A new decision framework to support recommendations by the National Institute for Health and Clinical Excellence (NICE) on the use of new drugs and devices in the NHS has been published by the Centre for Health Economics at the University of York.
NICE is increasingly making "fast track” recommendations to the NHS on the use of new drugs and devices soon after these products are first licensed, but such decisions are frequently being made when the evidence base to support these new technologies is limited, say the researchers. For example, there may be substantial uncertainty surrounding overall effectiveness, the patients most likely to benefit, the potential for harms and whether the new technologies actually represent best use of resources for the NHS.
However, one underused option available to NICE is to make an "only in research" recommendation, in which a drug or device can only be used in the context of an appropriately-designed programme of evidence development, they point out. Having further evidence can be valuable, but guidance on how best to balance the benefits of access to a new drug or technology with the value of further evidence about the product's performance has been lacking, they add.
The researchers, from York and Brunel Universities, say they have developed a "transparent, consistent and methodologically robust" approach to formalise a process which NICE can use to produce its "only in research" recommendation.
"Uncertainties about treatment effects can never be entirely eliminated but they can be reduced by further evidence. This in turn facilitates better decisions on patient outcomes and on best use of finite resources in the NHS, which are crucial at a time of financial constraint," said Professor Karl Claxton of the Centre for Health Economics at York, who led the project.
"Our research has established the key principles of what assessments are needed, as well as outlining how these assessments should be made. Our framework will enable NICE to make 'only in research' recommendations in an explicit and transparent manner," he added.
The team say that their work has implications for: - policy, eg, NICE guidance and drug pricing; - the process of appraisal, for example the greater involvement of research commissioners; and - methods of approval, eg, should additional information, evidence and analysis be required.
However, they add: "establishing the key principles of what assessments are required and how they might be informed has much wider relevance beyond NICE and the UK NHS."
The project is funded by the National Institute for Health Research Health Technology Assessment (NIHR HTA) programme.