scholarly journals Should the Government Be Allowed to Take Control over Your Car as Part of a Disaster Management Plan?

2020 ◽  
Vol 17 (21) ◽  
pp. 7780
Author(s):  
Yipeng Lv ◽  
Zafar Zafari ◽  
Boshen Jiao ◽  
Casey Chun ◽  
Lulu Zhang ◽  
...  
Keyword(s):  
Markov Model ◽  
Incremental Cost ◽  
Autonomous Vehicles ◽  
Credible Interval ◽  
The United States ◽  
Status Quo ◽  
Health States ◽  
Life Years ◽  
Hypothetical Scenario ◽  
The Government

Introduction: With the Safety Ensuring Lives Future Deployment and Research in Vehicle Evolution (SELF DRIVE) Act in the United States, there is a growing interest in autonomous vehicles (AVs). One avenue of innovation would be to use them to mobilize and coordinate response efforts during natural disasters. This study uses an earthquake response in an urban, developed setting as a hypothetical example case study. In this hypothetical scenario, private AVs would be mobilized to help rescue victims from collapsed structures. Methods: A Markov model compared an intervention arm with AVs to a status quo arm using a hypothetical cohort of American earthquake victims. The three possible health states were trapped but alive, rescued and alive, and dead. The cycle length of the Markov model was 6 h. Results: The cost of deploying AVs was $90,139 relative to $87,869 in status quo arm. Using AVs produced an incremental cost of $2269 (95% credible interval (CI) = $−12,985–$8959). Victims have 7.33 quality-adjusted life years (QALYs) in the intervention arm compared to 7.20 QALYs in the status quo arm, resulting in an incremental gain of 0.13 (95% CI = −0.73–2.19) QALYs. The incremental cost-effectiveness ratio (ICER) was $16,960/QALY gained (95% CI = cost-saving–$69,065/QALY). Discussion: The mobilization of private AVs in the setting of an earthquake has the potential to save money and reduce the loss of life. AVs may advance emergency management competencies.

scholarly journals Cost-effectiveness of Chloride-liberal versus Chloriderestrictive Intravenous Fluids among Patients Hospitalized in the United States

10.36469/9829 ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. 90-102
Author(s):  
Louise Perrault ◽  
Dilip Makhija ◽  
Idal Beer ◽  
Suzanne Laplante ◽  
Sergio Iannazzo ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Decision Tree ◽  
Markov Model ◽  
The United States ◽  
Sensitivity Analyses ◽  
Major Surgery ◽  
Health State ◽  
Health States ◽  
Life Years ◽  
The Us

Background: Patients developing acute kidney injury (AKI) during critical illness or major surgery are at risk for renal sequelae such as costly and invasive acute renal replacement therapy (RRT) and chronic dialysis (CD). Rates of renal injury may be reduced with use of chloride-restrictive intravenous (IV) resuscitation fluids instead of chloride-liberal fluids. Objectives: To compare the cost-effectiveness of chloride-restrictive versus chloride-liberal crystalloid fluids used during fluid resuscitation or for the maintenance of hydration among patients hospitalized in the US for critical illnesses or major surgery. Methods: Clinical outcomes and costs for a simulated patient cohort (starting age 60 years) receiving either chloride-restrictive or chloride-liberal crystalloids were estimated using a decision tree for the first 90-day period after IV fluid initiation followed by a Markov model over the remainder of the cohort lifespan. Outcomes modeled in the decision tree were AKI development, recovery from AKI, progression to acute RRT, progression to CD, and death. Health states included in the Markov model were dialysis free without prior AKI, dialysis-free following AKI, CD, and death. Estimates of clinical parameters were taken from a recent meta-analysis, other published studies, and the US Renal Data System. Direct healthcare costs (in 2015 USD) were included for IV fluids, RRT, and CD. US-normalized health-state utilities were used to calculate quality-adjusted life years (QALYs). Results: In the cohort of 100 patients, AKI was predicted to develop in the first 90 days in 36 patients receiving chloride-liberal crystalloids versus 22 receiving chloride-restrictive crystalloids. Higher costs of chloride-restrictive crystalloids were offset by savings from avoided renal adverse events. Chloride-liberal crystalloids were dominant over chloride-restrictive crystalloids, gaining 93.5 life-years and 81.4 QALYs while saving $298 576 over the cohort lifespan. One-way sensitivity analyses indicated results were most sensitive to the relative risk for AKI development and relatively insensitive to fluid cost. In probabilistic sensitivity analyses with 1000 iterations, chloride-restrictive crystalloids were dominant in 94.7% of iterations, with incremental cost-effectiveness ratios below $50 000/QALY in 99.6%. Conclusions: This analysis predicts improved patient survival and fewer renal complications with chloriderestrictive IV fluids, yielding net savings versus chloride-liberal fluids. Results require confirmation in adequately powered head-to-head randomized trials.

