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Table 1 Identified skills required and members of DevelopAKUre consortium

From: Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium

Expertise Partner
Leading clinical disease expertise and coordination Royal Liverpool University Hospital (RLUH) (consortium coordinator, study co-sponsor)
Leading scientific disease expertise University of Liverpool (study co-sponsor)
Patient perspective and network, advocacy AKU Society, United Kingdom
Operational clinical study expertise (including Data Management) to conduct complex international clinical studies in rare diseases PSR, the Netherlands
Clinical research from a regulatory perspective, managing regulatory authority interactions and applications, expertise in the investigational drug and pharmacovigilance Swedish Orphan Biovitrum (Sobi), Sweden
Medical monitoring expertise Cudos, the Netherlands
Biomarker analysis expertise Nordic Biosciences, Denmark
Inflammatory markers analysis expertise Universita degli studi di Siena, Italy
Genetic analysis expertise Biomedical Research Center of the Slovak Academy of Sciences, Slovakia
Clinical expertise and study conduct (in addition to RLUH) Hôpital Necker, France (study site)
Clinical expertise and study conduct (in addition to RLUH) National Institute of Rheumatic Diseases, Slovakia (study site)
Additional clinical study participants’ support and communication with study participants ALCAP, France