Predictors of orphan drug approval in the European Union.
Predictors of orphan drug approval in the European Union.
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DOI：
10.1007/s00228-007-0454-6
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发表时间：
2008-05
影响因子：
2.9
通讯作者：
Leufkens, Hubert G. M.
中科院分区：
文献类型：
--
作者：
Heemstra, Harald E.;de Vrueh, Remco L.;van Weely, Sonja;Bueller, Hans A.;Leufkens, Hubert G. M.
为了鼓励罕见病药物的开发，美国（1983 年）和欧盟（2000 年）引入了孤儿药立法。最近的文献讨论了可能影响欧盟新孤儿药产品开发的因素。本研究旨在确定潜在孤儿药在欧盟成功上市的预测因素。已经对随机选择的授权样本和尚未授权的孤儿药指定的匹配样本进行了比较。研究中的决定因素包括适应症、产品和申办者的特征。数据仅从公共领域收集。孤儿药批准与申办者之前获得另一种孤儿药批准的经验密切相关（OR = 17.3，95% CI = 5.6–53.1）。此外，与生物技术产品相比，现有的合成实体往往更有可能获得批准状态（OR = 3.9，95% CI = 0.9–16.6）。这项研究表明，公司开发孤儿药的经验是后续其他孤儿药授权的重要预测因素。这同样适用于现有的（合成的）分子，对此有很多知识可用。进一步的研究应针对那些未达到批准状态的指定孤儿药产品的临床开发计划的质量。
To encourage the development of drugs for rare diseases, orphan drug legislation has been introduced in the USA (1983) and in the EU (2000). Recent literature discusses factors that may influence the development of new orphan medicinal products in the EU. This study aims to identify predictors for successful marketing authorisation of potential orphan drugs in the EU. A comparison between randomly selected authorised and a matched sample of not-yet-authorised orphan drug designations has been performed. Determinants in the study included characteristics of the indication, of the product and of the sponsor. Data were collected from the public domain only. Orphan drug approval was strongly associated with previous experience of the sponsor in obtaining approval for another orphan drug (OR = 17.3, 95% CI = 5.6–53.1). Furthermore, existing synthetic entities compared to biotechnology products tended to have a higher likelihood of reaching approval status (OR = 3.9, 95% CI = 0.9–16.6). This study showed that experience of a company in developing orphan drugs is an important predictor for subsequent authorisation of other orphan drugs. The same applies for existing (synthetic) molecules, for which much knowledge is available. Further research should be directed towards studying the quality of the clinical development program of those designated orphan medicinal products not reaching approval status.
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DOI：
10.2471/blt.06.031518
发表时间：
2006-09-01
期刊：
Bulletin of the World Health Organization: International Journal of Public Health
影响因子：
--
作者：
Stolk, Pieter;Willemen, Marjolein JC;Leufkens, Hubert GM
通讯作者：
Leufkens, Hubert GM
影响因子：
120.1
作者：
Haffner, ME;Whitley, J;Moses, M
通讯作者：
Moses, M
影响因子：
9.3
作者：
Minghetti, P;Giudici, EM;Montanari, L
通讯作者：
Montanari, L
影响因子：
3.4
作者：
Joppi, R;Bertele, V;Garattini, S
通讯作者：
Garattini, S