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EU and US unite against rare diseases.

Article Excerpt
More than 50 million people in the EU and US alone suffer from rare diseases. The reluctance of drug manufacturers to commit R&D spend on these areas because of the low level of return, coupled with intense lobbying efforts of patient groups, has caused many regulatory agencies to introduce initiatives that have incentivized rare disease R&D. The number of drug approvals for rare disease has escalated since the implementation of these regulations, and the EU and US have now joined forces to improve the efficiency of the regulatory processes relating to rare disease drug development and to reduce the unmet need amongst their populations.

Rare diseases represent an important area of unmet medical need (Table 1). Globally, it is estimated that 5000-8000 distinct rare diseases exist, with approximately 30 million people in the EU and 25 million people in the US suffering from such conditions. (1-3) Most diseases are assessed by their impact on society as a whole and little attention is paid to rare diseases because of their low prevalence. Officially, rare diseases are defined as those affecting fewer than five in 10000 people in the EU and fewer than 200000 people in the US. (3) These disorders pose serious difficulties for sufferers and their families.

Table 1: Challenges concerning rare disease management.

Lack of information about ongoing clinical research.

Lack of general financial resources.

Limited knowledge of experts and support services.

Limited access to specialized healthcare services and to approved therapies.

Cost...

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