Cost-effectiveness of treating patients with advanced progressive pancreatic neuroendocrine tumor with everolimus versus sunitinib in the United States.

2012 ◽  
Vol 30 (4_suppl) ◽  
pp. 226-226
Author(s):  
Roman Casciano ◽  
Maruit Chulikavit ◽  
Allison Perrin ◽  
Zhimei Liu ◽  
Xufang Wang ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Adverse Events ◽  
Incremental Cost ◽  
Stable Disease ◽  
Indirect Comparison ◽  
The United States ◽  
Phase 3 ◽  
Physician Visits ◽  
Life Years ◽  
The Cost

226 Background: Everolimus and sunitinib were recently approved to treat patients with advanced, progressive pancreatic neuroendocrine tumors (pNETs). This analysis examines the projected cost-effectiveness of everolimus versus sunitinib in this setting from a US payer perspective. Methods: A lifetime Markov model was developed to simulate a cohort of advanced, progressive pNET patients and to estimate the cost per incremental life-years gained (LYG) and quality-adjusted life years (QALYs) gained when treating with everolimus as compared to sunitinib. Absent head-to-head trials, efficacy data were based on a weight-adjusted indirect comparison of the two agents using the respective phase 3 trial data. Disease or health states considered in the model included: stable disease without adverse events, stable disease with adverse events, disease progression, and death. Costs of anti-tumor therapies, symptomatic care drugs, and post-progression therapy were based on wholesale acquisition cost. Other costs including physician visits, tests, infusions, hospitalizations, adverse event costs, and end-of-life care costs were obtained from literature and/or standard sources such as the Healthcare Cost and Utilization Project and Medicare reimbursement rates. Utility inputs were based on a UK time trade-off study. Sensitivity analyses were conducted to test the model’s robustness. Results: In the base case analysis, the estimated gain of everolimus over sunitinib was 0.448 LYs (0.304 QALYs) at an incremental cost of $12,673, resulting in an incremental cost-effectiveness ratio (ICER) of $28,281/LYG ($41,702/QALY gained), which fell within the cost per QALY range for many other widely used oncology drugs. The analysis was sensitive to the uncertainty of the sunitinib trial results; however, a probabilistic sensitivity analysis showed the results were consistent across simulations. Conclusions: While the analysis is limited by its reliance on an indirect comparison of two phase 3 studies rather than a single head-to-head trial, everolimus is expected to be cost-effective relative to sunitinib in advanced pNET.

Evaluating the cost-effectiveness of hydrogel spacer in radiotherapy (RT) of prostate cancer (PC).

2018 ◽  
Vol 36 (6_suppl) ◽  
pp. 43-43
Author(s):  
Rahul Ramesh Khairnar ◽  
Joseph Levy ◽  
Mark Mishra
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Intensity Modulated Radiation Therapy ◽  
Cost Effective ◽  
Credible Interval ◽  
Phase Iii ◽  
Incremental Effectiveness ◽  
Life Years ◽  
The Cost ◽  
Fee Schedule

