The cost-effectiveness of iruplinalkib versus alectinib in anaplastic lymphoma kinase-positive crizotinib-resistant advanced non-small-cell lung cancer patients in China.

Background: Iruplinalkib is a second-generation anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor (TKI) with efficacy in patients with ALK-positive crizotinib-resistant advanced non-small cell lung cancer (NSCLC), which is independently developed by a Chinese pharmaceutical company. This study examined the cost-effectiveness of iruplinalkib versus alectinib in the Chinese healthcare setting. Methods: A partitioned survival model was developed to project the economic and health outcomes. Efficacy was derived using unanchored matching-adjusted indirect comparison (MAIC). Cost and utility values were obtained from the literature and experts' opinions. Deterministic and probabilistic sensitivity analyses (PSA) were carried out to evaluate the model's robustness. Results: Treatment with iruplinalkib versus alectinib resulted in a gain of 0.843 quality-adjusted life years (QALYs) with incremental costs of $20,493.27, resulting in an incremental cost-effectiveness ratio (ICER) of $24,313.95/QALY. Parameters related to relative efficacy and drug costs were the main drivers of the model outcomes. From the PSA, iruplinalkib had a 90% probability of being cost-effective at a willingness-to-pay threshold of $37,863.56/QALY. Conclusion: Compared to alectinib, iruplinalkib is a cost-effective therapy for patients with ALK-positive crizotinib-resistant advanced NSCLC.

Cost-Effectiveness Analysis of Fluocinolone Acetonide Intravitreal (FAI) Implant for Chronic Noninfectious Uveitis Affecting the Posterior Segment of the Eye (NIU-PS) in China.

INTRODUCTION: Chronic non-infectious uveitis affecting the posterior segment (NIU-PS), which can be recurrent and persistent for numerous years, mainly affects people of working age and significantly increases the risk of visual impairment. This study aimed to investigate the cost-effectiveness of fluocinolone acetonide intravitreal (FAI) implant in the treatment of patients with chronic NIU-PS from the Chinese healthcare perspective. METHODS: A Markov model with a 2-week cycle was constructed from the perspective of the Chinese healthcare system over a lifetime time horizon. The model consists of four health states: on-treatment, treatment failure, blindness, and death. The outcomes for effectiveness were based on the Chinese real-world study (RWS). Utilities and mortality rates were derived from published literature and standard sources. Costs were determined from the MENET website, prices of medical service items at local providers, published literature, and expert surveys. Outcomes were measured in quality-adjusted life years (QALYs). Sensitivity analyses were performed to account for the impact of uncertainty. RESULTS: It was estimated that in the base case, the FAI implant provided 0.43 incremental QALYs compared with the limited current practice (LCP) at an additional cost of $7503.72 (¥50,575.05), resulting in an incremental cost-effectiveness ratio (ICER) of $17,373.49 (¥117,097.33) per QALY gained. Parameters related to utility emerged as the primary influencers on the outcomes. In probabilistic sensitivity analysis (PSA), considering the willingness-to-pay (WTP) threshold of $19,072 (¥128,547) and $38,145 (¥257,094), the FAI implant had 67.70% and 99.50% probability of being cost-effective, respectively. As demonstrated in the scenario analysis, if the FAI implant aligns its price reduction with the average rate from the 2023 negotiation of the National Reimbursement Drug List (NRDL), it would result in lower costs and represent an absolute advantage. CONCLUSIONS: The FAI implant, which can effectively reduce the recurrence rate and maintain the incremental costs within the WTP limit, is likely to be cost-effective in treating chronic NIU-PS in China.

Clinical and Economic Evaluation of Ultrasound-Guided Radiofrequency Ablation vs. Parathyroidectomy for Patients with Primary Hyperparathyroidism: A Cohort Study.

