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

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.

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

Acupuncture in Medicine ◽

10.1136/acupmed-2012-010178 ◽

2012 ◽

Vol 30 (4) ◽

pp. 273-285 ◽

Cited By ~ 38

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.

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Economic evaluations of health care interventions in Oropharyngeal Dysphagia after Stroke: Protocol for a Systematic Review.

10.21203/rs.3.rs-376486/v1 ◽

2021 ◽

Author(s):

Sergio Marin ◽

Mateu Serra-Prat ◽

Omar Ortega ◽

Pere Clavé

Keyword(s):

Systematic Review ◽

Economic Evaluation ◽

Cost Effectiveness ◽

Oropharyngeal Dysphagia ◽

Cost Savings ◽

Economic Evaluations ◽

Stroke Patients ◽

Post Stroke ◽

Healthcare Interventions ◽

The Cost

Abstract Background and purpose: Oropharyngeal Dysphagia (OD) affects 40-81% of patients after stroke. A recent systematic review on the costs of OD and it’s main complications showed higher acute and long-term costs for those patients who developed OD, malnutrition and pneumonia after stroke. These results suggest that appropriate management of post-stroke OD could lead to reduction of clinical complications and significant cost savings. The purpose of this systematic review is to assess the available literature exploring the efficiency or cost-effectiveness of available healthcare interventions on the appropriate management of OD. Methods: A systematic review on economic evaluations of health care interventions on post-stroke patients with OD following PRISMA recommendations will be performed. MEDLINE, Embase, the National Health Service Economic Evaluation Database and the Cost-Effectiveness Analysis Registry Database will be searched and a subsequent reference check will be done. English and Spanish literature will be included without date restrictions. Studies will be included if they refer to economic evaluations or studies in which cost savings were reported in post-stroke patients suffering OD. Studies will be excluded if they are partial economic evaluation studies, if they refer to esophageal dysphagia, or if OD is caused by causes different from stroke. Evidence will be presented and synthetized with a narrative method and using tables. Quality evaluation will be done using Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement. Discussion: The protocol for this systematic review is the first step to assess the cost-effectiveness of the healthcare interventions that have been described as potential treatments for post-stroke OD. This systematic review will summarize the current evidence on the relation between cost and benefits associated with the appropriate management of OD in post-stroke patients. Systematic review registration: PROSPERO CRD42020136245

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Cost-of-illness and economic evaluations in axial spondyloarthritis

10.1093/med/9780198734444.003.0025 ◽

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.

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The cost-effectiveness of government actions to reduce sodium intake through salt substitutes in Vietnam

10.21203/rs.3.rs-52885/v1 ◽

2020 ◽

Author(s):

Colman Taylor ◽

Annet C Hoek ◽

Irene Deltetto ◽

Adrian Peacock ◽

Do Thi Phuong Ha ◽

...

Keyword(s):

Cost Effectiveness ◽

Sodium Intake ◽

Intervention Strategy ◽

Cost Savings ◽

Cost Effective ◽

Dietary Sodium ◽

Fish Sauce ◽

Regulatory Strategies ◽

Life Years ◽

The Cost

Abstract Background Dietary sodium reduction is recommended to reduce the burden of cardiovascular disease. In Vietnam food products including salt, fish sauce and bot canh contribute to ~70% of dietary sodium intake. Reduced sodium versions of these products can be produced by replacing some of the sodium chloride with potassium chloride. We aimed to assess the cost-effectiveness of three alternative approaches to introducing reduced sodium products onto the market with a view to lowering population sodium intake in Vietnam.Methods The three salt substitution strategies included voluntary, subsidised and regulatory approaches targeting salt, fish sauce and bot canh products. Costs were modelled using the WHO-CHOICE methodology. A Markov cohort model was developed to evaluate the cost-effectiveness of each strategy versus no intervention from the government perspective. The model linked each intervention strategy to assumed changes in levels of sodium intake and then to systolic blood pressure. Changes in SBP were linked to a probability of ischaemic heart disease or stroke. The model followed people over their lifetime to assess average costs and quality adjusted life years (QALYs) gained for each strategy. Results The voluntary salt substitution strategy was assumed to require no investment by government. Following ramp up (years 6+), the average annual costs for the subsidised and regulatory strategies were 21,808,968,902 ₫ (US$ 977,354) and 12,949,953,247 ₫ (US$ 580,410) respectively. Relative to no intervention, all three salt substitution strategies were found to be cost-effective. Cost savings were driven by reductions in strokes (32,595; 768,384; 2,366,480) and IHD events (22,830; 537,157; 1,648,590) for the voluntary, subsidised & regulatory strategies, respectively. The voluntary strategy was least cost-effective (-3,445 ₫ US$ -0.15; 0.009 QALYs gained) followed by the subsidised strategy (-43,189 ₫ US$ -1.86; 0.022 QALYs gained) and the regulatory strategy delivered the highest cost savings and health gains (-243,530 ₫ US$ -10.49; 0.074 QALYs gained). Conclusion This research shows that all three modelled salt substitution strategies would be good value for money relative to no intervention in Vietnam. The subsidised alternative would require the highest level of government investment, however the implementation costs will be exceeded by healthcare savings assuming a reasonable time horizon is considered.

