Wang Ting, McAuslane Neil, Goettsch Wim G, Leufkens Hubert G M, De Bruin Marie L
Centre for Innovation in Regulatory Science (CIRS), London, United Kingdom.
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, Netherlands.
Front Pharmacol. 2022 Jul 18;13:948161. doi: 10.3389/fphar.2022.948161. eCollection 2022.
The target product profile (TPP) outlines the desired profile of a target product aimed at a particular disease and is used by companies to plan clinical development. Considering the increasing importance of health technology assessment (HTA) in informing reimbursement decisions, a robust TPP needs to be built to address HTA needs, to guide an integrated evidence generation plan that will support HTA submissions. This study assessed current practices and experiences of companies in building HTA considerations into TPP development. An opinion survey was designed and conducted in 2019, as a cross-sectional questionnaire consisting of multiple-choice questions. The questionnaire provided a qualitative assessment of companies' strategies and experiences in building HTA considerations into the TPP. Eligible survey participants were the senior management of Global HTA/Market Access Departments at 18 top international pharmaceutical companies. 11 companies responded to the survey. All companies included HTA requirements in TPP development, but the timing and process varied. The key focus of HTA input related to health problems and treatment pathways, clinical efficacy/effectiveness, and safety. Variance of HTA methods and different value frameworks were identified as a challenge for development plans. Stakeholder engagement, such as HTA scientific advice, was used to pressure test the TPP. This research provides insight into current practice and potential opportunities for value-based drug development. It demonstrates the evolution of the TPP to encompass HTA requirements and suggests that the TPP could have a role as an iterative communication tool for use with HTA agencies to enhance an integrated evidence generation plan.
目标产品概况（TPP）概述了针对特定疾病的目标产品的理想概况，公司用其来规划临床开发。鉴于卫生技术评估（HTA）在为报销决策提供信息方面的重要性日益增加，需要构建一个完善的TPP来满足HTA需求，以指导一个将支持HTA申报的综合证据生成计划。本研究评估了公司在将HTA考量纳入TPP制定过程中的当前做法和经验。2019年设计并开展了一项意见调查，采用包含多项选择题的横断面问卷形式。该问卷对公司在将HTA考量纳入TPP方面的策略和经验进行了定性评估。符合条件的调查对象是18家顶级国际制药公司全球HTA/市场准入部门的高级管理人员。11家公司回复了调查。所有公司在TPP制定过程中都纳入了HTA要求，但时间安排和流程各不相同。HTA投入的关键重点涉及健康问题和治疗途径、临床疗效/有效性以及安全性。HTA方法的差异和不同的价值框架被确定为开发计划面临的一项挑战。利益相关者参与，如HTA科学建议，被用于对TPP进行压力测试。本研究为基于价值的药物开发的当前实践和潜在机会提供了见解。它展示了TPP的演变，以涵盖HTA要求，并表明TPP可作为一种迭代沟通工具，与HTA机构配合使用，以完善综合证据生成计划。
