Modelling the value of innovative treatments for Alzheimer’s disease in the United States

Makin, Charles; Neumann, Peter J.; Peschin, Sue; Goldman, Dana P.

doi:10.1080/13696998.2021.1927747

Cited by 9 publications

(7 citation statements)

References 37 publications

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“…While a small percentage of patients with dementia (8%) do not receive help from informal care providers, 30% of patients with dementia are dependent on three or more unpaid caregivers [ 3 ]. Interventions that target the underlying pathology of AD have the potential to lower this burden and provide immediate as well as long-term value to patients and their families [ 22 ].…”

Section: Discussionmentioning

confidence: 99%

“…Beyond the emotional toll, AD imposes a significant financial burden for people living with the disease and their caregivers [ 4 , 15 – 18 ]. Even though direct medical costs paid by the healthcare system may represent only 16% of total AD costs [ 19 ], the substantial socioeconomic costs resulting from AD are not traditionally included in health technology assessments [ 17 , 20 – 22 ]. Patients with AD eventually require multidisciplinary care from family and friends, caregiving professionals, and the medical community [ 17 ].…”

Section: Introductionmentioning

confidence: 99%

“…A breakthrough treatment that delays the onset or slows the progression of AD is likely to be more successful if diagnosis occurs at the early AD stage to reduce the probability of patients transitioning to a more severe AD health state. Such a therapy would reduce indirect and social care costs and delay direct medical costs [ 21 ], and may necessitate a revised framework for value assessments in AD [ 22 ].…”

Section: Introductionmentioning

confidence: 99%

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Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

et al. 2021

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Introduction: Alzheimer's disease (AD) is a chronic and progressive neurodegenerative disease that places a substantial burden on patients and caregivers. Aducanumab is the first AD therapy approved by the US Food and Drug Administration to reduce a defining pathophysiological feature of the disease, brain amyloid plaques. In the phase 3 clinical trial EMERGE (NCT02484547), aducanumab reduced clinical decline in patients with mild cognitive impairment (MCI) due to AD and mild AD dementia and confirmed amyloid pathology. Methods: We used a Markov modeling approach to predict the long-term clinical benefits of aducanumab for patients with early AD based on EMERGE efficacy data. In the model, patients could transition between AD severity levels (MCI due to AD; mild, moderate, and severe AD dementia) and care settings (community vs. institution) or transition to death. The intervention was aducanumab added to standard of care (SOC), and the comparator was SOC alone. Data sources for base-case and scenario analyses included EMERGE, published National Alzheimer's Coordinating Center analyses, and other published literature. Results: Per patient over a lifetime horizon, aducanumab treatment corresponded to 0.65 incremental patient quality-adjusted life-years (QALYs) and 0.09 fewer caregiver QALYs lost compared with patients treated with SOC. Aducanumab treatment translated to a lower lifetime probability of transitioning to AD dementia, a lower lifetime probability of transitioning to institutionalization (25.2% vs. 29.4%), delays in the median time to transition to AD dementia (7.50 vs. 4.92 years from MCI to moderate AD dementia or worse), and an incremental median time in the community of 1.32 years compared with SOC.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

et al. 2021

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“…The prevalence of AD continues to grow globally, and this is further compounded by its physical, emotional, and economic consequences on patients and their communities [103]. These consequences further substantiate the current imperative for therapeutic innovation [21,[104][105][106][107][108][109]. The aim of this review was to examine a particular genre of AD therapeutics in the form of amylin small peptide modulation (see Table 1).…”

Section: Discussionmentioning

confidence: 99%

Amylin and Secretases in the Pathology and Treatment of Alzheimer’s Disease

et al. 2022

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Alzheimer’s disease remains a prevailing neurodegenerative condition which has an array physical, emotional, and financial consequences to patients and society. In the past decade, there has been a greater degree of investigation on therapeutic small peptides. This group of biomolecules have a profile of fundamentally sound characteristics which make them an intriguing area for drug development. Among these biomolecules, there are four modulatory mechanisms of interest in this review: alpha-, beta-, gamma-secretases, and amylin. These protease-based biomolecules all have a contributory role in the amyloid cascade hypothesis. Moreover, the involvement of various biochemical pathways intertwines these peptides such that they have shared regulators (i.e., retinoids). Further clinical and translational investigation must occur to gain a greater understanding of its potential application in patient care. The aim of this narrative review is to evaluate the contemporary literature on these protease biomolecule modulators and determine its utility in the treatment of Alzheimer’s disease.

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“…Filling the knowledge gaps in terms of costs, utilities and mortality associated with AD, accounting for age, is crucial for accurate estimations of potential savings associated with reduced disease progression. However, to capture the full socioeconomic burden of AD, it is necessary to consider and apply a societal perspective that, in addition to direct medical costs, also incorporates indirect costs associated with caregiver burden and lost productivity [26,27]. It may also be relevant to include intangible costs associated with reduced quality of life for carers and family [28].…”

Section: How To Value the New Therapiesmentioning

confidence: 99%

People get ready! A new generation of Alzheimer's therapies may require new ways to deliver and pay for healthcare

Wahlberg,

Winblad,

Cole

et al. 2023

J Intern Med

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The development of disease‐modifying therapies (DMTs) for Alzheimer's disease (AD) has progressed over the last decade, and the first‐ever therapies with potential to slow the progression of disease are approved in the United States. AD DMTs could provide life‐changing opportunities for people living with this disease, as well as for their caregivers. They could also ease some of the immense societal and economic burden of dementia. However, AD DMTs also come with major challenges due to the large unmet medical need, high prevalence of AD, new costs related to diagnosis, treatment and monitoring, and uncertainty in the therapies’ actual clinical value. This perspective article discusses, from the broad perspective of various health systems and stakeholders, how we can overcome these challenges and improve society's readiness for AD DMTs. We propose that innovative payment models such as performance‐based payments, in combination with learning healthcare systems, could be the way forward to enable timely patient access to treatments, improve accuracy of cost‐effectiveness evaluations and overcome budgetary barriers. Other important considerations include the need for identification of key drivers of patient value, the relevance of different economic perspectives (i.e. healthcare vs. societal) and ethical questions in terms of treatment eligibility criteria.

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Modelling the value of innovative treatments for Alzheimer’s disease in the United States

Abstract: Medical writing support, under direction of the authors, was provided by MediTech Media, and was funded by Biogen.

Cited by 9 publications

References 37 publications

Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

Amylin and Secretases in the Pathology and Treatment of Alzheimer’s Disease

People get ready! A new generation of Alzheimer's therapies may require new ways to deliver and pay for healthcare

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