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The evolution of patient-focused drug development and Duchenne muscular dystrophy.
Expert Review of Pharmacoeconomics & Outcomes Research ( IF 2.3 ) Pub Date : 2020-02-26 , DOI: 10.1080/14737167.2020.1734454
Norah L Crossnohere 1, 2 , Ryan Fischer 3 , Emily Crossley 4 , Elizabeth Vroom 5 , John Fp Bridges 1, 2
Affiliation  

Introduction: There is a groundswell of interest from patient, industry, and groups to rigorously and transparently integrate patient-voice into regulatory decision-making. Patient-focused drug development (PFDD) is an approach established by the US Food and Drug Administration to systematically incorporate patient experiences into drug development and evaluation. It has created a demand for scientific advancement to measure and integrate patient voice into decision making.Areas covered: This narrative review describes the evolving nature of advocacy-regulatory relations preceding PFDD, characterizes current PFDD and other patient-engagement activities, and explores future opportunities for patient participation along the drug development pipeline. We present Duchenne muscular dystrophy as a case study to illustrate how PFDD is being operationalized by patient groups and regulators using both verbal and written data sources.Expert opinion: PFDD represents the most widespread approach yet to integrate the patient voice as a source of evidence to inform regulatory decision making. Regulatory approvals are just one frontier in drug development. On the horizon remain uncertainties in how patient experience can inform post-marketing surveillance, pricing, reimbursement, and health technology assessment. Patient-input may be particularly crucial to demonstrate the value of expensive first-generation rare disease treatments that confer meaningful benefits but do not meet traditional thresholds for cost-effectiveness.

中文翻译:

以患者为中心的药物开发和杜兴肌营养不良症的演变。

简介:患者,行业和团体的兴趣浓厚,他们希望将患者的声音严格透明地整合到监管决策中。以患者为中心的药物开发(PFDD)是美国食品和药物管理局(US Food and Drug Administration)建立的一种方法,用于将患者的经验系统地纳入药物开发和评估中。它引起了对科学进步的需求,以将患者的声音衡量并整合到决策中。涵盖的领域:本叙述性回顾描述了PFDD之前倡导-调节关系的发展性质,描述了当前PFDD和其他患者参与活动的特征,并探索了未来的机会让患者参与药物开发流程。我们以案例分析杜氏肌营养不良症为例,以说明患者组和监管者如何使用口头和书面数据来源来操作PFDD。专家意见:PFDD代表了迄今为止最广泛的方法,将患者声音整合为证据告知监管决策。监管批准只是药物开发的前沿领域之一。在患者体验如何为上市后监测,定价,报销和健康技术评估提供信息方面仍存在不确定性。病人的投入对于证明昂贵的第一代罕见病治疗的价值尤其重要,这种治疗可以带来有意义的好处,但不符合成本效益的传统阈值。专家意见:PFDD代表了迄今为止最广泛的方法,该方法整合了患者的声音作为证据,为监管决策提供了依据。监管批准只是药物开发的前沿领域之一。在患者体验如何为上市后监测,定价,报销和健康技术评估提供信息方面仍存在不确定性。病人的投入对于证明昂贵的第一代罕见病治疗的价值尤其重要,这种治疗可以带来有意义的好处,但不符合成本效益的传统阈值。专家意见:PFDD代表了迄今为止最广泛的方法,该方法整合了患者的声音作为证据,为监管决策提供了依据。监管批准只是药物开发的前沿领域之一。在患者体验如何为上市后监测,定价,报销和健康技术评估提供信息方面仍存在不确定性。病人的投入对于证明昂贵的第一代罕见病治疗的价值尤其重要,这种治疗可以带来有意义的好处,但不符合成本效益的传统阈值。在患者体验如何为上市后监测,定价,报销和健康技术评估提供信息方面仍存在不确定性。病人的投入对于证明昂贵的第一代罕见病治疗的价值尤其重要,这种治疗可以带来有意义的好处,但不符合成本效益的传统阈值。在患者体验如何为上市后监测,定价,报销和健康技术评估提供信息方面仍存在不确定性。病人的投入对于证明昂贵的第一代罕见病治疗的价值尤其重要,这种治疗可带来有意义的益处,但不符合成本效益的传统阈值。
更新日期:2020-02-26
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