Adaptive Design Clinical Trials and their Impact on the Health Economic Analysis of Healthcare Technologies

Overarching Research Aim

Can adaptive designs and health economics be used together to increase the efficiency of health technology assessments without compromising accuracy?

Project Summary

In the UK there are limited resources for both for new healthcare technologies and research. Bodies such as the National Institute for Health and Care Excellence (NICE) decide whether a technology should be funded by the National Health Service (NHS). To enable a decision to be made NICE need to know that the technology improves health - it is clinically effective - and that it is value for money – it is cost-effective.

Funding bodies such as the National Institute for Health Research (NIHR) also only want to fund research that is value for money. They only wish to pay for health technologies and research that give the biggest improvements in health for the amount of money spent. The methods of health economics, such as cost-effectiveness analysis and value of information analysis facilitate these decisions.

An adaptive design is a type of clinical trial that allows early examinations of the data at pre-specified time points during the trial. This information can be used to make modifications to the trial, such as stopping early. The use of adaptive designs is increasing in health research; however the changes made are often motivated by showing how clinically effective a treatment is. Evidence of cost-effectiveness is not considered when conducting early analyses of the data.

The aims of this research are to:

1. Identify the current methods used for the health economic analysis of adaptive design clinical trials.
2. Assess whether adjustments are required for a health economic analysis using a trial with an adaptive design.
3. Explore how health economics can be incorporated into the design and analysis of an adaptive design.
4. Identify the practical and ethical considerations when considering the health economic analysis of adaptive designs.

Public Involvement

This research is supported by the Health Economic Evaluation and Adaptive Designs (HEEAD) advisory panel. The panel is comprised of seven members of the public who provide regular input, guidance and a lay perspective to the on-going research.

Funding Acknowledgement

This is independent research arising from a Doctoral Research Fellowship (DRF-2013-08-013) supported by the National Institute for Health Research. The views expressed are those of the author and not necessarily those of the NHS, the National Institute for Health Research, the Department of Health or the University of Sheffield.