New guidance to aid cost-effectiveness analysis of new drugs
Posted: 17 January 2025 | Catherine Eckford (European Pharmaceutical Review) | No comments yet
The report aims to support development of economic models for health technology assessment (HTA) decision making, as there has been “little change in guidance on the use of surrogate endpoints in HTA since 2018”.
A new collaborative report provides best practice recommendations on using surrogate endpoints in health economic models to guide decisions about health technology assessment (HTA).
The National Institute of Health and Care Excellence (NICE) explained that HTA bodies assess evidence on the short-term effects of a drug provided by companies and drug developers. These surrogate endpoints (eg, biomarkers or intermediate outcome) are used to predict the long-term effectiveness of new treatments.
Clarity on surrogate endpoints for cost-effective analysis
Critically, the recommendations provide transparency and support for technology developers when using surrogate endpoints for analysing the cost-effectiveness of the drugs or devices they are developing. Guidance on this topic prior to publication of this report, has been “fragmented”, NICE stated.
The paper was developed following “the challenges arising from changes in pharmaceutical clinical trials and regulation,” the authors of the paper wrote.
The [surrogate endpoints report] was developed following the challenges arising from changes in pharmaceutical clinical trials and regulation”
They highlighted that products given conditional or accelerated approval by the European Medicines Agency (EMA) “between 2011 and 2018 have predominantly relied on non-validated surrogate endpoints… this means that there is often a lack of data on long-term effectiveness for new health technologies entering the market.”
Further, the authors emphasised that the issue “is particularly acute when the surrogate endpoint is novel and lacks clear links to the final outcome of interest.”
As such, the guidelines advise developers on how to choose and check the surrogate endpoints, select longer-term benefits of their product and how to display this data in evidence submissions, NICE added.
The new guidance was jointly produced by NICE alongside other international HTA agencies, including:
• 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.
Related topics
Clinical Development, Clinical Trials, Data Analysis, Drug Development, Drug Markets, Drug Safety, Industry Insight, Regulation & Legislation, Research & Development (R&D), Technology, Therapeutics
Related organisations
Australian Department of Health and Aged Care, Canada Drug Agency (CDA-AMC), Institute for Clinical and Economic Review (ICER), Institute for Technology Assessment in Health (IETS), National Health Care Institute (ZIN), National Institute for Health and Care Excellence (NICE), Rubix Health
