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European
ministers have provided an incentive for the development
of drugs for rare diseases that seems likely to benefit
the pharmaceutical industry, particularly the biotechnology
sector. The ministers in the EU responsible for single market
measures have set aside a sizeable budget to encourage industry
to produce therapies for rare disease, along the lines of
the ‘Orphan Drug’ legislation in the US. Since its introduction
in the US in 1984, biotechnology companies have made some
85% of all applications for special status. Indeed, some
firms have been created specifically to exploit the potential
of ‘orphan drugs’, such as Orphan Medical.
In
Europe, the draft legislation still has to be approved by
the European Parliament but it allows for:
- Exclusive
marketing rights for a minimum of six and a maximum of
ten years
- Complete
or partial waiver of the Euro200,000 EMEA fee for obtaining
EU-wide approval
- Access
to European Community funds for research into rare diseases
- Free
advice from the EMEA on clinical trial protocols
These
incentives are designed to allow companies some leeway to
develop drugs for the treatment of rare diseases. Similar
incentive schemes are also available in Japan and Australia.
They have been behind the development of products such as
the beta-interferons for multiple sclerosis, Enbrel, Epogen
and Prograf. Indeed, IMS HEALTH’s R&D
focus database has records on more than 220 marketed
and pipeline orphan drugs.
The
US Postition
In
the US, where orphan drug designation brings seven years
of marketing exclusivity, the original definition of a rare
disease was amended in 1984 to:
"Any disease or condition which (a) affects less than 200,000
persons in the US or (b) affects more than 200,000 persons
in the US but for which there is no reasonable expectation
that the cost of developing and making available in the
US a drug for such disease or condition will be recovered
from sales in the US of such a drug."
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