Byun Ji-Hye, Kwon Sun-Hong, Ha Ji-Hye, Lee Eui-Kyung
School of Pharmacy, Sungkyunkwan University, Suwon-si, Gyeonggi-do, South Korea.
Ministry of Food and Drug Safety, Cheongju-si, Chungcheongbuk-do, South Korea.
Ther Clin Risk Manag. 2016 Jun 13;12:965-74. doi: 10.2147/TCRM.S100438. eCollection 2016.
The benefit-risk balance for drugs can alter post approval owing to additional data on efficacy or adverse events. This study developed a quantitative benefit-risk assessment (BRA) model for statins using multicriteria decision analysis with discrete choice experiments and compared a recent BRA with that at the time of approval.
Following a systematic review of the literature, the benefit criteria within the statin BRA model were defined as a reduction in the plasma low-density lipoprotein cholesterol level and a reduction in myocardial infarction incidence; the risk criteria were hepatotoxicity (Liv) and fatal rhabdomyolysis (Rha). The scores for these criteria were estimated using mixed treatment comparison methods. Weighting was calculated from a discrete choice experiment involving 203 Korean patients. The scores and weights were integrated to produce an overall value representing the benefit-risk balance, and sensitivity analyses were conducted.
In this BRA model, low-density lipoprotein (relative importance [RI]: 37.50%) was found to be a more important benefit criterion than myocardial infarction (RI: 35.43%), and Liv (RI: 16.28%) was a more important risk criterion than Rha (RI: 10.79%). Patients preferred atorvastatin, and the preference ranking of cerivastatin and simvastatin was switched post approval because of the emergence of additional risk information related to cerivastatin.
A quantitative statin BRA model confirmed that the preference ranking of statins changed post approval because of the identification of additional benefits or risks.
由于疗效或不良事件的额外数据，药物的效益风险平衡在批准后可能会改变。本研究使用离散选择实验的多标准决策分析方法，为他汀类药物开发了一种定量效益风险评估（BRA）模型，并将近期的BRA与批准时的进行了比较。
在对文献进行系统综述后，他汀类BRA模型中的效益标准被定义为血浆低密度脂蛋白胆固醇水平降低和心肌梗死发病率降低；风险标准为肝毒性（Liv）和致命性横纹肌溶解（Rha）。使用混合治疗比较方法估计这些标准的得分。权重通过涉及203名韩国患者的离散选择实验计算得出。将得分和权重整合以产生代表效益风险平衡的总体值，并进行敏感性分析。
在这个BRA模型中，发现低密度脂蛋白（相对重要性[RI]：37.50%）是比心肌梗死（RI：35.43%）更重要的效益标准，而Liv（RI：16.28%）是比Rha（RI：10.79%）更重要的风险标准。患者更喜欢阿托伐他汀，由于与西立伐他汀相关的额外风险信息出现，西立伐他汀和辛伐他汀的偏好排名在批准后发生了变化。
定量他汀类BRA模型证实，由于发现了额外的益处或风险，他汀类药物的偏好排名在批准后发生了变化。
