A cost–utility analysis of thrombopoietin receptor agonists for treating pediatric immune thrombocytopenia purpura after failure of first-line therapies
Huimin Du
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, China
NHC Key Lab of Health Economics and Policy Research (Shandong University), Jinan, Shandong, China
Search for more papers by this authorJiamin Wang
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, China
NHC Key Lab of Health Economics and Policy Research (Shandong University), Jinan, Shandong, China
Search for more papers by this authorJoel Livingston
Department of Pediatrics, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada
Department of Pediatrics, University of Alberta and Stollery Children's Hospital, Edmonton, Alberta, Canada
Search for more papers by this authorZiyad Alrajhi
Department of Pediatrics, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada
King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
Search for more papers by this authorMelanie Kirby-Allen
Department of Pediatrics, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada
Search for more papers by this authorBrian Chan
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
KITE Research Institute, Toronto Rehabilitation Institute - University Health Network, Toronto, Ontario, Canada
Search for more papers by this authorRebecca Hancock-Howard
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorCorresponding Author
Peter C. Coyte
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Correspondence
Peter C. Coyte, Institute of Health Policy, Management and Evaluation, University of Toronto, Health Sciences Bldg, 155 College St, Suite 425, Toronto, ON, M5T 3M6, Canada.
Email: [email protected]
Search for more papers by this authorHuimin Du
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, China
NHC Key Lab of Health Economics and Policy Research (Shandong University), Jinan, Shandong, China
Search for more papers by this authorJiamin Wang
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, China
NHC Key Lab of Health Economics and Policy Research (Shandong University), Jinan, Shandong, China
Search for more papers by this authorJoel Livingston
Department of Pediatrics, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada
Department of Pediatrics, University of Alberta and Stollery Children's Hospital, Edmonton, Alberta, Canada
Search for more papers by this authorZiyad Alrajhi
Department of Pediatrics, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada
King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
Search for more papers by this authorMelanie Kirby-Allen
Department of Pediatrics, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada
Search for more papers by this authorBrian Chan
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
KITE Research Institute, Toronto Rehabilitation Institute - University Health Network, Toronto, Ontario, Canada
Search for more papers by this authorRebecca Hancock-Howard
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorCorresponding Author
Peter C. Coyte
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
Correspondence
Peter C. Coyte, Institute of Health Policy, Management and Evaluation, University of Toronto, Health Sciences Bldg, 155 College St, Suite 425, Toronto, ON, M5T 3M6, Canada.
Email: [email protected]
Search for more papers by this authorHuimin Du and Jiamin Wang are joint first authors.
Abstract
Background
Thrombopoietin receptor agonists (TPO-RAs) have emerged as a recommended treatment for children with persistent and/or chronic immune thrombocytopenic purpura (ITP). The purpose of this study was to evaluate the cost-effectiveness of TPO-RAs relative to treatment without TPO-RAs (non-TPO-RAs/usual care) for ITP in children who do not respond to first-line therapy and in whom splenectomy is not recommended in Ontario, Canada, from a hospital payer perspective.
Procedure
A 2-year Markov model with an embedded decision tree was used. Data on medications used, dose, response rate, bleeding, and emergency treatment events were collected from the Hospital for Sick Children in Toronto. The health outcomes were described in quality-adjusted life-years (QALYs). Health-state utilities were derived from the peer-reviewed literature. Scenario analyses, deterministic, and probabilistic sensitivity analyses were conducted. Economic costs were measured in 2021 Canadian dollars ($1.00 = US$0.80)
Results
TPO-RAs are estimated to result in an increased cost of $27,118 and a QALY gain of 0.21 compared to non-TPO-RAs over a 2-year horizon, resulting in an incremental cost-effectiveness ratio (ICER) of $129,133. In a 5-year scenario analysis, the ICER fell to $76,403. In the probabilistic sensitivity analysis, TPO-RAs exhibit a 40.0% probability of being cost-effective at a conventional ($100,000) willingness-to-pay threshold per QALY gained.
Conclusions
Further assessment of the long-term efficacy of TPO-RAs is warranted to obtain more precise long-term estimates. As the costs of TPO-RAs decline with the introduction of generic formulations, TPO-RAs may be increasingly cost-effective.
CONFLICT OF INTEREST STATEMENT
The authors declare that there is no conflict of interest.
Open Research
DATA AVAILABILITY STATEMENT
Data available on request from the authors.
Supporting Information
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