Navigating HTA Requirements: The Current Landscape of Alzheimer's Disease-Modifying Treatments
Author(s)
Nathalie Abi-Saleh, MS, PharmD1, Denise So, MS2, Victoria Molenkamp, MSc3.
1Thermo Fisher Scientific, Waltham, MA, USA, 2Thermo Fisher Scientific, Fareham, United Kingdom, 3Thermo Fisher Scientific, London, United Kingdom.
1Thermo Fisher Scientific, Waltham, MA, USA, 2Thermo Fisher Scientific, Fareham, United Kingdom, 3Thermo Fisher Scientific, London, United Kingdom.
OBJECTIVES: The prevalence of dementia worldwide is projected to reach 78 million by 2030. Alzheimer’s disease (AD), the most common form of dementia, accounts for up to 70% of all cases. As of June 2025, two disease-modifying treatments (DMTs) for AD have received authorisation in Europe: lecanemab and donanemab. Our research aims to provide an overview of DMT health technology assessment (HTA) appraisals and highlight the challenges faced by manufacturers when the clinical value and cost-effectiveness of DMTs for AD are under evaluation.
METHODS: We conducted a targeted search for published materials on the assessment of DMTs in AD, including HTA appraisals, press releases and reports in the European Union (EU)4 and the UK.
RESULTS: We identified two National Institute for Health and Care Excellence (NICE) appraisals, two Scottish Medicines Consortium (SMC) appraisals, and one NICE Innovation Laboratory report that highlighted potential challenges faced by manufacturers when DMTs are assessed for dementia. Key uncertainties identified were prevalence estimates for the eligible treatment population, long-term treatment benefit, whether trial outcomes are clinically meaningful and the additional cost of service implications. The report concluded that while multiple challenges may arise during the evaluation of DMTs, NICE’s approach is appropriate for their assessment. Both lecanemab and donanemab have been appraised by the SMC and NICE. Neither drug was recommended for use in Scotland or NICE, citing issues that matched the uncertainties identified in the 2023 NICE report. There are no published DMT appraisals for AD in France, Germany, Italy or Spain.
CONCLUSIONS: Our findings highlight a potential disconnect between HTA evidence requirements for DMTs in AD and the available evidence provided in appraisals. This is particularly relevant given that three additional appraisals for DMTs in AD are awaiting development at NICE at the time of writing, and more appraisals are expected in the future.
METHODS: We conducted a targeted search for published materials on the assessment of DMTs in AD, including HTA appraisals, press releases and reports in the European Union (EU)4 and the UK.
RESULTS: We identified two National Institute for Health and Care Excellence (NICE) appraisals, two Scottish Medicines Consortium (SMC) appraisals, and one NICE Innovation Laboratory report that highlighted potential challenges faced by manufacturers when DMTs are assessed for dementia. Key uncertainties identified were prevalence estimates for the eligible treatment population, long-term treatment benefit, whether trial outcomes are clinically meaningful and the additional cost of service implications. The report concluded that while multiple challenges may arise during the evaluation of DMTs, NICE’s approach is appropriate for their assessment. Both lecanemab and donanemab have been appraised by the SMC and NICE. Neither drug was recommended for use in Scotland or NICE, citing issues that matched the uncertainties identified in the 2023 NICE report. There are no published DMT appraisals for AD in France, Germany, Italy or Spain.
CONCLUSIONS: Our findings highlight a potential disconnect between HTA evidence requirements for DMTs in AD and the available evidence provided in appraisals. This is particularly relevant given that three additional appraisals for DMTs in AD are awaiting development at NICE at the time of writing, and more appraisals are expected in the future.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA244
Topic
Clinical Outcomes, Economic Evaluation, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Neurological Disorders, No Additional Disease & Conditions/Specialized Treatment Areas