Abstract
BackgroundClinical guidelines recommend a stepped-escalation treatment strategy for type 2 diabetes (T2DM). Across multiple treatment strategies varying in efficacy and costs, no clinical or economic studies directly compared them. This study aims to estimate and compare the cost-effectiveness of 10 commonly used pharmacologic combination strategies for T2DM.MethodsBased on Chinese guideline and practice, 10 three-stepwise add-on strategies were identified, which start with metformin, then switch to metformin plus one oral drug (i.e., sulfonylurea, thiazolidinedione, α-glucosidase inhibitor, glinide, or DPP-4 inhibitor) as second line, and finally switch to metformin plus one injection (i.e., insulin or GLP-1 receptor agonist) as third line. A cohort of 10,000 Chinese patients with newly diagnosed T2DM was established. From a healthcare system perspective, the Cardiff model was used to estimate the cost-effectiveness of the strategies, with clinical data sourced from a systematic review and indirect treatment comparison of 324 trials, costs from claims data of 1164 T2DM patients, and utilities from an EQ-5D study. Outcome measures include costs, quality-adjusted life-years (QALYs), incremental cost-effectiveness ratios (ICERs), and net monetary benefits (NMBs).ResultsOver 40-year simulation, the costs accumulated for a patient ranged from $7661 with strategy 1 to $14,273 with strategy 10, while the QALY gains ranged from 13.965 with strategy 1 to 14.117 with strategy 8. Strategy 7 was dominant over seven strategies (strategies 2~6, 9~10) with higher QALYs but lower costs. Additionally, at a willingness-to-pay threshold of $30,787/QALY (i.e., 3 times GDP/capita for China), strategy 7 was cost-effective compared with strategy 1 (ICER of strategy 7 vs. 1, $3371/QALY) and strategy 8 (ICER of strategy 8 vs. 7, $132,790/QALY). Ranking the strategies by ICERs and NMBs, strategy 7 provided the best value for money when compared to all other strategies, followed by strategies 5, 9, 8, 1, 3, 6, 10, 2, and 4. Scenario analyses showed that patients insist on pharmacologic treatments increased their QALYs (0.456~0.653) at an acceptable range of cost increase (ICERs, $1450/QALY~$12,360/QALY) or even at cost saving compared with those not receive treatments.ConclusionsThis study provides evidence-based references for diabetes management. Our findings can be used to design the essential drug formulary, infer clinical practice, and help the decision-maker design reimbursement policy.
Highlights
Clinical guidelines recommend a stepped-escalation treatment strategy for type 2 diabetes (T2DM)
Strategy 1 was associated with the lowest costs and lowest Qualityadjusted life-year (QALY) gains when compared with other nine strategies, whereas strategy 10 resulted in the highest costs but incrementally less QALY gains when compared with strategy 7 and strategy 8 (Table 2, Fig. 3)
Because the Incremental cost-effectiveness ratio (ICER) were ¥23,288 ($3371)/QALY when moving from strategy 1 to strategy 7 and ¥917,312 ($132,790)/QALY when moving from strategy 7 to strategy 8, strategy 7 was cost-effective compared with strategy 1 and strategy 8 at a willingness-to-pay threshold of ¥212,676 ($30,787)/ QALY (Table 2)
Summary
Clinical guidelines recommend a stepped-escalation treatment strategy for type 2 diabetes (T2DM). Across multiple treatment strategies varying in efficacy and costs, no clinical or economic studies directly compared them. This study aims to estimate and compare the cost-effectiveness of 10 commonly used pharmacologic combination strategies for T2DM. Type 2 diabetes (T2DM) as a chronic progressive disease imposes a substantial disease burden on patients and the healthcare system [1]. Chinese clinical guidelines advocate a stepwise failure-driven treatment strategy for blood glucose lowering that leads to the sequential addition of therapies [2]. Metformin is the preferred initial therapy, which is recommended to be maintained throughout the treatment [2]. If oral dual therapy fails to effectively control glucose, injections like insulin or glucagon-like peptide 1 (GLP-1) receptor agonist can be added onto metformin [2]
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