Mwayi Kachapila reflects on research into adaptive platform trials and the viability of including health economics to help adoption of potential interventions
“How long before *it* is available for the public?”
It’s a question that researchers get asked on a routine basis following clinical trials. If a trial shows promise, it’s only natural for the public to want to see that intervention be offered widely to benefit patients.
As health and life sciences researchers, the prospect of finding a drug, test or treatment that improves public health is a major motivator of our work. A drug, treatment or medical device does need to not only be effective though – it also needs to make economic sense. It’s no good having a treatment that is so prohibitively expensive that only the mega rich can afford it and doesn’t represent good value for taxpayers.
Here in the UK, the National Institute for Clinical Excellence (NICE) play a crucial role in deciding which experimental treatments being developed provide value for money for the taxpayer, and if the treatment costs are so high that it makes other healthcare unaffordable, that’s no good for anyone.
But how can we know if something is affordable or not?
Currently, the usual practice in health economics is to do an analysis of the cost and benefits of an intervention after the results of a trial, answering a question such as:
These questions and others are part of the evidence that needs to be gathered in order for an organisation like NICE to decide, is this good value for money.
Previously, research that analyses the economic considerations during a clinical trial have been conducted separately and after the fact, but with new research methods it now is possible and makes sense to develop ways to combine clinical and economic analysis of trials.
It’s no secret that drug development is hard, expensive and many promising drugs aren’t suitable for use to treat disease. Designing trials that can adapt if treatments don’t show the benefit they should has led to MAMS adaptive and adaptive platform trials, which are multi-arm, multi-stage trials that can validly bring together trial stages that historically were run separately.
MAMS adaptive trials such as the STAMPEDE trial for prostate cancer launched in 2005 demonstrated their potential, and the global pandemic saw – out of necessity – a significant increase in their use.
MAMS adaptive trials are here to stay, but until now there hasn’t been good evidence about whether building in economic analysis into adaptive trials is viable and helpful.
Now, in a research article published in Value in Health, a team from the University looked at examples of where health economic analysis has been built into adaptive and MAMS adaptive and adaptive trial designs.
We found just 17 published research articles across electronic databases. Four were results of economic evaluations, and 13 were economic evaluation protocols; and no trial reported using pre-trial economic evaluations to inform the trial designs.
However.
Doing some analysis of our own, we found that in 14 articles it was possible to estimate the costs and benefits of the interventions at the interim analysis stages. Despite this, there were only five interim cost-effectiveness analyses, and three of these informed decisions to drop or maintain trial arms.
Our paper makes a strong case for the incorporation of health economic analysis into MAMS-adaptive and adaptive platform trials.
Doing so could help researchers and funders direct our collective endeavour to ensure that more of the time, effort and resources we commit to curing disease and solving health problems is focused on solutions that work in the real world and can be of benefit to any patient who needs it.
Mwayi Kachapila
Research Fellow in Health Economics
Research Fellow in Health Economics
Staff profile page for Mr Mwayi Kachapila, Research Associate in Health Economics at the Institute of Applied Health Research, University of Birmingham.
