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Istituto Superiore di SanitàRome (Italy)
Avril Daly
Representing EURORDIS as a Partner in EUROPLAN
Chief Executive Officer of Fighting Blindness (Ireland)
Chair of the Genetic & Rare Disorders Organisation (GRDO)
13 May 2011, Eurordis General Assembly – Amsterdam
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
National Conferences
OBJECTIVES, METHODOLOGY and TOOLS
KEY FACTS & FIGURES and MAIN RESULTS
KEY MESAGES for each THEME
KEY QUESTIONS for PANEL DISCUSSION
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
EUROPLAN WP8
Title: “Promoting the recommendations for Rare Diseases National Plans and presenting the Commission Communication on Rare Diseases”.
WP leader: EURORDIS
Funding: 60% EC Contribution + 40% EURORDIS
Istituto Superiore di SanitàRome (Italy)
Commission Communication and Council
Recommendations on RD ensure that common policy
guidelines are shared everywhere in the EU
EUROPLAN Rrecommendations on RD National Plan within
EU MS link national efforts with a common strategy at EU level
Presenting the Commission Communication and Council Recommendations on RD
WP 8 objectives reflect this double combined approach
Reality check + Promoting the EUROPLAN
Recommendations on RD National Plans
EUROPLAN WP8 OBJECTIVES
Istituto Superiore di SanitàRome (Italy)
EUROPLAN WP8
EURORDIS supported the organisation of 15 National Conferences (instead10 planned in initial
contract) on Promoting National Plans or Strategies for Rare
Diseases based on the Commission Communication , the Council
Recommendations and EUROPLAN Recommendations and monitoring indicators
14 National Alliances and 1 Foundation for RDs, selected following a Call for Proposals
6 Advisors appointed by EURORDIS following an Expression of Interests: Simona Bellagambi, UNIAMO, Italy Britta Berglund, Rare Diseases Sweden, Sweden Avril Daly, GRDO, Ireland Dorica Dan, RONARD, Romania Mirjam Mann, ACHSE, Germany Christel Nourissier, Alliance Maladies Rares, France
Istituto Superiore di SanitàRome (Italy)
WP8 EURORDIS’ ADVISORS
MoU signed between EURORDIS and Advisors’ organisations where the reciprocal engagements are laid out
Advisors in charge of 2 to 3 national conferences
Liaise with Conference organisers / National Alliances: key contacts throughout the entire project
Liaise with EURORDIS and other Advisors to exchange best practices and difficulties
Supervise the Conference Agenda setting
Support to Conference Report drafting
Istituto Superiore di SanitàRome (Italy)
METHODOLOGY (1)
A methodology was developed for the organisation of the 15 National Conferences based on the following:
Promote Patient-run Multi-stakeholders approach Ensure that all interests at stake in each country were
involved in the debate that should eventually lead to advance the national policy on RDs
Promote EU & MSs integration + MSs exchanges Discuss the challenges of the transferability of the EU
Recommendations
A ‘supranational’ coordination was created while ensuring a close touch with the national situation (EURORDIS staff and 6 Advisors)
Istituto Superiore di SanitàRome (Italy)
METHODOLOGY (2)
Result-oriented / transferability and national situation Conducive to deliver suggestions to improve the existing
country policy on RDs, while analysing the transferability of European guidelines in the national system.
Common Framework for all Conferences. Same format for each conference Same layout and design of presentations Same documents downloadable on EURORDIS’ website Same format for final reports
Harmonised model of discussion, tackling the same issues, same concepts and terminology is strengthening the unity of RDs’ policy and its supporting community
Istituto Superiore di SanitàRome (Italy)
EUROPLAN CONFERENCESCOMMON FORMAT
Each National Conference is configured with Plenary Session and Workshops
Each Workshop is devoted to a specific Theme and reports to the plenary on that Theme
Conference organisers decided how each Theme and Sub-Theme is dealt with in the National Conference and on practical solutions or proposals emerged in that area
Therefore the Conference Final Report is based on the Workshops’ reports and cover the proposal emerged from each of the Workshops
Istituto Superiore di SanitàRome (Italy)
EUROPLAN NATIONAL CONFERENCES - THEMES
THEME 1: Methodology and Governance of a National Plan or National Strategy
THEME 2: Adequate Definition, Codification and Inventorying of Rare Diseases THEME 3: Research on Rare Diseases THEME 4: Standards of Care for RDs, Centres of
Expertise, European Reference Networks, Orphan
Drugs THEME 5: Patient Empowerment and Specialised
Services
Horizontal themes THEME 6: Sustainability of infrastructures and activities
in the field of RDs
THEME 7: Gathering the Expertise on Rare Diseases at the
EU level
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
www.eurordis.org National Rare Disease Policy
Istituto Superiore di SanitàRome (Italy)
TOOLS on EURORDIS’WEBSITEEUROPLAN & National Conferences
I. EU Policy Documents on Rare Diseases (EC Comm. RDs; Council Recos on RDs…)
II. EUROPLAN Guidance Documents for the National Plans (Recommendations, Indicators…)
III. EURORDIS Guidance Documents for the National Conferences: Conference Agenda Layout & Content outline Guidelines for WG Chairs and Rapporteurs Template Reports Conference Reports EURORDIS Policy Fact Sheets, Key Presentations,
etc…IV. EUROPLAN National Conferences’ Final Reports
and Conferences « Photo Wall »
Istituto Superiore di SanitàRome (Italy)
EUROPLAN NATIONAL CONFERENCE REPORTS
Each Final Report includes:
Report of the discussion and outcomes of the Working Groups (themes)An overall assessment of the usefulness of the EUROPLAN Recommendations for the advancement of a national strategy in the country.The transferability of the EUROPLAN Recommendations in your country.
This assessment results from the collection of the evaluation carried out at the end of each Workshop
and included in the Workshop reports…
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
15 National Conferences
KEY FACTS & FIGURES
MAIN RESULTS
KEY MESAGES by THEME
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEYS FACTS & FIGURES (1)
Istituto Superiore di SanitàRome (Italy)
KEYS FACTS & FIGURES (2)
COUNTRY CITY ORGANISERNUMBER of
PARTICIPANTSDATE ADVISOR
BULGARIA Plovdiv NAPRD National Alliance of People with RD
352 28-29-30 May Dorica Dan
CROATIA Dubrovnik Croatian Society for RD 180 17-18-19 September Christel Nourissier
DENMARK Copenhagen Rare Disorders Denmark 81 19 November Britta Berglund
FRANCE Paris Alliance Maladies Rares 153 30 September Christel Nourissier
GERMANY Berlin ACHSE 190 13-14 October Mirjam Mann
GREECE Athens PESPA 199 26-27 November Simona Bellagambi
HUNGARY Budapest HUFERDIS 138 15-16 October Dorica Dan
IRELAND Dublin GRDO 169 20 January 2011 Avril Daly
ITALY Florence UNIAMO 120 11-12-13 November Simona Bellagambi
NETHERLANDS The Hague VSOP 80 18-19-20 November Mirjam Mann
POLAND Krakow Foundation MATIO 64 22 October Christel Nourissier
ROMANIA Bucarest RONARD 178 18-19 June Dorica Dan
SPAIN Burgos FEDER 116 5-6 November Simona Bellagambi
SWEDEN Stockholm Sällsynta diagnoser 84 11 November Britta Berglund
UNITED KINGDOM
Manchester Genetic Alliance UK - Rare Diseases UK
86 16 November Avril Daly
Istituto Superiore di SanitàRome (Italy)
KEYS FACTS & FIGURES (3)
15 Conferences in a range of different countries:
Northern and Southern countries. New and old EU Members States, and even a EU
candidate country, Croatia. Countries of different sizes, from the largest EU
countries (Germany) to smaller countries like Denmark or Ireland.
All sorts of health systems were represented: from centralised ones (France) to systems with a greater degree of decentralisation (Italy, Spain, Germany and UK).
Spill-over effect in countries outside the scope of EUROPLAN Project: conferences in Austria, Finland, Switzerland, Russia, Armenia, Georgia.
Istituto Superiore di SanitàRome (Italy)
KEYS FACTS & FIGURES (4)
Over 2200 participants with representatives of main stakeholders groups (so distributed by % of attendance):
Public authorities: 14% (up to 22% in Spain)
Academics /researchers: 20% (up to 30% in Hungary)
Healthcare professionals: 14 % (up to 29% in France)
Industry: 10% (up to 22% in UK)
Patients: 39% (between 33% and 44%)
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
MAIN RESULTS about NATIONAL CONFERENCES (1)
1. Has effectively help present the two new EU key policy texts on RD - Commission Communication & Council Recommendation on Rare Diseases – reaching broader national audiences
2. Helped to make RD communities more aware that national efforts are “embedded” into a more comprehensive and long-term approach integrating EU and national levels.