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

Thrombosis and Haemostasis ◽

10.1055/s-0039-3401822 ◽

2019 ◽

Vol 120 (02) ◽

pp. 216-228 ◽

Cited By ~ 4

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.

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Abstract 1: Impact of Predicted Bleeding Risk on the Cost-Effectiveness of Direct Thrombin Inhibition vs. Heparin Monotherapy for Patients Undergoing PCI

Circulation Cardiovascular Quality and Outcomes ◽

10.1161/circoutcomes.4.suppl_1.a1 ◽

2011 ◽

Vol 4 (suppl_1) ◽

Author(s):

Amit P Amin ◽

Steven P Marso ◽

Sunil V Rao ◽

John Messenger ◽

John House ◽

...

Keyword(s):

Cost Effectiveness ◽

Cost Saving ◽

Major Bleeding ◽

Cost Effective ◽

Bleeding Risk ◽

Thrombin Inhibitors ◽

Published Data ◽

Life Years ◽

The Cost ◽

Predicted Risk

Introduction Direct thrombin inhibitors (DTI) reduce bleeding and are cost-saving in patients (pts) undergoing PCI when compared to unfractionated heparin (UFH) plus routine glycoprotein IIb/IIIa inhibition (GPI). Little is known about the cost-effectiveness of DTI alone vs. UFH alone, however. Methods We combined data on patient-level bleeding risk with several external data sources to estimate the economic impact of substituting DTI for UFH among unselected PCI patients. We used a validated model to predict the risk of major bleeding for 81,628 NCDR® CathPCI Registry® patients who received UFH only in 2004-06. DTI costs were estimated based on current FDA dosing guidelines and acquisition costs. The cost of major bleeding ($8722) was estimated based on data from the Mid America Heart Institute. The benefit of DTI vs. UFH alone was based on bleeding risk reduction from ISAR-REACT 3. A Markov model based on published data was used to estimate the loss in life expectancy associated with major bleeding. Results The overall rate of major bleeding in the reference population was 2.2%, the estimated cost of a major bleed was $8722, projected DTI costs were $641/pt and heparin costs were negligible ($4). Assuming that DTI use reduces the risk of bleeding by 33%, use of DTI for all patients was estimated to increase costs by $573/patient and was cost saving only for patients with a predicted risk of major bleeding >20% (0.16% of the overall population). When life years lost due to bleeding were included, at willingness-to-pay thresholds of $50K and $100K per life year gained, DTI was cost effective for patients with a bleeding risk ≥8% (2.5% patients) and ≥5% (7.9% of patients), respectively. Conclusions For patients undergoing PCI, substitution of DTI for UFH monotherapy is projected to increase costs for virtually all patients, but may be reasonably cost-effective for patients with a high predicted risk (≥5%) of bleeding.

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Cost of Current Iron Chelation Infusion Therapy and Cost-Effectiveness of Once-Daily Oral Deferasirox in Transfusion-Dependent Thalassemia Patients in Canada.

Blood ◽

10.1182/blood.v108.11.3349.3349 ◽

2006 ◽

Vol 108 (11) ◽

pp. 3349-3349

Author(s):

T.E. Delea ◽

K. El Ouagari ◽

O. Sofrygin

Keyword(s):

