scholarly journals Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Tamara Dangouloff ◽  
Camille Botty ◽  
Charlotte Beaudart ◽  
Laurent Servais ◽  
Mickaël Hiligsmann
Keyword(s):  
Cost Effectiveness ◽  
Spinal Muscular Atrophy ◽  
Annual Cost ◽  
Muscular Atrophy ◽  
Standard Of Care ◽  
Severe Form ◽  
Cost Of Care ◽  
Economic Evaluations ◽  
Life Years ◽  
The Cost

Abstract Background Spinal muscular atrophy (SMA) is a rare and devastating condition for which new disease-modifying treatments have recently been approved. Given the increasing importance of economic considerations in healthcare decision-making, this review summarizes the studies assessing the cost of SMA and economic evaluations of treatments. A systematic review of the literature in PubMed and Scopus up to 15 September 2020 was conducted according to PRISMA guidelines. Results Nine studies reporting the annual cost of care of patients with SMA and six evaluations of the cost-effectiveness of SMA treatments were identified. The average annual cost of SMA1, the most frequent and severe form in which symptoms appear before the age of 6 months were similar according to the different studies, ranged from $75,047 to $196,429 per year. The yearly costs for the forms of the later-onset form, called SMA2, SMA3, and SMA4, which were usually pooled in estimates of healthcare costs, were more variable, ranging from $27,157 to $82,474. The evaluations of cost-effectiveness of treatment compared nusinersen treatment against standard of care (n = 3), two treatments (nusinersen and onasemnogene abeparvovec) against each other and no drug treatment (n = 1), nusinersen versus onasemnogene abeparvovec (n = 1), and standard of care versus nusinersen with and without newborn screening (n = 1). The incremental cost-effectiveness ratio (ICER) of nusinersen compared to standard of care in SMA1 ranged from $210,095 to $1,150,455 per quality-adjusted life years (QALY) gained and that for onasemnogene abeparvovec ranged from $32,464 to $251,403. For pre-symptomatic patients, the ICER value ranged from $206,409 to $735,519. The ICERs for later-onset forms of SMA (2, 3 and 4) were more diverse ranging from $275,943 to $8,438,049. Conclusion This review confirms the substantial cost burden of standard of care for SMA patients and the high cost-effectiveness ratios of the approved drugs at the current price when delivered in post-symptomatic patients. Since few studies have been conducted so far, there is a need for further prospective and independent economic studies in pre- and post-symptomatic patients.

A Systematic Review of Cost-Effectiveness Analyses Alongside Randomised Controlled Trials of Acupuncture

2012 ◽  
Vol 30 (4) ◽  
pp. 273-285 ◽  
Author(s):  
Song-Yi Kim ◽  
Hyangsook Lee ◽  
Younbyoung Chae ◽  
Hi-Joon Park ◽  
Hyejung Lee
Keyword(s):  
Cost Effectiveness ◽  
Usual Care ◽  
Randomised Controlled Trials ◽  
Risk Of Bias ◽  
Economic Evaluations ◽  
Controlled Trials ◽  
German Studies ◽  
Life Years ◽  
Randomised Controlled ◽  
The Cost

Objective To summarise the evidence on the cost-effectiveness of acupuncture. Methods We identified full economic evaluations such as cost-effectiveness analysis (CEA), cost-utility analysis (CUA) and cost-benefit analysis (CBA) alongside randomised controlled trials (RCTs) that assessed the consequences and costs of acupuncture for any medical condition. Eleven electronic databases were searched up to March 2011 without language restrictions. Eligible RCTs were assessed using the Cochrane criteria for risk of bias and a modified version of the checklist for economic evaluation. The general characteristics and the results of each economic analysis such as incremental cost-effectiveness ratios (ICERs) were extracted. Results Of 17 included studies, nine were CUAs that measured quality-adjusted life years (QALYs) and eight were CEAs that assessed effectiveness of acupuncture based on improvements in clinical symptoms. All CUAs showed that acupuncture with or without usual care was cost-effective compared with waiting list control or usual care alone, with ICERs ranging from ¢3011/QALY (dysmenorrhoea) to ¢22 298/QALY (allergic rhinitis) in German studies, and from £3855/QALY (osteoarthritis) to £9951/QALY (headache) in UK studies. In the CEAs, acupuncture was beneficial at a relatively low cost in six European and Asian studies. All CUAs were well-designed with a low risk of bias, but this was not the case for CEAs. Conclusions Overall, this review demonstrates the cost-effectiveness of acupuncture. Despite such promising results, any generalisation of these results needs to be made with caution given the diversity of diseases and the different status of acupuncture in the various countries.

Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis

2021 ◽  
pp. jnnp-2021-326344
Author(s):  
Sophy TF Shih ◽  
Michelle Anne Farrar ◽  
Veronica Wiley ◽  
Georgina Chambers
Keyword(s):  
Gene Therapy ◽  
Cost Effectiveness ◽  
Spinal Muscular Atrophy ◽  
Newborn Screening ◽  
Markov Models ◽  
Muscular Atrophy ◽  
Early Gene ◽  
Sensitivity Analyses ◽  
Life Years ◽  
Late Treatment

ObjectiveTo assess cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) and early treatment with nusinersen or onasemnogene abeparvovec (gene therapy), compared with nusinersen without SMA screening.MethodsInformed by an Australian state-wide SMA NBS programme, a decision analytical model nested with Markov models was constructed to evaluate costs and quality-adjusted life-years (QALYs) from a societal perspective with sensitivity analyses.ResultsBy treating one presymptomatic SMA infant with nusinersen or gene therapy, an additional 9.93 QALYs were gained over 60 years compared with late treatment in clinically diagnosed SMA. The societal cost was $9.8 million for early nusinersen treatment, $4.4 million for early gene therapy and $4.8 million for late nusinersen treatment. Compared with late nusinersen treatment, early gene therapy would be dominant, gaining 9.93 QALYs while saving $360 000; whereas early nusinersen treatment would result in a discounted incremental cost-effectiveness ratio (ICER) of $507 000/QALY.At a population level, compared with no screening and late treatment with nusinersen, NBS and early gene therapy resulted in 0.00085 QALY gained over 60 years and saving $24 per infant screened (85 QALYs gained and $2.4 million saving per 100 000 infants screened). More than three quarters of simulated ICERs by probability sensitivity analyses showed NBS and gene therapy would be dominant or less than $50 000/QALY, compared with no screening and late nusinersen treatment.ConclusionNBS coupled with gene therapy improves the quality and length of life for infants with SMA and would be considered value-for-money from an Australian clinical and policy context.

Cost-of-illness and economic evaluations in axial spondyloarthritis

2016 ◽  
Author(s):  
Annelies Boonen
Keyword(s):  
Cost Effectiveness ◽  
Cost Of Illness ◽  
Axial Spondyloarthritis ◽  
Economic Evaluations ◽  
Low Back ◽  
Quality Adjusted Life Years ◽  
Life Years ◽  
Methodological Principles ◽  
The Cost ◽  
Quality Adjusted Life

Consideration of costs and budgets plays an increasingly important role in decisions on access to innovative technologies. When clinicians want to influence such decisions, it is essential to understand the information on the burden of the disease and the evidence on cost-effectiveness of technologies. This chapter provides guidance to understanding the key methodological principles of economic evaluations, and describes available evidence on these issues in axial spondyloarthritis (axSpA). In the prebiologics era, the cost-of-illness for society of ankylosing spondylitis was slightly lower than for rheumatoid arthritis, and substantially lower than chronic low back pain. Cost of sick leave and work disability accounted for up to 75% of total cost-of-illness. Treatment with biologics increased cost-of-illness substantially, but the important gain in quality-adjusted life years resulted in acceptable cost-effectiveness in patients with active disease. There remains a gap in knowledge about the cost-effectiveness of diagnosing and treating axSpA earlier.

65: The cost-effectiveness of prenatal screening for spinal muscular atrophy

2009 ◽  
Vol 201 (6) ◽  
pp. S37
Author(s):  
Sarah Little ◽  
Vanitha Janakiraman ◽  
Anjali Kaimal ◽  
Thomas Musci ◽  
Jeffrey Ecker ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Spinal Muscular Atrophy ◽  
Prenatal Screening ◽  
Muscular Atrophy ◽  
The Cost

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

2019 ◽  
Vol 120 (02) ◽  
pp. 216-228 ◽  
Author(s):  
Paolo Angelo Cortesi ◽  
Giancarlo Castaman ◽  
Gianluca Trifirò ◽  
Simona Serao Creazzola ◽  
Giovanni Improta ◽  
...  
Keyword(s):  
Health Care ◽  
Cost Effectiveness ◽  
Cost Saving ◽  
Budget Impact ◽  
Economic Evaluations ◽  
Extensive Literature ◽  
Haemophilia A ◽  
Old Patients ◽  
Life Years ◽  
The Cost

