Fiscal implications of newborn screening in the diagnosis of severe combined immunodeficiency.

In the United States, newborn screening (NBS) is currently recommended for identification of 31 debilitating and potentially fatal conditions. However, individual states determine which of the recommended conditions are screened. The addition of severe combined immunodeficiency (SCID) screening to the recommended NBS panel has been fully instituted by 18 states, with another 11 states piloting programs or planning to begin screening in 2014. Untreated, SCID is uniformly fatal by 2 years of age. Hematopoietic stem cell transplantation usually is curative, but the success rate depends on the age at which the procedure is performed. Short-term implementation costs may be a barrier to adding SCID to states' NBS panels. A retrospective economic analysis was performed to determine the cost-effectiveness of NBS for early (<3.5 months) versus late (≥3.5 months) treatment of children with SCID at 3 centers over 5 years. The mean total charges at these centers for late treatment were 4 times greater than early treatment ($1.43 million vs $365,785, respectively). Mean charges for intensive care treatments were >5 times higher ($350,252 vs $66,379), and operating room-anesthesia charges were approximately 4 times higher ($57,105 vs $15,885). The cost-effectiveness of early treatment for SCID provides a strong economic rationale for the addition of SCID screening to NBS programs of other states.

The economic burden of Medicare-eligible patients by multiple sclerosis type.

OBJECTIVE: Although the global rate of multiple sclerosis (MS) is low, a few studies have documented high costs. Costs are highly variable depending on MS stage. This study was designed to assess the economic burden of Medicare-eligible patients by MS type in the United States using a claims-based classification algorithm to examine cost variation by disease stage. METHODS: A sample of 2003 to 2006 Medicare patients was selected. Cases were classified as pre-existing progressive MS or pre-existing relapsing-remitting MS (RRMS); the latter were further subdivided into relapsing, remitting, or stable. RESULTS: The sample had 5044 MS subjects, of whom 34.4% had prevalent progressive MS and 65.6% had prevalent RRMS. There were many chronic, comorbid conditions. The mean all-cause Medicare expenditures (not including self-administered medications) per person-year for MS in 2006 were $23,630 for prevalent progressive patients and $5887 for prevalent RRMS patients. Within the RRMS type, Medicare expenditures per person per month in 2006 were $1418 for relapsing patients, $608 for remitting patients, and $331 for stable patients. CONCLUSIONS: There are substantial cost advantages to Medicare for keeping RRMS patients in a stable health state and in keeping them from advancing in disability severity. The overall cost advantage would be diminished by the large cost burden of comorbidity, which would likely remain fixed with improved MS therapies.

Estimation and validation of a multiattribute model of Alzheimer disease progression.

OBJECTIVES: To estimate and validate a multiattribute model of the clinical course of Alzheimer disease (AD) from mild AD to death in a high-quality prospective cohort study, and to estimate the impact of hypothetical modifications to AD progression rates on costs associated with Medicare and Medicaid services. DATA AND METHODS: The authors estimated sex-specific longitudinal Grade of Membership (GoM) models for AD patients (103 men, 149 women) in the initial cohort of the Predictors Study (1989-2001) based on 80 individual measures obtained every 6 mo for 10 y. These models were replicated for AD patients (106 men, 148 women) in the 2nd Predictors Study cohort (1997-2007). Model validation required that the disease-specific transition parameters be identical for both Predictors Study cohorts. Medicare costs were estimated from the National Long Term Care Survey. RESULTS: Sex-specific models were validated using the 2nd Predictors Study cohort with the GoM transition parameters constrained to the values estimated for the 1st Predictors Study cohort; 57 to 61 of the 80 individual measures contributed significantly to the GoM models. Simulated, cost-free interventions in the rate of progression of AD indicated that large potential cost offsets could occur for patients at the earliest stages of AD. CONCLUSIONS: AD progression is characterized by a small number of parameters governing changes in large numbers of correlated indicators of AD severity. The analysis confirmed that the progression of AD represents a complex multidimensional physiological process that is similar across different study cohorts. The estimates suggested that there could be large cost offsets to Medicare and Medicaid from the slowing of AD progression among patients with mild AD. The methodology appears generally applicable in AD modeling.

Willingness-to-pay for reductions in care need: estimating the value of informal care in Alzheimer's disease.

OBJECTIVE: To estimate the value of informal care in Alzheimer's disease using contingent valuation. METHODS: A questionnaire was administered to 517 primary carers in four countries (UK, Spain, Sweden, and US). Dichotomous choice and bidding game methods were used to elicit their willingness to pay for a reduction in care burden by 1 h per day, or a total elimination of care needs. Further, the relationship between carer willingness to pay and carer and patient characteristics including disease severity and income was examined. RESULTS: Carers spend on average about 7-9 h per day on giving care to their patient, of which 4-5 h constituted basic and instrumental ADL tasks. For a 1 h reduction in need for care per day, carers in the UK, Spain, Sweden, and US said that they were willing to pay pound105, pound121, pound59, and pound144 per month respectively. The willingness to pay was higher for carers with higher disposable income while the influence of other determinants varied across countries. About one-third of carers were not willing to pay anything for a reduction in care. CONCLUSIONS: Carers' stated willingness to pay for reductions in care giving time is substantial and comparable to the prices currently paid for treatments that achieve this benefit. Its determinants seem more directly related to carer status than directly to patient status and may vary by region and by cultural and sociologic factors.

Patient dependence and longitudinal changes in costs of care in Alzheimer's disease.

BACKGROUND/AIMS: To examine the incremental effect of patients' dependence on others, on cost of medical and nonmedical care, and on informal caregiving hours over time. METHODS: Data are obtained from 172 patients from the Predictors Study, a large, multicenter cohort of patients with probable Alzheimer disease (AD) followed annually for 4 years in 3 University-based AD centers in the USA. Enrollment required a modified Mini-Mental State Examination score >or=30. We examined the effects of patient dependence (measured by the Dependence Scale, DS) and function (measured by the Blessed Dementia Rating Scale, BDRS) on medical care cost, nonmedical care cost, and informal caregiving time using random effects regression models. RESULTS: A one-point increase in DS score was associated with a 5.7% increase in medical cost, a 10.5% increase in nonmedical cost, and a 4.1% increase in caregiving time. A one-point increase in BDRS score was associated with a 7.6% increase in medical cost, a 3.9% increase in nonmedical cost and an 8.7% increase in caregiving time. CONCLUSIONS: Both functional impairment and patient dependence were associated with higher costs of care and caregiving time. Measures of functional impairment and patient dependence provide unique and incremental information on the overall impact of AD on patients and their caregivers.