Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment.
Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment.
复制标题
DOI：
10.3389/fphar.2018.01263
复制
发表时间：
2018
影响因子：
5.6
通讯作者：
Pilc A
中科院分区：
文献类型：
--
作者：
Malinowski KP;Kawalec P;Trabka W;Sowada C;Pilc A
目的：评估不同欧盟成员国的孤儿药报销份额和报销决策协议，并确定报销几率受欧洲药品管理局（EMA）有条件批准或特殊情况或疾病类型的影响。方法：从EMA网站上收集目前已批准的孤儿药目录。对于每种药物，收集了有关有条件批准或特殊情况下批准的信息。报销情况可在国家报销或HTA机构网站上查询。选定国家之间的偿还决定的协议是用κ系数来衡量协议的评估。EMA有条件批准和特殊情况下批准的影响使用逻辑回归进行评估，并以优势比表示。结果：孤儿药报销比例从波兰的27%到丹麦的88%差异显著，平均值为51% （p < 0.0001）。关于报销状况，西班牙和意大利之间的一致性最高，一致性最显著，德国和英国之间的一致性最低，κ值分别为0.64和0.01。在法国、意大利和西班牙，有条件批准状态显著降低了77-80%的报销机会；然而，在特殊情况下批准仅在德国有显著影响，报销机会减少85%。疾病类型（肿瘤或代谢性）与有条件批准（p值为0.03 -肿瘤药物比其他分析药物更有可能获得有条件批准）和特殊情况（p值为0.02 -代谢性疾病药物更有可能在特殊情况下获得批准）显著相关。结论：获得报销孤儿药在欧盟国家之间差异很大。在偿还决定方面，意大利和西班牙之间的一致性最高，而德国和英国之间的一致性最低。在一些国家，有条件批准和在特殊情况下批准是报销的重要负面预测因素，它们与疾病类型（肿瘤或代谢性疾病）显著相关。
Objective: To assess shares of reimbursed orphan drugs and agreement in reimbursement decision-making in different European Union member states as well as to define odds for reimbursement influenced by the presence of conditional approval or exceptional circumstances granted by the European Medicines Agency (EMA) or by type of the disease. Methods: The list of authorized drugs with current orphan designations was collected from the website of the EMA. For each drug, the information regarding conditional approval or approval under exceptional circumstances was collected. The reimbursement statuses were available on national reimbursement or HTA agencies websites. The agreement for reimbursement decisions between selected countries was assessed using the κ coefficient for the measurement of agreement. The impact of the EMA's conditional approval as well as approval under exceptional circumstances was assessed using the logistic regression and presented as odds ratio. Results: The percentage of reimbursed orphan drugs varied significantly from 27% in Poland to 88% in Denmark, with an average value of 51% (p < 0.0001). Regarding the reimbursement status, the highest, substantial agreement was observed between Spain and Italy, and the lowest agreement was observed between Germany and England, with κ of 0.64 and 0.01, respectively. Conditional approval status significantly decreased the chance for reimbursement in France, Italy, and Spain by 77–80%; however, approval granted under exceptional circumstances had significant impact only in Germany with 85% decrease in chances for reimbursement. The type of the disease (oncology or metabolic) was significantly associated with both conditional approval (p of 0.03—oncology drugs were more likely to be conditionally approved then the rest of analyzed drugs) and exceptional circumstances (p of 0.02—drugs for metabolic diseases were more likely to be approved under exceptional circumstances). Conclusions: Access to reimbursed orphan drugs varies significantly across EU countries. The highest, substantial agreement in reimbursement decisions was observed between Italy and Spain and the lowest between Germany and England. Conditional approval and approval under exceptional circumstances were significant negative predictors of reimbursement in some countries and they were significantly associated with the type of the disease (oncology or metabolic).
登录
查看更多内容
影响因子：
5.6
作者：
Jakovljevic M;Lazarevic M;Milovanovic O;Kanjevac T
通讯作者：
Kanjevac T
影响因子：
11.5
作者：
Di Paolo, Antonello;Arrigoni, Elena
通讯作者：
Arrigoni, Elena
影响因子：
4.4
作者：
Denis, Alain;Mergaert, Lut;Simoens, Steven
通讯作者：
Simoens, Steven
影响因子：
2.9
作者：
Sarnola, Kati;Ahonen, Riitta;Timonen, Johanna
通讯作者：
Timonen, Johanna
DOI：
10.1080/20016689.2017.1297886
发表时间：
2017
期刊：
Journal of market access & health policy
影响因子：
--
作者：
Young KE;Soussi I;Hemels M;Toumi M
通讯作者：
Toumi M