Recommendations for collaborative paediatric research including biobanking in Europe: a Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) initiative

Abstract

Innovative research in childhood rheumatic diseases mandates international collaborations. However, researchers struggle with significant regulatory heterogeneity; an enabling European Union (EU)-wide framework is missing. The aims of the study were to systematically review the evidence for best practice and to establish recommendations for collaborative research. The Paediatric Rheumatology European Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) project enabled a scoping review and expert discussion, which then informed the systematic literature review. Published evidence was synthesised; recommendations were drafted. An iterative review process and consultations with Ethics Committees and European experts for ethical and legal aspects of paediatric research refined the recommendations. SHARE experts and patient representatives vetted the proposed recommendations at a consensus meeting using Nominal Group Technique. Agreement of 80% was mandatory for inclusion. The systematic literature review returned 1319 records. A total of 223 full-text publications plus 22 international normative documents were reviewed; 85 publications and 16 normative documents were included. A total of 21 recommendations were established including general principles (1-3), ethics (4-7), paediatric principles (8 and 9), consent to paediatric research (10-14), paediatric databank and biobank (15 and 16), sharing of data and samples (17-19), and commercialisation and third parties (20 and 21). The refined recommendations resulted in an agreement of >80% for all recommendations. The SHARE initiative established the first recommendations for Paediatric Rheumatology collaborative research across borders in Europe. These provide strong support for an urgently needed European framework and evidence-based guidance for its implementation. Such changes will promote research in children with rheumatic diseases.

Periodic fever syndromes: beyond the single gene paradigm

“…Research utilizing large numbers of clinical samples linked with relevant detailed clinical information is critical for targeting disease-specific biomarkers and validation of genetic risk factors of diseases, especially when studying gene variants with small effects. While initiatives began > 15 years ago in Europe to build the needed infrastructure for research in auto-inflammatory diseases [64], in Canada, our capacity to engage in research in this area is limited to a few centres that have the clinical expertise and resources for a local registry and/or biobank. It will be critical to develop population-based biobanks with a centralized longitudinal registry to support investigators and clinicians in their work.…”

Diagnosis and Treatment of Angiography Positive Medium to Large Vessel Childhood Primary Angiitis of Central Nervous System (p-cPACNS): An International Survey

“…Currently, diagnostic approaches and classification of cPACNS relies on suggested, but nor prospectively evaluated criteria, and treatment is based on mostly retrospective and relatively small case series and/or expert opinion (10). In such situations, establishing consensus on diagnostic and treatment plans to harmonize approaches and prospectively collect meaningful clinical datasets in relation to consensus treatment plans can be a helpful tool to generate evidence and allow improvement of outcomes in patients with rare diseases (11,(15)(16)(17).…”

“…The establishment of consensus treatment plans follows a structured approach, including surveying the current standard of care among experts as an early step (15)(16)(17). Here, we present results from an internationally shared online survey collecting information on diagnostic and therapeutic approaches of clinicians experienced with cPACNS.…”

Langfristentwicklungen in der Kinder- und Jugendrheumatologie

“…Daneben finden sich international mehrere Therapieempfehlungen, die evidenz- und konsensbasiert neue Therapiestrategien propagieren, z. B. jene vom American College of Rheumatology 56 und der SHARE-Initiative (Single Hub and Access point for paediatric Rheumatology in Europe) 57 . Im Rahmen von SHARE entwickelten Experten aus ganz Europa Empfehlungen zur Versorgung („best practice“) von Kindern und Jugendlichen mit JIA, Uveitis 58 , jSLE 59 60 , juvenilem Antiphospholipidsyndrom 61 , juvenilen Vaskulitiden 62 63 64 und autoinflammatorischen Erkrankungen 65 .…”