Recent breakthroughs in our understanding of the disease have begun to shed light on its complex nature and new treatments have begun to emerge including DMDTs (disease-modifying dementia treatments). These treatments aim to alter the course of disease progression and reduce its substantial impact.
While there are currently no licensed DMDTs in the UK, several are at varying stages of development and regulatory approval. Two of these, lecanemab, made by Eisai and Biogen and donanemab (made by Eli Lilly and Company), are scheduled to be appraised by NICE next year. Both treatments target mild cognitive impairment and mild dementia due to Alzheimer’s disease. Working with the NHS and patients, NICE have already begun work in readiness for the companies submitting their evidence.
This includes work done by NICE’s Health Technology Assessment Innovation Laboratory (HTA Lab) to identify the key issues that might arise during planned and future evaluations, based on current knowledge, publicly available evidence and in-depth discussions with researchers, patient groups and NHS colleagues.
The report has identified a number of issues that need to be considered as part of a robust appraisal process.
The key issues include the uncertainty over how many people may have mild cognitive impairment, and the current need for invasive tests to identify people who may be eligible for the new treatments.
Another key issue is that there is also no published data on the long-term treatment effectiveness of DMDTs at this stage. Extensive assumptions will be needed to extrapolate results from current research trials to infer the lifetime benefit of DMDTs in terms of quality and length of life.
Economic modelling considerations are also raised. DMDTs are expected to be given by intravenous infusions in hospital. The economic models will need to include these additional costs for this and any associated injection site reactions and complications. Additionally, monitoring for side effects known to be associated with using these drugs will need to be considered.
However, overall, this work found that despite these challenges, NICE’s methods and processes for technology appraisal continue to be appropriate for assessing these innovative new treatments.
Alzheimer’s disease, a form of dementia, is a degenerative brain disease that impacts memory, thinking abilities, and essential cognitive functions. It affects an estimated 850,000 people in the UK - 1 in every 14 people aged 65 years and over.
The development of disease-modifying treatments for dementia represents a long-awaited change in the management of a condition associated with a considerable burden of illness. The UK is at the forefront of this research, and these new treatments represent potentially significant advancements in the field.
It is important, however, that the value of these treatments in terms of their clinical and cost effectiveness compared to currently available options can be properly assessed - an essential step towards a future where Alzheimer's can be effectively treated or even prevented.