Making the economic case for cell and gene therapies

The paper

Gye A, Goodall S, De Abreu Lourenco R. Cost-effectiveness Analysis of Tisagenlecleucel Versus Blinatumomab in Children and Young Adults with Acute Lymphoblastic Leukemia: Partitioned Survival Model to Assess the Impact of an Outcome-Based Payment Arrangement. Pharmacoeconomics. 2023 Feb;41(2):175-186. doi: 10.1007/s40273-022-01188-w

What was this research about?

Government decisions about subsidising medicines or medical devices are based on Health Technology Assessments (HTA), which provide decision makers with the necessary information to understand the comparative harms, benefits and costs of new medical technologies. Understanding the cost effectiveness (or value for money) of new therapies is an important part of this decision support.

This paper provided evidence on the economic impacts of chimeric antigen receptor T cell (CAR-T) therapies, a new type of cancer treatment that alters the body’s own immune system to target cancer cells.

Research shows cell and gene therapies like CAR-T hold huge promise for patients – but they are also very expensive. It has previously been difficult to make the economic case for the government to subsidise cell and gene therapies as it’s still unclear what their long-term health outcomes will be.

Some economists have tried to overcome the uncertainty around estimating that longer-term cost-effectiveness by using ‘Outcome-based payment arrangements’ (OBA) in their modelling. OBAs are essentially where the amount the government pays for a technology is linked to how well it treats patients in practice.

For the first time, this paper assessed the value of OBAs, looking at the value of achieving complete remission in patients and its impact on long-term survival in young patients with acute lymphoblastic leukemia (ALL) treated with the CAR-T therapy tisagenlecleucel.

What did the paper show?

This research found only a modest impact on reducing uncertainty about cost-effectiveness when using OBAs. That is, our measure of cost-effectiveness does not alter greatly even when we link how governments pay for an intervention, like tisagenlecleucel, to the outcomes we see in practice.

What is the significance of these findings?

This was the first paper to examine the importance of incorporating an OBA into the structure of an economic model to help inform decision-making.

It shows that, to manage the financial impact of high-cost therapies such as cell and gene therapies, it is better to use financial caps or price-volume agreements as a more efficient approach than using OBAs.

What happens next?

The Centre for Health Economics Research and Evaluation (CHERE) has a highly experienced HTA team providing advice for the Medical Services Advisory Committee (MSAC) and the Pharmaceutical Benefits Advisory Committee (PBAC). 

The findings of this paper will help to inform how governments and stakeholders in healthcare provision think about approaches to funding cell and gene therapies.

Acknowledgement of Country

UTS acknowledges the Gadigal people of the Eora Nation, the Boorooberongal people of the Dharug Nation, the Bidiagal people and the Gamaygal people, upon whose ancestral lands our university stands. We would also like to pay respect to the Elders both past and present, acknowledging them as the traditional custodians of knowledge for these lands.