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)

Incentives

I. 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)

OR

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.

OR

If such a method does exist, that the medicinal product will be of significant benefit to those affected.

AND

Current status

• Orphan designations

(Westermark, 2011)

Current status

• Market authorizations

60 authorized orphan drugs (October 2011)

(KCE, 2010)