Integrative research and innovation strategy for rare diseases. Insights from the 5-year European joint programme on rare diseases, including analysis to inform recommendations for future actions.

ABSTRACT:

Background

The European Joint Programme on Rare Diseases (EJP RD) was an initiative that sought to integrate different rare disease strategies into a research ecosystem. This paper summarizes the work of the dedicated work package on Integrative Research and Innovation Strategy and outlines recommendations to facilitate alignment with National Plans and Strategies for Rare Diseases.

Methods

We carried out three periodic surveys (in 2020, 2021 and 2023) on the status of national plans and strategies (NP/NS) for rare diseases in EJP RD member countries. Using the feedback from the surveys and other sources, we developed an annual mapping of research and innovation needs and their alignment with the EJP RD programme. A review of the findings and progress from these approaches -

SUBMITTER: Sanchez-Gonzalez MDC 

PROVIDER: S-EPMC12532414 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature