PP08 Health Technology Assessment Of Autologous Chondrocyte Implantation

2018 ◽  
Vol 34 (S1) ◽  
pp. 69-69
Author(s):  
Hema Mistry ◽  
Martin Connock ◽  
Pamela Royle ◽  
Norman Waugh

Introduction:Microfracture (MF) has been the main intervention in symptomatic articular cartilage knee defects. Autologous chondrocyte implantation (ACI) has looked promising, but was not recommended by the UK National Institute for Health and Care Excellence (NICE) in 2015 due to the short-term follow-up data from trials.Methods:Most long-term data comes from observational studies. We provided new unpublished analyses to NICE based on survival data of these studies, with appropriate caveats. They included: a large ACI study by Nawaz with useful subgroup data by osteoarthritis Kellgren-Lawrence stage and previous repair attempts; a very large MF study by Layton, and a small RCT by Knutsen indicating MF was as ‘good’ as ACI. A Markov model explored the cost-effectiveness of ACI vs. MF. Different scenarios were explored: ACI or MF as a first procedure, followed by ACI or MF in those needing a second repair. A NHS England perspective was adopted. Health outcomes were expressed as quality-adjusted life-years (QALYs).Results:The revised base-case analysis, used a list price of £16,000 (EUR 17,380 in 2013 prices) for cells, used ACI failure data from Nawaz with no previous procedures for ACI, and pooled MF failure data from two studies-Saris and Knutsen. ACI was more expensive but provided more QALYs. The incremental cost-effectiveness ratio comparing ACI then MF with MF then ACI was £8,000 (EUR 8,690) per QALY. Various sensitivity analyses were conducted assuming a threshold of £20,000 (EUR 21,730) per QALY: previous repair attempts reduced success of ACI (£22,000 (EUR 23,900) per QALY); reducing cell costs, ACI improved its cost-effectiveness; and limiting intervention to patients with higher Kellgren-Lawrence score did not appear cost-effectiveness.Conclusions:The final NICE guidance published in October 2017 approved the use of ACI for patients who had no previous knee repairs, for people with minimal osteoarthritic damage to the knee, and for people with articular defects of over 2cm2.

2021 ◽  
Vol 9 (10_suppl5) ◽  
pp. 2325967121S0029
Author(s):  
Drake LeBrun ◽  
Jake Feingold ◽  
Stephanie Swenson-Buza ◽  
Simone Gruber ◽  
Elizabeth Dennis ◽  
...  

Objectives: Treatment options for articular cartilage lesions of the patella have evolved over the past several years due to the development of novel cell-based cartilage restoration techniques, including particulated juvenile allograft cartilage (PJAC) and matrix-induced autologous chondrocyte implantation (MACI). The objective of this study was to evaluate the cost -effectiveness of these modalities in the management of patellar cartilage defects. Methods: A Markov state-transition model was utilized to evaluate the cost-effectiveness of three strategies for patients with patellar chondral lesions: (1) nonoperative management, (2) PJAC, and (3) MACI. Probabilities, health utilities, and costs of surgical procedures and rehabilitation protocols were derived from institutional data and literature review. Effectiveness was assessed using quality-adjusted life-year (QALY). Cost-effectiveness was evaluated from societal and payer perspectives over a 15-year time horizon. The principal outcome measure was the incremental cost-effectiveness ratio (ICER). Sensitivity analyses were performed on pertinent model parameters to assess their effect on base case conclusions. Results: From a societal perspective, nonoperative management, PJAC, and MACI cost $4,140, $52,683, and $83,073 respectively. Nonoperative management, PJAC, and MACI were associated with 4.91, 7.07, and 7.79 QALYs gained, respectively. Therefore, PJAC and MACI were cost-effective relative to nonoperative management (ICERs $22,527/QALY and $27,456/QALY, respectively; Figure 1). Although MACI was more cost-effective than PJAC in the base case, this was strongly sensitive to the estimated probabilities of full versus intermediate benefit following PJAC and MACI (Table 1). If the probabilities of full and intermediate benefit following PJAC were assumed to be the same as those following MACI (i.e., PJAC and MACI were equally effective), then PJAC dominated MACI by being cheaper and more effective. At a $100,000/QALY willingness-to-pay threshold, MACI, PJAC, and nonoperative management were the preferred strategies in 63%, 33%, and 4% of the Monte Carlo probabilistic sensitivity analyses, respectively (Figures 2 and 3). Similar results were seen from a payer perspective. Conclusions: In the management of symptomatic patellar cartilage defects, PJAC and MACI were both cost-effective compared to nonoperative treatment in the management of symptomatic patellar cartilage defects; however, MACI was the preferred strategy in our base-case analysis. The cost-effectiveness of PJAC compared to MACI depended heavily on the probability of achieving full versus intermediate benefit after PJAC and MACI.