AbstractRecent evidence demonstrated that weekly prophylaxis with subcutaneous bispecific antibody (emicizumab) has shown higher efficacy in adolescent and adults patients affected by haemophilia A (HA) with inhibitor, compared with patients treated on demand or on prophylaxis with bypassing agents (BPAs). However, no economic evaluations assessing the value and sustainability of emicizumab prophylaxis have been performed in Europe. This study assessed the cost-effectiveness of emicizumab prophylaxis compared with BPA prophylaxis and its possible budget impact from the Italian National Health Service (NHS) perspective. A Markov model and a budget impact model were developed to estimate the cost-effectiveness and budget impact of emicizumab prophylaxis in HA patients with inhibitors. The model was populated using treatment efficacy from clinical trials and key clinical, cost and epidemiological data retrieved through an extensive literature review. Compared with BPAs prophylaxis, emicizumab prophylaxis was found to be more effective (0.94 quality adjusted life-years) and cost saving (–€19.4/–€24.4 million per patient lifetime) in a cohort of 4-year-old patients with HA and inhibitors who failed immune tolerance induction. In the probabilistic sensitivity analysis, emicizumab prophylaxis had always 100% probability of being cost-effective at any threshold. Further, the use of emicizumab prophylaxis was associated to an overall budget reduction of €45.4 million in the next 3 years. In conclusion, the clinically effective emicizumab prophylaxis can be considered a cost-saving treatment for HA with inhibitor patients. Furthermore, emicizumab treatment is also associated to a significant reduction of the health care budget, making this new treatment a sustainable and convenient health care option for Italian NHS.

scholarly journals The cost-effectiveness of using performance-based financing to deliver the basic package of health services in Afghanistan

2020 ◽  
Vol 5 (9) ◽  
pp. e002381
Author(s):  
Ahmad S Salehi ◽  
Josephine Borghi ◽  
Karl Blanchet ◽  
Anna Vassall
Keyword(s):  
Cost Effectiveness ◽  
Health Services ◽  
Unit Cost ◽  
Health Benefit ◽  
Standard Of Care ◽  
Child Health Services ◽  
Randomised Control Trial ◽  
Economic Evaluations ◽  
Performance Based Financing ◽  
The Cost

Performance-based financing (PBF) is a mechanism to improve the quality and the utilisation of health benefit packages. There is a dearth of economic evaluations of PBF in the ‘real world’. Afghanistan implemented PBF between 2010 and 2015 and evaluated the programme using a pragmatic cluster-randomised control trial. We conducted a cost-effectiveness analysis of the PBF programme in Afghanistan, compared with the standard of care, from the provider payer’s perspective. The incremental cost-effectiveness ratio of PBF compared with the standard of care was US$1242 per disability-adjusted life year averted; not cost-effective when compared with an opportunity cost threshold of US$349. Incentive payments were the main contributor to PBF financial cost (70%) followed by data verification (23%), staff time (5%) and administration (2%). The unit cost per case of antenatal care (ANC), skilled birth attendance (SBA) and postnatal care (PNC) services in the standard of care was US$0.96 (95% CI 0.92–1.0), US$4.8 (95% CI 4.1–6.3) and US$1.3 (95% CI 1.2–1.4), respectively, whereas the cost of ANC, SBA and PNC services per case in PBF areas were US$4.72 (95% CI 4.68–5.7), US$48.5 (95% CI 48.0–52.5) and US$5.4 (95% CI 5.1–5.9), respectively. To conclude, our study found that PBF, as implemented in the Afghan context, was not the best use of funds to strengthen the delivery of maternal and child health services. The cost-effectiveness of alternative PBF designs needs to be appraised before using PBF at scale to support health benefit packages. PBF needs to be considered in the context of funding the range of constraints that inhibit health service performance improvement.

