Epidemiology and economic burden of amyotrophic lateral sclerosis in the United States: a literature review

Berry, James; Blanchard, Marie; Bonar, Kerina; Drane, Emma; Murton, Molly; Ploug, Uffe Jon; Ricchetti-Masterson, Kristen; Savić, Nataša; Worthington, Emma; Heiman‐Patterson, Terry

doi:10.1080/21678421.2023.2165947

Cited by 13 publications

(7 citation statements)

References 41 publications

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“…12 Medication costs for ALS patients in the United States were reported as $2473 per year for those with commercial insurance, 13,14 but this increased to $7800 in 2018, the year following approval by the FDA of intravenous edaravone. 15 In our study, out-of-pocket costs were notably high for some patients, especially for those with Medicare coverage, at times leading to a decision not to start the treatment. As additional drugs become approved for ALS, financial considerations are likely to increase in importance, potentially impacting drug initiation and continuation.…”

Section: Discussionmentioning

confidence: 59%

Sodium phenylbutyrate–taurursodiol access, adherence and adverse event in patients with amyotrophic lateral sclerosis: Experience at one center in the United States

Mamarabadi,

Fafoutis,

Geronimo

et al. 2024

Muscle and Nerve

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Introduction/AimsSodium phenylbutyrate–taurursodiol (PB‐TURSO) was recently approved for treating amyotrophic lateral sclerosis (ALS). Third‐party payors' coverage policies are evolving, and adverse events are just being fully assessed. The goals of this study were to evaluate patients' experiences in obtaining and continuing PB‐TURSO and assess adverse events and medication adherence.MethodsMedical records of 109 ALS patients who were considered PB‐TURSO candidates by the treating physician at a tertiary ALS clinic from October 2022 to May 2023 were reviewed. Data was recorded for demographics, clinical, and insurance information. A survey was e‐mailed to patients asking about out‐of‐pocket expenses for PB‐TURSO, financial assistance, medication start and (if applicable) stop dates, and reasons for discontinuation.ResultsInsurance information was available for 91 patients [57 males (62%); mean age 64.8 years (range 25.7–88)]. Of 79 who applied for insurance approval, 71 (90%) were approved; however, 19 required 1–3 appeals. Among 73 patients with available data about medication status, 54 started PB‐TURSO and 19 did not, most commonly due to personal choice or out‐of‐pocket expenses. About 44% of patients (24/54) stopped taking PB‐TURSO, primarily due to adverse events. Monthly out‐of‐pocket expenses varied from $0 to $3500 and 36 patients qualified for financial assistance. Administrative and nursing staff devoted 7.2 hours/week to the insurance authorization process.DiscussionMost patients received insurance approval for PB‐TURSO, but one‐fourth required appeals. Some out‐of‐pocket costs were very high. Investment of staff time was substantial. These findings have implications for insurance coverage of, and adherence to, future ALS treatments.

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Section: Discussionmentioning

confidence: 59%

Sodium phenylbutyrate–taurursodiol access, adherence and adverse event in patients with amyotrophic lateral sclerosis: Experience at one center in the United States

Mamarabadi,

Fafoutis,

Geronimo

et al. 2024

Muscle and Nerve

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“…A recent study in New Zealand showed a mortality rate of 2.8/100,000 pop./year in the period 1992–2013 with 74% of the New Zealand population being made up of European and Caucasian descendants [ 68 ]. In the US population, very similar incidence and mortality rates are reported with the exception of Midwest where registered mortality from ALS is significantly higher [ 69 , 70 , 71 ].…”

Section: Discussionmentioning

confidence: 88%

Amyotrophic Lateral Sclerosis and Air Pollutants in the Province of Ferrara, Northern Italy: An Ecological Study

