Association Between Food and Drug Administration Approval and Disparities in Immunotherapy Use Among Patients With Cancer in the US.
Association Between Food and Drug Administration Approval and Disparities in Immunotherapy Use Among Patients With Cancer in the US.
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DOI：
10.1001/jamanetworkopen.2022.19535
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发表时间：
2022-06-01
影响因子：
13.8
通讯作者：
Boffa, Daniel J.
中科院分区：
文献类型：
--
作者：
Ermer, Theresa;Canavan, Maureen E.;Maduka, Richard C.;Li, Andrew X.;Salazar, Michelle C.;Kaminski, Michael F.;Pichert, Matthew D.;Zhan, Peter L.;Mase, Vincent;Kluger, Harriet;Boffa, Daniel J.
美国食品药品监督管理局（FDA）的药物批准是否与美国癌症患者新型癌症治疗使用差异的减少有关？在这项对402689例IV期非小细胞肺癌、肾细胞癌和黑色素瘤患者的队列研究中，黑人种族、西班牙裔、医疗补助保险、缺乏保险和家庭收入较低等因素与FDA批准检查点抑制剂之前和之后免疫治疗使用频率显著降低相关。这项研究发现，在FDA批准之前和之后，美国的免疫疗法使用是异质性的，这表明FDA的批准可能会缩小一些差距，但不一定会消除新疗法使用的差异。临床试验和同情使用协议为选定的患者提供了在美国食品和药物管理局（FDA）批准之前获得潜在挽救生命的治疗的机会。FDA的批准减少了许多准入障碍;然而，目前尚不清楚FDA的批准是否与美国癌症患者公平使用新型疗法的增加和使用差异的减少有关。评估FDA药物批准与在美国批准首个检查点抑制剂治疗癌症患者之前和之后，健康、社会人口统计学和社会经济阶层中免疫治疗使用差异之间的相关性。这项队列研究使用来自国家癌症数据库的数据，在FDA批准第一种检查点抑制剂疗法之前和之后，检查了免疫疗法在健康，社会人口和社会经济阶层的使用。纳入了2007年1月1日至2018年12月31日（具体年份因肿瘤类型而异）期间诊断为IV期非小细胞肺癌（NSCLC）、肾细胞癌（RCC）或皮肤黑色素瘤的402689例20岁或以上患者。患者健康状况（Charlson-Deyo comorbid评分和年龄）、社会人口学特征（性别、种族和民族）和社会经济学（基于居住地邮政编码的保险状况和家庭收入）特征。在FDA批准前4年和批准后3年，使用多变量logistic回归模型评估患者特征与接受免疫治疗的相关性。在402689例患者中（中位[IQR]年龄为68 [60-76岁]; 225081例男性[55.9%]），347233例患者患有NSCLC，43714例患者患有RCC，11742例患者患有黑色素瘤。共有47 527例患者（11.8%）为黑人，15 763例（3.9%）为西班牙裔，375 874例（93.3%）为非西班牙裔，335 833例（83.4%）为白色，16 553例（4.1%）为其他种族。在FDA批准前，6271例NSCLC患者（3.2%）、1155例RCC患者（4.8%）和504例黑素瘤患者（8.6%）接受了免疫治疗，而在FDA批准后，23908例NSCLC患者（15.6%）、3890例RCC患者（19.7%）和1143例黑素瘤患者（19.3%）接受了免疫治疗。在FDA批准之前，社会人口统计学和社会经济学特征与肿瘤类型的可变免疫治疗相关。例如，在NSCLC患者中，黑人患者接受免疫治疗的可能性低于白色患者（比值比[OR]，0.78; 95%CI，0.71 -0.85; P <0.001）;在肾细胞癌患者中，未投保的患者接受免疫治疗的可能性低于私人投保的患者（OR，0.31; 95%CI，0.20-0.48; P < .001）。在FDA批准后，大多数差异仍然存在，但有几个缩小了（例如，黑人NSCLC患者：OR，0.87 [95%CI，0.83-0.91; P < .001];未投保的RCC患者：OR，0.60 [95%CI，0.48-0.75; P < .001]）。尽管仍存在许多差异，但社会经济特征之间的一些差距似乎有所扩大（例如，最低收入与最高收入四分位数的NSCLC患者：OR，0.80; 95% CI，0.76-0.83; P < .001），并出现了新的差距（例如，黑人RCC患者：OR，0.82; 95% CI，0.72-0.93; P = .003）。在这项队列研究中，在FDA批准之前，NSCLC、RCC和黑色素瘤患者的许多社会人口统计学和社会经济学特征中存在免疫治疗使用的差异，包括在临床试验增加患者的重要时期。尽管FDA的批准与免疫疗法的使用显著增加有关，但差距仍然存在，这表明FDA的批准可能不会消除新疗法使用中的差异。这项队列研究使用国家癌症数据库的数据来评估美国食品药品监督管理局（FDA）批准的药物与美国IV期非小细胞肺癌，肾细胞癌和黑色素瘤患者使用免疫治疗的差异之间的关联。
Is drug approval by the Food and Drug Administration (FDA) associated with a reduction in disparities in novel cancer treatment use among patients with cancer in the US? In this cohort study of 402 689 patients with stage IV non–small cell lung cancer, renal cell carcinoma, and melanoma, factors such as Black race, Hispanic ethnicity, Medicaid insurance, lack of insurance, and lower household income were associated with significantly less frequent immunotherapy use before and after checkpoint inhibitors were approved by the FDA. This study found that immunotherapy use in the US before and after FDA approval was heterogeneous, suggesting that FDA approval may narrow some gaps but not necessarily eliminate disparities in the use of novel therapies. Clinical trials and compassionate use agreements provide selected patients with access to potentially life-saving treatments before approval by the Food and Drug Administration (FDA). Approval from the FDA decreases a number of access barriers; however, it is unknown whether FDA approval is associated with increases in the equitable use of novel therapies and reductions in disparities in use among patients with cancer in the US. To assess the association between FDA drug approval and disparities in the use of immunotherapy across health, sociodemographic, and socioeconomic strata before and after approval of the first checkpoint inhibitors for the treatment of patients with cancer in the US. This cohort study used data from the National Cancer Database to examine the use of immunotherapy across health, sociodemographic, and socioeconomic strata before and after FDA approval of the first checkpoint inhibitor