Dia 1 - KU Leuven

Weesziekten en weesgeneesmiddelen
• Weesziekte of zeldzame ziekte:
Prevalentie < 1/2000
Bv: mucoviscidose 1/5000, hemofilie 1/10.000
Maar: ziekte van Crohn 6/2000, ijzerstapelingsziekte
(hemochromatose) 1/200
• Weesgeneesmiddel: een geneesmiddel dat enkel
(vooral?) dient om een weesziekte te behandelen
Orphan drug regulation
United States: Orphan Drug Act (1983)
Europe (2000)
Market exclusivity for a period of 10 years
protects against a ‘similar’ drug being authorized in the EU for the same
therapeutic indication
II. Protocol and scientific assistance
wrt clinical and non-clinical aspects of drug development
III. Access to the centralized registration procedure
IV. Access to EU-funded research
V. Incentives undertaken on a national level
Fee reductions
such as full or partial EMA fee waivers
Criteria for orphan designation (Art. 3)
A medicinal product intended for
the diagnosis, prevention or
treatment of a life-threatening or
chronically debilitating condition
1° that affects not more that 5 in
10 000 people in the Community
(prevalence criterion)
2° that without incentives it would
be unlikely to generate sufficient
return the justify the investment
(return on investment criterion)
There is no satisfactory method of
diagnosis, prevention or treatment
of the condition.
If such a method does exist, that
the medicinal product will be of
significant benefit to those
Current status
• Orphan designations
(Westermark, 2011)
Current status
• Market authorizations
60 authorized orphan drugs (October 2011)
(KCE, 2010)

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