Cost-effectiveness of tositumomab and iodine I-131 tositumomab (Bexxar therapeutic regimen (BTR)), in treatment of non- Hodgkin lymphoma (NHL)

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 8089-8089 ◽  
Author(s):  
C. R. Flowers ◽  
J. C. Sambrook ◽  
A. Briggs ◽  
K. Osenenko ◽  
H. Wang ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
Alternative Therapies ◽  
Cost Of Care ◽  
Low Grade ◽  
Life Years ◽  
Weibull Models ◽  
Third Line ◽  
The Cost

8089 Background: BTR has demonstrated efficacy in NHL patients and it has potential to prolong time to progression (TTP) in relapsed/refractory, low grade, follicular, or transformed NHL. This study assessed effectiveness and cost-effectiveness of BTR compared to alternative therapies in first, second, and third line NHL therapy. Methods: Time-to-event models were constructed with 2 events: progression and death. Patient data from 8 BTR clinical trials were combined to fit Weibull models for TTP and overall survival (OS) by including FLIPI covariates. Estimates for BTR were compared with estimates for alternatives from Weibull models fitted to published TTP and BTR OS data by lines of therapy and measured in life-years (LY). Estimated pre-progression costs included drug costs, lab tests, monitoring, and adverse events; post progression costs included NHL costs until death, all valued in 2006 $US and discounted at 3%. Indirect comparisons yielded incremental cost-effectiveness ratios (ICER=Δ cost/Δ LYs) in each line of therapy. Results: As observed in the table , cost of care estimates in BTR were often comparable with alternative therapies, but typically LY gain favored BTR. Mostly in first and third line, a BTR strategy had an ICER less than the cost-effectiveness threshold of $50,000 per LY gained. Conclusion: Overall, a BTR strategy has a favorable cost-effectiveness profile to alternative strategies including rituximab maintenance (RXM) in first, second, and third line NHL therapy. Results imply both a possible survival gain with early BTR use, and the cost-effectiveness of BTR. This modeling approach can aid in clinical decision making regarding the sequence and timing of therapy for patients with follicular NHL. No significant financial relationships to disclose. [Table: see text]

scholarly journals 1209. Expanding an Economic Evaluation of the Veterans Affairs Initiative to Prevent Methicillin-Resistant Staphylococcus aureus Infections to Include Prevention of Gram-Negative Bacteria

2018 ◽  
Vol 5 (suppl_1) ◽  
pp. S366-S367
Author(s):  
Richard E Nelson ◽  
Michihiko Goto ◽  
Matthew Samore ◽  
Makoto Jones ◽  
Vanessa Stevens ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Savings ◽  
Veterans Affairs ◽  
Economic Evaluations ◽  
Published Data ◽  
Department Of Veterans Affairs ◽  
Gram Negative ◽  
Trade Offs ◽  
Life Years ◽  
The Cost

Abstract Background In October 2007, the Department of Veterans Affairs (VA) launched the National MRSA Prevention Initiative, a nationwide effort to reduce MRSA transmission through (1) universal screening, (2) contact isolation for MRSA+ patients, (3) institutional culture change that “infection prevention is everyone’s business,” (4) emphasis on hand hygiene, and (5) increased infection control resources. While the initiative focused on MRSA, recent evidence suggests that it also led to a significant decrease in hospital-onset (HO) Gram-negative rod (GNR) bacteremia. The objective of this analysis was to evaluate the cost-effectiveness and the budget impact of the initiative taking into account both MRSA and GNR infections. Methods We developed an economic model using published data on the rate of MRSA HAIs and HO-GNR bacteremia in the VA from October 2007 to September 2015, estimates of the attributable cost and mortality of these infections, and the costs associated with the intervention obtained through a microcosting approach. We explored several different assumptions for the rate of infections that would have occurred if the initiative had not been implemented. Effectiveness was measured in life-years (LYs) gained. Results We found that during fiscal years 2008–2015, the initiative resulted in an estimated 4,761–9,236 fewer MRSA HAIs and 1,447–2,159 fewer HO-GNR bacteremia. The initiative itself was estimated to cost $206 million over this 8-year period while the cost savings from prevented MRSA HAIs ranged from $75–165 million and from prevented HO-GNR bacteremia ranged from $42–62 million. The incremental cost-effectiveness of the initiative ranged from $12,146–$46,500/LY when just including MRSA HAIs and from $7,945–$24,387/LY when including HO-GNR bacteremia. The overall impact on the VA’s budget ranged from $200–$334 million. Conclusion An MRSA surveillance and prevention strategy in VA may have prevented a substantial number of MRSA and GNR infections. The savings associated with the prevented infections helped to offset some but not all of the cost of the initiative. Economic evaluations of these interventions can help decision makers understand the trade offs between increased cost and improved health that can come from such interventions. Disclosures All authors: No reported disclosures.