therapies. A total of 402 689 patients 20 years or older who were diagnosed with stage IV non–small cell lung cancer (NSCLC), renal cell carcinoma (RCC), or melanoma of the skin between January 1, 2007, and December 31, 2018 (specific years varied by tumor type), were included. Patient health (Charlson-Deyo comorbidity score and age), sociodemographic characteristics (sex, race, and ethnicity), and socioeconomic (insurance status and household income based on zip code of residence) characteristics. The association of patient characteristics with receipt of immunotherapy was evaluated in the 4 years before and the 3 years immediately after FDA approval using multivariable logistic regression modeling. Among 402 689 patients (median [IQR] age, 68 [60-76 years]; 225 081 men [55.9%]), 347 233 had NSCLC, 43 714 had RCC, and 11 742 patients had melanoma. A total of 47 527 patients (11.8%) were Black, 15 763 (3.9%) were Hispanic, 375 874 (93.3%) were non-Hispanic, 335 833 (83.4%) were White, and 16 553 (4.1%) were of other races. Before FDA approval, 6271 patients (3.2%) with NSCLC, 1155 patients (4.8%) with RCC, and 504 patients (8.6%) with melanoma received immunotherapy compared with 23 908 patients (15.6%) with NSCLC, 3890 patients (19.7%) with RCC, and 1143 patients (19.3%) with melanoma after FDA approval. Before FDA approval, sociodemographic and socioeconomic characteristics were associated with variable immunotherapy administration by tumor type. For example, among those with NSCLC, Black patients were less likely to receive immunotherapy than White patients (odds ratio [OR], 0.78; 95% CI ,0.71-0.85; P < .001); among those with RCC, uninsured patients were less likely to receive immunotherapy than privately insured patients (OR, 0.31; 95% CI, 0.20-0.48; P < .001). After FDA approval, most disparities persisted, but several narrowed (eg, Black patients with NSCLC: OR, 0.87 [95% CI, 0.83-0.91; P < .001]; uninsured patients with RCC: OR, 0.60 [95% CI, 0.48-0.75; P < .001]). Although many disparities remained, some gaps across socioeconomic characteristics appeared to widen (eg, patients with NSCLC in the lowest vs highest income quartile: OR, 0.80; 95% CI, 0.76-0.83; P < .001), and new gaps emerged (eg, Black patients with RCC: OR, 0.82; 95% CI, 0.72-0.93; P = .003). In this cohort study, disparities in immunotherapy use existed across a number of sociodemographic and socioeconomic characteristics among patients with NSCLC, RCC, and melanoma before FDA approval, including during the important period when clinical trials were accruing patients. Although FDA approval was associated with a significant increase in the use of immunotherapy, gaps persisted, suggesting that FDA approval may not eliminate disparities in the use of novel therapies. This cohort study uses data from the National Cancer Database to assess the association between drug approval by the Food and Drug Administration (FDA) and disparities in the use of immunotherapy among patients with stage IV non–small cell lung cancer, renal cell carcinoma, and melanoma in the US.
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DOI：
10.1200/jco.20.01605
发表时间：
2021-03-01
期刊：
Journal of clinical oncology : official journal of the American Society of Clinical Oncology
影响因子：
--
作者：
Borghaei H;Gettinger S;Vokes EE;Chow LQM;Burgio MA;de Castro Carpeno J;Pluzanski A;Arrieta O;Frontera OA;Chiari R;Butts C;Wójcik-Tomaszewska J;Coudert B;Garassino MC;Ready N;Felip E;García MA;Waterhouse D;Domine M;Barlesi F;Antonia S;Wohlleber M;Gerber DE;Czyzewicz G;Spigel DR;Crino L;Eberhardt WEE;Li A;Marimuthu S;Brahmer J
通讯作者：
Brahmer J
DOI：
10.1634/theoncologist.2015-0507
发表时间：
2016-05
期刊：
The oncologist
影响因子：
--
作者：
Kazandjian D;Suzman DL;Blumenthal G;Mushti S;He K;Libeg M;Keegan P;Pazdur R
通讯作者：
Pazdur R
影响因子：
3.2
作者：
Aristizabal, Paula;Singer, Jenelle;Martinez, Maria E.
通讯作者：
Martinez, Maria E.
影响因子：
10.9
作者：
Krimphove, Marieke J.;Tully, Karl H.;Trinh, Quoc-Dien
通讯作者：
Trinh, Quoc-Dien
影响因子：
158.5
作者：
Larkin, J.;Chiarion-Sileni, V.;Wolchok, J. D.
通讯作者：
Wolchok, J. D.