We speak to Claire Spencer (Senior Health Economist) to get the story behind the poster: ‘The Challenge of Demonstrating Cost Effectiveness in Alzheimer’s Disease in England: Case Study of a Hypothetical Emerging Treatment’. Claire worked with Catrin Treharne (Director – Health Economics), to research, develop, and co-author the poster for presentation at ISPOR.
In all, we presented 7 independent research posters at ISPOR Europe, from 7th–9th November 2022. In this series of interviews we sit down with poster authors to learn a bit more about their research.
What topic does your poster explore?
Our poster explores the challenges associated with demonstrating the cost-effectiveness of a treatment in Alzheimer’s disease (AD). The analysis aimed to identify potential cost-effective treatment profiles for a hypothetical emerging treatment in England.
In practice, this means understanding the maximum price a manufacturer could charge for a treatment with a certain treatment effect (defined as a hazard ratio on time to disease progression) and be considered cost-effective at standard willingness-to-pay thresholds compared with the current standard of care. While NICE committees would consider a range of factors in their decision making, for this analysis, we assumed that the decision to recommend or not recommend a product rested solely on the estimated incremental cost-effectiveness ratio (ICER).
What inspired you to pursue this topic?
Alzheimer’s is a progressive neurological disease that, along with dementia more broadly, is a leading cause of death in England. Indeed, dementia accounted for 11.6% of all registered deaths in February 2022. The prevalence of Alzheimer’s disease is also increasing in line with an ageing population.
Currently, NICE guidelines recommend the use of several symptomatic treatments in the absence of approved treatments that slow or stop the progression of the disease.
My personal inspiration for this topic came from my Master’s thesis, during which I supported Alzheimer’s Research UK in exploring the cost effectiveness of repurposing a medicine indicated primarily for depression and anxiety, for mild-stage late-onset AD. In order to explore the topic more broadly, we decided to use a hypothetical emerging treatment as the basis for our case study and model.
Despite dementia long being considered a graveyard for drug development, there are early signs that the tide might be turning in Alzheimer’s clinical research, with some promising clinical trial results published in recent months. As such, I felt that this would be an appropriate time to pursue this topic.
How did you and your co-authors work together to conduct the research and develop the poster?
Once we had decided the topic, Catrin and I developed the model structure, sourcing inputs from published literature.
Once we were happy with the structure and inputs and had generated results, we began to develop our abstract. During poster development we benefited from an independent review by Elizabeth Thurgar (Director – Health Economics) who gave us a fresh perspective and some useful feedback that we incorporated into the final version.
For you, what was the most interesting aspect of your research?
We were already conscious of the wider societal impact of AD before developing the model and were eager to explore this in our analysis.
We developed our base case analysis in line with the NHS and personal social services perspective on costs preferred by NICE. Therefore, we did not include costs that would likely be borne by patients or their caregivers.
Developing the poster gave us the opportunity to consider scenario analyses exploring the effect of including a broader perspective on costs and including carer health-related quality of life within the analysis. The inclusion of societal costs had a much bigger impact on results (reducing the ICER by approximately 13%) than the incorporation of carer disutility (this alone reduced the ICER by only 0.4%).
What was your most important conclusion?
Based on the results of the model that we developed, the maximum acceptable annual cost for a hypothetical emerging treatment in England was very low. This remained true even when large treatment effects for the new hypothetical therapy were tested.
To take an example: our analysis suggested that, at a willingness-to-pay threshold of £20,000, a treatment that slowed disease prgression by half (hazard ratio of 0.5) compared with standard of care would be considered cost-effective, with a maximum annual cost of £3,100.
This poses significant challenges for manufacturers of AD treatments, particularly when treatments for AD would not be expected to meet the criteria for a severity modifier considering the average age of the patient population.
What have you learnt that you can apply to future client projects?
The analysis has given me a thorough understanding of modelling approaches and supporting data sources in AD, which I hope to be able to apply to a client project in the future!
I am also now more aware of the challenges faced by manufacturers and the need to critically appraise whether current methods capture all elements of value for patients and caregivers.
View the poster here.
If you would like to discuss this research with the poster authors or to learn more about how we can support you with health economic modelling, and/or NICE reimbursement, email firstname.lastname@example.org.