Cultural, geographical and ethical questions when looking to enroll pediatric patients in rare disease clinical trials

Introduction: Rare diseases are becoming increasingly recognized worldwide as an important public health challenge and Orphan Drugs begin to play an important role in Research and Development. There are several major issues concerning conducting clinical trials in the field of Rare Diseases, especially in children, as 80% of those diseases are genetic in origin and involve, in most instances, children. Areas covered: This review concentrates on challenges associated with the patient recruitment process. The authors experience and opinion was complemented by a systematic search of relevant literature and includes ethical, cultural and geographical issues with rare diseases. The review presents current opinion and provides valuable support for planning and conducting clinical research in rare diseases. Expert opinion: The expert opinion section concentrates on importance of future rare disease clinical research in the context of understanding common diseases and developing new treatment options.