Addressing the limitations of the regulatory landscape in South Africa regarding advanced cell and gene therapies and related sectors involving human cells, tissues and organs

Authors

  • I M Viljoen Institute for Cellular and Molecular Medicine, and SAMRC Extramural Unit for Stem Cell Research and Therapy, Faculty of Health Sciences, University of Pretoria, South Africa https://orcid.org/0000-0002-8582-6404
  • M S Pepper Institute for Cellular and Molecular Medicine, and SAMRC Extramural Unit for Stem Cell Research and Therapy, Faculty of Health Sciences, University of Pretoria, South Africa https://orcid.org/0000-0001-6406-2380

DOI:

https://doi.org/10.7196/SAMJ.2025.v115i1.2629

Keywords:

Advanced Cell-Based Products, Gene Therapies, Regulatory Frameworks, South Africa, Medicines and Related Substances Act, National Health Act

Abstract

Advanced cell-based and gene therapy products emerged during the 1990s as new health product categories for treating and curing previously untreatable or incurable conditions. These products are complex, diverse and therapeutically specific, requiring specialised regulatory frameworks. During the last three decades, several jurisdictions have constructed specific regulatory frameworks to ensure these products’ safety, clinical efficacy and quality. As these are new and disruptive products, these frameworks are continuously evolving. However, South Africa (SA)’s regulatory frameworks for medicines, human biological materials and genetically modified organisms have not kept pace with scientific and technological developments, leaving regulatory gaps. We briefly describe these novel products and their regulatory frameworks, and propose a way forward in SA.

Author Biography

  • M S Pepper, Institute for Cellular and Molecular Medicine, and SAMRC Extramural Unit for Stem Cell Research and Therapy, Faculty of Health Sciences, University of Pretoria, South Africa

    Director, Institute for Cellular and Molecular Medicine;

    Director, SAMRC Extramural Unit for Stem Cell Research and Therapy;

    Research Professor, Dept. Immunology, Faculty of Health Sciences, University of Pretoria

    Professeur Associé, Department of Human Genetics and Development, Faculty of Medicine, University of Geneva

References

1. Constitution of the Republic of South Africa, 1996.

2. South Africa. Medicines and Related Substances Act No. 101 of 1965.

3. South Africa. Medicines and Related Substances Amendment Act No. 14 of 2015.

4. South Africa. National Health Act No. 61 of 2003.

5. South Africa. Genetically Modified Organisms Act No. 15 of 1997.

6. South Africa. Medicines and Related Substances Act, 1965. General Regulations. Government Gazette

No. 41064, 2017.

7. National Department of Agriculture Forestry and Fisheries, South Africa. Genetically Modified

Organisms Act of 1997. Regulations. Government Gazette 32966, 2010 as amended.

8. South Africa. National Health Act No. 61 of 2003. Regulations Relating to the Use of Human Biological

Material. Government Gazette No. 35099, 2012.

9. South Africa. National Health Act No. 61 of 2003. Regulations Relating to Blood and Blood Products. Government Gazette No. 35099, 2012.

10. South Africa. National Health Act No. 61 of 2003. Regulations Regarding the General Control of Human Bodies, Tissue, Blood Products and Gametes. Government Gazette No. 35099, 2012.

11. South Africa. National Health Act No. 61 of 2003. Regulations Relating to the Import and Export of Human Tissue, Blood, Blood Products, Cultured Cells, Stem Cells, Embryos, Foetal Tissue, Zygotes and Gametes. Government Gazette No. 35099, 2012.

12. South Africa. National Health Act No. 61 of 2003. Regulations Relating to Tissue Banks. Government Gazette No. 35099, 2012.

13. South Africa. National Health Act No. 61 of 2003. Regulations Relating to Stem Cell Banks. Government Gazette No. 35099, 2012.

14. European Union.Regulation (EC) No 726/2004 on laying down community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. 31 March 2004. Directive 2001/83/EC on the community code relating to medicinal products for human use. 6 November 2001.

15. European Union. Regulation (EC) No 1394/2007 on advanced therapy medicinal products. 30 December 2008.

16. United States of America. Federal Food, Drug, and Cosmetic Act as Amended through PL 117-328. Enacted December 29, 2022.

17. United States of America. Section 351 of the Public Health Services Act. Regulation of biological products, (1 July 1944, 1944).

18. United States of America. Section 361 of the Public Health Services Act. Regulation to control communicable diseases, (1944).

19. United States of America. 21 CFR Part 600 - Biological Products. General. 59 FR 54042 (1994).

20. United States of America. 21 CFR Part 1271 - Human cells, tissues, and cellular and tissue-based

products as amended. 21 CFR Part 1271 (2023).

21. Australia. Therapeutic Goods Act No. 21 of 1990. Compilation No. 85, 1 July 2024. Therapeutic Goods

Amendment (2009 Measures No. 3) Act No. 54, 2010, Pub L No. C2010A00054. 31 May 2010.

22. European Union. Regulation (EU) No 2024/1938 on standards of quality and safety for substances of human origin intended for human application and repealing Directives 2002/98/EC and 2004/23/

EC. 13 June 2024.

23. Japan. The Regenerative Medicine Promotion Act No.13 of 2013.

24. Japan. Act on Ensuring the Safety of Regenerative Medicine. Act No. 85 of 2013.

25. Japan. Act on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and

Medical Device. Act No. 145 of August 10, 1960.

26. USA. Food and Drug Administration Modernisation Act of 1997. Public Law 105-115. I l l STAT.

2296. 21 November 1997.

27. USA. The 21st Century Cures Act. 13 December 2016.

28. USA. Food and Drug Omnibus Reform Act of 2022. Public Law 117–328. 29 December 2022.

29. South Africa. Parliamentary Committee on Health. Submission by the SA Medical Association:

Concerns about Section 46 and Chapter 8 regulations. 2013. https://pmg.org.za/committee-

meeting/15989/ (accessed 31 August 2024).

30. Royal College of Nursing of the United Kingdom v Department of Health and Social Security [1981]

UKHL J0205-3AC 800 p 822 B-E: (1981).

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Published

2025-02-18

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Section

In Practice

How to Cite

1.
Viljoen IM, Pepper MS. Addressing the limitations of the regulatory landscape in South Africa regarding advanced cell and gene therapies and related sectors involving human cells, tissues and organs. S Afr Med J [Internet]. 2025 Feb. 18 [cited 2025 Feb. 19];115(1):e2629. Available from: https://samajournals.co.za/index.php/samj/article/view/2629