Cost Effectiveness ◽

Iron Overload ◽

Chelation Therapy ◽

Thalassemia Major ◽

Infusion Therapy ◽

Published Data ◽

Blood Transfusions ◽

Once Daily ◽

Life Years ◽

The Cost

Abstract Background: Deferasirox (Exjade®) is a recently approved once-daily oral chelator that has been shown to produce reductions in liver iron concentrations and serum ferritin similar to those with infusional deferoxamine (Desferal®), in patients with β-thalassemia major or sickle cell disease (SCD) and chronic iron overload from blood transfusions. The objective of this study was to estimate the cost of deferoxamine administration in Canada and evaluate the cost-effectiveness of deferasirox versus deferoxamine for chronic iron overload from blood transfusions from the Ontario provincial healthcare system. Methods: Cost of chelation therapy with deferoxamine was estimated from five major treatment centers in Canada. Costs comprised the material used and the time spent by hospital pharmacists in preparing deferoxamine infusions. Cost-effectiveness of the new oral iron chelator, deferasirox, which could replace deferoxamine, was estimated using a Markov model. The model estimates and compares the total lifetime costs and quality-adjusted life years (QALYs) related to the use of deferasirox or deferoxamine in patients with β-thalassemia major at risk of chronic iron overload from blood transfusions. Dosages of deferasirox and deferoxamine used in the model were those that have been shown to be similarly effective in such patients. Compliance with deferoxamine was based on analyses of health-insurance claims data from transfusion-dependent thalassemia patients. Probabilities of complications of iron overload and death by compliance with chelation were estimated from published studies. Because data on compliance with deferasirox in typical clinical practice are unavailable, we used published data on compliance with the oral chelator deferiprone versus deferoxamine. Costs of deferasirox, deferoxamine, and drugs used in the treatment of complications of iron overload were based on publicly available data sources. Utilities were based on a study of patient preferences for oral versus infusional chelation therapy, as well as published literature and assumption. Future costs and QALYs were discounted at 5% annually. Costs were adjusted to 2004 Canadian dollars. Results: Cost of yearly infusion therapy in Canada was estimated at $6,000 per patient, excluding deferoxamine drug cost. Cost-effectiveness of deferasirox compared to deferoxamine was estimated at $45,054 per QALY gained resulting mainly from a gain in 2.9 QALYs obtained at an expected lifetime cost of $130,058 per patient. Cost-effectiveness is sensitive to the estimated costs of deferoxamine and infusional therapy, as well as the quality of life benefit associated with oral versus infusional therapy, and is more favorable in younger patients. Conclusion: In patients with transfusion-dependent β-thalassemia, the cost-effectiveness of deferasirox versus deferoxamine is within the range generally considered acceptable in Canada.

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

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462320000902 ◽

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.

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Cost-effectiveness of teduglutide in adult patients with short bowel syndrome: Markov modeling using traditional cost-effectiveness criteria

American Journal of Clinical Nutrition ◽

10.1093/ajcn/nqz269 ◽

2019 ◽

Vol 111 (1) ◽

pp. 141-148 ◽

Cited By ~ 4

Author(s):

Vikram K Raghu ◽

David G Binion ◽

Kenneth J Smith

Keyword(s):

Cost Effectiveness ◽

Cost Saving ◽

Short Bowel Syndrome ◽

Adult Patients ◽

Sensitivity Analyses ◽

Published Data ◽

Model Parameters ◽

Short Bowel ◽

Life Years ◽

The Cost

ABSTRACT Background Adults with short bowel syndrome have a high mortality and significant morbidity due to unsuccessful attempts at rehabilitation that necessitate chronic use of parenteral nutrition (PN). Teduglutide is a novel therapy that promotes intestinal adaptation to improve rehabilitation but with a price >$400,000/y. Objective The current study evaluated the cost-effectiveness of using teduglutide in US adult patients with short bowel syndrome. Methods A Markov model evaluated the costs (in US dollars) and effectiveness (in quality-adjusted life years, or QALYs) of treatment compared with no teduglutide use, with a presumed starting age of 40 y. Parameters were obtained from published data or estimation. The primary effect modeled was the increased likelihood of reduced PN days per week when using teduglutide, leading to greater quality of life and lower PN costs. Sensitivity analyses were performed on all model parameters. Results In the base scenario, teduglutide cost $949,910/QALY gained. In 1-way sensitivity analyses, only reducing teduglutide cost decreased the cost/QALY gained to below the typical threshold of $100,000/QALY gained. Specifically, teduglutide cost would need to be reduced by >65% for it to reach the threshold value. Probabilistic sensitivity analysis favored no teduglutide use in 80% of iterations at a $100,000/QALY threshold. However, teduglutide therapy was cost-saving in 13% of model iterations. Conclusions Teduglutide does not meet a traditional cost-effectiveness threshold as treatment for PN reduction in adult patients with short bowel syndrome compared with standard intestinal rehabilitation. Subpopulations that demonstrate maximum benefit could be cost-saving, and complete nonuse could lead to financial loss. Teduglutide becomes economically reasonable only if its cost is substantially reduced.

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

Orphanet Journal of Rare Diseases ◽

10.1186/s13023-021-01695-7 ◽

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.

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