Proceedings: International Regulatory Considerations on Development Pathways for Cell Therapies

Abstract

Regenerative medicine is a rapidly evolving field that faces novel scientific and regulatory challenges. In September 2013, the International Workshop on Regulatory Pathways for Cell Therapies was convened to discuss the nature of these challenges and potential solutions and to highlight opportunities for potential convergence between different regulatory bodies that might assist the field's development. The workshop discussions generated potentially actionable steps in five main areas that could mitigate cell therapy development pathway risk and accelerate moving promising therapies to patients. These included the need for convergence of regulatory guidelines on donor eligibility and suitability of lines for use in clinical trials and subsequent commercialization for cell therapies to move forward on a global basis; the need to challenge and encourage investigators in the regenerative medicine field to share information and provide examples of comparability studies related to master cell banks; the need for convergence of guidelines across regulatory jurisdictions on requirements for tumorigenicity studies, based on particular cell types and on biodistribution studies; the need to increase transparency in sharing clinical trial information more broadly and disseminating results more rapidly; and the need to establish a forum for sharing the experiences of various approaches being developed to expedite regulatory approvals and access for patients to innovative cell and regenerative therapies in the different regulatory jurisdictions and to assess their potential strengths and weaknesses.

A novel technique for decellularization of allogenic nerves and in vivo study of their use for peripheral nerve reconstruction

“…However, they are imported from US and have high costs discouraging their employment in Europe. Moreover, American donor selection criteria do not always match the European regulations and standards [30].…”

A global comparative overview of the legal regulation of stem cell research and therapy: Lessons for South Africa

“…Two examples are the issues of donor eligibility and the suitability of stem cell lines for use in clinical trials and subsequent commercialisation. [52] The legal regulation of human tissue (governing the procurement, use and disposal of human tissue) and tissue establishments across jurisdictions is also diverse, creating additional obstacles.…”

Recent developments in ex vivo platelet production

“…On the other hand, the use of immortalized MKs previously established from leukemia patients presents a significant challenge to meet GMP-standards. Based on FDA regulations, the use of cell lines derived from non-GMP sources is unacceptable unless the cells possess unreplaceable benefit to humans (Feigal et al 2014). This condition indicates the importance of GMP-standards during the establishment of newly isolated immortalized MKs in the future.…”