The Potential of Disease Progression Modeling to Advance Clinical Development and Decision Making

IF 6.3 2区医学 Q1 PHARMACOLOGY & PHARMACY

Clinical Pharmacology & Therapeutics Pub Date : 2024-10-15 DOI:10.1002/cpt.3467

Mary Summer Starling, Lindsay Kehoe, Bruce K. Burnett, Phil Green, Karthik Venkatakrishnan, Rajanikanth Madabushi

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引用次数: 0

Abstract

While some model-informed drug development frameworks are well recognized as enabling clinical trials, the value of disease progression modeling (DPM) in impacting medical product development has yet to be fully realized. The Clinical Trials Transformation Initiative assembled a diverse project team from across the patient, academic, regulatory, and industry sectors of practice to advance the use of DPM for decision making in clinical trials and medical product development. This team conducted a scoping review to explore current applications of DPM and convened a multi-stakeholder expert meeting to discuss its value in medical product development. In this article, we present the scoping review and expert meeting output and propose key questions that medical product developers and regulators may use to inform clinical development strategy, appreciate the therapeutic context and endpoint selection, and optimize trial design with disease progression models. By expanding awareness of the unique value of DPM, this article does not aim to be technical in nature but rather aims to highlight the potential of DPM to improve the quality and efficiency of medical product development.

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疾病进展模型推进临床开发和决策的潜力。

虽然一些以模型为基础的药物开发框架在促进临床试验方面得到了广泛认可，但疾病进展建模（DPM）在影响医疗产品开发方面的价值尚未得到充分认识。临床试验转型计划组建了一支来自患者、学术界、监管部门和业界的多元化项目团队，以推动在临床试验和医药产品开发决策中使用 DPM。该团队进行了一次范围审查，以探索 DPM 的当前应用，并召开了一次多方利益相关者专家会议，讨论其在医疗产品开发中的价值。在本文中，我们介绍了范围界定审查和专家会议的成果，并提出了一些关键问题，供医疗产品开发人员和监管机构用于制定临床开发策略、了解治疗背景和终点选择，以及利用疾病进展模型优化试验设计。通过扩大对 DPM 独特价值的认识，本文并不以技术性为目的，而是旨在强调 DPM 在提高医疗产品开发质量和效率方面的潜力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Pharmacology & Therapeutics 医学-药学

CiteScore

12.70

自引率

7.50%

发文量

290

审稿时长

2 months

期刊介绍： Clinical Pharmacology & Therapeutics (CPT) is the authoritative cross-disciplinary journal in experimental and clinical medicine devoted to publishing advances in the nature, action, efficacy, and evaluation of therapeutics. CPT welcomes original Articles in the emerging areas of translational, predictive and personalized medicine; new therapeutic modalities including gene and cell therapies; pharmacogenomics, proteomics and metabolomics; bioinformation and applied systems biology complementing areas of pharmacokinetics and pharmacodynamics, human investigation and clinical trials, pharmacovigilence, pharmacoepidemiology, pharmacometrics, and population pharmacology.