Sponsorship bias in published pharmacoeconomic evaluations of national reimbursement negotiation drugs in China: a systematic review.

BACKGROUND: China's National Reimbursement Drug List (NRDL) has become the primary route for drug reimbursement in China. More recently, the authority has made pharmacoeconomic evaluation an integral part of the application for NRDL inclusion. The underlying financial conflict of interests (FCOI) of pharmacoeconomic evaluations, however, has the potential to influence evidence generated and thus subsequent decision-making yet remains poorly understood. METHODS: We searched for studies published between January 2012 and January 2022 on the 174 drugs added to the 2017-2020 NRDLs after successful negotiation. We categorised the study's FCOI status into no funding, industry funding, non-profit funding and multiple fundings based on authors' disclosure and assessed the reporting quality of included studies using the Consolidated Health Economic Evaluation Reporting Standards 2022 checklist. We compiled descriptive statistics of funding types and study outcomes using t-tests and χ2 tests and conducted multivariate regression analysis. RESULTS: We identified 378 records and our final sample included 92 pharmacoeconomic evaluations, among which 69.6% were conducted with at least one funding source. More than half (57.6%) of the evaluations reached favourable conclusions towards the intervention drug and 12.6% reached a dominant result of the intervention drug over the comparison from model simulation. The reporting quality of included studies ranged from 19 to 25 (on a scale of 28), with an average of 22.3. The statistical tests indicated that industry-funded studies were significantly more likely to conclude that the intervention therapy was economical (p<0.01) and had a significantly higher proportion of resulting target drug economically dominated the comparison drug (p<0.05). CONCLUSION: The study revealed that FCOI bias is common in published pharmacoeconomic evaluations conducted in Chinese settings and could significantly influence the study's economical results and conclusions through various mechanisms. Multifaceted efforts are needed to improve transparency, comparability and reporting standardisation.

Economic evaluation of tislelizumab versus chemotherapy as second-line treatment for advanced or metastatic esophageal squamous cell carcinoma in China.

Background and purpose: The latest RATIONALE-302 trial (NCT03430843) showed that tislelizumab therapy significantly improved overall survival benefits for patients with advanced or metastatic esophageal squamous cell carcinoma (ESCC) compared with traditional chemotherapy. This study aimed to compare the cost-effectiveness of tislelizumab versus chemotherapy as a second-line treatment for advanced or metastatic ESCC in China. Methods: A partitioned survival model was developed to predict patients' lifetime quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) from the Chinese healthcare payers' perspective. We extracted efficacy and safety data from the RATIONALE-302 trial and the local cost and resource use data from online databases and published studies. One-way sensitivity analysis (OWSA) and probabilistic sensitivity analysis (PSA) were performed to explore model uncertainty. Results: Compared with chemotherapy, tislelizumab generated a higher cost (US$ 10211.78 vs. US$ 7294.72) but yielded more QALY (0.78 vs. 0.51 QALYs). The ICER for tislelizumab was US$11073.85 per QALY gained. The PSA results indicated that the probability of tislelizumab being economical was 76% under a willingness-to-pay (WTP) threshold of 1.5 times per capita GDP ($17915) in China. Conclusion: Tislelizumab could be a promising cost-effective strategy as the second-line treatment for patients with ESCC compared with chemotherapy in the Chinese setting.

Cost-effectiveness of edaravone dexborneol versus edaravone for the treatment of acute ischemic stroke in China: Based on the TASTE study.

Background and purpose: The TASTE trial indicated that patients with acute ischemic stroke (AIS) using edaravone dexborneol have a significantly higher proportion of 90-day good functional outcomes (mRS 0-1) than those using edaravone. This study compared the cost-effectiveness of the aforementioned interventions in treating AIS in the Chinese setting, aiming to inform treatment decisions in clinical practice. Methods: A model combining a decision tree and a Markov model was developed to assess the cost-effectiveness of edaravone dexborneol versus edaravone for AIS over a 30-year time horizon from the Chinese healthcare system's perspective. Both efficacy and safety data were extracted from the TASTE study. Local costs and utilities were derived from publications and open-access databases; both cost and effectiveness were discounted at a rate of 5% per year. Sensitivity analyses were conducted to ensure robustness and identify the main drivers of the result. Results: Compared with edaravone, edaravone dexborneol for AIS was found to be cost-effective in the first year and highly cost-effective as the study time horizons extended. In the long term (30 years), edaravone dexborneol yielded a lifetime gain of 0.25 (0.07-0.45) quality-adjusted life years (QALYs) at an additional cost of CNY 2201.07 (-3,445.24-6,637.23), yielding an ICER of CNY 8823.41 per QALY gained under the willingness-to-pay (WTP) of 1.5 times per capita GDP (121,464 CNY). The result is robust in both deterministic and probabilistic sensitivity analysis (PSA) methods, with the advantage of the edaravone dexborneol strategy increasing over time. Specifically, the probability of edaravone dexborneol dominant dexborneol is 76.30%, 98.90%, and 99.50% over 1-, 5-, and 30-year time horizons. Conclusion: Both short- and long-term economic analyses suggest that edaravone dexborneol is highly likely to be a cost-effective alternative to treat AIS compared with edaravone in China.

Effect of the full coverage policy of essential medicines on medication adherence: A quasi-experimental study in Taizhou, China.

Objective: Different forms of full coverage policy of essential medicines (FCPEMs) have been adopted worldwide to lower medication expenditure and improve adherence. This study aims to analyse the effect of FCPEMs on patients' medication adherence in Taizhou city, China. Methods: This study was a quasi-experimental study and set treatment and control groups. We extracted Electronic Health Records (EHRs) for hypertension and diabetes 1 year before and after FCPEMs implementation and their medication adherence level assessed by physicians. We applied the propensity score matching (PSM) method to balance the bias between the two groups. Then, the descriptive analysis was used to compare the differences in the reported medication adherence. Using the Difference-In-Differences (DIDs) method, the fixed-effect model with the logistic regression was built to analyse the effects of FCPEMs. Results: 225,081 eligible patients were identified from the original database. In the baseline year, FCPEM covered 39,251 patients. After PSM, 6,587 patients in the treatment group and 10,672 patients in the control group remained. We found that the proportion of patients with high adherence in the treatment group increased by 9.1% (60.8 to 69.9%, P < 0.001) and that in the control group increased by 2.6% (62.5 to 65.2%, P < 0.001). The regression results showed that FCPEMs significantly increased patients' medication adherence (OR = 2.546, P < 0.001). Conclusion: FCPEMs significantly improved medication adherence. Socially disadvantaged individuals might benefit more from continuing FCPEM efforts. Expanding the coverage of FCPEMs to other medicines commonly used in patients with chronic diseases may be a promising strategy to manage chronic diseases and promote patient outcomes.