RATIONALE AND OBJECTIVES: To compare the clinical and economic effects of ultrasound (US)-guided radiofrequency ablation (RFA) with parathyroidectomy (PTX) for primary hyperparathyroidism (PHPT). MATERIALS AND METHODS: From April 2014 to April 2021, 123 PHPT patients who received US-guided RFA or PTX were studied. Propensity score (PS) matching was used to balance the baseline data of the two groups. The rates of cure, recurrent and persistent PHPT, and complications were compared. A Chinese healthcare system perspective cost minimization analysis was conducted. RESULTS: After PS matching, 37 patient pairs (1:1) were created for the two groups. Follow-up was 27.2 ± 10.6 months and 28.8 ± 16.1 months for the RFA and PTX groups, respectively. At the last follow-up, there was no evidence of differences regarding clinical cure rate between the two groups (RFA vs. PTX, 91.9% vs. 94.6%, p = 1.000). Recurrent PHPT did not develop in any patient. One patient in each group had persistent PHPT. The incidence of complications and side effects, except postoperative pain (RFA vs. PTX, 16.2% vs. 40.5%, p = 0.020), were no significant difference between the two groups (all, p > 0.05). The incremental cost was -$284.00; thus, RFA was more cost-effective. For patients with employee medical insurance or resident medical insurance, the incremental costs (RFA vs. PTX) were -$391.94 and -$49.43, respectively. CONCLUSION: There were no significant differences in efficacy and safety between RFA and PTX. As the incremental cost for RFA compared with PTX was negative, RFA may be used as a more cost-effective nonsurgical treatment alternative for PHPT.

Whether and How Disutilities of Adverse Events were Used in the Economic Evaluation of Drug Therapy for Cancer Treatment.

BACKGROUND: The disutilities of adverse events (AEs) are important inputs for cost-utility analysis (CUA), reflecting the impacts of AEs on health outcomes. Health technology assessment institutions and scholars have proposed recommendations for applying disutility values in economic evaluations. OBJECTIVES: This study aimed to identify the current use of disutilities of AEs as model parameters in the CUA of cancer drug therapy and to compare the discrepancies between the use of disutilities and published recommendations. METHODS: A systematic search was conducted on the PubMed, Web of Science, and Cochrane Library databases, as well as the official websites of the National Institute for Health and Care Research (NIHR), the Institute for Clinical and Economic Review (ICER), the Institute for Quality and Efficiency in Health Care (IQWiG), the Canadian Agency for Drugs and Technologies in Health (CADTH), and the Centre for Reviews and Dissemination (CRD) for CUAs of drug therapy for cancer published in English from January 2019 to April 2022. Information about the use of disutilities of AEs (whether and how disutilities were used, or why they were not used) in selected studies was extracted and compared with published recommendations. Descriptive analyses were used to summarize the results. RESULTS: A total of 467 CUAs were included, 54% (254/467) of which included disutilities of AEs in their model. The proportion that included these disutilities increased from 2019 to 2021, ranging from 47% (51/107) to 61% (116/190). Only 6% (15/254) of the CUAs using disutilities of AEs considered all five recommendations about the justification for inclusion and exclusion, description of values and sources, grades of AEs, calculation, and uncertainty analyses. Only 15% (72/467) provided a clear justification for inclusion and exclusion of disutilities of AEs, and 7% (17/254) did not provide values or sources. In total, 69% (175/254) of the analyses focused on AEs of grade 3 or greater, and 11% (28/254) applied utility decrements for grades 1 and 2. Disutilities of AEs were generally calculated using the incidence rates, which were clearly stated in 49% (65/132) of the analyses. Uncertainty analyses were conducted in 84% (214/254) of the CUAs. CONCLUSIONS: The current use of disutilities of AEs in CUAs shows some discrepancies with recommendations proposed in the literature. One is that detailed information about the use of disutilities of AEs was not reported and the other is that essential methods to analyze the impact of AEs on quality-adjusted life-years were not thoroughly conducted. Therefore, it is suggested that researchers should attach importance to the impact of AEs on health-related quality of life. Furthermore, an application process was developed for the disutilities of AEs to remind and guide researchers to correctly use the disutilities of AEs as parameters in the decision-analytic model.

