Scientific considerations for the regulatory evaluation of cell therapy products

Biologicals. 2017 Nov:50:20-26. doi: 10.1016/j.biologicals.2017.08.011. Epub 2017 Sep 6.

Abstract

Cell therapy involves the administration of a viable somatic cell preparation to a patient for the treatment of a disease or traumatic damage. Because cell therapies are complex and very different from traditional biological products, they present significant challenges for regulatory authorities, manufacturers, developers, health care providers, and patients involved in their application. Like other emerging areas of biomedical research and development, there are many issues where regulatory views and decisions among countries and regions may differ due to minimal scientific evidence to support safety and efficacy, and lack of experience with these novel treatments. A brief overview of the current regulatory landscape for cell-based therapies is presented, and the need for a global effort to develop a set of common principles that may serve to facilitate the regulatory evaluation and market availability of these products is identified. In addition, a number of elements that could form a core consensus package of requirements for evaluating human cell therapy products is presented in the supplemental material which should be read in conjunction with the manuscript.

Keywords: Regulatory evaluation; Scientific evidence; Stem cells; Therapy; Transplantation.

Publication types

  • Review

MeSH terms

  • Biomedical Research / methods*
  • Biomedical Research / standards
  • Biomedical Research / trends
  • Cell Culture Techniques / methods*
  • Cell- and Tissue-Based Therapy / methods*
  • Cell- and Tissue-Based Therapy / standards
  • Cell- and Tissue-Based Therapy / trends
  • Guidelines as Topic
  • Humans
  • Quality Control
  • Tissue Engineering / methods*
  • World Health Organization