10.36469/9870 ◽  
2013 ◽  
Vol 1 (3) ◽  
pp. 239-253 ◽  
Author(s):  
Jona T. Stahmeyer ◽  
Svenja Schauer ◽  
Siegbert Rossol ◽  
Hans Heinrich Wedemeyer ◽  
Daniel Wirth ◽  
...  

Background: About 400,000-500,000 people are infected with hepatitis C in Germany. Long-term consequences are the development of liver cirrhosis and hepatocellular carcinoma. The introduction of first generation protease inhibitors has significantly improved the treatment of hepatitis C genotype 1 patients. The aim of the study was to assess the cost-effectiveness of triple therapy with telaprevir in Germany. Methods: We used a Markov model on disease progression and natural history to assess the cost-effectiveness of triple therapy with telaprevir compared to standard treatment with pegylated interferon and ribavirin. Model structure and inputs were discussed with clinical experts. Deterministic and probabilistic sensitivity analyses were performed to verify the robustness of results. Results: The base-case analyses shows that triple therapy results in higher costs (untreated patients: €48,446 vs. €30,691; previously treated patients: €63,228 vs. €48,603) and better outcomes (untreated patients: 16.85 qualily of life years [QALYs] vs. 15.97 QALYs; previously treated patients: 14.16 QALYs vs. 12.89 QALYs). The incremental cost-effectiveness ratio (ICER) was €20,131 per QALY and €30,567 per life year gained (LYG) for previously untreated patients. ICER in treatment experienced patients was €7,664 per QALY for relapse patients, €12,506 per QALY for partial responders and €28,429 per QALY for null responders. Results were robust in sensitivity analyses. Conclusion: Although triple therapy with telaprevir leads to additional costs, there is a high probability of being cost-effective for different thresholds. This health economic analysis makes an important contribution to current debates on cost savings and efficient resource allocation in the German healthcare sector.


2021 ◽  
Author(s):  
Xueyan Luo ◽  
Wei Xu ◽  
Quan Yuan ◽  
Han Lai ◽  
Chunji Huang

BACKGROUND Mobile health (mhealth) technology is increasingly used in disease management. Using mhealth tools to integrate and streamline care was found to improve atrial fibrillation (AF) patients’ clinical outcomes. OBJECTIVE This study aimed to investigate the potential clinical and health economic outcomes of mhealth-based integrated care for AF from the perspective of a public healthcare provider in China. METHODS A Markov model was designed to compare outcomes of mhealth-based care and usual care in a hypothetical cohort of AF patients in China. The time horizon was 30 years with monthly cycles. Model outcomes measured were direct medical cost, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were conducted to examine the robustness of base-case results. RESULTS In the base-case analysis, mhealth-based care gained higher QALYs of 0.0818 with an incurred cost of USD1,778. Using USD33,438 per QALY (three times gross domestic product) as the willingness-to-pay threshold, mhealth-based care was cost-effective, with an ICER of USD21,739 per QALY. The one-way sensitivity analysis found compliance to mhealth-based care had the greatest impact on the ICER. In probabilistic sensitivity analysis, mhealth-based care was accepted as cost-effective in 80.91% of 10,000 iterations. CONCLUSIONS This study suggested that the use of mhealth technology in streamlining and integrating care for AF patients was cost-effective in China.


Nutrients ◽  
2019 ◽  
Vol 11 (6) ◽  
pp. 1235
Author(s):  
Anita E. Gandola ◽  
Livia Dainelli ◽  
Diane Zimmermann ◽  
Maznah Dahlui ◽  
Patrick Detzel

