Within the EJP RD (European Joint Programme on Rare Diseases), Work Package (WP) 20, "Accelerating the validation, use and development of innovative methodologies tailored for clinical trials in RDs", recognizes and addresses methodological challenges in clinical trials for rare diseases. These challenges include gaps in knowledge of best practices and innovative methodologies. Several actions have been implemented to overcome these gaps.
On one hand, WP 20 seeks to validate and apply innovative statistical methodologies previously developed from three FP7-funded projects (ASTERIX, InSPiRe, and IDeAl) via three demonstration projects. Additionally, two innovative projects address additional needs for trials in limited populations. On the other hand, WP 20 strives to disseminate these state-of-the-art innovative methodologies in these five projects through different scientific webinars.
WP 20 is pleased to publish a series of papers focusing on these developments in clinical trials for rare diseases in the Orphanet Journal of Rare Diseases, which will also be linked to their respective webinars.
WP 20's ambition is to reach the widest possible audience in the field of clinical trials for rare diseases and to expand knowledge and research on these topics through these publications.