Obtaining EQ-5D-3L utility index from the health status scale of traditional Chinese medicine (TCM-HSS) based on a mapping study.

BACKGROUND: Almost all traditional Chinese medicine (TCM) quality of life measures are non-preference-based measures (non-PBMs), which do not provide utilities for cost-utility analysis in pharmacoeconomic evaluation. Whereas the mapping has become a new instrument to obtain utilities, which builds a bridge between non-PBMs and PBMs. PURPOSE: To develop mapping algorithms from the health status scale of traditional Chinese medicine (TCM-HSS) onto the three-level EuroQol five-dimensional questionnaire (EQ-5D-3L). METHODS: The cross-sectional data were collected by questionnaire survey from a tertiary hospital visit population and community residents in China, and randomly divided into training and validation set by 2:1. Based on the training set, direct and indirect mapping methods (7 regression methods and 4 model specifications) were conducted to establish alternative models, which were comprehensively evaluated based on the validation set by mean absolute error, root mean square error, and Spearman correlation coefficient between predicted and observed values. Based on the whole sample, the preferred mapping algorithm was developed. RESULTS: A total of 639 samples were included, with an average age of 45.24 years and 61.66% of respondents were female. The mean EQ-5D-3L index was 0.9225 [SD = 0.1458], and the mean TCM-HSS index was 3.4144 [SD = 3.1154]. The final mapping algorithm was a two-part regression model including the TCM-HSS subscales, interaction terms, and demographic covariates (age and gender). The prediction performance was good. The mean error was 0.0003, the mean absolute error was 0.0566, the root mean square error was 0.1039, and 83.10% of the prediction errors were within 0.1; the Spearman correlation coefficient between predicted and observed EQ-5D-3L values was 0.6479. CONCLUSION: It is the first study to develop a mapping algorithm between the TCM-HSS and EQ-5D-3L, which demonstrates excellent prediction accuracy and estimates utility value for economic evaluation from TCM quality of life measures.

Effects of bDMARDs on quality of life in patients with psoriatic arthritis: meta-analysis.

OBJECTIVES: To determine the effects of biological disease-modifying anti-rheumatic drugs (bDMARDs) on the quality of life (QoL) among patients with psoriatic arthritis (PsA). DESIGN: Meta-analysis. DATA SOURCES AND ELIGIBILITY CRITERIA: PubMed, Web of Science, Cochrane Library, China National Knowledge Infrastructure, WanFang and VIP databases were searched to collect randomised controlled trials (RCTs), which were conducted to evaluate the effect of bDMARDs in the treatment of patients with PsA and reported QoL-related outcomes, from inception to November 2020 and updated on 19 February 2022. DATA EXTRACTION AND SYNTHESIS: Outcomes about Health Assessment Questionnaire Disability Index (HAQ-DI), Dermatology Life Quality Index, physical component summary and mental component summary of the Short Form 36, EuroQol Visual Analogue Scale, Psoriasis Area Severity Index (PASI) 50/75/90/100 were extracted by two reviewers independently. Data were pooled using the fixed or random effects methods and considered as mean difference (MD) or risk ratio with 95% CI. RESULTS: Out of 3190 articles screened, 37 RCTs (with 47 articles reported) were included. Pooled estimates showed that bDMARDs were superior versus placebo on all outcomes. Against methotrexate (MTX) and tofacitinib, bDMARDs showed no statistically significant advantages or significant disadvantages. Similar results were found for bDMARDs+MTX versus MTX. For HAQ-DI, the results of the subgroups of bDMARDs versus placebo, bDMARDs+MTX versus MTX, bDMARDs versus tofacitinib and bDMARDs versus MTX were -0.21 (MD, 95% CI, -0.23 to -0.18), -0.22 (MD, 95% CI, -0.58 to 0.14), -0.01 (MD, 95% CI, -0.05 to 0.04) and -0.03 (MD, 95% CI, -0.04 to -0.02), respectively. CONCLUSIONS: Compared with placebo, bDMARDs taken by patients with PsA appear to significantly improve the QoL. Compared with other therapeutic agents, more studies are required to confirm the effect of single and combined bDMARDs use further.

