A new white paper reveals how system change is required to ensure Australians can access the 2,000 cell and gene therapies (CGTs) currently in development globally and recommendations on how best to prepare the national health landscape to seize the opportunity.
The number of CGTs both in development and on market has grown exponentially in the past decade, presenting both opportunities and challenges for the health landscape as Australian patients expect access to technologies which often cater to unmet medical needs.
The report ‘Cell and Gene Therapies: Rising to the Challenge’, commissioned by Novartis and developed by EvoHealth, assessed what needs to happen within Australia to prepare for and accommodate the growing number of CGTs.
The report notes, “CGTs are not limited to a single disease or therapeutic area. They can treat not only inherited genetic disorders but also some cancers and viral infections. Many CGTs target rare diseases that have a high unmet need. These diseases often have limited or no treatment options, such as cystic fibrosis, some ocular and a range of neuromuscular conditions.”
Differing from traditional small molecule therapeutics, CGTs are not specifically a product, device or a service and can be a combination thereof, often with complex manufacturing, delivery, supply chains and high cost of goods. CGTs often target small patient populations with severe unmet need and can significantly change the course of the disease.
It goes on to state, “The majority of rare diseases (>80 per cent) are genetic in origin. These patients carry a significantly higher disease burden, and an unmet clinical need. By treating these conditions, CGTs will provide treatment options for the first time to many vulnerable Australians. This is what society expects.”
The report argues CGTs provide an opportunity for the Australian Government to address health inequities, and provides a list of seven key recommendations for Government to action, including:
- Establishing an Office for Rare Diseases as a portfolio agency of Department of Health;
- Leveraging recommendations from the National Strategic Action Plan for Rare Disease;
- Developing incentives for CGTs to create opportunities for trials, manufacturing and commercial success in Australia;
- Develop a national strategic approach to equitable and timely clinical care for CGTs via the National Health Reform Agreement framework;
- Establish a CGT expert advisory group to provide input and advice to the key evaluation bodies;
- Enhance the role of horizon scanning via the Health Technology Reference Group, or equivalent; and
- The Minister for Health to establish a cross-functional working group including Departments of Health (both Federal and State/Territory), industry, patients and academics to consider current Health Technology Assessment processes for CGTs.
The EvoHealth report is in line with AusBiotech’s work to the harness the opportunities presented by CGTs in Australia through the Regenerative Medicines Consortium Project, and the Australia Cell and Gene Catalyst (the Catalyst), which builds on work of the Consortium. These initiatives aim to provide Australian patients access to world-class advanced therapies while simultaneously cultivating a thriving cell and gene industry and end-to-end value chain in Australia.