Bridging East with West of Europe – a comparison of orphan drugs policies in Poland, Russia and the Netherlands

Rare disorders and orphan drugs are increasingly a topic of interest for the pharmaceutical industry as well as regulators and payers. The main reasons for the high interest are identified gaps in the access to medical treatment (or complete lack thereof) that rare disease patients often experience, difficulties in getting regulatory approval for new drugs as well as high prices associated with orphan drugs. HTA processes for orphan drugs are often subject to the same approach as drugs for common diseases, which creates exceptional situations due to small and heterogeneous patient groups, a lack of strong outcome data and high prices. This often leads to either failure to achieve reimbursement or granting of reimbursement without adequate evidence on cost-effectiveness. The HTA issues come on top of the existing difficulties to get new orphan drugs developed and licensed: less than 3% of the estimated 5000 to 8000 rare diseases have an actual medicinal treatment approved by EMA (1, 2). The number of new orphan drug approvals was ~ 14 per year in 2015-17 (3).