Exploring the usability of EUCERD core indicators for rare diseases

Area of Science:

• Public Health
• Health Services Research
• Rare Diseases Epidemiology

Background:

• The European Commission supported initiatives to develop health service indicators for rare diseases (RDs).
• The EUROPLAN project created 59 process and outcome indicators for monitoring National Plans on RDs.
• A large number of indicators posed challenges for practical application.

Purpose of the Study:

• To derive a manageable selection of core indicators from a larger set.
• To identify key indicators for orienting rare disease (RD) policies.
• To enhance knowledge sharing and comparability among European Member States regarding RDs.

Main Methods:

• A descriptive study was conducted within the "Working for RDs" Joint Action.
• The study aimed to select core indicators from the initial 59 EUROPLAN indicators.
• The selection process resulted in 21 core indicators adopted by the EU Committee of Experts on RDs.

Main Results:

• A subset of 21 core indicators was successfully derived from the initial 59 indicators.
• These core indicators were adopted by the European Union Committee of Experts on RDs in June 2013.
• The selected core indicators facilitate knowledge sharing and comparability across Member States.

Conclusions:

• The 21 core indicators provide a valuable tool for rare disease (RD) health service planning and evaluation.
• Core indicators enhance the ability of Member States to share knowledge and achieve comparability in RD policies.
• This selection represents a significant step towards more effective and coordinated rare disease management in Europe.