EndoCompass Project: Rare Diseases in Endocrinology

Area of Science:

• Endocrinology
• Rare Diseases
• Health Research Infrastructure

Background:

• Endocrine science is underrepresented in EU research funding, despite the importance of hormone health.
• A gap exists between the societal impact of endocrine disorders and their research prioritization.
• The EndoCompass project aims to define strategic research priorities in endocrine science.

Purpose of the Study:

• To identify and promote strategic research priorities in endocrine science.
• To address critical hormone-related health challenges.
• To develop a roadmap for rare endocrine disease research infrastructure.

Main Methods:

• Analysis of the EU CORDIS database (Horizon 2020).
• Expert evaluation of the European rare disease research landscape.
• Identification of structural needs and opportunities in research infrastructures.

Main Results:

• Priorities include harmonized data capture, patient-centered outcomes, innovative trial methods for rare diseases, and genomic diagnostics.
• Emphasis on bridging pediatric and adult care and fostering public-private partnerships.
• Development of sustainable research networks for rare endocrine diseases.

Conclusions:

• The EndoCompass project provides a roadmap for rare disease research infrastructure development.
• Coordinated European approaches are needed, integrating expertise across centers, age groups, and specialties.
• Strategic investment in sustainable rare disease research networks is supported by the findings.