3. Specific recommended actions and national measures have been integrated into the debate and the language of stakeholders participating to the Conferences .
4. Altogether, EUROPLAN Recommendations were broadly endorsed or validated and in many cases amended by the national audiences.
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
MAIN RESULTS about NATIONAL CONFERENCES (2)
6. There is a great variability among and within countries about the type of services provided to rare disease patients and the accessibility to these services. Hence, national conferences were modulated to propose a common format while, at the same time, leaving a certain degree of flexibility to the organisers to respond to the most pressing questions arising from their specific national context.
7. The EUROPLAN Conference provided an invaluable opportunity to have the all or most national stakeholders sitting around the same table to discuss the same agenda.
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
MAIN RESULTS about NATIONAL CONFERENCES (3)
8. The whole experience of EUROPLAN proved to be an outstanding awareness-raising exercise in the field of rare diseases at the national level and created momentum on Rare Disease National Plans across Europe.
9. This momentum should not be lost: the results of the EUROPLAN Conferences should converge into the work carried out in the country and serve as a “blue print” to develop/implement the National Plans.
10.To conclude: the National Conferences proved instrumental to achieve the expected outputs of the EUROPLAN project.
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. It is essential to concentrate policy actions on RDs around a National Plan (NP) or Strategy, to address specificities and to optimise resource and actions.
2. Integration of the measures of the NP into the national health system is crucial, as rare disease patients need to become part of the health and social system planning.
3. Mapping existing resources is a necessary process virtually in all countries.
4. Drawing up a NP should be an inclusive process i.e. a with a body responsible for the development and the implementation of the NP composed of all stakeholders including patients.
Area 1 – Methodology and Governance
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
5. NPs must transcend sectors, be comprehensive and include healthcare and social provisions.
6. NPs cannot function without resources attached. Sustaining the Rare Disease Strategy will be very important, particularly in terms of embedding the strategy into everyday healthcare services.
7. A monitoring system must be integrated into the Plan with a suitable representation of qualified patients and possibly using as a reference the EUROPLAN Indicators (to be refined based on experience).
Area 1 – Methodology and Governance
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. The EU definition of RDs is supported although with a few exceptions .
2. While awaiting ICD-11, it is suggested to introduce existing coding systems such as the ICD10 or the Orphanet code, possibly with cross-referring systems.
3. Day-to-day registration of rare diseases should be organised in such a way that the workflow, as far as possible, is integrated into existing administrative systems.
4. Patient registries sustainability must be achieved by optimising resources and reducing fragmentation and overlapping.
Area 2 – Definition, coding and inventorying
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. RDs must be a national priority in medical research and ad hoc national research measures should be dedicated to RDs.
2. RD research programmes must be supported primarily by adequate public funds, even though public-private partnerships were not excluded.
3. RD research should be multidisciplinary and cover all research areas, but chiefly focus on translational research.
4. Socio-economic research (i.e. research on quality of life, living conditions, etc.) was highlighted in most National Conferences as extremely important and should become a national priority.
Area 3 – Research on RDs
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
5. Qualified patients advocates must become fully-fledged research partners.
6. Centres of Expertise must play a pivotal role in closing the gap between research and care.
7. All National Conferences called for the creation of quality patient registries as a primary objective and a basic requirement in the development of RD research.
8. Multi-centre national and international studies are an absolute necessity for the organisation of clinical trials.
Area 3 – Research on RDs
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. Mapping out CoE (Centres of Expertise) is a necessary exercise, prior to any planning on CoE.
2. National Conferences called uniformly for common quality standards for the designation and the accreditation of CoE.
3. Proximity of care is a recurring theme with answers modulated according to the size of the country and the healthcare system existing therein.
– In smaller countries, CoE for all diseases is inappropriate, still valid for some diseases, when expertise exist + networks of medical experts could be established, while promoting cross-border cooperation at the same time.
– In larger countries or decentralised countries, vertical systems (local/regional/national) are preferable.
Area 4 – Centres of Expertise and European Reference Networks
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
4. Integration between medical and social levels must be strengthened further, with a necessary reinforcement of the social level.
5. Services acknowledged by Centres of Expertise as essential to provide to patients should be recognised and reimbursed.
