Challenges in demonstrating the value of disease-modifying therapies for Alzheimer’s disease

ABSTRACT

Introduction

Alzheimer’s disease (AD) is a complex neurodegenerative disease, affecting millions of people worldwide and imposing heavy economic burdens to societies. Currently, only symptomatic treatments are available for patients, but there is ongoing research on potential therapies that can modify the course of disease. The main objective of this work is to identify and explore the challenges surrounding decision modeling for economic evaluation of interventions for AD.

Areas covered

This article discusses the challenges in modeling the natural history of disease, particularly regarding the selection of disease progression and outcome measures, the inclusion of biomarker status in models, and the approach to model mortality. Challenges stemming from the use of long-term assumptions regarding treatment effects and the need for real-world evidence to fill data gaps are discussed. Lastly, the overwhelming economic impact of disease and the challenges in estimating these costs for modeling are addressed.

Expert opinion

Value assessment frameworks need to be reconsidered in order to demonstrate the full benefit of new disease-modifying therapies spanning beyond the scope of health systems. Data collection efforts that expand the evidence base, upon which economic models are based, will reduce the uncertainties surrounding the long-term outcomes of interventions in AD.

KEYWORDS:

• Alzheimer’s disease
• dementia
• modeling
• cost-effectiveness
• economic evaluation
• value assessment
• health policy

Article highlights

• The financial burden associated with AD is enormous. It has been estimated to amount to US$948 billion worldwide in 2016, with costs expected to increase by 15.94% every year resulting from an increase in the number of people living with AD and/or increases in per-person costs of care.

• The modeling of AD natural history, the effect of disease-modifying therapies and quantifying the associated costs have many challenges, both technically and in regards to insufficient or incomplete data.

• Data collection efforts to expand the evidence base, in which economic models are based upon, will reduce the uncertainties surrounding the long-term outcomes of interventions in AD and assist decision-makers estimate the long-term consequences when making regulatory and reimbursement decisions.

• Broadening the scope of cost-effectiveness evaluations to include the wider societal impact of AD can highlight the significance of new therapies in maximizing population health, but also in maximizing social welfare.

• Current approaches to value assessments need to be reconsidered if the full benefits of new therapies in AD are to be demonstrated beyond the scope of the healthcare payer perspective.

• Cross-sector collaboration across physicians, patient groups, manufacturers, and payers is required to validate the modeling of AD natural history and the effect of novel therapies, offset the challenges of budget silos in health care and help remove barriers to patient access to effective therapy.

Disclosure of interest

Peter Pemberton-Ross and Robin Thompson are employees and stock-holders of Biogen. Anders Gustavsson is a partner of Quantify Research, providing consultancy services to pharmaceutical companies and other private and public organizations and institutions. Melissa Gomez and Mahmoud Hashim are employees of Ingress-Health, providing consultancy services to pharmaceutical companies and other private institutions. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewers disclosure

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Declaration of interest

No potential conflict of interest was reported by the authors.

Additional information

Funding

Medical writing support and editing for this article were funded by Biogen and was provided by Ingress-Health.