An assessment of the impact of the European Union’s Orphan Drug legislation ten years after it was enacted, concludes it has boosted research and led to the increased availability of drugs to treat rare diseases.
The assessment, commissioned by the trade associations EuropaBio and the European Biopharmaceutical Enterprises (EBE) and carried out by the consultants, Office of Health Economics (OHE), shows that between 2000 and 2004, R&D investment in rare diseases in Europe increased by more than half, and then more than doubled between 2004 and 2008. Over the period from 2000-2008, total growth in investment was more than 200 per cent.
As a result R&D focused on finding new treatments for patients with rare diseases represents an increasingly significant proportion of the total investment in R&D in the biopharmaceutical industry.
Orphan Medicinal Products (OMP) Regulation, which came into force in April 2000, provides incentives for research, development and commercialisation of drugs for the diagnosis, prevention or treatment of life-threatening or seriously debilitating rare conditions.
Although individual rare diseases, including certain cancers, metabolic diseases, diseases of the nervous system and musculoskeletal disorders, affect fewer than 5 in every 10,000 people, in total such rare conditions directly or indirectly affect an estimated 30 million people in Europe.
Since the OMP legislation came into force the number of marketed treatments for rare diseases has increased from eight to 69. Whilst it has provided support to existing companies to invest in treatments for rare diseases, it has also helped to establish new companies in Europe focusing on researching new treatments for rare diseases.
Erik Tambuyzer, Chair of the EBE-EuropaBio Joint Task Force, said this is a clear example of how the right legislation can drive forward European healthcare innovation delivering benefit to individuals, to society and at the same time to the development of a sustainable and competitive economy. “While only a small percentage of rare diseases currently have a treatment, there is much room for more industry involvement in the field to develop more rare disease treatments.”
EBE and EuropaBio commissioned the research as part of their Joint Task Force on Rare Diseases & Orphan Medicinal Products. The Joint Task Force consists of companies that have either already developed and market, or intend to develop orphan medicines under Regulation EC/141/2000.
Links
Assessment of the Impact of Orphan Medicinal Products (OMPs) on the European Economy and Society. Office of Health Economics (OHE) Consulting.
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