6. Healthcare pathways are defined and adopted, based on best practices and expertise at national and international. “Case managers” should dedicate their attention to individual cases and follow them all along the healthcare pathway.
7. Mechanisms should be found to recognise and reimburse the interventions of paramedical and other specialist professionals involved in the care of RD patients.
Area 4 – Centres of Expertise and European Reference Networks
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
8. Promoting the participation of CoEs in European Reference Networks (ERN) should be integrated in the NP and become a quality criterion of the performance of each CoE.
9. Cross-border healthcare should be promoted, especially for small countries where the existing resources cannot cover all RDs and mobility of patients appears both as a need and a concern.
10. Screening policies must be supported by robust legal frameworks and by reliable implementation and evaluation. National Conferences are calling to extend the current neonatal screening programmes and to coordinate them.
Area 4 – Centres of Expertise and European Reference Networks
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. Activities to promote information and increase awareness on rare diseases to the public at large must be boosted.
2. Information sources for patients and professionals should be validated to ensure a consistent quality level.
3. To make healthcare professionals acquainted with rare diseases, training of professional doctors should start during the medical degree course and continue during their careers, with different degrees of specialisation.
4. Guidelines should be developed to align actions performed at different levels of care and by different healthcare professionals, with specific information for patients, families, caregivers, social workers and teachers.
Area 5 – Gathering expertise at EU level (Information and training)
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. Collaboration among EU countries and EU authorities is necessary to overcome the bottlenecks created by the scarce and fragmented expertise on orphan drugs at national level. The participation to the Common Scientific Assessment of the Clinical Added Value of ODs is widely supported as the right mechanisms to make this cooperation work in practice.
2. In order to manage compassionate provision of orphan drugs, systems of ‘temporary authorisations’ could be granted under certain conditions.
3. It is important to make compulsory at the national level the collection of data on the efficacy and tolerance of medicines under compassionate or early access and off-label use.
Area 5 – Gathering expertise at EU level (Orphan Drugs)
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. Firstly, patient empowerment is a holistic approach to each individual citizen.
2. Involvement of patient groups representatives in decision-making processes can be only achieved when patients are really enabled to participate to such processes. This means that patients need to be qualified to become fully-fledged partners in decision-making debates which affect them directly
3. The importance of specialised services for RD patients for patients and families must be recognised and financially supported by the community - in particular Respite Care Services and Therapeutic Recreative Programmes.
Area 6 – Patient Empowerment
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY MESSAGES by AREA
1. Need to devise strategies and mechanisms to recognise and integrate RD patients into existing social services (rehabilitation, access to social services and financial supports, integration into school and workplaces), while recognising their specificities and providing quality services in response to their needs.
2. Help lines should be included in the provisions of a NP on RD and financially supported to provide personalised information and orientation. Help lines should be set up according to quality standards, such as those developed in the framework of European Network of Help Lines.
Area 6 – Patient Empowerment
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
ACKNOWLEDGEMENTS TO
• EURORDIS Advisors:
Simona Bellagambi, Britta Berglund,
Avril Daly, Dorica Dan,
Mirjam Mann, Christel Nourrissier
• EURORDIS Staff:
Yann Le Cam as Chief ExecutiveValentina Bottarelli as our Project Manager in EuroPlanAriane Weinman as support to Managers & AdvisorsJustine Evans as Web Content ManagerPatrice Régnier as Finance Director
• The 14 National Alliances and Fundacio Matjo of Poland who organised National Conferences
• ISS colleagues and all EuroPlan Partners
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
PANEL DISCUSSION
KEY QUESTIONS FOR DISCUSSIONon
IMPACT and USEFULNESS of NATIONAL CONFERENCES
and SUGGESTIONS for 2012-2014
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY QUESTIONS FOR DISCUSSION
Were the national conferences useful:In promoting national plans in these 15 countries?
To disseminate and promote the understanding of the EU policy framework on rare diseases?
To bring all stakeholders together?
To do a reality check of EUROPLAN Recommendations?
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
KEY QUESTIONS FOR DISCUSSION
Overall, are the EUROPLAN Recommendations relevant, understood and well accepted?
Can we sort out a common core of recommendations and other recommendations which need to be further adapted to national situations?
Which lessons can we learn from these 15 conferences regarding adjustment national strategies on rare diseases according to country population size and GNP dedicated to health?
Istituto Superiore di SanitàRome (Italy)Istituto Superiore di SanitàRome (Italy)
EUROPLAN
THANK YOU!
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