Surrogate Endpoints Guidance: New International Report Enhances HTA Practices

By João L. Carapinha

January 24, 2025

The National Institute for Health and Care Excellence (NICE) published a new report that provides surrogate endpoints guidance. This guidance offers best practices for utilizing surrogate endpoints in health economic models to inform health technology assessment (HTA) decisions. The aim is to clarify how surrogate endpoints can predict long-term health outcomes from short-term effects, which is particularly crucial when long-term data is not available. The report results from a collaborative effort among several global health technology assessment agencies.

What you need to know

The new report addresses the previously fragmented guidance on using surrogate endpoints in HTA, providing clarity and support for technology developers and HTA agencies. It outlines methods for selecting, validating, and incorporating surrogate endpoints into health economic models, enhancing predictions of long-term health benefits. This surrogate endpoints guidance results from a collaborative effort among multiple international HTA agencies, including NICE, Canada’s Drug Agency, ICER in the US, and others. The report assists HTA committees in understanding the relationship between short-term surrogate endpoints and long-term health outcomes, which is crucial for evaluating the value of new treatments.

Surrogate endpoints are biomarkers or intermediate outcomes that predict a treatment’s effect on a final clinical outcome. This concept is critical in health technology assessment as it allows the evaluation of treatments based on shorter-term data when long-term outcomes are not yet available. The report aligns with regulatory standards and HTA guidance, drawing from published methods in HTA manuals, regulatory guidelines, and statistical validation. This ensures that the guidance is grounded in established practices and methodologies.

Implications

The new guidance significantly impacts health economics and outcomes research by providing a standardized approach to using surrogate endpoints. This can enhance the accuracy and reliability of cost-effectiveness analyses, which are crucial for decision-making in healthcare resource allocation. By clarifying the relationship between surrogate endpoints and long-term health outcomes, this guidance can lead to more informed decisions about the reimbursement and adoption of new treatments. The long-term effects of many drugs are vital for determining their overall value to patients and health systems. The report may lead to modular updates in HTA methods, ensuring that the guidance remains current and integrated into ongoing HTA processes, further enhancing the evaluation of health technologies.

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