SHARE: results and what to come next !

1. SHARE: results and what to come next!
Bas Vastert and Nico Wulffraat,
pediatric rheumatologists, PhD
ENCA Meeting, PRES Congress Genua, 2016

2. - Background
- Results up to 2016
- Next steps and challenges
Outline

3. SHARE: Single Hub and Access point for paediatric Rheumatology in
Europe
EU (EAHC) funded project running from 09-2012 until 12-2015
Aiming to improve care and research collaborations for paediatric
rheumatology in Europe and beyond
Background

4. OBJECTIVES of the SHARE project
• Identifying specific needs in each EU country
• Best practices for diagnosis and treatment of PRD
• Create central platform for exchange of information,
data and samples
• Identify best practices for obtaining ethical consent
• Implementation of results into training programmes
• Involve parents/patients as stakeholders

5. • WP4: Addressing the need for health care for PRD
throughout Europe
• WP5: development of best practices on diagnosis and
treatment for PRD
• WP6: Reinforcing the network (patient information / website)
• WP7: Barriers between nations for data collection and data
sharing
Output workpackages

6. WP4: Addressing the need for health care for PRD throughout
Europe
Output:
- 1-2 manuscripts describing the current situation in Europe
Output workpackage 4

7. WP4: Addressing the need for health care for PRD throughout
Europe
- Providing the opportunity to zoom in on individual countries
- Influence health care authorities
- Collaborate with national patient organisations
Opportunities workpackage 4

8. WP4: The need for optimal care in Europe
•3 surveys: National PRINTO co-ordinators/PReS
country representatives, Centre directors and individual
members
WP6: Reinforcing the network (patient information and
participation)
•Extensive update of network and information
•Patient Survey prepared by ENCA and Wulffraat
•Circulated by parent organisations with help of centers
•Data collection Web based at Printo website
WP4: Doctors view
WP6: Patients view

9. WP6 patient survey on PRD across Europe
Topics in patient survey
•Referrals
•Paediatric rheumatology Team and Cooperation
• Transition of care
• Use of webportals, help lines, medical reports
• Access to (expensive) medication
• Pain management
• Participation in research
• Financial aspect
• School issues
• Special rehabilitation and employment issues

10. Patient survey respondents (n=457), per
country of origin

11. Patient survey respondent, country of origin
Replies pooled per region: WEST-EAST and North-South
No replies from: Bulgaria, Croatia, Greece, Poland,
Rumenia, Bosnia, Estonia

12. Patient Demographics
JIA Subtype

13. Was your child referred to a paediatric rheumatologist within 6
weeks of reporting the initial symptoms to your doctor?

14. How long was it between referral
to the paediatric rheumatologist
and the first clinic appointment?

15. WP5: doctors survey Acces to (pediatric)
rheumatologists
percentage of PRD patients in your country managed by general paediatricians?
West Europe East Europe Latin America Other Countries Total p-value
N 14 8 3 8 33 0.12
< 25% 8 (57.1%) 2 (25.0%) 1 (33.3%) 4 (50.0%) 15 (45.5%)
25-50% 5 (35.7%) 4 (50.0%) 0 (0.0%) 1 (12.5%) 10 (30.3%)
> 50% 0 (0.0%) 1 (12.5%) 0 (0.0%) 2 (25.0%) 3 (9.1%)
not known 1 (7.1%) 1 (12.5%) 2 (66.7%) 1 (12.5%) 5 (15.2%)
percentage of PRD patients in your country managed by adult rheumatologists?
West Europe East Europe Latin America Other Countries Total p-value
N 14 8 3 8 33 0.11
< 25% 12 (85.7%) 7 (87.5%) 1 (33.3%) 3 (37.5%) 23 (69.7%)
25-50% 0 (0.0%) 1 (12.5%) 1 (33.3%) 3 (37.5%) 5 (15.2%)
> 50% 0 (0.0%) 0 (0.0%) 0 (0.0%) 0 (0.0%) 0 (0.0%)
not known 2 (14.3%) 0 (0.0%) 1 (33.3%) 2 (25.0%) 5 (15.2%)
percentage of PRD patients in your country are managed by paediatric rheumatologists?
West Europe East Europe Latin America Other Countries Total p-value
N 14 8 3 8 33 0.10
< 25% 0 (0.0%) 0 (0.0%) 0 (0.0%) 3 (37.5%) 3 (9.1%)
25-50% 4 (28.6%) 2 (25.0%) 1 (33.3%) 1 (12.5%) 8 (24.2%)
> 50% 10 (71.4%) 5 (62.5%) 1 (33.3%) 3 (37.5%) 19 (57.6%)
not known 0 (0.0%) 1 (12.5%) 1 (33.3%) 1 (12.5%) 3 (9.1%)