This study evaluated the cost-effectiveness of the consumption of a milk powder product fortified with potassium (+1050.28 mg/day) and phytosterols (+1200 mg/day) to lower systolic blood pressure and low-density lipoprotein cholesterol, respectively, and, therefore, the risk of myocardial infarction (MI) and stroke among the 35–75-year-old population in Malaysia. A Markov model was created against a do-nothing option, from a governmental perspective, and with a time horizon of 40 years. Different data sources, encompassing clinical studies, practice guidelines, grey literature, and statistical yearbooks, were used. Sensitivity analyses were performed to evaluate the impact of uncertainty on the base case estimates. With an incremental cost-effectiveness ratio equal to international dollars (int$) 22,518.03 per quality-adjusted life-years gained, the intervention can be classified as very cost-effective. If adopted nationwide, it would help prevent at least 13,400 MIs, 30,500 strokes, and more than 10,600 and 17,100 MI- and stroke-related deaths. The discounted cost savings generated for the health care system by those who consume the fortified milk powder would amount to int$8.1 per person, corresponding to 0.7% of the total yearly health expenditure per capita. Sensitivity analyses confirmed the robustness of the results. Together with other preventive interventions, the consumption of milk powder fortified with potassium and phytosterols represents a cost-effective strategy to attenuate the rapid increase in cardiovascular burden in Malaysia.


2019 ◽  
Vol 40 (Supplement_1) ◽  
Author(s):  
P Lindgren ◽  
E Hagstrom ◽  
B Van Hout ◽  
G Villa ◽  
M Urbich ◽  
...  

Abstract Background/Introduction Elevated low-density lipoprotein cholesterol (LDL-C) is one of the most important modifiable risk factors for atherosclerotic cardiovascular disease (ASCVD). Evolocumab, a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, is indicated for the reduction of CV risk by lowering LDL-C. Purpose Assess the cost-effectiveness of evolocumab added to standard of care (SoC), maximally tolerated lipid-lowering treatment, in two patient populations for which evolocumab is reimbursed in Sweden: (1) patients with ASCVD with LDL-C ≥2.5 mmol/L on SoC, and (2) heterozygous familial hypercholesterolemia (HeFH) patients without ASCVD with LDL-C ≥3.0 mmol/L on SoC. Methods A previously published Markov model was adapted to the Swedish context. The model incorporated real-world CV event (CVE) rates (myocardial infarction, ischemic stroke and CV death). In patients with ASCVD, a CVE rate of 6.3/100 patient-years was obtained from Swedish national registries. In HeFH patients without ASCVD, a CVE rate of 4.5/100 patient-years was obtained from a national screening program in the Netherlands. ASCVD patient characteristics were obtained from Swedish national registries. HeFH patient characteristics were obtained from the RUTHERFORD-2 clinical trial. The model used an evolocumab LDL-C reduction of 59%, as observed in the FOURIER CV outcomes clinical trial, and the relationship between LDL-C lowering and CVE reduction from the Cholesterol Treatment Trialists' Collaboration (CTTC) 2010 meta-analysis (base case) or FOURIER (scenario). An annual evolocumab list price (before discount) of SEK 48,759 [€ 4,632] (1 SEK = € 0.095) was considered. Costs and health outcomes were evaluated over a lifetime horizon from a societal perspective. Results In the base case, for patients with ASCVD with LDL-C ≥2.5 mmol/L on SoC, the addition of evolocumab was associated with: a 0.30 reduction in the lifetime per-patient CVE rate, increased costs of SEK 413,835 and increased quality-adjusted life years (QALY) of 0.67, yielding an incremental cost-effectiveness ratio (ICER) of SEK 615,393 [€ 58,462] per QALY gained. In the base case, for HeFH patients without ASCVD with LDL-C ≥3.0 mmol/L on SoC, the addition of evolocumab was associated with: a 0.57 reduction in the lifetime per-patient CVE rate, increased costs of SEK 701,200 and increased QALY of 1.39, yielding an ICER of SEK 503,710 [€ 47,852] per QALY gained. In the scenario analysis, ICER were SEK 539,846 [€ 51,285] and SEK 462,961 [€ 43,981] per QALY, respectively. Conclusions These results indicate the addition of evolocumab to SoC may be considered cost-effective in Sweden. Indeed, based on these data, the Swedish Dental and Pharmaceutical Benefits Agency (TLV) recently granted expanded reimbursement for evolocumab (submission 2138/2018), which led to a positive national recommendation in the patient populations described above. Acknowledgement/Funding This study was sponsored by Amgen.


