A small, US-based biotech company specializing in rare diseases contracted Premier Research to manage their global pediatric and adult Lipid Storage Disease program. The initial request from the client included the following services for two interventional pediatric studies and one interventional adult study: Project Management, Medical Management including Safety Committee oversight, Pharmacovigilance, Biostatistics, Medical Writing, Data Operations utilizing our preferred EDC system and Regulatory services in Europe (including UK and France) for Competent Authority and Ethics Committee submissions.
The first adult interventional study successfully completed enrollment and study start-up activities in the second study are near completion. Study start-up activities for the first pediatric interventional study have been completed; and Premier Research and the client are currently working with the sites to provide support and creative solutions for identifying and recruiting patients with the extremely rare, fatal, and often undiagnosed disease. Additionally, Premier Research is partnering with the client to expand the study scope to include other regions to further facilitate enrollment and global awareness of the disease and trial.
Our successful collaboration with the client during study start-up has led to an expansion of the partnership to include additional adult and pediatric interventional studies and a retrospective adult chart review; as well as an expansion of services to include monitoring and site management in the US and Europe.