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Orphan drug legislation hailed as success

Legislation governing Orphan Medicinal Products in Europe has had a positive impact, according to a joint EBE and EuropaBio task force

EBE (European Biopharmaceutical Enterprises) and EuropaBio's Joint Rare Diseases and Orphan Medicinal Products Task Force have 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. 

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 five in every 10,000 people, in combination, rare diseases may directly or indirectly affect an estimated 30m people in Europe. 

Since coming into force the OMP legislation has increased the availability of medicines to treat patients with rare diseases from just eight 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; between 2004 and 2008, it more than doubled. Additionally, R&D that is 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 companies that have either already developed and currently market, or intend to develop, orphan medicines under Regulation EC/141/2000. Together, the 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, commented: "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."

The full report can be downloaded from EuropaBio

29th November 2010

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