43 Background: A hydrogel rectal spacer (HRS) is an FDA-approved medical device used to increase the separation between the rectum and the prostate. A recent phase III trial demonstrated a small reduction in the incidence of RT toxicities associated with use of HRS. We conducted a cost-effectiveness analysis of HRS use in PC patients undergoing intensity modulated radiation therapy (IMRT). Methods: A multi-state Markov model was constructed to examine the cost-effectiveness of HRS in men with localized PC receiving IMRT in the US (arms: IMRT alone vs. IMRT + HRS). Subgroups included delivery site of IMRT (hospital vs. ambulatory) and baseline sexual function (SF) (general population vs. those with good SF). Based on previous studies, recurrence and survival were assumed equal for both arms. Data on SF, gastrointestinal and genitourinary toxicities incidence, as well as potential risks associated with HRS implantation were obtained from a recently published clinical trial. Health utilities and costs were derived from the literature and 2018 Physician Fee Schedule. Quality-adjusted life years (QALYs) and costs were modeled for a 5-year period from receipt of RT. Probabilistic sensitivity analysis (PSA) and value-based threshold analysis were conducted. Costs and utilities were discounted at 3% annually. Results: The per-person 5-year incremental cost for HRS administered in a hospital was $4,008 and the incremental effectiveness was 0.0273 QALYs. The incremental cost-effectiveness ratio (ICER) was $146,746 (95% credible interval from PSA $125,638 – $178,049) for PC patients undergoing HRS insertion in a hospital vs. $73,359 ($66,732 – $86,767) for patients undergoing HRS insertion in an ambulatory facility. For men with good SF, the ICER was $55,153 ($46,002 – $76,090) and $26,542 ($17,399 – $46,044) in hospital vs. ambulatory facility. Conclusions: This study is the first to evaluate the cost-effectiveness of HRS based on long-term toxicity data. Based on the current Medicare Physician Fee Schedule, HRS is cost-effective in men with good SF at a willingness to pay threshold of $100,000 and it is marginally cost-effective for the entire population depending on the facility where the HRS is inserted.

scholarly journals Forecasting US ivacaftor outcomes and cost in cystic fibrosis patients with the G551D mutation

2016 ◽  
Vol 47 (6) ◽  
pp. 1697-1705 ◽  
Author(s):  
Piyameth Dilokthornsakul ◽  
Ryan N. Hansen ◽  
Jonathan D. Campbell
Keyword(s):  
Cystic Fibrosis ◽  
Usual Care ◽  
Budget Impact ◽  
Lifetime Cost ◽  
Credible Interval ◽  
Genetic Mutation ◽  
Forced Expiratory Volume ◽  
Health States ◽  
Payer Perspective ◽  
Life Years

Ivacaftor, a breakthrough treatment for cystic fibrosis (CF) patients with the G551D genetic mutation, lacks long-term clinical and cost projections. This study forecasted outcomes and cost by comparing ivacaftor plus usual care versus usual care alone.A lifetime Markov model was conducted from a US payer perspective. The model consisted of five health states: 1) forced expiratory volume in 1 s (FEV1) % pred ≥70%, 2) 40%≤ FEV1 % pred <70%, 3) FEV1 % pred <40%, 4) lung transplantation and 5) death. All inputs were extracted from published literature. Budget impact was also estimated. We estimated ivacaftor's improvement in outcomes compared with a non-CF referent population.Ivacaftor was associated with 18.25 (95% credible interval (CrI) 13.71–22.20) additional life-years and 15.03 (95% CrI 11.13–18.73) additional quality-adjusted life-years (QALYs). Ivacaftor was associated with improvements in survival and QALYs equivalent to 68% and 56%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $3 374 584. The budget impact was $0.087 per member per month.Ivacaftor increased life-years and QALYs in CF patients with the G551D mutation, and moved morbidity and mortality closer to that of their non-CF peers. Ivacaftor costs much more than usual care, but comes at a relatively limited budget impact.

scholarly journals The Trade-Off between Optimizing Flight Patterns and Human Health: A Case Study of Aircraft Noise in Queens, NY, USA

2018 ◽  
Vol 15 (8) ◽  
pp. 1753 ◽  
Author(s):  
Zafar Zafari ◽  
Boshen Jiao ◽  
Brian Will ◽  
Shukai Li ◽  
Peter Muennig
Keyword(s):  
Indirect Costs ◽  
Ground Level ◽  
Status Quo ◽  
Current Status ◽  
Decision Analytic Model ◽  
Health States ◽  
Life Years ◽  
Automation Systems ◽  
The U.S

