PDF(Pubmed)
Abstract：
UNASSIGNED: Many cancer drugs are approved under the US Food and Drug Administration (FDA) accelerated approval pathway based on preliminary evidence. It is unclear how this limited evidence is integrated into the National Comprehensive Cancer Network (NCCN) guidelines, which are common references for clinicians and are used by public and private payers to determine reimbursement for oncology treatments.
UNASSIGNED: To analyze the NCCN guidelines\' assessments for cancer drug indications that received FDA accelerated approval compared with cancer drug indications that received FDA regular approval.
UNASSIGNED: This cross-sectional study analyzes FDA-approved indications for cancer drugs that were granted accelerated approval from program inception in 1992 to June 30, 2022. For each drug, the FDA-approved labeling was reviewed to identify all indications. All analyses were performed at the drug-indication level.
UNASSIGNED: The exposure was FDA regulatory status as of October 2022, including regular approval, accelerated approval, accelerated approval converted to regular approval, and withdrawn accelerated approval.
UNASSIGNED: The level of evidence and consensus (category 1, 2A, 2B, and 3) and treatment preference (preferred, alternative preferred, other recommended, and useful in certain circumstances) ratings assigned by NCCN committees as of February 2023.
UNASSIGNED: A total of 315 oncology indications for 100 drugs were analyzed. These indications included 156 (50%) with regular approval, 60 (38%) with accelerated approval, 78 (49%) with accelerated approval that was converted to regular approval, and 21 (13%) with withdrawn accelerated approvals. Among all indications, 105 (33%) were rated by the NCCN as having category 1 evidence, 185 (59%) with category 2A, 6 (2%) with category 2B, and 2 (1%) with category 3 evidence. Compared with indications with regular approval, those with accelerated approval were less frequently assigned category 1 evidence (47% vs 3%; P < .001) and were less often listed as preferred treatment options (58% vs 40%; P = .008). Among the 21 withdrawn accelerated approval indications, 8 (38%) remained in the NCCN guidelines, with most having level 2A evidence ratings.
UNASSIGNED: This study found that cancer drug indications with accelerated approval were less likely to be assigned high-level evidence ratings and preferred status in the NCCN guidelines compared with indications with regular approval; most accelerated and regular approval drugs had low-quality evidence ratings but high levels of consensus among oncologists on NCCN committees. Greater clarity on the thresholds and definitions of evidence levels would make the NCCN guidelines more useful to clinicians, patients, and payers.
UNASSIGNED: To analyze the NCCN guidelines\' assessments for cancer drug indications that received FDA accelerated approval compared with cancer drug indications that received FDA regular approval.
UNASSIGNED: This cross-sectional study analyzes FDA-approved indications for cancer drugs that were granted accelerated approval from program inception in 1992 to June 30, 2022. For each drug, the FDA-approved labeling was reviewed to identify all indications. All analyses were performed at the drug-indication level.
UNASSIGNED: The exposure was FDA regulatory status as of October 2022, including regular approval, accelerated approval, accelerated approval converted to regular approval, and withdrawn accelerated approval.
UNASSIGNED: The level of evidence and consensus (category 1, 2A, 2B, and 3) and treatment preference (preferred, alternative preferred, other recommended, and useful in certain circumstances) ratings assigned by NCCN committees as of February 2023.
UNASSIGNED: A total of 315 oncology indications for 100 drugs were analyzed. These indications included 156 (50%) with regular approval, 60 (38%) with accelerated approval, 78 (49%) with accelerated approval that was converted to regular approval, and 21 (13%) with withdrawn accelerated approvals. Among all indications, 105 (33%) were rated by the NCCN as having category 1 evidence, 185 (59%) with category 2A, 6 (2%) with category 2B, and 2 (1%) with category 3 evidence. Compared with indications with regular approval, those with accelerated approval were less frequently assigned category 1 evidence (47% vs 3%; P < .001) and were less often listed as preferred treatment options (58% vs 40%; P = .008). Among the 21 withdrawn accelerated approval indications, 8 (38%) remained in the NCCN guidelines, with most having level 2A evidence ratings.
