ABSTRACT
AIMS: Chronic lung allograft dysfunction (CLAD), a common complication of lung transplantation, is the leading cause of death for lung transplant recipients. While data on lung transplant costs are available, the impact of CLAD on healthcare resource use (HRU) and cost is not well understood. The primary objective was to quantify the HRU and costs of CLAD in the US using real-world data. METHODS: A longitudinal retrospective analysis was performed of commercial claims data from the IQVIA PharMetrics Plus database for patients aged 18-64 who underwent lung transplantation between January 1, 2006 and September 30, 2018. Lung transplantation was identified using International Classification of Disease and Common Procedure Terminology procedure codes. Patients studied were observable for at least 12 months before and after transplantation. Patients who developed CLAD were identified using novel, diagnosis codes for incident lung disease at least one year following transplantation. Descriptive analyses were conducted to assess the study's outcomes prior to and following a CLAD diagnosis. All-cause HRU and costs, the study's primary outcomes, leading up to and following CLAD diagnosis were calculated. RESULTS: Among 129 transplant patients who developed CLAD, healthcare costs were substantially higher in the year following diagnosis ($198,113), compared to the year leading to diagnosis ($85,276). Inpatient admissions were responsible for most costs in years 1 and 2 following diagnosis ($99,372 and $83,348 respectively). Drug costs were higher in the 12 months post-index, compared to the 12 months pre-index ($3,600 vs $2,527). LIMITATIONS: Claims data do not include clinical data, have limits determining loss of follow-up, and do not provide granularity to determine disease severity. Also, there is no ICD-10-CM code specific to CLAD or BOS. CONCLUSIONS: CLAD after lung transplant is associated with substantial HRU and costs. Further work is needed to develop interventions that reduce this impact.
Subject(s)
Insurance , Lung Transplantation , Allografts , Health Care Costs , Humans , Lung , Lung Transplantation/adverse effects , Retrospective Studies , United StatesABSTRACT
Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. Objective: To compare the manufacturer's proprietary CUA model to the model published by the Institute for Clinical and Economic Review (ICER), and to update the manufacturer's model with long-term follow-up data and some key ICER assumptions. Study design: We updated a recent CUA evaluating value for money in cost per incremental Quality-adjusted Life Year (QALY) of onasemnogene abeparvovec versus nusinersen (Spinraza®) or best supportive care (BSC) in symptomatic SMA1 patients, and compared it to the ICER model. Setting/Perspective: USA/Commercial payer Participants: Children aged <2 years with SMA1. Interventions: Onasemnogene abeparvovec, a single-dose gene replacement therapy, versus nusinersen, an antisense oligonucleotide, versus BSC. Main outcome measure: Incremental-cost effectiveness ratio and value-based price using traditional thresholds for general medicines in the US. Results: Updated survival (undiscounted) predicted by the model was 37.60 years for onasemnogene abeparvovec compared to 12.10 years for nusinersen and 7.27 years for BSC. Updated quality-adjusted survival using ICER's utility scores and discounted at 3% were 13.33, 2.85, and 1.15 discounted QALYs for onasemnogene abeparvovec, nusinersen, and BSC, respectively. Using estimated net prices, the discounted lifetime cost/patient was $3.93 M for onasemnogene abeparvovec, $4.60 M for nusinersen, and $1.96 M for BSC. The incremental cost per QALY gained for onasemnogene abeparvovec was dominant against nusinersen and $161,648 against BSC. These results broadly align with the results of the ICER model, which predicted a cost per QALY gained of $139,000 compared with nusinersen, and $243,000 compared with BSC (assuming a placeholder price of $2 M for onasemnogene abeparvovec), differences in methodology notwithstanding. Exploratory analyses in presymptomatic patients were similar. Conclusion: This updated CUA model is similar to ICER analyses comparing onasemnogene abeparvovec with nusinersen in the symptomatic and presymptomatic SMA populations. At a list price of $2.125 M, onasemnogene abeparvovec is cost-effective compared to nusinersen for SMA1 patients treated before age 2 years. When compared to BSC, cost per QALY of onasemnogene abeparvovec is higher than commonly used thresholds for therapies in the USA ($150,000 per QALY).
ABSTRACT
Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy for SMA1. Study design: A Markov model was used to estimate the incremental cost-effectiveness ratio (ICER), expressed as cost/quality-adjusted life year ($/QALY), of AVXS-101 versus nusinersen over a lifetime. Survival, healthcare costs and QALYs were estimated using natural history data for SMA patients who achieved motor milestones (sitting/walking). Health utility weights were obtained from the CHERISH trial. Setting: USA; commercial payer perspective Participants: SMA1 infants Interventions: AVXS-101 was compared to nusinersen. Main outcome measure: The primary outcome was the ICER. Results: Expected survival (undiscounted) over a lifetime predicted by the model was 37.20 life years for AVXS-101 and 9.68 for nusinersen (discounted QALYs, 15.65 and 5.29, respectively). Using a potential AVXS-101 price range ($2.5-5.0M/treatment), the average lifetime cost/patient was $4.2-6.6M for AVXS-101 and $6.3M for nusinersen. The ICER range was (-$203,072) to $31,379 per QALY gained for AVXS-101 versus nusinersen, indicating that AVXS-101 was cost-effective with prices of ≤$5M. Conclusion: Single-dose AVXS-101 was cost-effective compared to chronic nusinersen for SMA1 patients.
