Report - Cost-effectiveness: recommendations for surrogate endpoints

The National Health Care Institute and similar government organisations in England, Canada, the United States, Colombia and Australia have elaborated together how professionals can best deal with surrogate endpoints. This is a type of endpoint that is increasingly used to demonstrate the effectiveness of a treatment. 

Challenge to determine the relationship between effectiveness and costs

To determine whether a treatment really works, researchers look at different results. These are the so-called endpoints. In clinical trials, an easily observable effect is often used to measure the effectiveness of a treatment. For example, the blood pressure going down. We call this a surrogate endpoint. This does say something about the effect, but does not always provide insight into the effect of a treatment on the longer term, e.g. longer life. Surrogate endpoints are also not always well scientifically substantiated based. This makes it difficult to determine the effectiveness and also cost-effectiveness of a treatment. Cost-effectiveness is the ratio between what a treatment achieves for the patient and the cost. If the cost-effectiveness is not clear, it may take longer for a treatment to be included in the basic benefit package.

Explanations and tips for professionals on dealing with surrogate endpoints

In this report, the 6 organisations provide recommendations to better investigate and substantiate surrogate endpoints. The recommendations are  intended for professionals, such as technology developers, consultants, policymakers and academics developing  health economics models. The 6 organisations also describe how they themselves deal with surrogate endpoints when determining cost-effectiveness.