What do transformational shifts mean in terms of ultimate outcomes and ‘value’ of medicines?

Emerging ‘one-time’ curative therapies create important new options for patients living with difficult and incurable health conditions. While cost remains central to the discussion, it’s important to reset how we view these transformational approaches and consider what these shifts mean in terms of ultimate outcomes and ‘value’.

The Federal Government’s recent approval and funding of Australia’s first cell immunotherapy (Kymriah) should be viewed as a historical step forward in the provision of transformational treatments to Australian patients:

  • Importantly, it marked the arrival of and access to one of the most advanced forms of personalized medicines now being made available.
  • We are now in completely new territory when it comes to the formal assessment and review of these therapies. There is no question that these highly complex technologies are set to change how we review and fund medicines and health technologies moving forward.
  • That future starts now with a review of the National Medicines Policy.

An estimated 289 novel cell and gene therapies are in development worldwide – nearly half for cancer.[1] The US leads the way with access – approving the first therapy in late 2017. Five therapies have now been approved in a period of less than 2 years. Four are cancer therapies.

Early trial outcomes with these therapies highlight the potential return on these innovative approaches – significant improvement in survival rates and a reduction in the need for long-term chronic treatments and care.2 Patients could be spared pain and suffering from enduring multiple non-curative therapies; and the healthcare system could potentially benefit from reductions in the cumulative cost of hospital care and ongoing treatments.

As we head towards the Federal Election, addressing cancer has been a top priority for the major political parties – through access to clinical trials, medical scans, specialist consultations and medicines approved by the PBAC. We are working to harness this interest and direct it to the review of outdated policies and the creation of systems and processes that can accommodate the future of treatment for Australian patients. For Australians it will take us closer to world-class, sustainable access to new cancer therapies as they arrive.

Our view is that time is of the essence. It is critical we work collaboratively and quickly together to set up a clear framework for the future that can assess and review the next generation of therapies in an effective and efficient way.  Our National Medicines Policy is a good place to start.

A review of the National Medicines Policy (NMP) will need to involve a multi stakeholder approach including consumers, clinicians, industry and other supply chain participants. Healthcare consumers are fundamental to the NMP and community expectations should be a part of the review and outcomes.

Activating change is not a quick fix – a long-term view is essential. Transformative treatment involves navigating a complex array of public and private providers across multiple parts of the healthcare sector.

And the future is not just in the treatment of cancer – the innovative medicines industry is also investigating cell therapy in organ transplantation to eliminate the need for lifelong immunosuppressants. Our sector will also bring this therapy into earlier and earlier stages of disease and in the process reduce the cost of treatment and the burden of disease on patients and their families and the wider healthcare system. Some of these genomic discoveries may also even take place in Australia.[2]

The promise of successful, one-time treatment has the potential to positively affect millions of lives[3]  and we are pleased to see momentum is now underway in transformative treatment for Australians.

[1] Medicines in development for Cell Therapy and Gene Therapy. https://www.phrma.org/medicines-in-development-for-cell-and-gene-therapy

2. Hampson, G. Gene therapy: evidence, value and affordability in the US health care system