Using Cerebrospinal Fluid Biomarker Testing to Target Treatment to Patients with Mild Cognitive Impairment: A Cost-Effectiveness Analysis

Abstract

ObjectiveCerebrospinal fluid (CSF) biomarkers are shown to facilitate a risk identification of patients with mild cognitive impairment (MCI) into different risk levels of progression to Alzheimer’s disease (AD). Knowing a patient’s risk level provides an opportunity for earlier interventions, which could result in potential greater benefits. We assessed the cost effectiveness of the use of CSF biomarkers in MCI patients where the treatment decision was based on patients’ risk level.MethodsWe developed a state-transition model to project lifetime quality-adjusted life-years (QALYs) and costs for a cohort of 65-year-old MCI patients from a US societal perspective. We compared four test-and-treat strategies where the decision to treat was based on a patient’s risk level (low, intermediate, high) of progressing to AD with two strategies without testing, one where no patients were treated during the MCI phase and in the other all patients were treated. We performed deterministic and probabilistic sensitivity analyses to evaluate parameter uncertainty.ResultsTesting and treating low-risk MCI patients was the most cost-effective strategy with an incremental cost-effectiveness ratio (ICER) of US$37,700 per QALY. Our results were most sensitive to the level of treatment effectiveness for patients with mild AD and for MCI patients. Moreover, the ICERs for this strategy at the 2.5th and 97.5th percentiles were US$18,900 and US$50,100 per QALY, respectively.ConclusionBased on the best available evidence regarding the treatment effectiveness for MCI, this study suggests the potential value of performing CSF biomarker testing for early targeted treatments among MCI patients with a narrow range for the ICER.Electronic supplementary materialThe online version of this article (doi:10.1007/s41669-017-0054-z) contains supplementary material, which is available to authorized users.

How relevant are social costs in economic evaluations? The case of Alzheimer’s disease

“…From the 27 studies selected [ 40 – 66 ], three of them [ 47 , 51 , 57 ] included both cost–utility analysis (CUA) and Cost–Effectiveness Analysis (CEA), using other outcome measures in addition to QALYs. The remaining 24 studies included only CUA.…”

“…Ten studies were made using data from the United Kingdom [ 41 , 43 , 47 , 48 , 51 , 55 , 57 , 59 , 60 ], six from the United States [ 40 , 46 , 56 , 64 – 66 ], two from Spain [ 45 , 51 ], and another two from France [ 42 , 49 ]. Other studies used data from Denmark [ 50 ], Switzerland [ 52 ], Germany [ 53 ], Norway [ 54 ], Canada [ 58 ], The Netherlands [ 62 ], and Taiwan [ 63 ].…”

“…Fifteen studies evaluated a pharmacological intervention [ 44 , 46 , 49 , 51 – 55 , 58 – 61 , 63 – 65 ], whereas another seven studies evaluated a diagnostic or screening procedure [ 40 , 42 , 43 , 45 , 56 , 62 , 66 ]; four studies assessed a non-pharmacological therapy [ 41 , 48 , 50 , 57 ], and one evaluated a medical procedure [ 47 ]. With respect to the perspective used in the studies, most of the studies used both the health care payer and the societal perspective as the main points of view of the analyses [ 45 , 48 , 49 , 51 – 56 , 58 , 60 , 61 , 63 , 64 ].…”

“…Four studies used the health care provider as the main perspective, but included the societal perspective in the sensitivity analysis [ 41 , 44 , 47 , 59 ]. In another three studies, the main perspective was the societal one, with the health care payer perspective included in the sensitivity analysis [ 40 , 50 , 66 ].…”

“…The time horizon of the 22 studies widely differed, from 1 year or less [ 41 , 44 , 47 , 48 , 51 , 57 , 62 ] to lifetime [ 40 , 43 , 56 , 65 ]. Moreover, the discount rate for costs and outcomes varied from 3% [ 6 – 66 ] to a maximum of 5% [ 58 ].…”