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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



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