Blanchette Christopher M, Nunes Anthony P, Lin Nancy D, Mortimer Kathleen M, Noone Joshua, Tangirala Krishna, Johnston Stephen, Gutierrez Benjamin
University of North Carolina, Charlotte, NC, USA;
Optum Epidemiology, Waltham, MA, USA;
Drugs Context. 2015 Feb 10;4. doi: 10.7573/dic.212272. eCollection 2015.
Risk evaluation and mitigation strategies (REMS), as mandated by the US Food and Drug Administration (FDA) for medications with the potential for harm, are increasingly incorporating rigid protocols for patient evaluation, but little is known about compliance with these programs. Despite the inherent limitations, data on administrative claims may provide an opportunity to investigate adherence to these programs.
We assessed adherence to liver function test (LFT) requirements included in the REMS program for bosentan through use of administrative claims. Patients observed in the Optum Research Database who were initiators of bosentan from November 20, 2001 to March 31, 2013 were included. Adherence to LFTs was calculated using pharmacy claims for bosentan dispensation and medical claims for laboratory services, and was assessed at the time of drug initiation and within specified time intervals throughout follow-up.
Of 742 patients, 523 (70.5%) had ≥1 qualifying LFT. Among patients with ≥12 dispensations, claims for LFTs at individual dispensations were 53.2-64.0%. Median proportion of dispensations with ≥1 LFT was 0.8 among patients with ≥6 (interquartile range, 0.7-1.0) or ≥12 (0.7-0.9) dispensations. Adherence was 90-100% for 33.3% of all initiators, whereas 29.3% of initiators were non-adherent (defined as <50% of on-therapy LFTs).
Analyses of administrative claims suggest that the REMS program for bosentan may not have adequately guaranteed adherence to the program's monthly monitoring of LFTs. Such investigations of existing REMS programs may provide insight on how to accomplish more successful evaluation of REMS.
美国食品药品监督管理局（FDA）要求对具有潜在危害的药物实施风险评估和缓解策略（REMS），此类策略越来越多地纳入严格的患者评估方案，但对于这些方案的依从性了解甚少。尽管存在固有局限性，但行政索赔数据可能为调查对这些方案的依从性提供契机。
我们通过行政索赔评估了波生坦REMS方案中肝功能测试（LFT）要求的依从性。纳入了在Optum研究数据库中观察到的、于2001年11月20日至2013年3月31日开始使用波生坦的患者。使用波生坦配药的药房索赔和实验室服务的医疗索赔计算LFT的依从性，并在开始用药时以及整个随访期间的特定时间间隔内进行评估。
在742例患者中，523例（70.5%）进行了≥1次符合要求的LFT。在有≥12次配药的患者中，每次配药时LFT的索赔率为53.2% - 64.0%。在有≥6次（四分位间距，0.7 - 1.0）或≥12次（0.7 - 0.9）配药的患者中，有≥1次LFT的配药的中位数比例为0.8。所有开始用药者中有33.3%的依从性为90% - 100%，而29.3%的开始用药者不依从（定义为治疗期间LFT的<50%）。
行政索赔分析表明，波生坦的REMS方案可能未充分保证对该方案每月LFT监测的依从性。对现有REMS方案的此类调查可能为如何更成功地评估REMS提供见解。
