SP0021 DELIVERING FUTURE GLOBAL RESEARCH CHALLENGES IN PAEDIATRIC RHEUMATOLOGY
Authors: Nicolino Ruperto
Journal: Annals of the Rheumatic Diseases (2019)

<h3></h3> The treatment of pediatric rheumatic diseases has improved tremendously in the last 20 years thanks to appropriate legislative initiatives, the existence of very large collaborative networks and the availability of new potent medications. In particular there has been a tremendous improvement in a sizable proportion of patients with juvenile idiopathic arthritis (JIA) with over 3500 children enrolled in trials with registrative purposes. For JIA now the tendency is to concentrate on oral therapies and on drugs targeting specific JIA categories. Also in juvenile systemic lupus erythematosus (SLE) the childhood counterpart of SLE there are several potential targets to be tested in children. In addition the field of paediatric rheumatology has provided an excellent example of large scale academic studies which, through the Paediatric Rheumatology International Trials Organisation (PRINTO) have enrolled over 38,000 children from over 60 countries Further improvement in future years will stem from a better definition of the paediatric disease entities, the discovery of laboratory and imaging biomarkers that could help the tuning of therapy, a smoother implementation of future clinical trials, a more standardized link between academia (clinical and basic science), regulatory authorities, learned societies such as the Pediatric Rheumatology European Society (PRES) and family organisations for the planning of future trials. This lecture will present a general overview and a perspective for the future (mainly academic) work to align clinical data collection with basic researchers, PRES, and family associations in order to foster and facilitate drug evaluation in pediatric rheumatic diseases as well as academic research. <h3>References:</h3> [1] Ruperto N, Martini A. Current and future perspectives in the management of juvenile idiopathic arthritis. Lancet Child Adolesc Health 2018;2:360–370. [2] Consolaro A, Giancane G, et al for PRINTO. Phenotypic variability and disparities in treatment and outcomes of childhood arthritis throughout the world: an observational cohort study. Lancet Child Adolesc Health. 2019 Feb 25. pii: S2352–4642(19)30027–6. doi: 10.1016/S2352–4642(19)30027–6. [Epub ahead of print]. [3] Ruperto N, Martini A, for PRINTO. Networking in pediatrics: the example of the Pediatric Rheumatology International Trials Organisation (PRINTO). Archives Dis Child 2011;96:596–601. [4] Ruperto N*, Eichler I*, et al. A European network of paediatric research at the European Medicines Agency (Enpr–EMA). Archives Dis Child 2012;97(3):185–188. [5] Ruperto N, Vesely R, Saint–Raymond A, Martini A for PRINTO. Impact of the European paediatric legislation in pediatric rheumatology: past present and future. Ann Rheum Dis 2013; 72:1893–189. [6] Brunner HI, Martini A, Lovell DJ, Ruperto N, on behalf of PRINTO–PRCSG. Clinical trials in children and adolescents with systemic lupus erythematosus –methodological aspects, regulatory landscape and future opportunities. Ann Rheum Dis 2019;78:162–170. <h3>Disclosure of Interests:</h3> Nicolino Ruperto Grant/research support from: The Gaslini Hospital, where NR works as full-time public employee, has received contributions (&gt; 10.000 USD each) from the following industries in the last 3 years: BMS, Eli-Lilly, GlaxoSmithKline, F Hoffmann-La Roche, Janssen, Novartis, Pfizer, Sobi. This funding has been reinvested for the research activities of the hospital in a fully independent manner, without any commitment with third parties., Consultant for: Received honoraria for consultancies or speaker bureaus (&lt; 10.000 USD each) from the following pharmaceutical companies in the past 3 years: Ablynx, AbbVie, Astrazeneca-Medimmune, Biogen, Boehringer, Bristol-Myers Squibb, Eli-Lilly, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, SanofiServier, Sinergie, Sobi and Takeda., Speakers bureau: Received honoraria for consultancies or speaker bureaus (&lt; 10.000 USD each) from the following pharmaceutical companies in the past 3 years: Ablynx, AbbVie, Astrazeneca-Medimmune, Biogen, Boehringer, Bristol-Myers Squibb, Eli-Lilly, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, SanofiServier, Sinergie, Sobi and Takeda.