School of Health Policy and Management, York University, 4700 Keele Street, Toronto, ON M3J 1P3, Canada.
Aust Health Rev. 2023 Apr;47(2):203-207. doi: 10.1071/AH22280.
Objective To examine the potential therapeutic value of new medicines approved in the US and both approved and not approved in Australia. Methods A list of new medicines approved by the US Food and Drug Administration (FDA) between 1 January 2015 and 31 December 2020 was assembled and it was determined which of these medicines were also approved in Australia. Three metrics - first in class, priority review and therapeutic rating by two independent organisations - were used to determine the potential therapeutic value of the medicines. The percent of medicines with and without potential significant therapeutic value was compared using each of the three metrics. Results A total of 273 drugs were approved by the FDA, of which 147 (53.8%) were approved by the Therapeutic Goods Administration, the Australian regulator. For each of these three metrics, the percent of medicines with and without potential significant therapeutic value approved in Australia was the same: first in class (yes vs no: Chi-squared P = 0.8562), priority review (yes vs no: Chi-squared P = 0.4593), therapeutic rating (major/moderate vs little/no: Chi-squared P = 0.9006). Some of the 126 drugs not approved may be therapeutically important. Conclusions New medicines approved in the US between 2015 and 2020 without potential significant therapeutic value are as likely to be introduced into Australia as drugs with potential significant therapeutic value. Some potentially valuable drugs may not have been submitted for approval in Australia by the companies making them.
目的 考察美国批准和澳大拉西亚（澳大利亚和新西兰）批准和未批准的新药的潜在治疗价值。
方法 收集了美国食品和药物管理局（FDA）在 2015 年 1 月 1 日至 2020 年 12 月 31 日期间批准的新药清单，并确定了这些药物中哪些也在澳大利亚获得批准。使用三个指标——首创性、优先审查和两个独立组织的治疗评级——来确定药物的潜在治疗价值。使用这三个指标中的每一个来比较具有和不具有潜在重要治疗价值的药物的百分比。
结果 共有 273 种药物被 FDA 批准，其中 147 种（53.8%）被澳大利亚监管机构治疗商品管理局批准。对于这三个指标中的每一个，具有和不具有潜在重要治疗价值的药物在澳大利亚的批准比例相同：首创性（是与否：卡方检验 P=0.8562）、优先审查（是与否：卡方检验 P=0.4593）、治疗评级（主要/中度与小/无：卡方检验 P=0.9006）。未批准的 126 种药物中的一些可能具有治疗意义。
结论 在 2015 年至 2020 年期间，美国批准的没有潜在重要治疗价值的新药与具有潜在重要治疗价值的药物一样有可能被引入澳大利亚。一些潜在有价值的药物可能没有被生产它们的公司提交给澳大利亚批准。
