The Cost-Effectiveness of Stem Cell Transplantations from Unrelated Donors in Adult Patients with Acute Leukemia

Costa, Vânia; McGregor, Maurice; Laneuville, Pierre; Brophy, James M.

doi:10.1111/j.1524-4733.2007.00180.x

Cited by 26 publications

(42 citation statements)

References 45 publications

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“…In another study, conducted by Esperou et al in 2004 on 85 patients from five transplant centers, the mean cost for the first 6 months after transplantation, restricted to a specific protocol, was € 76,000. 35 The average cost for initial hospitalization for UCBT in Canada 22 and in the USA 21 was consistent with that in our series. However in both studies, only costs up to 100 days were included whereas in other studies costs were considered over a much longer period.…”

Section: Discussionsupporting

confidence: 77%

“…21,22 The acquisition cost of two cord blood units is one of the main limitations of using dUCBT. The median cost for the first 100 days after UCBT was estimated to be around US$ 80,407 in Canada in 2007 22 and around US$ 137,564 in the USA in 2009, 21 not including the cost for the donor search. In a more recent study from the Erasmus University, 23 the cost of UCBT over 1 year was estimated to be around € 250,000.…”

Section: Introductionmentioning

confidence: 99%

“…The role of complications in increasing costs of transplantation has been described previously. In 2007, Costa et al 22 reported a cost increment of US$ 2,716 for chronic GVHD and US$ 10,576 for relapse. The additional cost for chronic GVHD reported by Esperou et al 35 was much higher, at around € 20,000, but also included costs due to transplant-related complications and infections.…”

mentioning

confidence: 99%

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Cost-effectiveness and clinical outcomes of double versus single cord blood transplantation in adults with acute leukemia in France

Labopin¹,

Ruggeri²,

Gorin³

et al. 2013

Haematologica

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IntroductionAllogeneic stem cell transplantation (HSCT) is effective treatment for patients with various hematologic disorders. It is, however, a complex, resource-intense and costly procedure. [1][2][3][4] The cost of HSCT has been previously evaluated, mainly in the setting of HLA identical sibling 5 and matched unrelated donor transplants.The first studies on the cost-efficacy of HSCT compared allogeneic HSCT to chemotherapy in patients with acute leukemia. 7,8 Despite the high cost of HSCT, the results demonstrated the advantages of the procedure due to the impact on long-term survival adjusted to the quality of life. In the first published studies, the estimated costs for HSCT varied greatly (Online Supplementary Table S1) depending on the country in which they were performed, type of donor, transplant center procedures and year of transplantation. 5,7,[9][10][11] A more recent comparative study of autologous and allogeneic HSCT for patients transplanted for hematologic malignancies in the USA estimated a 100-day total cost of US$ 203,026 for allogeneic HSCT. 12 Unrelated donor cord blood transplantation (UCBT) has become a widely accepted transplant modality in the absence of an HLA-matched donor. [13][14][15][16] However, the delay of engraftment and the increased risk of graft failure remain problems in adults transplanted with a single cord blood unit. The possibility of using two cord blood units has extended the use of UCBT to patients for whom a single unit containing a minimum of 2.5x10 7 /kg total nucleated cells is not available. [17][18][19] Studies have been performed comparing outcomes after single (s) UCBT and double (d) UCBT,20 but, none focused on a homo- open-access paper. doi:10.3324/haematol.2013.092254 The online version of this article has a Supplementary Appendix. Manuscript received on May 27, 2013. Manuscript accepted on October 14, 2013 Double cord blood transplantation extends the use of cord blood to adults for whom a single unit is not available, but the procedure is limited by its cost. To evaluate outcomes and cost-effectiveness of double compared to single cord blood transplantation, we analyzed 134 transplants in adults with acute leukemia in first remission. Transplants were performed in France with reduced intensity or myeloablative conditioning regimens. Costs were estimated from donor search to 1 year after transplantation. A Markov decision analysis model was used to calculate qualityadjusted life-years and cost-effectiveness ratio within 4 years. The overall survival at 2 years after single and double cord blood transplants was 42% versus 62%, respectively (P=0.03), while the leukemia-free-survival was 33% versus 53%, respectively (P=0.03). The relapse rate was 21% after double transplants and 42% after a single transplant (P=0.006). No difference was observed for non-relapse mortality or chronic graft-versus-host-disease. The estimated costs up to 1 year after reduced intensity conditioning for single and double cord blood transplantation were € 165,253 and €191,...

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Section: Discussionsupporting

confidence: 77%

Section: Introductionmentioning

confidence: 99%

mentioning

confidence: 99%

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Cost-effectiveness and clinical outcomes of double versus single cord blood transplantation in adults with acute leukemia in France

Labopin¹,

Ruggeri²,

Gorin³

et al. 2013

Haematologica

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“…The pragmatic search using Google and Google Scholar found two papers deemed relevant to the primary aim of the review. 182,183 Costa et al 182 conducted a cost-effectiveness evaluation of unrelated stem cell transplantation for adults with acute leukaemia (ALL and acute myeloid leukaemia) structured around a 20-year Markov model. The study concluded that the two forms of transplantation considered (cord blood and bone marrow/peripheral blood stem cells) were cost-effective compared with no transplantation.…”

Section: Resultsmentioning

confidence: 99%

“…The 'cure point' was assumed to represent the time at which patients are assumed to no longer be at risk of disease relapse. The study by Costa et al 182 assumed that at 5 years post transplantation the patient will be free of any procedural mortality…”