The effects of berberine on inflammatory markers in Chinese patients with metabolic syndrome and related disorders: a meta­analysis of randomized controlled trials.

BACKGROUND: A meta-analysis of randomized controlled trials (RCTs) was conducted to systematically evaluate the effects of berberine on the inflammatory markers of metabolic syndrome (MetS) and related disorders. METHOD: Databases that were searched from inception to October 2020 included PubMed, Web of Science, the Cochrane Library, CNKI, VIP, WanFang Data, and ClinicalTrials.gov. Two reviewers independently selected articles and extracted data. The pooled evaluations were entered and analyzed in Review Manager 5.3. RESULTS: Of the 7387 publications screened, 52 studies were included, and the related trials involved 4616 patients. Pooled estimates showed that the use of berberine could significantly reduce the concentration level of C-reactive protein (CRP) [standardized mean difference (SMD) = - 1.54, 95% confidence intervals (CI) - 1.86, - 1.22, p < 0.05], tumor necrosis factor-α (TNF-α) [SMD = - 1.02, 95% CI - 1.27, - 0.77, p < 0.05], and interleukin 6 (IL-6) [SMD = - 1.17, 95% CI - 1.53, - 0.81, p < 0.05] among patients with MetS and related disorders. However, it did not affect the level of interleukin 1ß (IL-1ß) [SMD = - 0.81, 95% CI - 1.80, 0.17, p = 0.11]. CONCLUSION: Overall, the use of berberine in patients with MetS and related disorders appeared to significantly decrease several inflammatory markers. Further multi-center and rigorous investigations with larger patient populations are encouraged to confirm the effect of berberine on MetS and related disorders.

Efficacy and safety of PEG-asparaginase versus E. coli L-asparaginase in Chinese children with acute lymphoblastic leukemia: a meta-analysis.

BACKGROUND: Multiagent chemotherapy is the primary treatment for acute lymphoblastic leukemia (ALL), of which asparaginases including Escherichia coli L-asparaginase (E. coli L-Asp) and pegylated-asparaginase (PEG-Asp), are cornerstone components. The study aimed to conduct a meta-analysis to compare the efficacy and safety of PEG-Asp with E. coli L-Asp in Chinese children with ALL. METHODS: A systematic literature search was conducted to collect randomized controlled trials (RCTs) on PEG-Asp versus E. coli L-Asp in Chinese children with ALL. Two reviewers independently selected articles and extracted data. Risk-of-bias assessment was conducted with Cochrane recommendation tool. Pooled estimates and risk ratios (RRs) with 95% confidence intervals (CIs) were calculated for all outcomes in Review Manager 5.3. RESULTS: Out of the 470 publications screened, 15 studies were included, involving 1,194 patients. Pooled estimates showed that there were no significant differences in complete responses (CR), overall response rate (ORR), gastrointestinal symptoms, and coagulation abnormalities rate between the PEG-Asp and E. coli L-Asp groups (all P>0.05). Hypersensitivity (RR =0.63; 95% CI, 0.40-1.01; Ρ=0.05) and hepatic injury rate (RR =0.45; 95% CI, 0.27-0.75; Ρ=0.002) were lower in the PEG-Asp group. The frequency of administration and length of hospital stay of patients in the PEG-Asp group were less than those in the E. coli L-Asp group (both Ρ<0.0001). CONCLUSIONS: Current evidence pointed out a similar efficacy in the two groups. The PEG-Asp group showed a lower hypersensitivity and hepatic injury rate. In addition, using PEG-Asp decreased the frequency of administration and the length of hospital stay, which, to some extent, might reduce patients' burden caused by medical resource consumption.