The path to successful commercialization of cell and gene therapies: empowering patient advocates

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• 作者：Gerhard Bauer¹ ; Mohamed Abou-El-Enein² ; ^{mohamed.abou-el-enein@charite.de} ; Alastair Kent³ ; Brian Poole⁴ ; Miguel Forte⁴ ; ⁵
• 关键词：affordability ; cell and gene therapies ; clinical trials commercialization ; patient advocacy ; regulation ; reimbursement
• 刊名：Cytotherapy
• 出版年：2017
• 出版时间：February 2017
• 年：2017
• 卷：19
• 期：2
• 页码：293-298
• 全文大小：135 K
• 卷排序：19

文摘

Often, novel gene and cell therapies provide hope for many people living with incurable diseases. To facilitate and accelerate a successful regulatory approval and commercialization path for effective, safe and affordable cell and gene therapies, the involvement of patient advocacy groups (PAGs) should be considered early in the development process. This report provides a thorough overview of the various roles PAGs play in the clinical translation of cell and gene therapies and how they can bring about positive changes in the regulatory process, infrastructure improvements and market stability.