NICE: International collaboration provides new guidance on the use of surrogate endpoints in cost-effectiveness analysis
NICE, in collaboration with other international health technology assessment agencies, has published a new report which offers guidance on best practice for using surrogate endpoints in health economic models to guide health technology assessment (HTA) decisions.
When looking at the evidence of effectiveness of new treatments, our independent committees look at the long-term effects on health that a drug might have. 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 this reduction in blood pressure will lead to it preventing heart attacks or strokes. The short-term effect – lowering blood pressure – is a ‘surrogate endpoint’ and it is used to predict the longer-term effect – preventing strokes and heart attacks.
The new report outlines how and when these surrogate endpoints can best be used to help judge the value of technologies to patients.
When companies submit their evidence to us, they may not have the longer-term data and may rely instead on showing an effect against the short-term outcome. The NICE committees need to know and understand the relationship between surrogate endpoints, and long-term health outcomes, to evaluate 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 the NHS is determined by these long-term effects.
Until now, guidance on using surrogate endpoints in health technology assessments has been fragmented. The new guidance will provide clarity and support for technology developers when using surrogate endpoints for analysing the cost-effectiveness of the drugs or devices they are developing.
The report outlines how to select and check the surrogate endpoints, the selection of the longer-term benefits and how to display them in evidence submissions.
The recommendations are based on published methods in HTA manuals, regulatory guidelines, statistical validation and experience of HTA agencies in decision-making when using surrogates.
The project activity was led and coordinated by NICE. Other 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.
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