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) ODA : tgv thalidomide schandaal (eind jaren 50). Nood om meer efficacy aan te tonen => cost van development stijgt  meer focus op grote populations  ODA ontstaan om die trend tegen te gaan in de tien jaar voor de regulation: US: slechts 10 en EU: slechts 8 GMen voor zeldzame ziekten

Incentives Market exclusivity for a period of 10 years protects against a ‘similar’ drug being authorized in the EU for the same therapeutic indication Protocol and scientific assistance wrt clinical and non-clinical aspects of drug development Access to the centralized registration procedure Access to EU-funded research Incentives undertaken on a national level + Fee reductions such as full or partial EMA fee waivers 3 uitzonderingen op market exclusivity: first, the sponsor’s consent; second, a lack of supply; third, if a new product, although similar, could be demonstrated to be ‘clinically superior’ — that is, “safer, more effective or otherwise clinically superior” to the product already on the market. Protocol assistance : wrt clinical and non-clinical aspects of drug development fee waivers (depending on the size of the sponsor)

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) Note : As there is a time lag between the applications being submitted and the Committee of Orphan Medicinal Products (COMP) opinion being issued, and between the COMP opinion being issued and the decision being made by the European Commission (EC), the number of submissions and the number of decisions in a given year may not completely correspond. It should also be noted that 74 products in total have been removed from the register at the request of sponsors — either for administrative reasons or when development was discontinued.

Current status Market authorizations 60 authorized orphan drugs (October 2011) OPM: diegene die eerste OD status kregen (2000-2001): 10jaar is verlopen! (KCE, 2010)