exist. Appropriate arrangements for referrals of patients from other Member States established within a framework. Appropriate capacities to diagnose, to follow-up and manage patients with evidence of good outcomes so far as applicable.

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Pilot reference networks (centres of reference) for rare diseases

The Work Plan 2006 for the implementation of the EU public health programme, introduces as a priority in the area of rare diseases: to develop European Networks of Centres of

Reference for Rare Diseases. According to this priority some Projects have been selected for funding:

European Centres of Reference Network for Cystic Fibrosis with the Klinikum der Johann Wolfgang Goethe-Universität (DE) as Project Leader, European Network of Centres of Reference for Dysmorphology with The University of Manchester (UK) as Project Leader, Patient Associations and Alpha1 International Registry with the Stichting Alpha1 International Registry (NL) as Project Leader, European Porphyria Network: providing better healthcare for patients and their families with the Assistance Publique - Hôpitaux de Paris (FR) as Project Leader, Establishment of a European Network of Rare Bleeding Disorders, with the Università degli Studi di Milano (IT) as Project Leader.European network of paediatric Hodgkin’s lymphoma – European-wide organisation of quality controlled treatment with the University of Leipzig (D) as Project Leader.European Network of Reference for Rare Paediatric Neurological Diseases (NEUROPED) with the European Network for Research on Alternating Hemiplegia (AT) as Project Leader.A reference network for Langerhans cell histiocytosis and associated syndrome in EU with Assistance Publique Hôpitaux de Paris (FR) as Project Leader.

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The European Commission Task Force on Rare Diseases

The Task Force is assisted by a Scientific Secretariat which was set up to contribute to the development of public health action in the field of rare diseasesThe main objectives of the Task Force on Rare Diseases are to identify morbidity and mortality indicators for rare diseases, set up a common framework in the field of public health for rare diseases, and to produce an electronic newsletter. To better emphasize the importance of and action in the field of rare diseases, the European Commission set up an advisory structure: the Task Force on Rare Diseases (RDTF).The RDTF publishes a monthly electronic newsletter on the EC's Rare Diseases actions: ORPHANews Europehttp://www.orpha.net/actor/EuropaNews/2006/060316.html36 members representing EU Projects (Health Programme and FP7) and Member States.

http://www.orpha.net/actor/cgi-bin/OAhome.php?Ltr=EuropaNews

http://www.orpha.net/actor/EuropaNews/2006/060316.html

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Revision of the International Classification of Diseases (ICD)

The WHO has launched the process of revision of the International Classification of Diseases (ICD) -10 to prepare the new ICD-11 which should be ready around 2015. The EC is very involved on the process from the side of the Rare Diseases.

Discussions on the revision and improvement of the ICD will be also launched for the mental health disorders.

The EU Task Force on Rare Diseases recognised as WHO Advisory Group on Rare Diseases.

5 860 rare diseases listed in Orphanet but only 250 having a code in the ICD-10

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A proposal for a Communication and a proposal for a Council Recommendation on rare diseases

There is probably no other area in public health in which 27 national approaches could be considered to be so inefficient and ineffective as with rare diseases. The reduced number of patients for these diseases and the need to mobilise resources could be only efficient if done in a coordinated European way.

Article 152 provides for the adoption by qualified majority by the Council of Recommendations, on the basis of Commission proposals, for the purposes set out in that article.

These Recommendations are the only legislative tool provided for in Article 152 on public health except for the few areas where measures or incentive measures may be adopted (see Article 152.4).

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DG SANCO priorities on rare diseasesA proposal for a Communication and a proposal for a Council

Recommendation on rare diseases

Recommendations are without legal force but are negotiated and voted on according to the appropriate procedure. Recommedations differ from regulations, directives and decisions, in that they are not binding for Member States. Though without legal force, they do have a political weight.

The Recommendation is an instrument of indirect action aiming at preparation of legislation in Member States, differing from the Directive only by the absence of obligatory power.

In order to coordinate the position of the Member States in respect of this important field, it is considered that a Recommendation is the appropriate legal instrument.