Objectives: Airports in the U.S. have gradually been transitioning to automated flight systems. These systems generate new flight paths over populated areas. While they can improve flight efficiency, the increased noise associated with these novel flight patterns potentially pose serious health threats to the overflown communities. In this case study, we estimated the monetary benefits relative to health losses associated with one significant change in flight patterns at LaGuardia Airport, year-round use of “TNNIS Climb”, which happened in 2012 as a result of flight automation in New York City. Prior to that, the use of the TNNIS Climb was limited to the U.S. Open tennis matches. Methods: We developed a decision-analytic model using Markov health states to compare the costs and quality-adjusted life years (QALYs) gained associated with the limited use of TNNIS (old status quo) and the year-round use of TNNIS (current status quo). The TNNIS Climb increases airplane noise to above 60 decibels (dB) over some of the most densely populated areas of the city. We used this increased exposure to noise as the basis for estimating ground-level health using data from sound monitors. The total costs (including both direct and indirect costs), QALYs, and the incremental cost-effectiveness ratio (ICER) were estimated for the limited versus the year-round use of the TNNIS Climb. Results: The incremental lifetime costs and QALYs per person exposed to noise associated with the limited versus the year-round use of TNNIS was $11,288, and 1.13, respectively. Therefore, the limited use of TNNIS had an ICER of $10,006/QALY gained relative to the year-round of TNNIS. Our analyses were robust to changes in assumptions and data inputs. Conclusions: Despite increases in efficiency, flight automation systems without a careful assessment of noise might generate flight paths over densely populated areas and cause serious health conditions for the overflown communities.

An assessment of the human health impact of seven leading foodborne pathogens in the United States using disability adjusted life years

2015 ◽  
Vol 143 (13) ◽  
pp. 2795-2804 ◽  
Author(s):  
E. SCALLAN ◽  
R. M. HOEKSTRA ◽  
B. E. MAHON ◽  
T. F. JONES ◽  
P. M. GRIFFIN
Keyword(s):  
United States ◽  
Foodborne Pathogens ◽  
Health Impact ◽  
The United States ◽  
Health State ◽  
Foodborne Disease ◽  
Health States ◽  
Disability Adjusted Life ◽  
Life Years ◽  
Safety Interventions

SUMMARYWe explored the overall impact of foodborne disease caused by seven leading foodborne pathogens in the United States using the disability adjusted life year (DALY). We defined health states for each pathogen (acute illness and sequelae) and estimated the average annual incidence of each health state using data from public health surveillance and previously published estimates from studies in the United States, Canada and Europe. These pathogens caused about 112 000 DALYs annually due to foodborne illnesses acquired in the United States. Non-typhoidal Salmonella (32 900) and Toxoplasma (32 700) caused the most DALYs, followed by Campylobacter (22 500), norovirus (9900), Listeria monocytogenes (8800), Clostridium perfringens (4000), and Escherichia coli O157 (1200). These estimates can be used to prioritize food safety interventions. Future estimates of the burden of foodborne disease in DALYs would be improved by addressing important data gaps and by the development and validation of US-specific disability weights for foodborne diseases.

scholarly journals Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy

Circulation ◽  
2020 ◽  
Vol 141 (15) ◽  
pp. 1214-1224 ◽  
Author(s):  
Dhruv S. Kazi ◽  
Brandon K. Bellows ◽  
Suzanne J. Baron ◽  
Changyu Shen ◽  
David J. Cohen ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
The United States ◽  
Quality Adjusted Life Year ◽  
Life Years ◽  
The Us ◽  
Amyloid Cardiomyopathy ◽  
Quality Adjusted Life

