SHARE: results and what to come next!

Bas Vastert and Nico Wulffraat, pediatric rheumatologists, PhD

ENCA Meeting, PRES Congress Genua, 2016

- Background

- Results up to 2016

- Next steps and challenges

Outline

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

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

• 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

WP4: Addressing the need for health care for PRD throughout Europe

Output: - 1-2 manuscripts describing the current situation in Europe

Output workpackage 4

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

WP4: The need for optimal care in Europe•3 surveys: National PRINTO co-ordinators/PReS country representatives, Centre directors and individual membersWP6: 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

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

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

Patient survey respondent, country of origin

Replies pooled per region: WEST-EAST and North-SouthNo replies from: Bulgaria, Croatia, Greece, Poland, Rumenia, Bosnia, Estonia

Patient DemographicsJIA Subtype

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

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

WP5: doctors survey Acces to (pediatric) rheumatologistspercentage 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%)

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

Methods literature review

Date literature review: June 30th, 2013

- 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

Methods: consensus finding

• Nominal group technique• Consensus reached when ≥ 80% agreement• Focused on evidence based recommendations

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

Selection JIA specific statements:general aspects1. 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.

How to get access and reimbursement for therapies for PRD?

Dutch situation:1: EMA registration: automatically in the process for negotiating

reimbursement2: 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

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

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

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

LegalBarriers

Process barriers

Patient related

barriers

Research partner barriers

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

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

questions

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