孤儿药与非孤儿药在传染病中的临床试验评估——一项为期11年的分析【2010-2020】。

Q2 Medicine Perspectives in Clinical Research Pub Date : 2023-04-01 Epub Date: 2022-07-23 DOI:10.4103/picr.picr_137_21
Palvi Kudyar, Mahanjit Konwar, Zoya Khatri, Nithya Jaideep Gogtay, Urmila Mukund Thatte
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引用次数: 0

摘要

背景:1983年美国《孤儿药法案》为罕见病新疗法的发展提供了动力。几项研究的重点是随着时间的推移被指定为孤儿的人数。然而,很少有人关注能够获得批准的临床试验,尤其是针对传染病的临床试验。材料和方法:确定了2010年1月至2020年12月31日美国食品药品监督管理局(FDA)批准的所有新药(孤儿药和非孤儿药),批准细节取自每种药物的US-FDA标签和总结报告。每项关键试验都根据其设计进行了表征。我们使用卡方检验测试了药物批准类型与试验特征的相关性,并产生了95%置信区间的粗略比值比。结果:在批准的1122种药物中,84种是用于传染病的,其中18种是孤儿药,66种是非处方药。共有35项关键试验支持了18种孤儿药的批准,而115项关键试验则支持了66种非孤儿药。孤儿药入选/试验的中位参与者人数为89人,而非孤儿药为452人(P<0.0001)。13/35(37%)孤儿药与69/115(60%)非孤儿药进行了盲试(P=0.029);对15/35(42%)孤儿药与100/115(87%)非处方药进行了随机分组(P<0.0001),20/35(57%)孤儿药在II期获得批准,而8/115(6%)非处方药物获得批准(P<0.00001)。
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Evaluation of clinical trials done for orphan drugs versus nonorphan drugs in infectious diseasesan eleven year analysis [2010-2020].

Background: The 1983 US Orphan Drug Act provided impetus for the development of new therapies for rare diseases. Several studies focused on the number of orphan designations over time. However, very few focused on clinical trials that lead to their approval, particularly for infectious diseases.

Materials and methods: All new drug approvals (orphan and non-orphan) by the US Food and Drug Administration (FDA) from January 2010 to December 31, 2020, were identified and details of approvals were taken from the US-FDA labels and summary reports for each drug. The pivotal trials for each were characterized based on their design. We tested the association of the type of drug approval with respect to the characteristics of trial using Chi-square test and generated crude odds ratios with 95% confidence intervals.

Results: From the total 1122 drugs approved, 84 were for infectious diseases, of which 18 were orphan drugs and 66 were nonorphan. A total of 35 pivotal trials supported 18 orphan drug approvals, while 115 pivotal trials supported 66 nonorphan drugs. The median number of participants enrolled/trial for orphan drugs was 89, while for nonorphan drugs, it was 452 (P < 0.0001). Blinding was done for 13/35 (37%) orphan drugs versus 69/115 (60%) nonorphan drugs (P = 0.029); randomization was done for 15/35 (42%) orphan drugs versus 100/115 (87%) nonorphan drugs (P < 0.0001) and 20/35 (57%) of the orphan drugs got approval in phase II versus 8/115 (6%) of nonorphan drugs (P < 0.00001).

Conclusion: A significant number of orphan drugs get approval based on early phase, nonrandomized, and unblinded with a smaller sample size as compared to nonorphan drugs.

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来源期刊
Perspectives in Clinical Research
Perspectives in Clinical Research Medicine-Medicine (all)
CiteScore
2.90
自引率
0.00%
发文量
41
审稿时长
36 weeks
期刊介绍: This peer review quarterly journal is positioned to build a learning clinical research community in India. This scientific journal will have a broad coverage of topics across clinical research disciplines including clinical research methodology, research ethics, clinical data management, training, data management, biostatistics, regulatory and will include original articles, reviews, news and views, perspectives, and other interesting sections. PICR will offer all clinical research stakeholders in India – academicians, ethics committees, regulators, and industry professionals -a forum for exchange of ideas, information and opinions.
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