Advances in Regenerative Medicine (RM) and Gene Therapy:

In December 1967, when Dr Christiaan Barnard and a team of surgeons performed the first human heart-to-heart transplant, the medical landmark made front-page news around the world. Today, advances in regenerative medicine (RM) and gene therapy are so routine that they are typically only covered by specialist scientific and industry journals.

However, governments, healthcare providers (HCPs), and medical professionals cannot afford to ignore the latest developments in RM and gene therapy because they are one of the most promising pathways to ensuring better lifelong health and quality of life for all citizens. Treatments for genetic disorders, tissue regeneration techniques, and organ replacements are just some of the areas where RM and gene therapy are already helping to shift healthcare in developed countries from symptomatic to curative treatments that are less costly in the long term. Prevention is the ultimate objective, aiming to minimize healthcare costs, and precision medicine – especially with RM and gene therapy playing an increasingly important role – can be leveraged in combating rare diseases and non-communicable diseases (NCDs) such as cancers.

Two accelerating trends mean that governments must act now to maximize the potential benefits of RM and gene therapy before it is too late. First, healthcare budgets worldwide are spiraling out of control in both developed and developing countries. The reasons are complex and interconnected. They include increased life expectancy, the rise of non-communicable and rare diseases as a global health burden, and the impact of COVID-19. RM and gene therapy are relevant to all these challenges, providing an increasing number of treatments that are more cost-effective and deliver better long-term outcomes for patients than conventional medicine.

Second, the remarkable advances in RM and gene therapy are connected to the emergence of digital scientific and medical technologies and processes such as gene editing and AI-enabled surgical devices. These technologies are evolving at such speed that governments cannot afford to sit on the sidelines while other countries secure a head start in investing in RM and gene therapy, from the laboratory to the hospital bedside. The policy-making and budgetary dilemma, which we examine in this report, is how to balance the high initial R&D costs of RM and gene therapy against the considerable long-term cost benefits.