Cost Effectiveness of Hematopoietic Stem Cell Transplantation Compared with Transfusion Chelation for Treatment of Thalassemia Major

Abstract

Hematopoietic stem cell transplantation (HSCT) is the only cure for thalassemia major (TM), which inflicts a significant 1-time cost. Hence, it is important to explore the cost effectiveness of HSCT versus lifelong regular transfusion-chelation (TC) therapy. This study was undertaken to estimate incremental cost per quality-adjusted life-year (QALY) gained with the intervention group HSCT, and the comparator group TC, in TM patients. A combination of decision tree and Markov model was used for analysis. A hospital database, supplemented with a review of published literature, was used to derive input parameters for the model. A lifetime study horizon was used and future costs and consequences were discounted at 3%. Results are presented using societal perspective. Incremental cost per QALY gained with use of HSCT as compared with TC was 64,096 (US$986) in case of matched related donor (MRD) and 1,67,657 (US$2579) in case of a matched unrelated donor transplantation. The probability of MRD transplant to be cost effective at the willingness to pay threshold of Indian per capita gross domestic product is 94%. HSCT is a long-term value for money intervention that is highly cost effective and its long-term clinical and economic benefits outweigh those of TC.

Cost-Effectiveness of Therapeutic Use of Safety-Engineered Syringes in Healthcare Facilities in India

“…A recent systematic review carried out for assessing the quality of economic evaluations in India reveals that the majority of studies used a one-time GDP per capita as the cost-effectiveness threshold [74]. Moreover, in the Indian context, recent cost-effectiveness studies used per capita GDP as the threshold [75][76][77][78]. Lastly, the methodological guidelines of India's health technology assessment (HTA) agency recommend the use of a one-time per capita GDP as the criterion for costeffectiveness [79].…”

Access to hematopoietic stem-cell transplantation in India

“…These differences necessitate cost-effectiveness analyses to guide policy makers to allocate resources for HSCT in India. Data from India on such analysis for a few disorders[2021] are also available.…”

Can any new thalassemia therapy achieve a better than 97.4% cure rate?

“…Although costs significantly vary among countries, the estimated cost of an allogeneic HCT is almost invariably much lower than that of conventional medical therapy. [16][17][18][19] Although the economic value of the gene therapy product has been estimated, 20 the proposed price 21 is clearly above the available resources in many countries, at least considering the present reimbursement modalities. A single study comparing HCT vs gene therapy costs has been published, but the transplant costs reported are very different to those reported in other countries.…”

“…A single study comparing HCT vs gene therapy costs has been published, but the transplant costs reported are very different to those reported in other countries. [16][17][18][19] For the above discussed issues and the gene therapy product production limitation a gene therapy access priority algorithm and prolonged documented follow up are urgently needed. 22 The present multicenter study by Yuelin He and co-workers 5 with its large number of patients and outstanding results offers further contribution to this debate and stands, in certain conditions, as the benchmark for next coming new therapies.…”

“…Cost issue and resources availability should be taken in consideration. Although costs significantly vary among countries, the estimated cost of an allogeneic HCT is almost invariably much lower than that of conventional medical therapy 16‐19 …”