THE AVAILABILITY AND EXPENDITURE OF ORPHAN MEDICINES IN POLAND

Author(s)

Sujkowska G, Jagodzinska-Kalinowska K, Matusewicz W
Agency for Health Technology Assessment and Tariff System, Warsaw, Poland

OBJECTIVES: The aim of the present analysis was to identify the level of the availability and total expenditure of medicines for rare diseases with  European authorization and orphan designation. In Poland all innovative medical technologies and services claiming public money founding have to be assessed by Agency for Health Technology Assessment (AOTM).  Pharmacoeconomic evaluations of new therapies are required for all reimbursement  decisions and orphan drug manufacturers cannot be exempted from providing a full pharmacoeconomic or HTA  reports. The criteria of assessment connected with clinical and cost effectiveness (threshold is 3xGDP for ICUR/QALY) are the same for all kind of drugs.   METHODS: All orphan designation admitted by European Medicines Agency (EMA) until the end of 2014  were reviewed and analyzed from the official website of EMA. Among 792 EMA’s orphan registrations studied 78 (9,8%) applied to orphan drugs. We compared the outcomes with reimbursement list officially  published by Ministry of Health. Then it was checked what was the share of orphan drugs in  overall reimbursement spending. RESULTS: At the end of 2014 there were 28 orphan drugs available on the reimbursement list (36% of designed by EMA). The total public payer reimbursement spending was €2.41bn in 2012 and €2.26bn in 2013. Orphan drugs have only accounted for a small percentage of the overall drug budget in polish health care system (1,5% in 2012 and 3,2% in 2013). CONCLUSIONS: In the literature we can find opinions that the relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. In Poland reimbursement was awarded to the minority of orphan drugs designed by EMA. Very strict requirements in order to ensure compatibility with law directives could potentially influence negative reimbursement decisions for orphan drugs.

Conference/Value in Health Info

2015-05, ISPOR 2015, Philadelphia, PA, USA

Value in Health, Vol. 18, No. 3 (May 2015)

Code

PSY72

Topic

Economic Evaluation

Topic Subcategory

Cost/Cost of Illness/Resource Use Studies

Disease

Rare and Orphan Diseases

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