EBE and EuropaBio hail EU Orphan Medicinal Products Regulation a success
Brussels, 29 November 2010
Today EBE and EuropaBio’s Joint Rare Diseases and Orphan Medicinal Products Task Force released an assessment of the EU’s Orphan Medicinal Products (OMP) Regulation evaluating the extent to which this legislation has impacted research into rare diseases and the availability of new medicines to treat rare diseases over the last ten years.
The OMP regulation EC 141/2000, which came into force in April 2000, provided incentives for research, development and bringing to the market of medicines intended for the diagnosis, prevention or treatment of rare life-threatening or seriously debilitating conditions. Although individual rare diseases, which include certain cancers, metabolic diseases, diseases of the nervous system and musculoskeletal disorders, affect fewer than 5 in every 10,000 people, in combination, rare diseases may directly or indirectly affect an estimated 30 million people in Europe.
Since coming into force the OMP legislation has increased the availability of medicines to treat patients with rare diseases from just 8 in 2000 to 69 today. It has provided support to companies investing in treatments for rare diseases, but importantly it has also helped to establish new companies in Europe focusing on researching new treatments for rare diseases.
The assessment, commissioned from the Office of Health Economics (OHE) Consulting, reveals that between 2000 and 2004, R&D investment in rare diseases in Europe increased by more than half and more than doubled between 2004 and 2008. Over the entire 2000-2008 period, the total growth in investment amounted to more than 200%. 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.
Commenting on the analysis, Dr 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 numerous benefits to individuals, to society and at the same time to the development of a sustainable and competitive economy. And while only a small percentage of rare diseases currently already have a treatment, there is much room for more industry involvement in the field to develop more rare disease treatments.”
As part of the work of their Joint Task Force on Rare Diseases & Orphan Medicinal Products, EBE and EuropaBio commissioned this report to support the assessment of the impact of the regulation and to raise awareness of the pressing need to improve patient access to treatments for rare disease across the EU. The Joint Task Force comprises of companies that have either already developed and market, or intend to develop orphan medicines under Regulation EC/141/2000. Together, the many members of the Joint Task Force represent a large proportion of orphan drugs currently available on the EU market.
Kevin Loth, Senior Director, External Relations, Europe, Celgene Corporation, who also led the development of the assessment on behalf of the Task Force concluded: “The EU OMP Regulation has simultaneously boosted EU innovation and brought new therapies to market for patients with rare and debilitating diseases who otherwise have few or no treatment options. As an industry we play an ever-increasing role in helping patients, who suffer from rare disease and so far had little hope of treatment. However, there is still much work to be done to ensure that all patients who can benefit from innovative therapies have broad access to them.”
For further information please contact:
Executive Director, EBE
T: + 32 2 626 2561
Healthcare Biotech Director, EuropaBio
T: +32 2 735 0313
Communications Manager, EuropaBio
Tel: +32 2 739 11 84; GSM: +32 476 60 71 35 Email:email@example.com
EBE (European Biopharmaceutical Enterprises) is the European trade association that represents biopharmaceutical companies of all sizes operating in Europe. It has 65 member companies, which are engaged in the research, development, manufacturing and marketing of new medicinal products using biotechnology. EBE also operates as the biotechnology arm of EFPIA, the European pharmaceutical industry Federation.
EuropaBio is the European Association for Bioindustries, bringing together bioscience companies from all fields of research and development, testing, manufacturing and distribution of biotechnology products. It has 66 corporate and 7 associated members, 4 BioRegions and 22 National Biotechnology Associations representing some 1800 small and medium sized enterprises.