STATUS OF ORPHAN DRUGS AND REIMBURSEMENT RECOMMENDATIONS AND DECISIONS IN ANALYSED EUROPEAN COUNTRIES
Author(s)
Kawalec P, Malinowski K
Jagiellonian University Medical College, Krakow, Poland
OBJECTIVES: The aim was to investigate a connection between reimbursement recommendations and decisions in case of drugs with orphan status. We examined if the rare or ultra-rare status of the disease could influence the reimbursement recommendations and decisions in considered countries. METHODS: We analysed the Orphanet database to collect data on all drugs with orphan status authorized by the European Medicines Agency. The following HTA Agencies were considered: NICE (England), G-BA (Germany), AOTMiT (Poland), ZIN (Netherlands), TLV (Sweden), SMC (Scotland), AWMSG (Wales), HAS (France) and data on HTA recommendations and reimbursement status for these drugs were collected. Descriptive statistics using counts and percentages were employed to analyse data, then we calculated odds for gaining positive or negative recommendations in each agency. Agreement between type of recommendation and final reimbursement decision was also investigated for each analysed country for orphan as well as ultra-orphan drugs using kappa coefficient of agreement. RESULTS: We analysed data on 101 drugs with orphan status; 87 of drugs were used for rare diseases and 14 for ultra rare diseases. Statistical analysis revealed that odds for positive recommendations for drugs for ultra rare disease varied from 0.4 to 0.8 compared to drugs for rare diseases only, although no statistical significance was observed. The agreement between recommendation and reimbursement status varied from 0.1036 for AWMSG to 1 for TLV in case of orphans (without ultra-orphans) and from -0.2857 for SMC to 0.5 for AOTMiT. Among all agencies, only for AOTMiT and TLV in subsample of orphans the agreement between recommendation and reimbursement status was significantly higher than 0. No significant difference in agreement between subsample of orphan drugs (without ultra-orphans) and of ultra-orphans only was observed for any of agencies. CONCLUSIONS: The distribution of recommendation types and reimbursement decisions was not associated with the orphan or ultra orphan status of considered drugs.
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PSY133
Topic
Economic Evaluation, Health Policy & Regulatory, Health Service Delivery & Process of Care
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Prescribing Behavior, Reimbursement & Access Policy
Disease
Rare and Orphan Diseases