Multi-stakeholder sessions on major innovation topics in rare disease clinical trials.

Background: The European Joint Programme on Rare Diseases aims to enhance the rare diseases research ecosystem by bringing together stakeholders such as research funders, institutions and patient organizations. Work Package 20 focuses on the validation, use and development of innovative methodologies for rare disease clinical trials. This paper reports on the outcomes of a retreat held in April 2023, where areas for innovation and educational needs in rare disease clinical trials were discussed in multi-stakeholder sessions.

The impact of allocation bias on test decisions in clinical trials with multiple endpoints using multiple testing strategies.

Background: Considering multiple endpoints in clinical trials provide a more comprehensive understanding of treatment effects and may lead to increased power or reduced sample size, which may be beneficial in rare diseases. Besides the small sample sizes, allocation bias is an issue that affects the validity of these trials. We investigate the impact of allocation bias on testing decisions in clinical trials with multiple endpoints and offer a tool for selecting an appropriate randomization procedure (RP).

Defining a Uterine Extraction Score based on a Volume/Access Ratio in Total Hysterectomy: a retrospective cohort study.

Background: Regardless of the technique used, extraction of the uterus is a crucial step in hysterectomy. There is currently no scoring system to predict its feasibility.

Objectives: Our main objective was to determine a predictive score of uterine extraction feasibility to optimise surgical planning of total hysterectomy.

Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations.

Background: The conduct of rare disease clinical trials is still hampered by methodological problems. The number of patients suffering from a rare condition is variable, but may be very small and unfortunately statistical problems for small and finite populations have received less consideration. This paper describes the outline of the iSTORE project, its ambitions, and its methodological approaches.