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The examination of orphan drugs in selected countries i.e., Belgium, Denmark, England, France, Germany, Italy, Poland, Scotland, Spain, Sweden, The Netherlands, and Wales revealed that the percentage of reimbursed orphan drugs varied among the countries from the lowest in Poland to the highest in Denmark. Analysis of agreement in decision-making regarding reimbursement of orphan drugs and HTA recommendations among analysed countries revealed that the highest, substantial agreement in reimbursement decisions was observed between Italy and Spain, and the highest agreement in recommendations was observed between England and Scotland. Study of the EMA-issued type of authorisation of analysed orphan drugs showed that the conditional approval significantly decreased the chance for reimbursement in France, Italy, and Spain; however the approval granted under the exceptional circumstances had similar impact only in Germany. Analysis of impact of the type of the disease treated with the specific orphan drugs revealed that the drugs for metabolic diseases were more likely to be approved under exceptional circumstances, but had lesser odds for being conditionally approved when compared to other drugs for non-metabolic and non-oncologic diseases. The opposite was observed for drugs used in treatment of patients with oncologic diseases. In-depth study of reimbursement decision-making pr ; ocess among EU-CEE countries i.e., Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, and Slovakia revealed that some of the considered countries already established separate regulations on reimbursement of orphan drugs; in case of some of these countries higher ICER values for orphans are used; in Lithuania and Romania, no formal HTA process was employed. The examination of orphan drugs in selected countries showed that the share of reimbursed orphan drugs varied significantly across the countries, but it was not associated either with GDP or GDP per capita. Analysis of agreement in decision-making regarding reimbursement of orphan drugs revealed that the lowest, slight agreement in reimbursement decisions was observed between Estonia and Latvia, and the highest, substantial agreement, between Estonia and Lithuania. Additionally, in Czechia, Lithuania and Slovakia, EMA’s conditional approval significantly decreased the chances for reimbursement. What is more, in Croatia, Estonia, Hungary, and Lithuania, drugs for oncological diseases had significantly greater chances for reimbursement. Detailed analysis of HTA recommendation-making process revealed that in Hungary, Latvia, and Slovakia, a positive recommendation was associated with a positive reimbursement decision, while a negative recommendation, with a lack of reimbursement. The study of ; subset of orphan drugs used in treatment of oncology diseases showed that the highest share of drugs with any recommendation was observed in Poland, and the lowest, in Latvia and Romania. The share of reimbursed drugs was the lowest in Latvia and the highest in Poland. Analysis of public expenditures revealed that the reimbursement of oncology orphan drugs is associated with a growing burden for the public budget, with an average 3-year increase in expenses of 68%, as compared with an increase of only 8.5% in the total GDP and of 9.3% in GDP per capita among EU-CEE countries. The total expenditures on the reimbursement of oncology orphan drugs varied among countries and was highly correlated with the total national GDP but not with GDP per capita. Expenditures per capita also were not significantly correlated with GDP per capita.
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Rada Dyscypliny Nauki o zdrowiu
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Last modified:
May 24, 2024
In our library since:
May 24, 2024
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9
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10
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http://dl.cm-uj.krakow.pl:8080/publication/5120
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| Edition name | Date |
|---|---|
| ZB-139807 | May 24, 2024 |
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