Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA; School of Pharmacy, Utrecht University, Utrecht, the Netherlands.
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, the Netherlands.
Eur J Cancer. 2024 Nov;212:114330. doi: 10.1016/j.ejca.2024.114330. Epub 2024 Sep 12.
Cancer drugs initially granted regulatory approval for one disease may also prove useful in treating other diseases. In these cases, manufacturers may seek to add supplemental indications to their drug labels. The extent to which manufacturers seek supplemental indications for cancer drugs may differ in the US and EU.
To investigate how manufacturers have pursued supplemental indications for cancer drugs approved in the US vs. EU.
We performed a retrospective observational cohort study of cancer drugs first approved in the US and EU from 2005-2022 to compare the rate at which supplemental indications are added to drug labels and the times to first, second, and third supplemental indications.
Among 129 cancer drugs approved in both the US and EU from 2005-2022, approval occurred an average of 0.79 years earlier in the US. These drugs had a total of 145 indications at the time of approval in the US and 144 indications in the EU. Supplemental indications were granted at a rate of 0.22 per drug-year in the US, compared to 0.17 per drug-year in the EU (incidence rate ratio=1.30, 95 % confidence interval [CI]: 1.06-1.60, p = 0.01). This resulted in a total of 221 approved supplemental indications in the US and 152 approved supplemental indications in the EU through October 1, 2023. The times to the first and second supplemental indications were similar across the two jurisdictions, while the time to the third supplemental indication was shorter in the US (hazard ratio: 2.06, 95 % CI: 1.03-4.14, p = 0.04).
Manufacturers obtained supplemental indications for cancer drugs in the US at a higher rate than in the EU, which could be due to variable regulatory requirements and/or different market forces.
最初获得监管批准用于一种疾病的癌症药物也可能在治疗其他疾病方面证明有用。在这些情况下，制造商可能会寻求在其药物标签上添加补充适应症。在美国和欧盟，制造商为癌症药物寻求补充适应症的程度可能有所不同。
调查制造商在美国和欧盟批准的癌症药物中寻求补充适应症的情况。
我们对 2005 年至 2022 年期间首次在美国和欧盟批准的癌症药物进行了回顾性观察队列研究，以比较将补充适应症添加到药物标签的速度以及首次、第二次和第三次补充适应症的时间。
在 2005 年至 2022 年期间在美国和欧盟批准的 129 种癌症药物中，在美国的平均批准时间提前了 0.79 年。这些药物在美国获得批准时共有 145 种适应症，在欧盟获得批准时共有 144 种适应症。在美国，每药物年批准补充适应症的速度为 0.22 个，而在欧盟为 0.17 个/药物年（发病率比=1.30，95%置信区间[CI]：1.06-1.60，p=0.01）。这导致截至 2023 年 10 月 1 日，在美国共批准了 221 项补充适应症，在欧盟共批准了 152 项补充适应症。在这两个司法管辖区，获得第一个和第二个补充适应症的时间相似，而在美国获得第三个补充适应症的时间更短（风险比：2.06，95%CI：1.03-4.14，p=0.04）。
制造商在美国获得癌症药物补充适应症的速度高于欧盟，这可能是由于监管要求和/或不同的市场力量存在差异。