In the case of the proposed Communication on rare diseases, it is considered necessary to accompany that Communication with a Proposal for a Council Recommendation on rare diseases.

http://en.wikipedia.org/wiki/Legal_force

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DG SANCO priorities on rare diseasesA proposal for a Communication and a proposal for a Council Recommendation on rare

diseases

After the preparation of the First Draft of the Public Consultation on a European Action in the Field of Rare Diseases, the first presentation of the strategic orientations of this Consultation took place in the EU Task Force on Rare Diseases on 20 June, 2007.

The RDTF decided to appoint a Drafting Group to support the European Commission on a voluntary basis in the preparation of the text for the Public Consultation. This Drafting Group consisted of six experts in the field of rare diseases. The Drafting Group met on 11 July and 15 October, 2007.

The First Draft of the Public Consultation on a European Action in the Field of Rare Diseases prepared by the Drafting Group and the European Commission was submitted for discussion with the most relevant stakeholders in some relevant meetings.

The 4th European Conference on Rare Diseases was organised in Lisbon on 26-28 November, 2007, under the Portuguese Presidency of the Council. During the conference, the Public Consultation regarding European Action in the Field of Rare Diseases was officially launched by Andrzej Ryś

(Director of Public Health at the European Commission) in the opening speech of the Conference.

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In total, 584 responses were received, including contributions from 15 Member States. Key outcomes were that stakeholders want a comprehensive approach to the rare diseases problem in the EU. All the answers consider that the national or regional level is insufficient to offer alternatives to the problem. The EU level is retained in a 100% of answers as the most appropriate. There was a general support for a new overarching, strategic and coherent framework for rare diseases policy in the near future. The vast majority supported the ten broad priorities proposed by the Commission.

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To improve information, identification and knowledge on rare diseasesTo support implementation of National Plans for Rare DiseasesTo improve prevention, diagnosis and care of patients with Rare DiseasesTo develop national/regional centres of expertise and establish EU reference networks To ensure equal access to all EU patients to orphan drugsTo gather at European level the limited and scattered expertise on rare diseases.To accelerate research and developments in the field of Rare Diseases and Orphan DrugsTo empower patients with Rare Diseases at individual and collective levelTo develop the international cooperation on rare diseasesTo coordinate the policies and initiatives at EU level

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Actions to improve information, identification and knowledge on rare diseases

The EU definition of rare disease based on a prevalence of less than 5 per 10,000 is maintained An EU or an international project exploring an incidence based definition of rare diseases will be launchedThe EU will contribute to the ongoing process of revision of the ICD (International Classification of Diseases) in order to ensure appropriate codification and classification of rare diseases in the future ICD-11. A working group will be supported for all the period of this revision The EU will establish since 2009 an Inventory of Rare Diseases to be periodically updated The database Orphanet will be supported using appropriate financial instruments from the Health Programme or the FP7 The support to the disease information networks through the Health Programme and the FP7 should be pursued

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Action to support implementation of National Plans for Rare Diseases

The Member States are invited to establish national or regional action plans for RD in order to implement the actions suggested in the Commission Communication and the Council Recommendation and to provide an annual report on the progress made toward this objective

The Commission will provide European guidelines for the elaboration of these action plans for RD (EUROPLAN Project selected for funding on 2007). Appropriate international conferences will be organised (18th November 2008 in Paris)

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To improve prevention, diagnosis and care of patients with Rare Diseases

Development of e-Health in the field of RD using on-line and electronic tools Creation of a help line unique EU-wide number for information and social services on rare diseases a 116 number An evaluation of possible population screening (including neonatal screening) strategies for Rare Diseases will be launched To launch a European series Patient Leaflets on some rare diseases in all the EU languages Provide support toward harmonization of quality testing and counselling for rare genetic diseases through initiatives such as EuroGentest

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Actions to develop national/regional centres of expertise and establish EU reference networks

To repertory in an EU list the existing Centres of Expertise identified throughout the Member States by the end of 2010;To establish a procedure for designation and accreditation methodology of EU Reference Networks for Rare Diseases (according to the future Directive on Cross- border health care);To provide adequate, long-term public funding to Centres of Expertise in order to ensure their sustainability and continuity of care for patients;To provide adequate, long-term public funding to European Reference Networks Centres of Expertise in order to ensure their sustainability and continuity of care for patients;To recommend inclusion in the National Plan for Rare Diseases provisions on the recognition and funding of Centres of expertise and their participation in European Reference Networks;To recommend the adoption of national initiatives in the National Plans for Rare Diseases on specialised social services;To provide financial support to networks of specialised social service;