Background: In patients with transthyretin amyloid cardiomyopathy, tafamidis reduces all-cause mortality and cardiovascular hospitalizations and slows decline in quality of life compared with placebo. In May 2019, tafamidis received expedited approval from the US Food and Drug Administration as a breakthrough drug for a rare disease. However, at $225 000 per year, it is the most expensive cardiovascular drug ever launched in the United States, and its long-term cost-effectiveness and budget impact are uncertain. We therefore aimed to estimate the cost-effectiveness of tafamidis and its potential effect on US health care spending. Methods: We developed a Markov model of patients with wild-type or variant transthyretin amyloid cardiomyopathy and heart failure (mean age, 74.5 years) using inputs from the ATTR-ACT trial (Transthyretin Amyloidosis Cardiomyopathy Clinical Trial), published literature, US Food and Drug Administration review documents, healthcare claims, and national survey data. We compared no disease–specific treatment (“usual care”) with tafamidis therapy. The model reproduced 30-month survival, quality of life, and cardiovascular hospitalization rates observed in ATTR-ACT; future projections used a parametric survival model in the control arm, with constant hazards reduction in the tafamidis arm. We discounted future costs and quality-adjusted life-years by 3% annually and examined key parameter uncertainty using deterministic and probabilistic sensitivity analyses. The main outcomes were lifetime incremental cost-effectiveness ratio and annual budget impact, assessed from the US healthcare sector perspective. This study was independent of the ATTR-ACT trial sponsor. Results: Compared with usual care, tafamidis was projected to add 1.29 (95% uncertainty interval, 0.47–1.75) quality-adjusted life-years at an incremental cost of $1 135 000 (872 000–1 377 000), resulting in an incremental cost-effectiveness ratio of $880 000 (697 000–1 564 000) per quality-adjusted life-year gained. Assuming a threshold of $100 000 per quality-adjusted life-year gained and current drug price, tafamidis was cost-effective in 0% of 10 000 probabilistic simulations. A 92.6% price reduction from $225 000 to $16 563 would be necessary to make tafamidis cost-effective at $100 000/quality-adjusted life-year. Results were sensitive to assumptions related to long-term effectiveness of tafamidis. Treating all eligible patients with transthyretin amyloid cardiomyopathy in the United States with tafamidis (n=120 000) was estimated to increase annual healthcare spending by $32.3 billion. Conclusions: Treatment with tafamidis is projected to produce substantial clinical benefit but would greatly exceed conventional cost-effectiveness thresholds at the current US list price. On the basis of recent US experience with high-cost cardiovascular medications, access to and uptake of this effective therapy may be limited unless there is a large reduction in drug costs.

Creole Nationalism and Mexican Liberalism

1973 ◽  
Vol 15 (2) ◽  
pp. 139-190 ◽  
Author(s):  
D. A. Brading
Keyword(s):  
Social Reform ◽  
The United States ◽  
Status Quo ◽  
Political Support ◽  
Presidential Power ◽  
Military Dictatorship ◽  
Porfirio Diaz ◽  
The Status ◽  
Classic Liberalism ◽  
The Government

Absolute monarchy, military dictatorship, domination by the privileged orders, union with the United States, communism, the preponderance of the Aztecs: all these aberrations have their apostles, their writers and their conspirators. Meanwhile the government, without a policy, power or political support, survives by the general inertia and is reduced to preserving the status quo.-Mariano Otero to Dr. MoraAlthough one recent influential textbook on Latin America has characterized the decades immediately following the achievement of independence as “the long wait,” in Mexico at least, these years were marked by an intense political and ideological conflict which defined the direction of its future (Halperín Donghi, 1969: 134-206). The most perceptive student of the epoch, Edmundo O'Gorman (1960) traces within the confused welter of pronunciamientos and manifestos two great forces: the search for a providential leader and the desire for some form of democratic populism. An analysis of ideology cannot be separated from a consideration of society. The presidential power created by Benito Juárez and perpetuated by Porfirio Díaz operated outside the strict legal confines of the Constitution. At the same time, the failure of classic liberalism to express popular aspirations retarded social reform for over half a century. Without the sanction of theory, few such demands could be translated into law.