16. WP5: development of best practices on diagnosis and
treatment for PRD
Output:
- 8-10 manuscripts for best practices in each PRD (+ periodic
fevers)
- Providing the first international, multi-disciplinary consensus
and evidense based diagnostic and treatment guidelines for
JDM, childhood SLE, childhood vasculitides!
- Providing opportunities for professionals and patient
organizations to improve national / local acces to
care/ expert advise / new therapies
Workpackage 5

17. Methods literature review
Date literature review: June 30th
, 2013

18. - Paper selection on predefined criteria
- JIA: 172 papers
- JDM: 108
- SLE/APS: 128 / 15
- Scleroderma: 52 / 37
- All papers scored by 2 experts independently on validity and level
of evidence. In case of disagreement: 3rd expert
- Results grouped and developed into recommendations
- Web-based survey to pre-test recommendations
Methods: from evidence to recommendations

19. Methods: consensus finding
• Nominal group technique
• Consensus reached when ≥ 80% agreement
• Focused on evidence based recommendations

20. Selection cross-cutting statements
1. Clear guidelines of referral to Paediatric Rheumatology are
needed for all centers in fostering the prompt and early
diagnosis and management of PedRD.
2. Children with rheumatic disease should be cared for by
peadiatric rheumatologists. This may be done by means of
shared care, which must include a good mechanism for
communication between teams.
1. Patients should be seen by a paediatric rheumatologist and,
depending on the clinical presentation of the PRD, members
of a multidisciplinary team. This team should consist as a
minimum of a nurse, specialised in PRD, a physio /
ocupational therapist and acces to a psychologists or
psychosocial worker

21. Selection JIA specific statements:
general aspects
1. New JIA patients should be seen in a specialised
center for PRD within 4 weeks after referral.
2. New JIA patients and those started on a new therapy,
should be reviewed within 2-3 months after initiation
of therapy to evaluate compliance, side effects and
progression of disease.
3. Response to ongoing therapy should be assessed
every 3-6 months, preferably by using standardized
disease activity tools, for example the JADAS.

22. How to get access and reimbursement for therapies for PRD?
Dutch situation:
1: EMA registration: automatically in the process for
negotiating reimbursement
2: Off label: for example rituximab for JDM
“advise” needed from the scientific community..
=> SHARE fulfills the criteria for such advise!
Subsequently:
Negotiations start with health care authorities, pharma
and insurance companies on pricing /reimbursement
Opportunities WP5

23. WP 6 Reinforcing the network
Extensive Update of PRINTO website
•Update of patient information
•Translation into EU languages
•Engaging active patient participation (ENCA)
Output and opportunities WP6

24. WP7: barriers between nations for data
collection and sharing
(1) to identify best practices for ethical consent, data and sample
collection
(2) to improve quality assurance in data and sample collection
(3) to identify ethical issues on data and sample collection and
the exchange of these data and samples between European
centres for carrying out research

25. Roadmap: Identifying barriers between nations for data
and specimen collection and sharing
Proposal for an
international
collaborative
research project
Recommendations for
international
collaborative research
in pediatric diseases
Evidence synthesis part 1:
Systematic literature review
Evidence synthesis part 2:
Practical evidence gathering
 Test proposals Germany/Europe
 Interviews with Ethics Boards
 Research partner barriers - survey
 Patient related barriers - survey
Legal
Barriers
Process
barriers
Patient
related
barriers
Research
partner
barriers

26. Output WP7: barriers between
nations for data collection and
sharing
-Multiple papers on data sharing, sample sharing,
international collaboration, differences in procedures
for obtaining ethical approval for new studies
- Influence European policy makers in science and
health care to critisize current differences in ethical
approval bodies throughout Europe

27. The SHARE project provides:
- mapping of the current situation in EU from both doctor and
patient perspective (WP 4)
- recommendations for diagnosis and treatment of PRD and
thereby facilitate improvement and uniformity of care for PRD
patients throughout Europe and beyond (WP 5)
- guidance for diagnosis and treatment, the best practices can,
and need to, be used to improve acces to treatment and care
in individual countries (WP 5)
- an update and improvement of patient information via the
renewed website (WP6)
- opportunities to take away hurdles for international
collaboration in research (WP 7)
Conclusions

28. questions
?