OBJECTIVES: In Poland there is currently no specific pathway for orphan drugs assessment. Nevertheless the planned amendment of the Reimbursement Act is to include more flexible approach to orphan drugs with price justification instead of standard cost per QALY criterion for assessment. The aim of this research is to review current situation of orphan drugs in Poland and to assess if shift in health technology assessment (HTA) approach is desired.
METHODS: Orphan drugs with EMA approval since beginning of 2012 were identified. HTA reports of Polish HTA Agency (AOTMiT) were searched for each case. Date, decision and decision drivers were extracted from reports. Final Minister of Health (MoH) decisions on reimbursement status were also collected.
RESULTS: Fifty two orphan drugs were granted EMA approval since 2012. Sixteen of them were submitted for AOTMiT assessment, with 4 still under evaluation. Of the 12 assessed interventions only 2 (Adempas® and Signifor®) were granted positive and 1 (Adcetris®) positive with restrictions recommendations. Final MoH decision was positive for Adcetris®, Adempas® and in addition for Opsumit®, which was negatively assessed by AOTMiT (clinical benefit demonstrated; positive recommendation possible providing price reduction). All 3 drugs were reimbursed under time-limited drug programs (programs specific for expensive drugs). Weak clinical evidence and lack of cost-effectiveness were commonly cited reasons for negative AOTMiT recommendations.
CONCLUSIONS: Patient access to orphan drugs in Poland is very limited. Only 6% of orphan drugs approved in EU in last 5 years were reimbursed in Poland. Given the high cost of orphan drugs and the difficulties encountered in providing sufficient effectiveness evidence, these products routinely exceed the limits of threshold for cost-effectiveness in Poland. Holding orphan drugs to the same standards for decision-making as ”standard” therapeutics causes patient access to be jeopardized. There is a strong need for shift in approach for orphan drugs assessment in Poland.