OBJECTIVES: Alzheimer's disease (AD) is a devastating neurodegenerative condition with no curative treatments currently available. Whilst existing therapies may provide symptomatic relief, emerging Disease Modifying Treatments (DMTs) aim to slow progression of the underlying disease pathology in early Alzheimer's/mild cognitive impairment (MCI). These DMTs provide the potential to significantly improve patient (and societal) outcomes, but present challenges for value demonstration and sustainable budget impact. The aim of this study is to identify key market access challenges for Alzheimer's DMTs and explore recommendations to support reimbursed patient access across global healthcare systems.

METHODS: Research has been undertaken with 10 HTA representatives from UK, Germany, Brazil, Saudi Arabia and Greece to explore key challenges/opportunities for the evaluation of Alzheimer’s DMTs, and recommendations for value demonstration to optimize patient access.

RESULTS: HTA representatives identified priority challenges for value demonstration/patient access with Alzheimer’s DMTs, including uncertain prevalence, unclear impact on disease course, and uncertainty on the relevant surrogate outcomes for disease progression/final outcomes. Additional challenges include the assessment of subgroup effects, long-term efficacy (including QoL impact) and extrapolating long-term clinical/economic benefits within pharmacoeconomic analysis. Similarities and differences can be identified between disparate countries, driven by healthcare system archetype and economic/population factors.

CONCLUSIONS: HTA representatives acknowledge the clinical importance of patient access for Alzheimer’s DMTs, and the potential value of improved outcomes in terms of relieving pressure on health/social care systems; work is needed to bridge the gap between evidence available, and predicted clinical/economic impact. Priority requirements to support value demonstration/minimize uncertainty are assurance on the size of the target population (according to accepted diagnostic standards and healthcare capacity), validation of the correlation between surrogate endpoints and final outcomes, defining the minimum important difference for slowing disease progression, and academic consensus on the optimal approach for pharmacoeconomic analysis of Alzheimer’s DMTs.