We used the publicly available data to identify all new drugs (new molecular entities and novel biologic agents) approved by the China's National Medical Products Administration (NMPA) between January 1, 2016 and December 31, 2020, with initial indications for solid tumor. Meanwhile, we assessed whether the drug was granted with one of expedited programs in NMPA pathways and designations to accelerate drug approval (special review, priority review, conditional approval, urgently needed overseas drugs, and breakthrough therapy). Notably, drugs that were later approved for additional indications were not considered in this study.
The launch price and postlaunch price of drugs were extracted from the trade name and generic name recorded in the Hospital Information System (HIS). To estimate monthly treatment cost of a drug, we used the prescription and dosing information from the NMPA-approved label. Monthly treatment costs were calculated over an average of 30 days on the basis of the dosage schedule for an adult patient weighing 60 kg with a body surface area of 1.70 m2. The cost of all regimes was adjusted to provide the price per 4-week period (33.3% increase for 3-week treatment cycles and 100% increase for 2-week treatment cycles). Drug prices were converted to US dollars at the exchange rate as of August 29, 2022.
To quantify the clinical benefit from the pivotal clinical trials supporting regulatory approval, we applied two value frameworks developed by ASCO and ESMO, namely the American Society of Clinical Oncology Value Framework (ASCO-VF) version 2 (6 (link)), and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) version 1.1 (8 (link)). Scores were assessed by one reviewer and checked by a second one, with any discrepancies resolved by a senior reviewer. In contrast to ESMO-MCBS, ASCO-VF was not planned to score single-arm studies and was therefore only suitable for phase II or III randomized clinical trials. In cases in which multiple pivotal clinical trials have been done and yield different clinical benefit scores for a given drug, the highest score was considered. Consistent with the developer of the value frameworks, meaningful clinical benefit was defined as a grade of A or B (for the curative setting) or 4, 5 (for the palliative setting) using ESMO-MCBS, whereas ASCO-VF did not clearly define what score was deemed “meaningful value.” Cherny et al. (14 (link)) recommended that the optimal threshold score of 45 or higher was proposed for recognizing substantial benefit for ASCO-VF by generating receiver operating characteristic (ROC) curves. Nevertheless, given the differences in construction and goals of ASCO-VF and ESMO-MCBS, they might yield some discordance in a cohort of studies. Thus, we split scores at the 75th percentile of ASCO-VF scores as the cutoff score for subsequent analyses, referring to the meaningful value achieved of ESMO-MCBS as a grade of 4, 5, B, or A (15 (link)).
The launch price and postlaunch price of drugs were extracted from the trade name and generic name recorded in the Hospital Information System (HIS). To estimate monthly treatment cost of a drug, we used the prescription and dosing information from the NMPA-approved label. Monthly treatment costs were calculated over an average of 30 days on the basis of the dosage schedule for an adult patient weighing 60 kg with a body surface area of 1.70 m2. The cost of all regimes was adjusted to provide the price per 4-week period (33.3% increase for 3-week treatment cycles and 100% increase for 2-week treatment cycles). Drug prices were converted to US dollars at the exchange rate as of August 29, 2022.
To quantify the clinical benefit from the pivotal clinical trials supporting regulatory approval, we applied two value frameworks developed by ASCO and ESMO, namely the American Society of Clinical Oncology Value Framework (ASCO-VF) version 2 (6 (link)), and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) version 1.1 (8 (link)). Scores were assessed by one reviewer and checked by a second one, with any discrepancies resolved by a senior reviewer. In contrast to ESMO-MCBS, ASCO-VF was not planned to score single-arm studies and was therefore only suitable for phase II or III randomized clinical trials. In cases in which multiple pivotal clinical trials have been done and yield different clinical benefit scores for a given drug, the highest score was considered. Consistent with the developer of the value frameworks, meaningful clinical benefit was defined as a grade of A or B (for the curative setting) or 4, 5 (for the palliative setting) using ESMO-MCBS, whereas ASCO-VF did not clearly define what score was deemed “meaningful value.” Cherny et al. (14 (link)) recommended that the optimal threshold score of 45 or higher was proposed for recognizing substantial benefit for ASCO-VF by generating receiver operating characteristic (ROC) curves. Nevertheless, given the differences in construction and goals of ASCO-VF and ESMO-MCBS, they might yield some discordance in a cohort of studies. Thus, we split scores at the 75th percentile of ASCO-VF scores as the cutoff score for subsequent analyses, referring to the meaningful value achieved of ESMO-MCBS as a grade of 4, 5, B, or A (15 (link)).
Free full text: Click here
