{"title":"探讨批次间差异对建立吸入粉末药物产品药代动力学生物等效性的潜在影响。","authors":"Shuhui Li, Kairui Feng, Jieon Lee, Yuqing Gong, Fang Wu, Bryan Newman, Miyoung Yoon, Lanyan Fang, Liang Zhao, Jogarao V S Gobburu","doi":"10.1002/psp4.13276","DOIUrl":null,"url":null,"abstract":"<p><p>Batch-to-batch variability in inhalation powder has been identified as a potential challenge in the development of generic versions. This study explored the impact of batch-to-batch variability on the probability of establishing pharmacokinetic (PK) bioequivalence (BE) in a two-sequence, two-period (2 × 2) crossover study. A model-based parametric simulation approach was employed, incorporating batch-to-batch variability through the relative bioavailability (RBA) ratio. In the absence of batch variability, recruiting a total of 48 subjects in a 2 × 2 crossover study with the reference formulation resulted in a 95% probability of concluding BE. However, this probability decreased to 80% with a 5% batch difference in RBA and further declined to 30% with a 10% batch difference. With a 10% batch difference, the required number of subjects to achieve an 80% probability of concluding BE increased to 84. When considering product differences between the reference and the test formulations, an additional 10% batch difference reduced the study power from 97% to 30% for a T/R bioavailability ratio of 100% in a 2 × 2 crossover study with 48 subjects. As a result, the substantial impact of batch-to-batch variability on the study power and type I error of the PK BE study may pose significant challenges for the development of generic Advair Diskus due to its degree of PK batch-to-batch variability. Therefore, alternative PK BE study designs and guidelines are needed to adequately address the influence of batch-to-batch variability in products like Advair Diskus.</p>","PeriodicalId":10774,"journal":{"name":"CPT: Pharmacometrics & Systems Pharmacology","volume":" ","pages":""},"PeriodicalIF":3.1000,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Exploration of the potential impact of batch-to-batch variability on the establishment of pharmacokinetic bioequivalence for inhalation powder drug products.\",\"authors\":\"Shuhui Li, Kairui Feng, Jieon Lee, Yuqing Gong, Fang Wu, Bryan Newman, Miyoung Yoon, Lanyan Fang, Liang Zhao, Jogarao V S Gobburu\",\"doi\":\"10.1002/psp4.13276\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Batch-to-batch variability in inhalation powder has been identified as a potential challenge in the development of generic versions. 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引用次数: 0
摘要
吸入粉剂的批次间变异性被认为是开发仿制药的潜在挑战。本研究探讨了在一项两序列、两周期(2 × 2)交叉研究中,批次间变异性对建立药代动力学(PK)生物等效性(BE)概率的影响。研究采用了一种基于模型的参数模拟方法,通过相对生物利用度(RBA)比值纳入了批次间的变异性。在不存在批次变异的情况下,使用参比制剂进行 2 × 2 交叉研究,共招募 48 名受试者,得出 BE 结论的概率为 95%。然而,当 RBA 的批次差异为 5%时,这一概率降至 80%;当批次差异为 10%时,这一概率进一步降至 30%。当批次差异为 10%时,得出 BE 结论的概率为 80%所需的受试者人数增加到 84 人。考虑到参比制剂和试验制剂之间的产品差异,在一项有 48 名受试者参加的 2 × 2 交叉研究中,当 T/R 生物利用率比为 100%时,额外的 10%批次差异会使研究功率从 97% 降至 30%。因此,批间变异性对 PK BE 研究的研究功率和 I 型误差的重大影响可能会给仿制药安乃近的开发带来重大挑战,因为它的 PK 批间变异性程度很高。因此,有必要制定替代的 PK BE 研究设计和指南,以充分解决批间变异性对安乃近(Advair Diskus)等产品的影响。
Exploration of the potential impact of batch-to-batch variability on the establishment of pharmacokinetic bioequivalence for inhalation powder drug products.
Batch-to-batch variability in inhalation powder has been identified as a potential challenge in the development of generic versions. This study explored the impact of batch-to-batch variability on the probability of establishing pharmacokinetic (PK) bioequivalence (BE) in a two-sequence, two-period (2 × 2) crossover study. A model-based parametric simulation approach was employed, incorporating batch-to-batch variability through the relative bioavailability (RBA) ratio. In the absence of batch variability, recruiting a total of 48 subjects in a 2 × 2 crossover study with the reference formulation resulted in a 95% probability of concluding BE. However, this probability decreased to 80% with a 5% batch difference in RBA and further declined to 30% with a 10% batch difference. With a 10% batch difference, the required number of subjects to achieve an 80% probability of concluding BE increased to 84. When considering product differences between the reference and the test formulations, an additional 10% batch difference reduced the study power from 97% to 30% for a T/R bioavailability ratio of 100% in a 2 × 2 crossover study with 48 subjects. As a result, the substantial impact of batch-to-batch variability on the study power and type I error of the PK BE study may pose significant challenges for the development of generic Advair Diskus due to its degree of PK batch-to-batch variability. Therefore, alternative PK BE study designs and guidelines are needed to adequately address the influence of batch-to-batch variability in products like Advair Diskus.