Economic Evaluation And Cost-Effectiveness Threshold In Practice Of Decision-Making On Orphan Drug Reimbursement In Poland

Abstract

Use of economic evaluations and cost-effectiveness thresholds for orphan drugs raise concerns and discussions worldwide. Orphan drugs undergo a full pharmacoeconomic evaluation and assessment in Poland. The goal of this study was to find out how important the reimbursement criterion of cost-effectiveness threshold (3xGDP per capita for QALY/LYG gained) is in case of orphan drugs in Poland. Documents published by the Agency for Health Technology Assessment and Tariff System (AHTAPol) and Minister of Health (MoH), pertaining to all orphan drugs considered for reimbursement between 2015 and 2016, were analysed. Inferences from published sources were used when ICUR values (cost-effectiveness indicators) were not disclosed due to confidentiality. ICUR value was calculated in 30 cases out of 32 orphan drug reimbursement applications. Positive recommendations of AHTAPol’s President (RAP) were issued for 30% of cost-ineffective cases. Negative RAP were issued for 13% of cost-effective cases. Positive MoH’s reimbursement decisions were issued for 33% of cost-ineffective cases, while 17% of cost-effective cases did not receive positive MoH reimbursement decisions. Risk sharing schemes (RSS) were proposed in 22 applications, of which 13 cases assessed by AHTAPol’s President were found as insufficient. For 10 applications, RSS were not proposed (for 7 of them AHTAPol’s President suggested necessity of RSS). The main reasons for negative RAP in cost-effective cases included lack of evidence of clinical effectiveness and uncertainty of estimations. The main reasons for positive RAP in cost-ineffective cases included limited therapeutic options, the only therapy available and proven clinical-effectiveness. Despite existence of formal cost-effectiveness threshold and no special formal approach to reimbursement of orphan drugs, results of pharmacoeconomic analysis do not determine final reimbursement decisions in Poland. Cost-effectiveness cannot be considered as a crucial criterion in decision-making on orphan drug reimbursement in Poland.