Regenerative medicines: A new regulatory paradigm for South Africa

Abstract

Clinicians are increasingly using regenerative medicines to repair, replace, regenerate or rejuvenate lost, damaged or diseased genes, cells, tissues or organs. In South Africa, access to these novel gene therapies and cell and tissue-based products is limited. The human leukocyte antigen (HLA) diversity and a paucity of suitable HLA-identical unrelated donors, results in limited access to haematopoietic stem and progenitor cell transplantation (HSPCT). Cell-based products could increase this access. Genetic diversity can also manifest in local or region-specific rare congenital disorders, and in vivo gene therapies hold the promise of developing treatments and cures for these debilitating disorders. South Africa has a disproportionate mortality rate due to non-natural causes, with many surviving with permanent injuries and disabilities. Tissue-engineered cell-based products have the potential to restore many of those affected and improve quality of life and productivity. These factors create an urgency for South Africa to develop regenerative medicines to address the country's unique needs and to provide access to these new and innovative treatment modalities. Achieving this objective requires a well-coordinated effort by multiple stakeholders and role players. A critical component of a regenerative medicine ecosystem is establishing an enabling regulatory framework for these new classes of medicines. Here we provide a brief profile of South Africa, including its genetic diversity, economy, the impact of the burden of disease, health policy and the healthcare system. We address the regulation of medicines, how the existing framework can accommodate regenerative medicines, and the steps needed to establish a future regulatory framework.

Keywords: Cell-based products; Hospital exemption; Regenerative medicine; Regulatory framework; South Africa.