Jessica Sue, BSc, MHSM
ISCT ANZ LRA Committee Member
Quality Manager
Sydney Cord Blood Bank
Sydney Children’s Hospitals Network
The ISCT Australia New Zealand (ANZ) Legal & Regulatory Affairs Committee (LRA), in collaboration with the Global Regulatory Task Force, is proud to present an on-demand, open-access webinar: ‘Progress in Advanced Cellular and Gene Therapies in South Africa’.
Chaired by ISCT ANZ LRA member Gabrielle O’Sullivan, the webinar featured an esteemed panel of individuals prominent in the CGT field and who are working to progress equitable access to advanced therapy medicinal products (ATMP) in the region:
- Dr. Theo Gerdener, Hematologist at Oncolab, Medical Director DKMS Africa, South Africa
- Dr. Candice Hendricks, Pediatric Hematologist and Clinician Scientist, Processing Director Alberts Cellular Therapy, University of Pretoria, South Africa
- Prof. Michael Pepper, Professor and Director of Institute for Cellular and Molecular Medicine, Facility of Health Scientists, University of Pretoria, South Africa
- Ignatius Viljoen, Business Development and GxP Consultant, ArexCell International, South Africa and Malawi
The panelists were interviewed by three early stage professionals:
- Candice Herg-Sagar, Cellular Therapy Scientist, Alberts Cellular Therapy, South Africa
- Murray Logan, Scientific Manager/Consultant, Institute for Cellular and Molecular Medicine, University of Pretoria, and Altera BioSciences, South Africa
- Mirja Krause-Onwukwe, Service Operations and Quality Manager, Hudson Institute of Medical Research, Australia (ISCT ANZ LRA member)
There are two JACIE-accredited centers in South Africa for hematopoietic stem cell transplantation (HSCT), and an additional one for collection and cell processing. There is also a strong and diverse research environment with a focus in clinical translation. Currently, HSCT remains the only approved cell therapy in South Africa, but the recent treatment of the first patients with CAR-T cell therapies in clinical trial marks a significant milestone in patient access to such therapies. However, South Africa is an ethnically diverse, middle-income country with a high disease burden and unequal health care. Other factors impacting CGT in particular include regulatory uncertainty, limited local production capacity, gaps in workforce, and high costs which affect the development and clinical translation of ATMPs, as well as equitable patient access. In an attempt to address these challenges and progress equitable access to CGT in the region, the BloodSA National Cell and Gene Therapy Working Party was formed, of which the interviewees are members, along with other stakeholders.
In the first segment of the webinar, the interviewing panel (Candice, Murray and Mirja) present a further overview of the current state of CGT in South Africa and highlight critical issues for consideration. A summary of five proposed key areas, along with strategic approaches that could effectively address the known challenges, is given:
- Regulatory pathways – alignment with global standards
- Manufacturing and scalability – consider decentralized production models
- Market access and reimbursement – consider outcome based reimbursement models
- Ecosystem and collaboration – develop opportunities for cross sector collaboration
- Workforce and training – develop specialist training to address gaps
The regulatory challenges are expanded by the interviewing panel, who emphasize the need to update the legislative definitions for ATMPs. Additionally, the clinical trial framework from the South African Health Products Regulatory Authority (SAHPRA) is noted to have been more focused on drug manufacturing as opposed to CGT, which limits utility. Further, when considering genetic modifications in the context of ATMPs, applications are managed by the Department of Agriculture, Land Reform and Rural Development, whose primary focus is agriculture. This, combined with outdated terminology in the relevant act governing GMOs, results in barriers to appropriate and effective regulations in this space.
In the second segment of the webinar, we hear from the interviewees (Dr. Candice Hendricks, Ignatius Viljoen, Prof. Michael Pepper, and Dr. Theo Gerdener) about what they see are the most significant challenges and issues, along with potential solutions and opportunities. Dr. Hendricks outlines how appropriate regulation of cell therapies in the region is critical, and this is a key mandate of the aforementioned CGT Working Party, along with providing a platform for collaboration between clinicians and scientists. Equitable access is the critical factor, which Dr. Hendricks feels can be supported by organizations conducting clinical trials in South Africa committing resources to ensure access to public sector patients. Ignatius Viljoen reiterates the issues with the current regulatory framework that has been built for mass produced molecule based medicines. This particularly impacts regulation of autologous CGT products that are highly personalized, and contributes to restriction of patient access whilst also hampering research and product development. Prof. Pepper discusses how an absence of regulatory clarity gives ambiguity to what is permissible in CGT treatment, resulting in unproven and unauthorized practices – and how science can be ahead of the law. He suggests a two pronged approach: review of existing legislation to ensure prioritization of patient safety, and prospective rewrite of legislation to ensure it is fit for purpose. Dr. Theo Gerdener discusses the collaboration with DKMS and South Africa, that aims to reduce the time taken to procure unrelated donors as well as associated costs, which are both major barriers to patients being able to undergo HSCT in South Africa. He also outlines the need to build skills locally in nursing and clinical staff and what measures are being taken to increase capacity for training. Achievement of locally manufactured, off the shelf allogeneic products is also discussed as an aim, that could reduce costs and increase patient access in the region.
This overview provides a brief summary of just some of the key points discussed – be sure to watch the webinar now at this link for detailed insights into this significant topic: https://www.isctglobal.org/webinar/progress-in-advanced-cellular-and-gene-therapies-in-south-africa.
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