EU Orphan Medicines Regulation Review

The purpose of the project was to delver an external report that evaluates the broader economic and societal impact of the Orphan Medicine Product (OMP) Regulation in Europe

CRA Approach

  • Set out a range of indicators to be used to assess the impact of the Regulation on society by improving the treatment and health of patients
  • Reviewed the existing literature on the impact of the Regulation
    • Analysed the existing studies on the impact of the Regulation
    • Compared the experience of the US and EU
  • Carried out a literature review using academic and open source databases
  • Conducted 14 interviews with wider stakeholder groups

Client Impact

  • Provided an evaluation of the societal and economic impact of the European Union (EU) Regulation on Orphan Medicinal Products (OMP)
  • Provided recommendations for the future of OMP regulation to ensure rare diseases remain a priority going forward
  • Report published on EFPIA website
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