UNASSIGNED: This study found that cancer drug indications with accelerated approval were less likely to be assigned high-level evidence ratings and preferred status in the NCCN guidelines compared with indications with regular approval; most accelerated and regular approval drugs had low-quality evidence ratings but high levels of consensus among oncologists on NCCN committees. Greater clarity on the thresholds and definitions of evidence levels would make the NCCN guidelines more useful to clinicians, patients, and payers.
摘要：
■根据初步证据，许多癌症药物是根据美国食品和药物管理局（FDA）加速批准途径批准的。目前尚不清楚这些有限的证据是如何被整合到国家综合癌症网络（NCCN）指南中的。这是临床医生的常用参考，公共和私人付款人用于确定肿瘤治疗的报销。
■分析获得FDA加速批准的癌症药物适应症与获得FDA定期批准的癌症药物适应症的NCCN指南评估。
■这项横断面研究分析了FDA批准的癌症药物的适应症，这些药物从1992年计划开始到2022年6月30日获得了加速批准。对于每种药物，我们审查了FDA批准的标签,以确定所有适应症.所有分析均在药物适应症水平进行。
■截至2022年10月，暴露量为FDA监管状态，包括定期批准，加速审批,加速审批转换为定期审批，撤销加速审批。
■证据和共识的水平（类别1，2A，2B,和3)和治疗偏好(首选，替代首选，其他推荐,并且在某些情况下有用)截至2023年2月由NCCN委员会分配的评级。
■共分析了100种药物的315种肿瘤学适应症。这些适应症包括156（50％）经定期批准，60（38%），加速批准，78(49%)，加速批准，转换为定期批准，21人（13%）被撤销加速审批。在所有适应症中，105人(33%)被NCCN评为有1类证据，185（59%），2A类，6（2%），2B类，和2（1％）与第3类证据。与常规批准的适应症相比，那些获得加速批准的患者的1类证据分配频率较低(47%vs3%;P<.001),并且被列为首选治疗方案的频率较低(58%vs40%;P=.008).在21个撤回的加速批准适应症中，8(38%)留在NCCN指南中，大多数具有2A级证据评级。
这项研究发现，与常规批准的适应症相比，加速批准的癌症药物适应症在NCCN指南中被赋予高水平的证据评级和首选状态的可能性较小；大多数加速和常规批准的药物具有低质量的证据评级，但NCCN委员会的肿瘤学家之间的共识较高。证据水平的阈值和定义更加清晰，将使NCCN指南对临床医生更有用，病人,和付款人。
■分析获得FDA加速批准的癌症药物适应症与获得FDA定期批准的癌症药物适应症的NCCN指南评估。
■这项横断面研究分析了FDA批准的癌症药物的适应症，这些药物从1992年计划开始到2022年6月30日获得了加速批准。对于每种药物，我们审查了FDA批准的标签,以确定所有适应症.所有分析均在药物适应症水平进行。
■截至2022年10月，暴露量为FDA监管状态，包括定期批准，加速审批,加速审批转换为定期审批，撤销加速审批。
■证据和共识的水平（类别1，2A，2B,和3)和治疗偏好(首选，替代首选，其他推荐,并且在某些情况下有用)截至2023年2月由NCCN委员会分配的评级。
■共分析了100种药物的315种肿瘤学适应症。这些适应症包括156（50％）经定期批准，60（38%），加速批准，78(49%)，加速批准，转换为定期批准，21人（13%）被撤销加速审批。在所有适应症中，105人(33%)被NCCN评为有1类证据，185（59%），2A类，6（2%），2B类，和2（1％）与第3类证据。与常规批准的适应症相比，那些获得加速批准的患者的1类证据分配频率较低(47%vs3%;P<.001),并且被列为首选治疗方案的频率较低(58%vs40%;P=.008).在21个撤回的加速批准适应症中，8(38%)留在NCCN指南中，大多数具有2A级证据评级。
这项研究发现，与常规批准的适应症相比，加速批准的癌症药物适应症在NCCN指南中被赋予高水平的证据评级和首选状态的可能性较小；大多数加速和常规批准的药物具有低质量的证据评级，但NCCN委员会的肿瘤学家之间的共识较高。证据水平的阈值和定义更加清晰，将使NCCN指南对临床医生更有用，病人,和付款人。
