Rare Disease Special Interest Group

Mission Statement

To provide an interactive and collaborative forum to discuss, develop and improve the use of epidemiological approaches for rare disease research.


  • To create a central data repository for definitions and regulatory environments for rare disease therapeutic products
  • To identify epidemiological approaches for natural history of rare diseases and safety and long-term outcomes of therapeutic products that treat rare diseases
  • To identify existing data sources and professional and research networks that may be considered for rare disease research
  • To identify issues unique to rare disease pharmacoepidemiology, and to explore methodological approaches to address these issues
  • To serve as a resource to the ISPE membership by providing expertise in rare disease pharmacoepidemiology and educational content in the area

Meeting Schedule

Monthly teleconferences and face-to-face meetings during ISPE mid-year and annual meetings

SIG homepage on ISPE Exchange (login required)

SIG leader(s)

Chair Jasmanda Wu
Co-Chair Federica Pisa