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

ABSTRACT:

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.

Methods

Multi-stakeholder sessions covered the topics: Future Educational System, Randomization in Rare Disease Clinical Trials, Endpoints in Rare Disease Clinical Trials and Using History Course Data. The sessions began with expert presentations to set t

SUBMITTER: Bodden D 

PROVIDER: S-EPMC11657468 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature