Innovations in cancer therapy, particularly the influx of new drugs have yielded high expectations of transform treatment of the disease from all healthcare stakeholders (1, 2). Nevertheless, dramatic rise in drug costs has recently highlighted a vigorous debate over whether cancer drugs prices, especially for that of targeted drugs and immunotherapies, commensurate with their value to patients, within reach of who need them not only in developed country, but also in developing country like China with scarce resources and rising demand for health services (3, 4).

To our knowledge, not all people know the price of everything but the value of nothing. It has never been more important to assess the value of new cancer drugs, and several organizations including the American Society of Clinical Oncology (ASCO), European Society for Medical Oncology (ESMO), Institute for Clinical and Economic Review (ICER), National Comprehensive Cancer Network (NCCN), and the pan-Canadian Oncology Drug Review Expert Review Committee (pCODRERC) have recently taken a step forward in this endeavor, developing tools for value assessment (5–11). All these tools have been designed for value assessment with the aim of weighing up the balance between efficacy, toxicity, quality of life and costs. Despite of their different conceptual definition of “value,” Bentley et al. (12) reported that the ASCO and ESMO tools demonstrated convergent validity and inter-rater reliability for value assessment for new cancer drugs.

In recent years, regulatory reforms have led to the introduction of a series of expedited programs to accelerate development, review, and approval of new drugs in China (13). Here, we overview the landscape of new cancer drugs approved by NMPA for solid cancer between 2016 and 2020 in China, describe the value of these drugs and further explore whether value is related to the drug price.

To the best of our knowledge, this is the first study in China to comprehensively evaluate the value of new cancer drugs using ASCO-VF and ESMO-MCBS, and to investigate the correlation between price of new drugs and their clinical benefits. In our review of all new cancer drugs approved by NMPA for solid cancer between 2016 and 2020, approximately half of new drugs achieved meaningful clinical benefit according to ESMO-MCBS. We found that all new drugs had a wide range of ASCO-VF scores and only fair association between ASCO-VF and ESMO-MCBS, which was consistent with previous studies (15, 16).

About three-quarters of new drugs were listed in the National Reimbursement Drug List (NRDL). In contrast to the increasing prices of cancer drugs in the years after approval in the US, the daily treatment cost of cancer agents has fallen in China, especially for targeted therapies and branded products (17). However, we found no significant correlation between price and clinical benefit according to the two frameworks. With rising demand for health services in China, prices should be better aligned with value, especially for expensive cancer drugs. Nearly half of cancer drug indications approved in China had shown OS benefit (18). Lack of a clear association between price and clinical benefit indicates that value frameworks can help not only identify drugs with low or uncertain clinical benefit that should be targeted for price negotiations, but also therapies with evidence of higher clinical benefit to improve access to benefit drugs (19, 20).

In 2015, China's government proposed to establish an open and transparent price negotiation mechanism with multi-party participation for some patented, high-priced drugs and exclusively produced drugs. In the same year, the first round of national-level drug pricing negotiations was launched (21). The dimensions of national price-negotiation cancer drugs include the value of drugs and the affordability of China's healthcare system funds. China's government has sufficient bargaining power. Since 2017, the government has started annual centralized price negotiation, which has sharply fallen drug prices compared with launch prices, resulting in increased affordability of expensive cancer drugs (22). However, as new clinical trials were conducted, results of post-approval clinical trials might lead to dynamic changes in value frameworks scores. Moreover, the qualifications of medical insurance payment tend to be strict. For new indications of cancer drugs, the out-of-pocket spending of patients have not been reduced, leading to increase the financial toxicity of patients. Therefore, the government needs to routinely monitor the impacts of shifts in medicine on resource utilization.

This study has several limitations. Most breakthrough-designated drugs were approved based on single-arm or non-randomized trials, resulting in the level of evidence for breakthrough therapies was inferior to that for non-breakthrough therapies when assessing the value through value frameworks (23). Thence, it is highly unfair to assess clinical benefit in this situation. Furthermore, treatment effects are known to be heterogeneous, and some patients can benefit greatly from drugs with a low value score (24). This was one of the limitations of the study, which did not evaluate the value of all new drugs. It is complicated to precisely define value of a drug, and our assessment relied entirely on data reported of clinical trial, not taking into account other factors that may influence value of a drug. Secondly, the association between ASCO-VF and ESMO-MCBS was only fair based on our findings. The ASCO-VF and the ESMO-MCBS have shared the goal of assisting clinicians and patients to measure the relative benefits of new cancer drugs (23). Nevertheless, due to the differences in the frameworks' inherent designs, especially the method and indicators of the frameworks differ greatly, resulting in greatly divergences in scores and grades (14, 16). In addition, we used scores at the 75th percentile of ASCO-VF scores as a threshold for comparisons. Nevertheless, changing this cutoff score will influence the degree of correlation between the two value frameworks. Meanwhile, we did not consider the duration of treatment when calculating the costs of a drug. But most of drugs in the palliative setting, so as long as response to treatment continues, monthly drug costs could be used. Furthermore, we did not investigate whether all cancer drugs that were approved by the NMPA were also approved in other countries. Therefore, our study needs to be followed by further study to assess the value in other countries especially emerging or developing countries comprehensively, promoting the situation of other countries in terms of access to oncology medicines with value assessment.

In summary, ASCO-VF and ESMO-MCBS are important tools for assessing value of cancer drugs, although the correlation between these frameworks is fair. Based on the available evidence, not all new drugs met the meaningful threshold according to ASCO-VF or ESMO-MCBS. The price of a drug was not significantly related to the level of clinical benefit, and the cost could not justify its value. Policy makers requires to improve the alignment between drug prices and clinical benefits in order to provide optimal cancer treatments for patients.

The original contributions presented in the study are included in the article/Supplementary material, further inquiries can be directed to the corresponding author.

Conceptualization and writing—review and editing: JL and QJ. Methodology: JL and SO. Software and writing—original draft preparation: JL. Data curation: HW, XQ, and RP. Formal analysis: JL, SO, and SW. Visualization: HW and XQ. Supervision and funding acquisition: QJ. All authors have read and agreed to the published version of the manuscript.