International collaboration provides new guidance on the use of surrogate endpoints in cost-effectiveness analysis

Recommendations will help organizations that appraise new drugs establish whether their use will result in longer-term benefits for patients

Four health technology assessment agencies, including Canada’s Drug Agency, have published new guidance on the use of surrogate endpoints in cost-effectiveness analysis to inform decision-making when evaluating new medicines.

When looking at the evidence of effectiveness of new treatments, the expert committees at Canada’s Drug Agency look at the long-term effects a drug might have on health. For example, when assessing a new drug that lowers blood pressure, we will not only consider whether the drug lowers blood pressure, but also whether it prevents people who take it from having heart attacks or strokes. The short-term effect is a “surrogate endpoint” and it is used to predict the longer-term effect.

When surrogate endpoints are used in drug submissions filed with us for reimbursement review, our committees may not have all the evidence they need at the time of their deliberations. The committees need to know and understand the relationship between short-term effects and long-term outcomes to make predictions about the long-term health effects that new drugs are likely to have. This is important because, for many drugs, most of their value to patients and health systems is determined by these predicted long-term effects.

Until now guidance on using surrogate endpoints in health technology assessment has been fragmented. The new guidance will provide clarity and support for pharmaceutical companies when using surrogate outcomes for analyzing the cost-effectiveness of the drugs they are developing.

The project activity was led and coordinated by the National Institute for Health and Care Excellence (NICE) in the UK. Members of the working group included:

• Canada’s Drug Agency (CDA-AMC)
• the Institute for Clinical and Economic Review (ICER) in the US
• the Australian Department of Health and Aged Care
• the National Health Care Institute (ZIN) in the Netherlands
• the Institute for Technology Assessment in Health (IETS) in Colombia
• Rubix Health in the US.

Canada’s Drug Agency has considered this guidance on surrogate endpoints in the development of our first-ever methods guide, which focuses on the appraisal of clinical evidence for a drug submitted by a sponsor to our drug reimbursement review program. This methods guide is posted for consultation until January 28, 2025.