2017年的癌症治疗:面对不稳定医疗体系的挑战,更多治疗方法的承诺

L. Bosserman
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引用次数: 0

摘要

过去的一年可能会被人们铭记为减轻癌症负担的突破性进展之一,美国食品和药物管理局(FDA)发表了一些具有里程碑意义的“研究成果”。值得注意的批准包括首个car(嵌合抗原受体t细胞)免疫疗法——用于b细胞前体急性淋巴细胞白血病的tisagenlecleucel (Kymriah)和用于复发或难治性大b细胞淋巴瘤的axicabtagene ciloleucel (Yescarta);美国批准的首个癌症生物仿制药贝伐单抗(bevacizumab- awb, Mvasi)用于多种癌症;neratinib (Nerlynx)作为早期人类表皮生长因子受体2 (HER2)过表达/扩增的乳腺癌的延伸辅助治疗,avelumab (Bavencio)用于转移性默克尔细胞癌的治疗。但是,我们对这些进步的兴奋无疑会被在扩大获得更优质医疗保健的机会、试点更有效的支付模式和巩固提供系统方面的持续挑战所冲淡。有效的生物、免疫和靶向治疗费用的迅速上涨也缓和了我们的兴奋。随着首个用于her2阳性乳腺癌和胃肠道癌症的靶向生物仿制药曲妥珠单抗-dkst (Ogivri)获批,我们可以期待随着时间的推移,这种具有显著临床疗效的靶向治疗的价格可能会下降20%-30%。我们知道,已获批的生物仿制药已经证明了临床有效性,并且在参考生物制剂中也可以看到类似的次要生物多样性我们也希望生物仿制药和参比化合物之间日益激烈的竞争将导致生产方法的改进,从而进一步降低价格,使更多的患者能够获得这些高效疗法。此外,FDA首次批准下一代测序(NGS) FoundationOne专业测试,以及医疗保险和医疗补助服务中心(CMS)迅速宣布将支付该测试的费用,这使我们更接近于了解哪些患者最有可能或不会从昂贵且有毒的靶向治疗中受益。随着许多临床试验研究哪些突变预测个体或靶向药物组合的哪些风险,批准和CMS覆盖政策将帮助我们提高对患者的价值;当我们能够推荐最有益的治疗方法,避免无效的治疗方法。最后,批准DigniCap头皮冷却系统用于所有实体肿瘤化疗患者是非常重要的。在保险覆盖范围尚未确定的情况下,这可能会影响一些患者接受化疗,否则他们可能会放弃化疗以避免脱发(另见第346-e348页)。
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Cancer care in 2017: the promise of more cures with the challenges of an unstable health care system
This past year will likely be remembered as one of breakthrough advances in reducing the burden of cancer, with some landmark “rsts” coming out of the US Food and Drug Administration (FDA). Among the notable approvals were the rst CART [chimeric antigen receptor T-cell] immunotherapies – tisagenlecleucel (Kymriah) for B-cell precursor acute lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for relapsed or refractory large B-cell lymphoma; the rst US-approved biosimilar for cancer, bevacizumab-awwb (Mvasi) for multiple types of cancer; and rst-time approvals for neratinib (Nerlynx) as an extended adjuvant therapy for early-stage human epidermal growth factor receptor 2 (HER2)-overexpressed/amplied breast cancer, and avelumab (Bavencio) for the treatment of metastatic Merkel cell carcinoma. But our excitement about those advances will undoubtedly be tempered by the continued challenges in expanding access to better quality health care, piloting more e™ective payment models, and consolidating delivery systems. Our excitement has also been tempered by the rapid rise in the cost of e™ective biologic, immunologic, and targeted therapies. With the approval of trastuzumab-dkst (Ogivri), the rst targeted biosimilar for HER2-positive breast and gastrointestinal cancers, we can look forward to price decreases possibly in the 20%-30% range over time from a targeted therapy with remarkable clinical eŸcacy. We know that approved biosimilars have demonstrated clinical eŸcacy along with similar minor biologic diversity that is also seen in the reference biologic.1 We can also hope that increasing competition among biosimilar and reference compounds will lead to improvements in production methodologies that can allow further price reductions so that even more patients can gain access to these highly e™ective therapies. In addition, the rst FDA approval for the next-generation sequencing (NGS) FoundationOne proling test and the rapid announcement by the Centers for Medicare & Medicaid Services (CMS) that it will cover the cost of that testing brings us a step closer to knowing which patients most likely will or won’t benet from costly and toxic targeted therapies. Along with the many clinical trials studying which mutations predict which eŸcacies of individual or combinations of targeted agents, the approval and CMS coverage policy will help us improve value to our patients; when we can recommend the most benecial therapies and avoid futile ones. Finally, the approval for the DigniCap Scalp Cooling System for patients on chemotherapy for all solid tumors is of great importance. Pending coverage availability, it may in¦uence some patients to get chemotherapy they might otherwise have forgone to avoid hair loss (see also pp. e346-e348).
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