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

Abstract

Gene- and cell-based therapies (GCTs) offer potential new treatment options for unmet medical needs. However, the use of conventional regulatory requirements for medicinal products to approve GCTs may impede patient access and therapeutic innovation. Furthermore, requirements differ between jurisdictions, complicating the global regulatory landscape. We provide a comparative overview of regulatory requirements for GCT approval in five jurisdictions and hypothesize on the consequences of the observed global differences on patient access and therapeutic innovation.

Health Care Evolves From Reactive to Proactive

“…These considerations underscore the importance of maintaining a focus on preventive approaches that complement therapeutic interventions, as our mechanistic armamentarium evolves from discoveries of the biological bases of health and disease . Although molecular innovation has been impressive, the translation of those discovery‐based insights into preventive therapies for individual patients, which are scalable to populations, must remain a priority .…”

Process Improvement for Maximized Therapeutic Innovation Outcome

“…However, while these technological approaches are poised to transform the outcomes of debilitating and deadly conditions, their penetration to the management of patients and populations is hindered by regulatory structures and regulations that have not kept pace. Indeed, there is marked variation in marketing, clinical practice guidelines, local and central regulation, as well as reimbursement policies across national jurisdictions . In that context, the majority of research and development activities in this field are still undertaken locally by academic developers and small and medium‐sized enterprises .…”

“…Indeed, there is marked variation in marketing, clinical practice guidelines, local and central regulation, as well as reimbursement policies across national jurisdictions . In that context, the majority of research and development activities in this field are still undertaken locally by academic developers and small and medium‐sized enterprises . These consideration highlight the need for improved coordination across medical and regulatory communities .…”

“…In that context, the majority of research and development activities in this field are still undertaken locally by academic developers and small and medium‐sized enterprises . These consideration highlight the need for improved coordination across medical and regulatory communities . Moreover, there is a need to build platforms for knowledge sharing, collaboration and learning among academia, developers, and regulatory authorities .…”

“…These consideration highlight the need for improved coordination across medical and regulatory communities . Moreover, there is a need to build platforms for knowledge sharing, collaboration and learning among academia, developers, and regulatory authorities . New models of precompetitive collaboration should be utilized to increase research efficiencies, while collaborations between regulatory agencies and interactions with developers need to be strengthened and harmonized .…”