Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs

2021 2020 2021 Barkholt, 2019, Regulatory watch: European regulatory experience with advanced therapy medicinal products, Nat. Rev. Drug Discov., 18, 8, 10.1038/nrd.2018.200 Carvalho, 2017, Regulatory and scientific advancements in gene therapy: state-of-the-art of clinical applications and of the supporting european regulatory framework, Front. Med. (Lausanne), 4, 182, 10.3389/fmed.2017.00182 Elsallab, 2020, Mitigating deficiencies in evidence during regulatory assessments of advanced therapies: A comparative study with other biologicals, Mol. Ther. Methods Clin. Dev., 18, 269, 10.1016/j.omtm.2020.05.035 Abou-El-Enein, 2016, Overcoming challenges facing advanced therapies in the EU market, Cell Stem Cell, 19, 293, 10.1016/j.stem.2016.08.012 Bravery, 2019, EU market authorisation strategy: Lessons from the first 22 ATMP submitted to the EMA, Cell Gene Ther. Insights, 5, 759, 10.18609/cgti.2019.088 Lapteva, 2020, Clinical development of gene therapies: The first three decades and counting, Mol. Ther. Methods Clin. Dev., 19, 387, 10.1016/j.omtm.2020.10.004 Bennett, 2020, Regulatory considerations in the development of gene therapies for neurological disorders in the EU region: An industry perspective, Cell Gene Ther. Insights, 6, 877, 10.18609/cgti.2020.100 Salmikangas, 2015, Manufacturing, characterization and control of cell-based medicinal products: Challenging paradigms toward commercial use, Regen. Med., 10, 65, 10.2217/rme.14.65 van der Loo, 2016, Progress and challenges in viral vector manufacturing, Hum. Mol. Genet., 25, R42, 10.1093/hmg/ddv451 Salzman, 2018, Addressing the value of gene therapy and enhancing patient access to transformative treatments, Mol. Ther., 26, 2717, 10.1016/j.ymthe.2018.10.017 2017 Drago, 2020 Faulkner, 2017, Stretching and challenging the boundaries of law: Varieties of knowledge in biotechnologies regulation, Minerva, 55, 209, 10.1007/s11024-017-9326-0 Halioua-Haubold, 2017, Regulatory considerations for gene therapy products in the US, EU, and Japan, Yale J. Biol. Med., 90, 683 Nagai, 2017, New Japanese regulatory frameworks for clinical research and marketing authorization of gene therapy and cellular therapy products, Curr. Gene Ther., 17, 17, 10.2174/1566523217666170406123231 Parca, 2018, Proposed regulatory framework for advanced therapy products in brazil. Health surveillance under debate: Society, science & technology, Visa Em Debate, 6, 15, 10.22239/2317-269x.01078 Bruce, 2020 2017 2019 Caraballo, 2020, The role of raw materials in the manufacturing of cell and gene therapy medicinal products: Practices and challenges, Cell Gene Ther. Insights, 6, 229, 10.18609/cgti.2020.031 2019 2019 2020 2020 Eglovitch, 2020 2020 2020 Liberti, 2015, Adaptive licensing and facilitated regulatory pathways: A survey of stakeholder perceptions, Clin. Pharmacol. Ther., 98, 477, 10.1002/cpt.140 Jokura, 2018, Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union, J. Tissue Eng. Regen. Med., 12, e1056 Eichler, 2015, From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients, Clin. Pharmacol. Ther., 97, 234, 10.1002/cpt.59 2019 2018 2018 2020 2020 2019 2018 Abou-El-Enein, 2018, Registry contributions to strengthen cell and gene therapeutic evidence, Mol. Ther., 26, 1172, 10.1016/j.ymthe.2018.04.007 Fritsche, 2019, Post-marketing safety and efficacy surveillance of cell and gene therapies in the EU: A critical review, Cell Gene Ther. Insights, 5, 1505, 10.18609/cgti.2019.156 Corbett, 2017, Innovative regenerative medicines in the EU: A better future in evidence?, BMC Med., 15, 49, 10.1186/s12916-017-0818-4