Antonioni

Govoni

Brancaleoni

et al. 2023

IJERPH

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The etiopathogenesis of amyotrophic lateral sclerosis (ALS) is still largely unknown, but likely depends on gene–environment interactions. Among the putative sources of environmental exposure are air pollutants and especially heavy metals. We aimed to investigate the relationship between ALS density and the concentration of air pollution heavy metals in Ferrara, northern Italy. An ecological study was designed to correlate the map of ALS distribution and that of air pollutants. All ALS cases diagnosed between 2000 and 2017 (Ferrara University Hospital administrative data) were plotted by residency in 100 sub-areas, and grouped in 4 sectors: urban, rural, northwestern and along the motorway. The concentrations of silver, aluminium, cadmium, chrome, copper, iron, manganese, lead, and selenium in moss and lichens were measured and monitored in 2006 and 2011. Based on 62 ALS patients, a strong and direct correlation of ALS density was observed only with copper concentrations in all sectors and in both sexes (Pearson coefficient (ρ) = 0.758; p = 0.000002). The correlation was higher in the urban sector (ρ = 0.767; p = 0.000128), in women for the overall population (ρ = 0.782, p = 0.000028) and in the urban (ρ = 0.872, p = 0.000047) population, and for the older cohort of diagnosed patients (2000–2009) the assessment correlated with the first assessment of air pollutants in 2006 (ρ = 0.724, p = 0.008). Our data is, in part, consistent with a hypothesis linking copper pollution to ALS.

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“…Current symptomatic treatments for ALS patients include riluzole, a glutamate-receptor antagonist, and edaravone, a free radical scavenger (Lamptey et al, 2022). None of the medications halt or reverse ALS progression (Berry et al, 2023).…”

Section: Current Diagnosis and Treatmentmentioning

confidence: 99%

Recent Breakthroughs in the Diagnosis and Treatment of Neurodegenerative Disorders

Curtis,

Goodman

2023

J Stud Res

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This paper covers recent breakthroughs in diagnosis and treatment of neurodegenerative disorders, specifically diagnostic biomarkers and disease modifying treatments for Alzheimer's Disease (AD), Parkinson's Disease (PD) and Amyotrophic Lateral Sclerosis (ALS). Biomarkers in the brain, spinal fluid and blood are helping identify the pathologic misfolded proteins that specifically characterize these neurodegenerative disorders. The signature proteins include beta-amyloid and hyperphosphorylated tau (P-tau) in AD, alpha-synuclein in PD, and recently discovered in ALS, TDP-43. As new biomarkers are validated, there is increasing optimism that innovative disease modifying treatments will prevent, reverse or delay the progression of these neurodegenerative diseases. The growing trend of FDA approval of medications through the Accelerated Approval program based on biomarker indicators is expediting drug development for AD, PD, and ALS. Using this pathway, the FDA has granted conditional approval for an AD treatment that lowers brain amyloid and another AD treatment that is associated in studies with slowing of both cognitive and functional loss. The FDA also granted conditional approval for an ALS treatment demonstrating reduction in a neurodegeneration biomarker. The FDA granted Breakthrough Device designation for a blood test as a diagnostic biomarker for AD and the recent breakthrough in the ability to diagnose PD with an accurate biomarker is a major milestone. Continued collaboration among the drug discovery and clinical research communities is needed to accelerate our understanding of the underlying biology and develop disease modifying treatments for these neurodegenerative disorders.

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Epidemiology and economic burden of amyotrophic lateral sclerosis in the United States: a literature review

Cited by 13 publications

References 41 publications

Sodium phenylbutyrate–taurursodiol access, adherence and adverse event in patients with amyotrophic lateral sclerosis: Experience at one center in the United States

Sodium phenylbutyrate–taurursodiol access, adherence and adverse event in patients with amyotrophic lateral sclerosis: Experience at one center in the United States

Amyotrophic Lateral Sclerosis and Air Pollutants in the Province of Ferrara, Northern Italy: An Ecological Study

Recent Breakthroughs in the Diagnosis and Treatment of Neurodegenerative Disorders

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