ClinicoEconomics and Outcomes Research (May 2022)
Cost-Effectiveness and Budget Impact Analyses of Tisagenlecleucel in Pediatric and Young Adult Patients with Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia from the Singapore Healthcare System Perspective
Abstract
Xiao Jun Wang,1 Yi-Ho Wang,2 Matthew Jian Chun Ong,1 Christina Gkitzia,3 Shui Yen Soh,4 William Ying Khee Hwang5– 7 1Novartis Singapore Pte Ltd, Singapore; 2Novartis Asia Pacific Pharmaceuticals Pte Ltd, Singapore; 3Novartis Pharma AG, Basel, Switzerland; 4KK Women’s & Children’s Hospital, Singapore; 5National Cancer Centre Singapore, Singapore; 6Singapore General Hospital, Singapore; 7Duke-NUS Medical School, SingaporeCorrespondence: Xiao Jun Wang, Novartis Singapore Pte Ltd, 20 Pasir Panjang Road, #10-25/28 Mapletree Business City (West Tower), 117439, Singapore, Tel +65 67226010, Email [email protected]: Children and young adults with relapsed or refractory (r/r) acute lymphoblastic leukemia (ALL) have poor survival due to ineffective therapy options. The newly approved chimeric antigen receptor T-cell (CAR-T) therapy, tisagenlecleucel, has demonstrated improved survival but at a high up-front cost. The study aims to evaluate the cost-effectiveness and budget impact of tisagenlecleucel versus salvage chemotherapy regimen (SCR) or blinatumomab (BLN) for the treatment of pediatric and young adult patients with relapsed/refractory B-cell ALL from the Singapore healthcare system perspective.Patients and Methods: A three-health state partitioned survival model was constructed to analyze the cost-effectiveness of tisagenlecleucel vs SCR/BLN with/without allogenic hematopoietic stem cell transplantation (allo-HSCT) over a lifetime period. Clinical efficacy for tisagenlecleucel, SCR and BLN were based on pooled data from ELIANA, ENSIGN and B2101J trials, the study by von Stackelberg et al 2011, and MT103-205 respectively. Medical costs from pre-treatment until terminal care, including treatment, side effects, follow-up, subsequent allo-HSCT and relapse, were considered. Incremental cost-effectiveness ratios (ICERs) were estimated as the incremental costs per quality-adjusted life-year (QALY) gain. Additionally, the financial impact of tisagenlecleucel introduction in Singapore was estimated, comparing the present treatment scenario (without tisagenlecleucel) with a future scenario (with tisagenlecleucel), over 5 years.Results: In the base-case analysis, tisagenlecleucel treatment demonstrated cost-effectiveness with an ICER of S$45,840 (US$34,762) per QALY (vs SCR) and S$51,978 (US$39,315) per QALY (vs BLN). The estimated budget ranges from S$477,857 (US$361,438) to S$1.4 million (US$1.05 million) annually for the initial 5 years.Conclusion: Tisagenlecleucel is likely to be a cost-effective treatment option with limited budget implications while treating r/r ALL patients who have failed at least 2 lines of prior therapies.Keywords: tisagenlecleucel, acute lymphocytic leukemia, partition survival model, cost-effectiveness, budget impact, Singapore
