EXPEDITED REGULATORY PATHWAYS AND REIMBURSEMENT DECISIONS FOR INNOVATIVE CANCER DRUGS IN CHINA
Author(s)
Qi Li, BsC1, Nan Peng, PhD2, Chen Mu, BsC1, guoxian Lu, BsC1, Ruolan Wei, BsC1, Haijing Guan, PhD3, Dongning Yao, PhD1;
1Nanjing Medical University, School of Pharmacy, Nanjing, China, 2School of Pharmaceutical Science and Technology,Tianjin University, Tianjin, China, 3Beijing Tiantan Hospital, Capital Medical University, Department of Pharmacy, Beijing, China
1Nanjing Medical University, School of Pharmacy, Nanjing, China, 2School of Pharmaceutical Science and Technology,Tianjin University, Tianjin, China, 3Beijing Tiantan Hospital, Capital Medical University, Department of Pharmacy, Beijing, China
OBJECTIVES: In the context of increasingly aligned regulatory and reimbursement evidence, we examined the relationship between expedited regulatory designation and inclusion in the National Reimbursement Drug List (NRDL) for innovative cancer drugs in China.
METHODS: We included cancer drug indications that entered China’s NRDL negotiations for the first time between 2017 and 2024. Indications were classified according to the presence of expedited regulatory designation at the time of market authorisation, including priority review, conditional approval, and breakthrough therapy designation. Analyses were conducted at the indication level using univariable analyses and multivariable logistic regression models. Covariates included negotiation year, company type, tumour type, innovation status, overall survival (OS) or progression-free survival (PFS) benefit, pivotal phase III trial support, line of therapy, and monthly treatment cost.
RESULTS: A total of 205 indications from 156 drugs entered NRDL negotiations during the study period, of which 137 (66.8%) achieved reimbursement. Among all evaluated indications, 117 received priority review, 63 were conditionally approved, and 15 were granted breakthrough therapy designation. The reimbursement rate for conditionally approved indications was significantly lower than that of non-conditionally approved indications (25.0% vs 42.0%, p = 0.013). Multivariable logistic regression analysis showed no significant association between expedited regulatory designation as a composite exposure and NRDL inclusion. However, at the individual pathway level, conditionally approved drugs were less likely to be reimbursed compared with non-conditionally approved drugs. In contrast, indications demonstrating OS or PFS benefit in pivotal trials were more likely to be reimbursed, while higher monthly treatment cost was associated with a significantly lower probability of inclusion.
CONCLUSIONS: Based on the available evidence, expedited regulatory designation did not appear to be independently associated with reimbursement decisions for innovative cancer drugs in China. Reimbursement decisions were more closely aligned with demonstrated clinical benefit and treatment cost.
METHODS: We included cancer drug indications that entered China’s NRDL negotiations for the first time between 2017 and 2024. Indications were classified according to the presence of expedited regulatory designation at the time of market authorisation, including priority review, conditional approval, and breakthrough therapy designation. Analyses were conducted at the indication level using univariable analyses and multivariable logistic regression models. Covariates included negotiation year, company type, tumour type, innovation status, overall survival (OS) or progression-free survival (PFS) benefit, pivotal phase III trial support, line of therapy, and monthly treatment cost.
RESULTS: A total of 205 indications from 156 drugs entered NRDL negotiations during the study period, of which 137 (66.8%) achieved reimbursement. Among all evaluated indications, 117 received priority review, 63 were conditionally approved, and 15 were granted breakthrough therapy designation. The reimbursement rate for conditionally approved indications was significantly lower than that of non-conditionally approved indications (25.0% vs 42.0%, p = 0.013). Multivariable logistic regression analysis showed no significant association between expedited regulatory designation as a composite exposure and NRDL inclusion. However, at the individual pathway level, conditionally approved drugs were less likely to be reimbursed compared with non-conditionally approved drugs. In contrast, indications demonstrating OS or PFS benefit in pivotal trials were more likely to be reimbursed, while higher monthly treatment cost was associated with a significantly lower probability of inclusion.
CONCLUSIONS: Based on the available evidence, expedited regulatory designation did not appear to be independently associated with reimbursement decisions for innovative cancer drugs in China. Reimbursement decisions were more closely aligned with demonstrated clinical benefit and treatment cost.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR141
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling, Reimbursement & Access Policy
Disease
SDC: Oncology