2019 ◽  
Vol 21 (Supplement_3) ◽  
pp. iii68-iii69
Author(s):  
X Armoiry ◽  
P Auguste ◽  
C Dussart ◽  
J Guyotat ◽  
M Connock

Abstract BACKGROUND The addition of novel therapy “Tumor-Treating fields” (TTF) to standard radio-chemotherapy with Temozolomide (TMZ) has recently shown superiority over conventional TMZ regimen in patients with glioblastoma. Despite the clinical benefit of TTF, there is a strong concern regarding the cost of this new treatment. A first cost-effectiveness analysis, which was published in 2016, was based on effectiveness outcomes from an interim analysis of the pivotal trial and used a “standard” Markov model. Here, we aimed to update the cost-effectiveness evaluation using a partitioned survival model design and using the latest effectiveness data. MATERIAL AND METHODS A partitioned survival model was developed with three mutually exclusive health states: stable disease, progressive disease, and dead. Parametric models were fitted to the Kaplan-Meier data for overall and progression-free survival. These generated clinically plausible extrapolations beyond the observed data. The perspective of the French national health insurance was adopted and the time horizon was 20 years. Base case results were expressed as cost/life-years (LY) gained (LYG). Secondary analyses were undertaken, with the results presented as cost/per quality adjusted life years (QALY) gained. Last, we undertook deterministic and probabilistic sensitivity analyses. RESULTS After applying 4% annual discounting of benefits and costs, the base case model generated incremental benefit of 0.507 LY at a incremental cost of €258,695 yielding an incremental cost effectiveness ratio (ICER) of €510,273 / LYG. Secondary analyses yielded an ICER of €667,173/QALY. Sensitivity analyses and bootstrapping methods showed the model was relatively robust. The model was sensitive to TTF device costs and the parametric model fitted to the Kaplan-Meier data for overall survival. The cost-effectiveness acceptability curve showed TTF has 0% of being cost-effective under conventional thresholds. CONCLUSION Using a partitioned survival model, uprated costs and more mature survival outcomes, TTF when compared to standard radio-chemotherapy with TMZ is not likely to be cost-effective. This has major implications in terms of access of newly eligible patients


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4962-4962
Author(s):  
Khalid El Ouagari ◽  
Kristen Migliaccio-Walle ◽  
Helen Lau ◽  
Duygu Bozkaya

Abstract Abstract 4962 Introduction: Guidelines for the treatment of MDS recommend iron chelation therapy (ICT) in iron-overloaded lower-risk patients with MDS and candidates for stem cell transplantation. In particular, recent reports indicate that ICT may improve overall survival (OS) in transfusion-dependent patients with low or intermediate-1 (int-1) MDS as per international prognostic scoring system (IPSS) criteria. Deferasirox is a once-daily oral chelator, with easy administration and potentially better compliance. The goal of this study is to evaluate the cost-effectiveness of deferasirox compared to receiving no chelation therapy in transfusion-dependent patients with lower-risk MDS from a Canadian healthcare system perspective. Methods: A Markov model was developed to evaluate the cost-effectiveness of deferasirox compared to receiving no chelation therapy in transfusion-dependent patients with lower-risk (eg, IPSS low or int-1) MDS. The data used in the model were obtained from published or presented studies. Model outcomes, including life years (LY) gained, quality-adjusted life years (QALYs) gained, developing complications of iron overload, progressing to acute myeloid leukemia (AML), death, and direct medical costs of ICT, transfusion, complications and AML, were estimated for each treatment group based on a simulation of 1000 patient lives. Finally, incremental cost-effectiveness ratios (ICER) were calculated as the ratio of total medical costs to LY and QALY gains. Extensive one-way sensitivity analyses were performed to examine the effects of changes in key model parameters. Probabilistic sensitivity analyses were also performed. The outcomes of the model were evaluated over a 20-year time frame and discounted annually at the rate of 5%. Costs are reported in 2009 Canadian dollars (CAD$). Results: Under base case assumptions, patients receiving deferasirox were less likely to progress to cardiac disease, AML, and death compared to patients receiving no chelation therapy. Adding deferasirox was projected to increase OS by 4.46 years (undiscounted); discounting for time, OS was projected to be increased by 2.93 years. Furthermore, undiscounted QALYs were increased by 4.20 years and discounted QALYs, by 2.99 years. The clinical benefits of deferasirox are obtained at an additional expected discounted total lifetime cost of CAD$185,429. The incremental cost-effectiveness ratios were therefore estimated to be CAD$62,001/QALY gained and CAD$63,286/LY saved. Deterministic sensitivity analyses showed the base case results to be robust with respect to variations in assumptions and estimates. The cost-effectiveness acceptability curve shows that deferasirox was preferred to no treatment in 96% of simulations when the willingness to pay for a QALY was CAD$100,000. Conclusion: The results of our analysis indicate that deferasirox offers a cost-effective treatment option for patients with lower-risk MDS as the ICER is within the thresholds that are considered acceptable (ie, $50,000 to $100,000 per QALY gained), from a Canadian healthcare system perspective. Additional clinical studies are ongoing to evaluate event-free survival with deferasirox in patients with lower-risk MDS and transfusional iron overload. Disclosures: El Ouagari: Novartis: Employment. Migliaccio-Walle: Novartis: Research Funding. Lau: Novartis: Employment. Bozkaya: Novartis: Research Funding.


