OBJECTIVES:

Orphan drugs are usually more expensive than the non-orphan medicines. Thus, the general access to these pharmaceuticals is often limited. The aim of this research is to evaluate the accessibility of orphan drugs in Hungary in 2022.

METHODS:

We collected all the pharmaceuticals that had been listed as an orphan drug by the European Medicines Agency (EMA) in 2022. Then we analysed the accessibility and reimbursement status of these pharmaceuticals in the official database of the National Health Insurance Fund.

We identified two major reimbursement technics. If a general reimbursement decision is granted by the Government, there is no need for any further evaluation on a patient level. While during the individual reimbursement the decision is made case by case. This technic puts significant administrative pressure on the system.

RESULTS:

131 drugs had orphan marketing authorization by EMA in 2022. Out of these 131 medicines the Hungarian patients have access to 55 (42%) with any type of reimbursement. 45 medicines were available with individual reimbursement (82%) and 10 with general reimbursement (18%). The average length from the EMA approval till the start of the general reimbursement was 350 days.

The three most common therapeutic areas in which these orphan-drugs were available were haematology (n=16, 29%), metabolism disorders (n=10, 18%) and neurology (n=7, 13%).

CONCLUSIONS:

The general access to orphan drugs seems to be limited in Hungary, while throughout the individual reimbursement thousands of Hungarians get access to these innovative pharmaceuticals. From the Payer’s point of view the individual reimbursement can guarantee stricter control on the spendings, but on the other hand it puts enormous administrative pressure on the system. It is worth considering that some of these individually reimbursed pharmaceuticals should be reimbursed generally.