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Actions to ensure equal access to all EU patients to orphan drugs

To explore additional incentives at national or European level to strengthen research into rare diseases and development of orphan medicinal products, and Member State familiarity with these products A European guideline should be developed clarifying responsibilities in a Compassionate Use situation (Commission, EMEA)The Commission should present, a report to the Council and the Parliament identifying bottlenecks on orphan drugs access (delays, marketing, access, reimbursement, prices, etc.) every two years, proposing the necessary legislative modifications in order to guarantee equal access to orphan drugs throughout the EU on the basis of a COMP/EUACRD European collaborative scientific assessment (Commission, EMEA)A method for the assessment of the Therapeutic Added Value of Orphan Drugs should perform a common scientific assessment of the TAV for each Orphan Drug and deliver an opinion document (Commission, EMEA)

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Actions to gather at European level the limited and scattered expertise on rare diseases

The Health Programme and the FP7 will continue to support, in a coordinated way, registries, databases and biobanks on rare diseases with appropriate financial tools for a sustainable funding when necessary The Commission will establish, by Commission Decision, a publicly accessible EU Register of Rare Diseases patient registers databases and biobanks defining criteria for register accreditation and qualification and the access to data or samples. EU registering will become mandatory for publicly funded or co-funded repositories under the Health Programme and the FP7 Specific support to further research into biomarkers should also be given to encourage long-term follow-up, and the acquisition of robust evidence on clinical effectiveness

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Actions to accelerate research and developments in the field of Rare Diseases and Orphan Drugs

The EU Advisory Committee on RD and the Committee for Orphan Medicinal Products (COMP) in the EMEA (European Medicines Agency) will address to the FP7 biannual recommendation on research priorities on RDTo create an adequate EU mechanism through the FP7 to specifically support the clinical research development of potential orphan medicinal products up to the end of phase II and to the specific Genetic and molecular characterisation for the more than 4 000 diseases when feasibleTo launch the creation of a public-private foundation for RD, the European Research Foundation for Rare Diseases

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Actions to empower patients with Rare Diseases at individual and collective level

The Health Programme will continue to integrate the support to the patient’s organisations as a priority for action (Commission, Member States)

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Actions to develop the international cooperation on rare diseases

An international cooperation framework on rare diseases with other countries (e.g. US, Canada, Japan, Singapore, Australia,…) will be adopted (Commission)

A proposal of resolution on an international action in the field of rare diseases will be submitted by the European Commission to the World Health Assembly (Commission)

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Actions to coordinate the policies and initiatives at EU level

An EU Advisory Committee on Rare Disease (EUACRD) will be created, by Commission Decision, in order to advice the European Commission. Members should be nominated by the national authorities. Experts leading Health Programme and FP initiatives should also be members. Observers from the patient’s organisations and from the industry will also be proposed.

The European Conferences on Rare Diseases will be organised every two or three years. Funds will be provided by the Health Programme (Commission).

A Rare Diseases Fund could be created (?) in the Financial Perspectives (2014-2020) (Commission).

A feasibility study for the best governance practices of Rare Diseases at EU level will be launched in 2009 by the Health Programme (Commission).

Every four years the Commission should produce an Implementation report on the Commission Communication and Council Recommendation addressed to the Council, the Parliament, the Social and Economic Committee and the Committee of the Regions (Commission)

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June 2007: First Draft in consultation with Task Force

August 2007: Final Draft

September 2007: Process of consultation starts with specialised bodies

November 2007-February 2008: Public consultation

March/June 2008: Impact Assessment

August 2008: Adoption of Communication and Recommendation Drafts

Octobre 2008: Interservice Consultation in the European Commission

5th November 2008 (?): Adoption by the Commission

December 2008: Start of discussion in the European Parliament, Council, Economic and Social Committe and Committe of the Regions

First Semester 2009: Expected adoption of the Recommendation by the Czech Presidency of the Council

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DG SANCO priorities on rare diseasesWeb site

http://ec.europa.eu/health/ph_threats/non_com/rare_diseases_en.htm

http://ec.europa.eu/health/ph_threats/non_com/rare_diseases_en.htm

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