Value assessment of NMPA-approved new cancer drugs for solid cancer in China, 2016–2020

Abstract

BackgroundWhether the high cost of cancer drugs is commensurate with their value to patients, which has become the focus of public concern. We aimed to assess the value of new cancer drugs approved for solid cancer in China and to explore the association between price and value of drugs.MethodsWe identified all new drugs for solid tumor that approved by the China's National Medical Products Administration (NMPA) between 2016 and 2020. The value of these drugs was assessed according to the American Society of Clinical Oncology Value Framework (ASCO-VF) and the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). We calculated Cohen's κ statistic to describe agreement between the two frameworks. Spearman's correlation coefficient was used to evaluate the correlation between price and value of drugs.ResultsBetween 2016 and 2020, 37 new drugs were approved by the NMPA for solid tumor and we could evaluate the value of 28 drugs (76%). Eight (29%) of drugs were approved for non-small-cell lung cancer and 6 (21%) for breast cancer. ASCO-VF scores had a range of −20 to 110.1, and the median score was 43.3 (inter-quartile range 27.1–58.35). Only seven drugs (25%) met the ASCO-VF cutoff score. By the ESMO-MCBS, 13 drugs showed a meaningful value. Agreement between these two frameworks thresholds was only fair (κ = 0.515, P < 0.05). We found no statistically significant correlation between launch price of drugs and clinical benefit according to both frameworks.ConclusionsNot all NMPA-approved new cancer drugs had meaningful value as measured by ASCO-VF or ESMO-MCBS. There was no significant correlation between drug price and the level of clinical benefit.

Hepatic cavernous hemangioma developed in non-small cell lung cancer patients after receiving Camrelizumab treatment: two case reports

“…Based on the KEYNOTE-189/KEYNOTE-407 studies, the U.S. FDA has approved pembrolizumab in combination with pemetrexed/platinum-based chemotherapy for first-line treatment of stage IV non-squamous/non-small cell lung cancer (NSCLC) patients who are negative for EGFR/ALK mutations ( 16 ). In China, based on the CameL and Rational series studies, the National Medical Products Administration (NMPA) has approved the use of Camrelizumab or Tislelizumab in combination with Pemetrexed and Carboplatin for first-line treatment of unresectable locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) patients who are negative for EGFR/ALK mutations ( 17 ). Additionally, the IMpower150 study has demonstrated that the quadruple combination of immune checkpoint inhibitors (ICI), Platinum-based doublet chemotherapy, and anti-angiogenic therapy significantly improves the overall survival of stage IV non-squamous NSCLC patients who are negative for EGFR/ALK mutations compared to chemotherapy alone (OS: 19.2 months vs 14.7 months; HR=0.78, 95% CI: 0.64-0.96) ( 18 ).…”

The correlation between the costs and clinical benefits of national price-negotiated anticancer drugs for specific cancers in China

“…Despite clinical endpoints being commonly employed to measure clinical efficacy, multiple frameworks have been proposed to reflect the clinical benefits, with ASCO-VF and ESMO-MCBS being applied most frequently. These frameworks had different conceptual definitions of “value” along with divergent scoring systems; however, the results of their value assessment were unified, demonstrating convergent validity and inter-rater reliability [ 13 ]. Based on these frameworks, previous studies also suggested that drug costs were either unrelated or had only weak associations with treatment outcomes, both in China and most other countries with or without negotiation policy [ 13 , 20 , 21 , 38 ], which is in line with our findings.…”

“…These frameworks had different conceptual definitions of “value” along with divergent scoring systems; however, the results of their value assessment were unified, demonstrating convergent validity and inter-rater reliability [ 13 ]. Based on these frameworks, previous studies also suggested that drug costs were either unrelated or had only weak associations with treatment outcomes, both in China and most other countries with or without negotiation policy [ 13 , 20 , 21 , 38 ], which is in line with our findings. This could be a consequence of the interplay between price negotiation and profit-oriented enterprises or patient’s benefit-driven government.…”

“…The average price discount for innovative drugs surpassed 50% after market entry [ 12 ]. According to prior studies, lung cancer (LC) and breast cancer (BC) were the most common neoplastic indications included in NRDL [ 13 , 14 ], and the ones showing the highest incidence among males and females in China, respectively [ 1 , 4 ]. Thus, we aimed to study the value of price-negotiated anticancer drugs targeted to LC and BC.…”

“…Several similar studies were conducted to estimate the prices and clinical benefits of new anticancer drugs in China and elsewhere, either based on traditional clinical endpoints or on aforementioned value frameworks. Most results indicated that anticancer costs were independent or weakly associated with improvement in clinical benefits [ 7 , 13 , 20 - 23 ], while few studies had converse findings [ 24 ]. However, little attention has been paid to the relation between clinical value and costs on specific cancer sites before and after the implementation of drug price negotiation.…”