OP141 Health Technology Assessment In India: Current Scenario And Way Forward

2020 ◽  
Vol 36 (S1) ◽  
pp. 1-2
Author(s):  
Komal Shah ◽  
Somen Saha ◽  
Priya Kotwani ◽  
Malkeet Singh ◽  
Kirti Tyagi
Keyword(s):  
Public Health ◽  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Cochrane Library ◽  
Economic Evaluations ◽  
Healthcare Budget ◽  
Life Years ◽  
The Cost

IntroductionIndia has introduced health technology assessment (HTA) as a tool for improving the allocation of health resources. The core mandate of HTA in India (HTAIn) is to undertake critical appraisal of available technologies, identify cost-effective interventions, and help the government pursue evidence-informed decisions regarding public health expenditures. We conducted a systematic review to assess economic evaluation studies published in the last four years from India.MethodsEconomic evaluations published from September 2015 to September 2019 were identified by searching various databases, including PubMed, Scopus, Embase, The Cochrane Library, and CINAHL according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guideline. Cost-effectiveness studies and HTAs reported or conducted in India were included. Two independent reviewers performed the final selection of studies by assessing the full-text articles and conducted the data extraction. Differences of opinions were resolved through discussion and mutual consensus.ResultsAfter screening 2,837 articles, seventy met the inclusion criteria and were selected. The articles predominantly used secondary data (70%) to evaluate the cost effectiveness of an innovation. Among the technologies assessed, fifty-seven percent were curative in nature and most commonly addressed infectious diseases (27%), closely followed by non-communicable diseases, and maternal and child health. Principally, the cost effectiveness of a technology was expressed in terms of disability-adjusted or quality-adjusted life-years. Only two studies reported negative findings.ConclusionsHTA can play a pivotal role in equipping policy makers and public health payers to make appropriate decisions for healthcare budget allocations when mapped with the true disease burden of the population. It is important to highlight negative results and to create a national repository of HTA studies to facilitate faster adoption of best practices in India.

scholarly journals Cost-Effectiveness Assessment of Different Glucosamines in Patients with Knee Osteoarthritis: a Simulation Model Adapted to Germany

2021 ◽  
Vol 14 ◽  
Author(s):  
Olivier Bruyère ◽  
Johann Detilleux ◽  
Jean-Yves Reginster
Keyword(s):  
Cost Effectiveness ◽  
Simulation Model ◽  
Area Under The Curve ◽  
Cost Effective ◽  
Health Utility ◽  
Economic Evaluations ◽  
Individual Health ◽  
Life Years ◽  
The Cost ◽  
Utility Scores

Background: The use of symptomatic slow-acting drugs for osteoarthritis (OA) (e.g., glucosamine, chondroitin) is largely debated in the scientific literature. Indeed, multiple formulations of these agents are available, both as pharmaceutical-grade products and as nutritional supplements, but while all preparations may claim to deliver a therapeutic effect, not all are supported by clinical evidence. Moreover, few data are available regarding the cost-effectiveness of all these formulations. Usually, access to individual patient data is required to perform economic evaluations of treatments, but it can be difficult to obtain. We previously developed a model to simulate individual health utility scores from aggregated data obtained from published OA trials. Objective: In the present study, using our new simulation model, we investigated the cost-effectiveness of different glucosamines used in Germany. Methods: We used our validated model to simulate the utility scores of 10 published trials that used different glucosamine preparations. Using the simulated utility scores, the quality-adjusted life years (QALYs) were calculated using the area-under-the-curve method. We used the 2018 public costs of glucosamine products available in Germany to calculate the Incremental Cost/Effectiveness Ratio (ICER). We performed analyses for pharmaceutical-grade Crystalline Glucosamine Sulfate (pCGS) and other formulations of glucosamine (OFG). A cost-effectiveness cut-off of 30,000 €/QALY was considered. Results: Of 10 studies in which utility was simulated, four used pCGS, and six used OFG. The ICER analyses showed that pCGS was cost-effective compared to a placebo, with an ICER of 4489 €/QALY at month 3, 4112 €/QALY at month 6 and 9983 €/QALY at year 3. The use of OFG was not cost-effective at any of the time points considered. Conclusion: Using our previously published model to simulate the individual health utility scores of patients, we showed that, in the German context, the use of pCGS could be considered cost-effective, while the use of OFG could not. These results highlight the importance of the formulation of glucosamine.

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