Global Regulatory Differences for Gene‐ and Cell‐Based Therapies: Consequences and Implications for Patient Access and Therapeutic Innovation

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GCTs represent a new class of medicinal products.1 These therapies are developed at the frontline of biotechnological innovation and could offer new treatment options in disease areas with limited treatment availability.2 However, the number of GCTs that are currently available to patients remains rather limited, despite substantial advances in this field.4 The paucity of available GCTs is often attributed to hurdles to translate GCTs from bench to bedside, but the regulatory landscape for marketing approval of medicinal products is also considered a barrier to GCT development, as current regulatory requirements for medicines are not tailored to GCT development.2 For instance, randomized‐controlled clinical trial (RCT) design is preferred to assess medicinal products for approval,7 but invasive delivery methods, small patient populations, and a potential lack of comparator treatments and clinical endpoints complicate RCT design for GCTs.2 Developers also face hurdles to meet manufacturing and quality standards. Lots are often small, with potentially high variability between lots.8 In addition, GCTs often originate from clinical practice and are largely developed by academic hospitals and small biotechnology companies,9 who often do not have experience with regulatory procedures.11
The global regulatory environment is also complex because regulatory frameworks for GCTs differ between jurisdictions, including requirements for approval.13 In 2007, new legislation for GCTs was implemented in the European Union (EU),2 and more recently, in Japan in 2014.14 The United States (US) and other jurisdictions currently regulate GCTs based on existing laws for biologics and by explicating the specific requirements for GCTs in scientific guidelines.16 However, how these various approaches to regulate approval of GCTs compare and affect patients and therapeutic innovation is unknown. Therefore, we first provide a comparative overview of how GCTs are regulated as medicinal products by comparing 1) legal provisions and guidance for approval of GCTs as medicinal products, 2) entry criteria for medicinal product regulations, and 3) criteria for approval in Canada, the EU, Korea, Japan, and the US. Second, we hypothesize on the consequences and potential implications of the observed regulatory differences between jurisdictions on patient access and therapeutic innovation.
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