Drago Daniela, Foss-Campbell Betsy, Wonnacott Keith, Barrett David, Ndu Adora
Biogen, Inc., Global Safety and Regulatory Sciences, Cambridge, MA, USA.
American Society of Gene and Cell Therapy, Policy and Advocacy, Milwaukee, WI, USA.
Mol Ther Methods Clin Dev. 2021 Apr 5;21:524-529. doi: 10.1016/j.omtm.2021.04.001. eCollection 2021 Jun 11.
The rapid expansion of the gene therapy pipeline in recent years offers significant potential to treat diseases with great unmet medical need. However, the unique nature of these therapies poses challenges to regulating them within traditional frameworks, even when developing in a single country. Various factors exacerbate the issues in commercializing products across regions, including the lack of established regulatory frameworks for developing gene therapy products in many jurisdictions. While some countries have established separate regulatory frameworks for advanced therapies/regenerative medicine products, differences exist between them. Recommended solutions to overcome these hurdles include fostering convergence among countries with separate regulatory frameworks for these products and utilizing reliance and recognition for countries without such frameworks. Additionally, regulators who choose to establish new dedicated frameworks for regulating gene therapies should consider the inclusion of key elements such as expedited regulatory pathways that offer early engagement with regulators, innovative clinical trial design, and adequate post-market confirmatory studies. Increasing the alignment of regulatory pathways across countries will be crucial to facilitating the development of, and access to, gene therapies on a global scale.
近年来基因治疗产品线的迅速扩张为治疗存在巨大未满足医疗需求的疾病提供了巨大潜力。然而，这些疗法的独特性质给在传统框架内对其进行监管带来了挑战，即使是在单个国家开展研发也是如此。各种因素加剧了跨地区产品商业化的问题，包括许多司法管辖区缺乏针对基因治疗产品研发的既定监管框架。虽然一些国家已经为先进疗法/再生医学产品建立了单独的监管框架，但它们之间存在差异。克服这些障碍的建议解决方案包括促进对这些产品有单独监管框架的国家之间的趋同，并对没有此类框架的国家采用依赖和认可的方式。此外，选择为基因治疗建立新的专门监管框架的监管机构应考虑纳入关键要素，如提供与监管机构早期接触的加速监管途径、创新的临床试验设计以及充分的上市后确证性研究。加强各国监管途径的一致性对于在全球范围内促进基因治疗的开发和可及性至关重要。
