Abstract
The aim was to evaluate if the European Medicines Agency (EMA) conditional or exceptional approval for orphan drugs were influenced by the type of the disease for which the orphan drug was for in 2017. The list of authorised drugs with current orphan designations was collected from EMA website and status of registration for the considered drugs was assessed. The impact of the disease type on the conditional approval as well as exceptional circumstances was assessed using the logistic regression and presented as odds ratio (OR) showing the odds for conditional approval or exceptional circumstances for metabolic diseases or oncologic diseases compared to other diseases. All ORs were presented with 95% confidence intervals (Cis) rounded to two decimal places and corresponding p-value rounded to four decimal places. A total of 95 orphan drugs were recorded among which 22 were for metabolic diseases, 36 for oncologic diseases and 37 for others. Conditional approval was performed for 0, 9, and 5 drugs respectively and the exceptional circumstances were identified for 7, 2 and 5 drugs respectively. Odds for conditional approval were 96% lower for metabolic drugs compared to the other drugs (excluding oncologic; OR of 0.04; CI: 0.00006 - 0.67; p=0.0092) and 10 times higher for oncologic drugs compared to the other drugs (excluding metabolic; OR of 10.02; CI: 1.58 - 287.77; p=0.0060). The odds for exceptional circumstances were 7 times higher for metabolic drugs compared to others (excluding oncologic; OR of 7.14; CI: 1.49 - 36.03; p=0.0128) and 84% lower for oncologic compared to others (excluding metabolic; OR of 0.16; CI: 0.02 – 0.90; p=0.0316). A significant influence in approval type by the type of disease for orphan drugs was revealed. For metabolic orphans significantly more often exceptional approval was issued by EMA in the analysed year.
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