2015 ◽  
Vol 36 (4) ◽  
pp. 438-444 ◽  
Author(s):  
Raghu U. Varier ◽  
Eman Biltaji ◽  
Kenneth J. Smith ◽  
Mark S. Roberts ◽  
M. Kyle Jensen ◽  
...  

OBJECTIVEClostridium difficile infection (CDI) places a high burden on the US healthcare system. Recurrent CDI (RCDI) occurs frequently. Recently proposed guidelines from the American College of Gastroenterology (ACG) and the American Gastroenterology Association (AGA) include fecal microbiota transplantation (FMT) as a therapeutic option for RCDI. The purpose of this study was to estimate the cost-effectiveness of FMT compared with vancomycin for the treatment of RCDI in adults, specifically following guidelines proposed by the ACG and AGA.DESIGNWe constructed a decision-analytic computer simulation using inputs from the published literature to compare the standard approach using tapered vancomycin to FMT for RCDI from the third-party payer perspective. Our effectiveness measure was quality-adjusted life years (QALYs). Because simulated patients were followed for 90 days, discounting was not necessary. One-way and probabilistic sensitivity analyses were performed.RESULTSBase-case analysis showed that FMT was less costly ($1,669 vs $3,788) and more effective (0.242 QALYs vs 0.235 QALYs) than vancomycin for RCDI. One-way sensitivity analyses showed that FMT was the dominant strategy (both less expensive and more effective) if cure rates for FMT and vancomycin were ≥70% and <91%, respectively, and if the cost of FMT was <$3,206. Probabilistic sensitivity analysis, varying all parameters simultaneously, showed that FMT was the dominant strategy over 10, 000 second-order Monte Carlo simulations.CONCLUSIONSOur results suggest that FMT may be a cost-saving intervention in managing RCDI. Implementation of FMT for RCDI may help decrease the economic burden to the healthcare system.Infect Control Hosp Epidemiol 2014;00(0): 1–7


2002 ◽  
Vol 146 (3) ◽  
pp. 283-294 ◽  
Author(s):  
GM Vidal-Trecan ◽  
JE Stahl ◽  
I Durand-Zaleski

OBJECTIVE: To examine the cost-effectiveness of therapeutic strategies for patients with toxic thyroid adenoma. DESIGN: A decision analytic model was used to examine strategies, including thyroid lobectomy after a 3-month course of antithyroid drugs (ATDs), radioactive iodine (RAI), and lifelong ATDs followed by either RAI (ATD-RAI) or surgery (ATD-surgery) in patients suffering severe drug reactions. METHODS: Outcomes were measured in quality-adjusted life years. Data on the prevalence of co-incident thyroid cancer, complications and treatment efficacies were derived from a systematic review of the literature (1966-2000). Costs were examined from the health care system perspective. Costs and effectiveness were examined at their present values. Discounting (3% per year), variations of major cost components, and every variable for which disagreements exist among studies or expert opinion were examined by sensitivity analyses. RESULTS: For a 40-year-old woman, surgery was both the most effective and the least costly strategy (Euro 1391),while ATD-RAI cost the most (Euro 5760). RAI was more effective than surgery if surgical mortality exceeded 0.6% (base-case 0.001%). RAI become less costly for women of more than 72 years (more than 66 in discounted analyses). For women of 85, ATD-RAI may be more effective than RAI and have an inexpensive marginal cost-effectiveness ratio (Euro 4975) if lifelong follow-up results in no decrement in quality of life. CONCLUSIONS: Age, surgical mortality, therapeutic costs and patient preference must all be considered in choosing an appropriate therapy.


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