Section: Implications For Model Conceptualisationmentioning

confidence: 99%

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

104

143

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BackgroundThe National Institute for Health and Care Excellence (NICE) commissioned a ‘mock technology appraisal’ to assess whether changes to its methods and processes are needed. This report presents the findings of independent research commissioned to inform this appraisal and the deliberations of a panel convened by NICE to evaluate the mock appraisal.MethodsOur research included reviews to identify issues, analysis methods and conceptual differences and the relevance of alternative decision frameworks, alongside the development of an exemplar case study of chimeric antigen receptor (CAR) T-cell therapy for treating acute lymphoblastic leukaemia.ResultsAn assessment of previous evaluations of regenerative medicines found that, although there were a number of evidential challenges, none was unique to regenerative medicines or was beyond the scope of existing methods used to conceptualise decision uncertainty. Regarding the clinical evidence for regenerative medicines, the issues were those associated with a limited evidence base but were not unique to regenerative medicines: small non-randomised studies, high variation in response and the intervention subject to continuing development. The relative treatment effects generated from single-arm trials are likely to be optimistic unless it is certain that the historical data have accurately estimated the efficacy of the control agent. Pivotal trials may use surrogate end points, which, on average, overestimate treatment effects. To reduce overall uncertainty, multivariate meta-analysis of all available data should be considered. Incorporating indirectly relevant but more reliable (more mature) data into the analysis can also be considered; such data may become available as a result of the evolving regulatory pathways being developed by the European Medicines Agency. For the exemplar case of CAR T-cell therapy, target product profiles (TPPs) were developed, which considered the ‘curative’ and ‘bridging to stem-cell transplantation’ treatment approaches separately. Within each TPP, three ‘hypothetical’ evidence sets (minimum, intermediate and mature) were generated to simulate the impact of alternative levels of precision and maturity in the clinical evidence. Subsequent assessments of cost-effectiveness were undertaken, employing the existing NICE reference case alongside additional analyses suggested within alternative frameworks. The additional exploratory analyses were undertaken to demonstrate how assessments of cost-effectiveness and uncertainty could be impacted by alternative managed entry agreements (MEAs), including price discounts, performance-related schemes and technology leasing. The panel deliberated on the range of TPPs, evidence sets and MEAs, commenting on the likely recommendations for each scenario. The panel discussed the challenges associated with the exemplar and regenerative medicines more broadly, focusing on the need for a robust quantification of the level of uncertainty in the cost-effective estimates and the potential value of MEAs in limiting the exposure of the NHS to high upfront costs and loss associated with a wrong decision.ConclusionsIt is to be expected that there will be a significant level of uncertainty in determining the clinical effectiveness of regenerative medicines and their long-term costs and benefits, but the existing methods available to estimate the implications of this uncertainty are sufficient. The use of risk sharing and MEAs between the NHS and manufacturers of regenerative medicines should be investigated further.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Cost-effectiveness of Tisagenlecleucel vs Standard Care in High-risk Relapsed Pediatric Acute Lymphoblastic Leukemia in Canada

et al. 2020

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IMPORTANCE Tisagenlecleucel, a chimeric antigen receptor T-cell therapy for relapsed or refractory pediatric acute lymphoblastic leukemia, has been approved for use in multiple jurisdictions. The public list price is US $475 000, or more than CaD $600 000. Assessing the cost-effectiveness of tisagenlecleucel is necessary to inform policy makers on the economic value of this treatment.OBJECTIVE To assess the value for money of tisagenlecleucel compared with current standard care for tisagenlecleucel-eligible pediatric patients with acute lymphoblastic leukemia under unknown long-term effectiveness. DESIGN, SETTING, AND PARTICIPANTSA cost-utility analysis of tisagenlecleucel compared with current standard care using a Canadian population-based registry of pediatric patients with acute lymphoblastic leukemia was performed. Results from 3 pooled single-arm tisagenlecleucel clinical trials and a provincial pediatric cancer registry were combined to create treatment and control arms, respectively. The population-based control arm consisted of patients meeting clinical trial inclusion and exclusion criteria, starting at second relapse. Multistate and individual-level simulation modeling were combined to predict patient lifetime health trajectories by treatment strategy. Tisagenlecleucel efficacy was modeled across long-term cure rates, from 10% to 40%, to account for limited information on its long-term effectiveness. Uncertainty was tested with 1-way and probabilistic sensitivity analysis. Data were collected in September 2017, and analysis began in December 2017.EXPOSURES Tisagenlecleucel compared with current standard care for tisagenlecleucel-eligible patients.MAIN OUTCOMES AND MEASURES Relative health care costs, survival gains, and quality-adjusted life-years (QALYs) between tisagenlecleucel and current standard care. RESULTSThe treatment and control arms were modeled on 192 and 118 patients, respectively. The mean (SD) age of control individuals was 10 (4.25) years, and the mean (SD) age of the pooled clinical trial sample was 11 (6) years. The control individuals had 78 boys (66%), and the pooled clinical trial sample had 102 boys (53%).

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The Cost-Effectiveness of Stem Cell Transplantations from Unrelated Donors in Adult Patients with Acute Leukemia

Cited by 26 publications

References 45 publications

Cost-effectiveness and clinical outcomes of double versus single cord blood transplantation in adults with acute leukemia in France

Cost-effectiveness and clinical outcomes of double versus single cord blood transplantation in adults with acute leukemia in France

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Cost-effectiveness of Tisagenlecleucel vs Standard Care in High-risk Relapsed Pediatric Acute Lymphoblastic Leukemia in Canada

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