Mestre-Ferrandiz, J., Garau, M., O'Neill, P. and Sussex, J.
This report examines the effect of the European Union’s 1999 Regulation on Orphan Medicinal Products (OMPs) on the European economy and society. This study collected data on several indicators of activity, completed a confidential survey of companies developing OMP, concluded four case studies and undertook a literature review. The result is a rich tableau of data and information that describe the effects of the OMP Regulation in some detail. Findings include the following.
- Ten of the 18 companies surveyed stated that the Regulation was 'decisive' in R&D decision making. The ten-year period of market exclusivity was identified as the single most important aspect of the OMP Regulation, followed by having access to the centralised procedure.
- The number of medicines available in Europe to treat rare diseases has risen from only eight in 2000 to 68 by the end of 2009. More than 500 drugs in total had received orphan status by the end of 2009.
- OMP R&D investment grew by 209%, from €150,000 million in 2000 to over €490,000 million in 2008. New companies have been formed and OMP-related industry employment in the EU grew by 161% between 2000 and 2008, higher than in pharmaceuticals overall in Europe.
- Societal effects of the OMP Regulation have been important -- including, for example, improvements in treatment that positively affect the lives of both patient and caregiver and the development of research networks that create synergies, potentially accelerating innovation.