OBJECTIVES: Cost-utility analysis (CUA) has become increasingly important in the National Reimbursement Drug List (NRDL) price negotiations in China. Quality-adjusted life year (QALY) is the most preferred effectiveness indicators by Chinese payer. This review aims to evaluate the study design and QALY gains of negotiated oncology drugs.

METHODS: A systematic review was conducted to identify the latest China CUA studies of the 2020 NRDL listed oncology drugs. Data sources included PubMed, Embase, Web of Science, CNKI, WanFang and SinoMed. Descriptive analyses were conducted to summarize the corresponding QALY gains.

RESULTS: Sixty-four studies (115 results) were included, involving 18 drugs (13 targeted therapies, 4 biologics and 1 chemotherapy) out of 42 negotiated oncology drugs. Active comparators were used in 91% of studies. Markov model (85%), healthcare perspective (67%) and 10-year time horizon (57%) were the most common choice for study design. 89% of studies obtained utility parameters from published literature and 8.7% from clinical trials. In terms of the original source of utility parameter, only 28% of them included Chinese population. The mean QALY gain was 0.597 [SD: 0.79]. CUA in breast cancer presented the highest average QALY gain (1.837), followed by myeloid leukemia (1.480) and non-small cell lung cancer (0.504). Overall, the average QALY gain was 0.453 compared with best supportive care/placebo and 0.610 compared with active comparators. QALY gains increased over longer time horizon, with a mean of 0.251 below 5 years, 0.475 for 10-20 years, and 1.293 for 20 years and beyond.

CONCLUSIONS: The study design of identified studies was generally aligned with China Pharmacoeconomics Guidelines. Nevertheless, the methods used for estimating QALY gains were not transparently reported, and majority of utility values were still based on international data. This study reveals the importance of conducting more domestic research and obtain high-quality data applicable to Chinese population.