How ‘surrogate outcomes’ influence long-term health outcomes

As part of NICE’s assessment of the clinical and cost effectiveness of new medicines we look at the long-term effects on health that a drug might have. For example, when assessing a new drug that lowers blood pressure, NICE will not only consider whether the drug is effective, but also whether it prevents people who take it from having heart attacks or strokes in the future. 

When new drugs are approved, they don’t always have all evidence for their longer-term effects available. However, when we know and understand the relationship between short-term effects, and long-term outcomes, we can use cost-effectiveness models 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 the National Health Service (NHS) is determined by these predicted long-term effects. We refer to short-term effects as ‘surrogate outcomes’ as it’s a replacement for the longer-term effect.  

We are building our understanding of the relationship between surrogate outcomes and how they influence longer-term health outcomes. This has led to an increase in drugs that are approved based on improving a surrogate outcome in clinical trials. If we can assess new drugs based on their ability to improve the surrogate outcome, we can get new treatments to people who need them faster, as we don’t have to wait for the evidence that the drug also improves long-term outcomes before making a recommendation. 

However, NICE, and similar organisations abroad, do need to be certain that the drug’s effect on the surrogate outcome will result in longer-term benefits for patients. When such evidence is missing this can make it difficult for organisations like NICE to use cost-effectiveness models to predict what the long-term benefits of the drug are likely to be. This can make it challenging to recommend such drugs for use in the NHS. 

NICE is working with a group of organisations who make recommendations for the use of new drugs in other countries to develop more guidance on the use of surrogate outcomes when analysing cost-effectiveness. The guidance will help pharmaceutical companies understand how surrogate outcomes should be used when analysing cost-effectiveness of the drugs they are developing.  
 
The international organisations NICE is collaborating with are: 

• Scottish Medicines Consortium (SMC), 

• Canadian Agency for Drugs and Technologies in Health (CADTH), 

• Health Technology Assessment international (HTAi) Global Policy Forum,  

• Australian Department of Health and Aged Care,  

• the National Healthcare Institute (ZIN), 

• Colombian Institute for Technology Assessment in Health (IETS). 

NICE is also working with several international organisations to explore developing a joint scientific advice procedure that offers advice on the proposed use of new surrogate outcomes in analysing cost-effectiveness.  

The organisations are:  

• Institute for Clinical and Economic Review (ICER),  

• Canada’s Drugs and Health Technologies Agency (CADTH),  

• Health Technology Assessment international (HTAi) Global Policy Forum,  

• All Wales Theraputics and Toxicology Centre (AWTTC),  

• Australian Department of Health and Aged Care.  

Work on these projects began in January and will continue over the next year. You can keep up to date on progress through the NICE website and social media channels.