A Risk-Based Assessment for Determining the Pharmacokinetic Comparability Requirements of Biologic-Device Combination Products Administered by Subcutaneous Injection.

IF 5 3区 医学 Q1 PHARMACOLOGY & PHARMACY
Mark Klitgaard Nøhr, Shaik Rizwan Waheed, Rasmus Juul Kildemoes, Vibeke Hatorp, Tine Aggerholm Bækdal, Charlotte Lindorff Adrian, Lars Peter Korsholm, Eva Lisby Arp-Hansen, Helle Holst, Scott W Roberts
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Abstract

The development of new large molecule drug therapies along with the innovation of biologic-device combination products such as prefilled syringes, autoinjectors and pen injectors have significantly impacted the treatment of new diseases and has improved the process of administering parenteral medicines. To support the regulatory approval of a new biologic-device combination products or subsequent chemistry, manufacturing and control changes impacting a combination product, sponsor companies must thoroughly assess the potential impact to product quality, safety and efficacy. In this report, a risk-based process to determine the potential impact to product quality, safety, and efficacy as well as corresponding regulatory actions supporting a chemistry, manufacturing and control change is presented. The risk assessment includes the standardized assessment of a) chemistry, manufacturing and control risk factors, potential responses and appropriately weighted scoring; b) pharmacokinetic risk factors, potential responses and appropriately weighted scoring; and c) the use of a 2-dimensional risk grid to combine the chemistry, manufacturing and control risks and pharmacokinetic risks to provide a regulatory recommendation. Three case studies (two clinical case studies and a post-approval case study) are provided to demonstrate the assessment process and capabilities.

Abstract Image

基于风险的评估,用于确定皮下注射给药的生物制剂-器械组合产品的药代动力学可比性要求。
新型大分子药物疗法的开发以及预灌封注射器、自动注射器和笔式注射器等生物制剂-器械组合产品的创新,对新疾病的治疗产生了重大影响,并改善了肠外药物的给药过程。为支持监管机构批准新的生物制剂-器械组合产品或影响组合产品的后续化学、生产和控制变更,申办公司必须彻底评估对产品质量、安全性和有效性的潜在影响。本报告介绍了一种基于风险的流程,用于确定对产品质量、安全性和疗效的潜在影响,以及支持化学、生产和控制变更的相应监管措施。风险评估包括以下方面的标准化评估:a) 化学、生产和控制风险因素、潜在应对措施和适当加权评分;b) 药物动力学风险因素、潜在应对措施和适当加权评分;c) 使用二维风险网格将化学、生产和控制风险与药物动力学风险结合起来,以提供监管建议。提供了三个案例研究(两个临床案例研究和一个批准后案例研究),以展示评估过程和能力。
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来源期刊
AAPS Journal
AAPS Journal 医学-药学
CiteScore
7.80
自引率
4.40%
发文量
109
审稿时长
1 months
期刊介绍: The AAPS Journal, an official journal of the American Association of Pharmaceutical Scientists (AAPS), publishes novel and significant findings in the various areas of pharmaceutical sciences impacting human and veterinary therapeutics, including: · Drug Design and Discovery · Pharmaceutical Biotechnology · Biopharmaceutics, Formulation, and Drug Delivery · Metabolism and Transport · Pharmacokinetics, Pharmacodynamics, and Pharmacometrics · Translational Research · Clinical Evaluations and Therapeutic Outcomes · Regulatory Science We invite submissions under the following article types: · Original Research Articles · Reviews and Mini-reviews · White Papers, Commentaries, and Editorials · Meeting Reports · Brief/Technical Reports and Rapid Communications · Regulatory Notes · Tutorials · Protocols in the Pharmaceutical Sciences In addition, The AAPS Journal publishes themes, organized by guest editors, which are focused on particular areas of current interest to our field.
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