Global Regulatory Perspective for MSCs

Mesenchymal stromal cell (MSC)-based therapies have potential in a range of disease indications, and both MSC therapies themselves as well as the applicable global regulatory frameworks governing their use are evolving. MSC therapies are making the transition from experimental and unlicensed therapies for use in named patients to medicinal products that must be granted a marketing authorization to be placed on the global pharmaceutical markets for the benefit of larger patient populations. Medicinal product legislation requires the demonstration of quality, safety, and efficacy through defined strategies around manufacturing, nonclinical studies, and clinical trials. This chapter discusses in particular the medicinal product legislation applicable to MSC-based products being developed for commercialization and provides an overview of key considerations in the different regulatory jurisdictions globally.