Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, USA.
Value Health. 2018 Jul;21(7):809-821. doi: 10.1016/j.jval.2018.01.003. Epub 2018 Mar 21.
First, to quantify the median time from European Union (EU)-wide approval to first use (launch) for a sample of cancer medicines and number of launches in Belgium, Estonia, Scotland, and Sweden as of June 2015. Second, to assess whether longer times to launch or lack of launches affected medicines with high or low expected additional clinical benefit. Third, to identify possible determinants of the probability of a cancer medicine to be launched.
Correlation between time to launch and a set of variables hypothesized to affect launch was tested using a complementary log-log model for a sample of 46 cancer medicines that obtained EU-wide marketing authorization between 2000 and 2014.
In median, for a sample of 24 cancer medicines that obtained marketing authorization between 2010 and 2014, the expected time from EU-wide marketing authorization to first use of a medicine was shortest in Sweden, 3.1 months, followed by Scotland (9.3 months), Belgium (14.8 months), and Estonia (27.8 months). Median times to launch were longer for the entire sample of 46 cancer medicines that obtained marketing authorization between 2000 and 2014. In the all-country model, medicines with shorter times to submission for reimbursement, local manufacturers headquarter (or local sales representative), and a Food and Drugs Administration priority review or a combination of expedited approval programs and medicines launched in Scotland and Sweden were associated with a higher hazard of launch. Longer times since EU-wide approval initially correlate with an increased hazard but as time further elapses they negatively affect the hazard of launch.
Median times from marketing authorization to first use of cancer medicines were shorter for medicines launched between 2010 and 2014 versus sample-wide (2000-2014). In Estonia, more medicines than in the other countries were not yet launched at the end of the observation period. There was no correlation between Prescrire and European School of Medical Oncology Magnitude of Clinical Benefit Scale ratings of added clinical value and time to launch.
首先，量化一批癌症药物自欧盟（EU）批准上市至首次使用（投放市场）的中位时间，并统计截至 2015 年 6 月，比利时、爱沙尼亚、苏格兰和瑞典的投放市场数量。其次，评估投放市场的时间是否更长或缺乏投放市场是否会影响具有高或低预期附加临床效益的药物。第三，确定影响癌症药物投放市场的概率的可能决定因素。
使用互补对数 - 对数模型，对 2000 年至 2014 年间获得欧盟批准上市的 46 种癌症药物样本，检验了与投放市场时间相关的一系列假设变量的相关性。
在中位水平上，对于获得 2010 年至 2014 年期间上市许可的 24 种癌症药物样本，从欧盟批准上市至药物首次使用的预期时间最短的是瑞典，为 3.1 个月，其次是苏格兰（9.3 个月）、比利时（14.8 个月）和爱沙尼亚（27.8 个月）。获得 2000 年至 2014 年期间上市许可的 46 种癌症药物的总体样本中位时间更长。在所有国家的模型中，提交报销申请的时间较短、药物制造商总部（或当地销售代表）在本地、食品和药物管理局优先审查或加速审批计划和苏格兰、瑞典的联合药物与更高的投放市场风险相关。从欧盟批准上市后时间越短，初始风险增加，但随着时间的进一步推移，它们会对投放市场的风险产生负面影响。
与样本范围（2000-2014 年）相比，2010 年至 2014 年投放市场的癌症药物的营销授权至首次使用的中位时间更短。在爱沙尼亚，在观察期末，未投放市场的药物数量多于其他国家。Prescrire 和欧洲医学肿瘤学会临床获益量表的附加临床价值评分与投放市场时间之间没有相关性。
