New Report on Emerging Approaches to Economic Evaluations of Tumour-Agnostic Drugs

Canada’s Drug Agency has published a new report on an economic modelling approach used in evaluations of tumour-agnostic drugs that treat cancer.

The introduction of tumour-agnostic drugs continues to impact cancer treatment and brings additional complexity to the health technology assessment (HTA) process for these products. With a growing number of these products coming to Canada, our new report on Bayesian hierarchical models of basket trials aims to provide clarity around methods used in economic evaluations of these therapies and highlights areas where more research is needed.

About Tumour-Agnostic Drugs and Basket Trials

Tumour-agnostic drugs may be used to treat cancer regardless of where it is located in the body, because they target a specific mutation or biomarker that is present in the tumour. Genomic testing can help identify these biomarkers and determine whether a tumour-agnostic drug may be effective for a given patient.

In many instances, tumour-agnostic drugs are studied in a basket trial. A basket trial is a type of clinical trial that assesses how well a treatment works across different cancer types that have the same type of mutation or biomarker. Patients with the same kind of cancer and the same mutation or biomarker are grouped together in “‘baskets”’ to assess the effectiveness of the treatment.

Challenges in Economic Evaluations

There is an increasing use of basket trial designs, specifically for oncology products. Incorporating the results of basket trials into economic evaluation can be challenging due to several factors, including differences among cancer types, limited sample sizes, lack of comparator drugs, use of surrogate end points, and measurement of study outcomes by disease site.

Bayesian hierarchical models form a statistical framework that accounts for the differences among cancer types while borrowing information across cancer types in basket trials. This report was developed to provide an overview of basket trials and the information they provide, describe the use of Bayesian hierarchical models to inform economic evaluations, and understand key aspects of the appraisal process.

While these methods are still associated with a high degree of uncertainty, the report highlights some specific approaches to understand and characterize that uncertainty. This information can help HTA bodies properly incorporate research findings from high-quality basket trials in economic appraisals.

The report and accompanying Viewpoint have been published in the Canadian Journal of Health Technologies.