The translational gap for gene therapies in low- and middle-income countries
(Doxzen et al. 2024)
“Gene therapies are designed to address the root cause of disease. As scientific understanding of disease prevention, diagnosis, and treatment improves in tandem with technological innovation, gene therapies have the potential to become safe and effective treatment options for a wide range of genetic and nongenetic diseases. However, as the medical scope of gene therapies expands, consideration must be given to those who will benefit and what proactive steps must be taken to widen development and access potential, particularly in regions carrying a high disease burden.”
The analysis delves into the crucial roles played by academia, government, and the commercial sector in the implementation of gene therapy in low- and middle-income countries (LMICs). It starts by highlighting the academic sphere's role as an incubator for specialized workers, including technicians, computational biologists, and social scientists. The number of these workers has been steadily increasing, indicating a growing capacity within LMICs. Academic institutions also collaborate with research hospitals, enabling the training of clinical specialists and the formation of multidisciplinary teams essential for clinical trials.
Moving to the government sphere, it is portrayed as both an enabler and a gatekeeper in the gene therapy landscape. Governments can prioritize specific diseases, incorporate gene therapy into national plans, and provide regulatory frameworks. However, regulating gene therapy products poses challenges due to their unique nature, requiring governments to adapt existing regulations or design new ones. Market access and cost coverage are also under government oversight, impacting the accessibility of gene therapies.
The commercial sphere, characterized by high costs and regulatory complexities, plays a vital role in scaling production and navigating regulatory regimes. Companies invest heavily in gene therapy development, manufacturing, and market access. They experiment with various corporate models to maintain profitability and improve access to gene therapies. Collaboration between academia and industry is emphasized, with universities focusing on basic research and companies handling later-phase clinical trials and product scaling.
The interactions among these spheres are dynamic and critical for the development, approval, and accessibility of gene therapies. Governments fund academic research, influence regulatory processes, and interact with the private sector through incentivization. Academia and industry collaborate in research and development, clinical validation, and market access. Governments also play a crucial role in training and maintaining the healthcare workforce necessary for gene therapy implementation.
The analysis also addresses shared areas of capacity building in LMICs essential for gene therapy success. These include health facilities, research and development infrastructure, manufacturing capacity, workforce training, community engagement, financial mechanisms, and policy and regulatory frameworks. Access to healthcare remains a challenge, especially in rural areas, highlighting the need for coordinated care models and improved distribution of treatment centers.
Research and development infrastructure in LMICs must capture biological and social diversity to ensure the safety and efficacy of gene therapies across populations. Long-term monitoring of gene therapy recipients is crucial for detecting adverse events and evaluating treatment efficacy. Capacity building efforts should focus on developing domestic manufacturing capacity, training a skilled healthcare workforce, engaging communities, securing funding, and establishing robust policy and regulatory frameworks.
In conclusion, implementing gene therapy in LMICs requires a concerted effort across academia, government, and the commercial sector. Collaboration, capacity building, and addressing challenges in healthcare access, research infrastructure, manufacturing, workforce training, community engagement, finance, and policy are key to realizing the potential of gene therapies and ensuring equitable access to these life-saving treatments on a global scale.
Doxzen, Kevin W., et al. "The Translational Gap for Gene Therapies in Low- and Middle-income Countries." Science Translational Medicine, 2024, https://doi.org/adn1902.