Cost-Effectiveness of Rituximab Post CVP Induction for the Treatment of Low-Grade NHL.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4486-4486
Author(s):  
John C. Hornberger ◽  
Carolina Reyes ◽  
Ashwini Shewade ◽  
David Loecke ◽  
Nancy Valente
Keyword(s):  
Cost Effectiveness ◽  
B Cell ◽  
Progression Free Survival ◽  
The United States ◽  
Sensitivity Analyses ◽  
Low Grade ◽  
Health States ◽  
Economic Implications ◽  
Expected Gain ◽  
Life Years

Abstract Background: Extended Rituximab (R) therapy after CVP (cyclophosphamide, vincristine, prednisone) induction in the first-line treatment of low-grade, CD20-positive, B-cell NHL significantly prolongs progression-free survival (PFS). A societal cost-effectiveness analysis is performed to estimate projected lifetime clinical and economic implications of this treatment. Methods: A Markov model with 3 health states (progression-free, post-progression, and death) was developed to estimate the direct medical costs and quality-adjusted life-years (QALYs) for a representative patient cohort with low-grade, CD20-positive, B-cell NHL. Kaplan-Meier estimates of PFS and overall survival (OS) up to 4 years are obtained from the ECOG/CALGB intergroup E1496 trial. After 4 years, transition rates are assumed to be the same in both arms. Costs include drug and administration costs, adverse events, treatment of relapses, and end-of-life costs. Incremental costs associated with R are based on Medicare reimbursement rates and published drug prices. Utilities are derived from the literature and a 3% discount rate is employed. Results: Projected mean OS is 1.2-yrs longer for patients assigned to extended R therapy versus observation alone (15.2 v 14.0 yrs). Extended R therapy is estimated to cost $42,822 on average, with an expected gain of 0.85 years of quality-adjusted survival. Over a lifetime, the cost per QALY gained is $57,515. Sensitivity analyses performed on all variables contributing to the model results showed that utility for follicular NHL patients (range: 0.68–1) and number of R infusions (range: 8–17) most influence the incremental cost-effectiveness ratio (ICER). Conclusion: The ICER of extended R dosing following CVP induction remains well below the threshold considered acceptable for oncology in the United States. Additional analysis of long term follow-up data will be useful for further evaluation of clinical and economic implications of this treatment for low-grade, CD20-positive, B-cell NHL patients.

scholarly journals Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US

2020 ◽  
Vol 18 (1) ◽  
Author(s):  
Praveen Thokala ◽  
Matt Stevenson ◽  
Varun M. Kumar ◽  
Shijie Ren ◽  
Alexandra G. Ellis ◽  
...  
Keyword(s):  
Health Care ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Motor Function ◽  
Muscular Atrophy ◽  
Sensitivity Analyses ◽  
Short Term ◽  
Health States ◽  
Life Years ◽  
Term Data

Abstract Background Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of treatment. Nusinersen is a disease-modifying therapy for patients with SMA. Objective The aim of this study was to estimate the cost-effectiveness of nusinersen compared to best supportive care (BSC) in patients diagnosed with infantile-onset SMA in the US. Methods A de novo economic model was developed with the following health states: “permanent ventilation”, “not sitting”, “sitting”, “walking”, and “death”. Short-term data were sourced from the pivotal clinical trials and studies of nusinersen (ENDEAR and SHINE). Motor function milestones achieved at the end of follow-up in the clinical trials were assumed to be sustained until death. Mortality risks were based on survival modelling of relevant published Kaplan–Meier data. Costs, life years (LYs), and quality-adjusted life years (QALYs) were discounted at 3% per annum, and the analyses were performed from a US health care sector perspective. Scenario analyses and sensitivity analyses were conducted to assess the robustness of the results to key parameters. Results In our base-case analysis, nusinersen treatment achieves greater QALYs and more LYs (3.24 and 7.64, respectively) compared with BSC (0.46 QALYs and 2.40 LYs, respectively), resulting in an incremental cost per QALY gained of approximately $1,112,000 and an incremental cost per LY gained of $590,000 for nusinersen compared to BSC. The incremental cost effectiveness ratios did not fall below $990,000 per QALY gained in scenario and sensitivity analyses. Results were most sensitive to the length of survival, background health care costs, and utility in the “not sitting” and “sitting” health states. Conclusions The estimated incremental cost-effectiveness of nusinersen from a US health care sector perspective exceeded traditional cost-effectiveness thresholds. Cost-effectiveness was dependent on assumptions made regarding survival, costs, utilities, and whether the motor function milestones were sustained over lifetime. Given the relatively short-term effectiveness data available for the treatment, a registry to collect long-term data of infantile-onset SMA patients is recommended.

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