Paumgartten Francisco José Roma
Escola Nacional de Saúde Pública, Fiocruz. Av. Brasil 4036/913, Manguinhos. 21040-361. Rio de Janeiro, RJ, Brasil.
Cien Saude Colet. 2019 Sep 26;24(10):3783-3792. doi: 10.1590/1413-812320182410.28522017. eCollection 2019.
In April 2017, the National Sanitary Surveillance Agency (ANVISA-Brazil) approved lenalidomide (LEN) for multiple myeloma (MM) and myelodysplastic syndrome. ANVISA had rejected the first application in 2010, and denied a request for reconsideration in 2012. The reason for rejection was the lack of comparative effectiveness studies proving that LEN was more effective than thalidomide (THAL), a strictly controlled drug regulated by Federal law 10.651/2003 and dispensed to patients (at no costs) through public health system units and hospitals. ANVISA unexplained retreat on the LEN approval for marketing was an unquestionable triumph of the lobbying that ensued the denial, at the forefront of which were politicians, Congress members, patient organizations and medical societies. Two randomized (phase III) trials and three observational (case-control and population-based cohort) compared the effectiveness of THAL- versus LEN-based therapies in MM. Overall, these studies showed no difference in efficacy between LEN- and THAL-based therapies. LEN caused less neuropathy, and more severe hematologic adverse effects. It is much costlier than THAL, and substitution of THAL by LEN shall raise considerably public healthcare costs in Brazil.
2017年4月，巴西国家卫生监督局（ANVISA）批准来那度胺（LEN）用于治疗多发性骨髓瘤（MM）和骨髓增生异常综合征。ANVISA曾在2010年驳回了首次申请，并于2012年拒绝了复议请求。驳回的原因是缺乏比较有效性研究，无法证明LEN比沙利度胺（THAL）更有效，THAL是一种受联邦法律10.651/2003严格管制的药物，通过公共卫生系统单位和医院（免费）发放给患者。ANVISA在LEN上市批准问题上无缘无故的退缩，无疑是在遭到拒绝后游说活动的胜利，其中最积极的是政治家、国会议员、患者组织和医学协会。两项随机（III期）试验和三项观察性（病例对照和基于人群的队列）研究比较了基于THAL和基于LEN的疗法在MM中的有效性。总体而言，这些研究表明基于LEN和基于THAL的疗法在疗效上没有差异。LEN引起的神经病变较少，但血液学不良反应更严重。它比THAL贵得多，用LEN替代THAL将大幅提高巴西的公共医疗成本。
