FDA局长在JPM放话：效率与国家优先！美国药物监管正在急速转向

Core Viewpoint - The article discusses the recent changes in FDA's drug approval processes under the leadership of Marty Makary, emphasizing the need for modernization and efficiency in the pharmaceutical industry, particularly in the context of competition with China [3][5][8]. Group 1: FDA's New Drug Approval Processes - The FDA is revising its drug approval logic, allowing a single pivotal clinical trial to suffice for registration if the statistical design is robust, moving away from the previous requirement of two trials [5]. - Makary opposes traditional animal testing, advocating for the use of computational models and organ-on-chip technologies instead, as many drugs that pass animal tests fail in humans [7]. - The FDA is breaking down the traditional phase I, II, III trial structure, allowing for continuous trials and Bayesian statistical methods, enabling real-time data assessment [7]. Group 2: Competition and Efficiency - The U.S. is facing competition in biomedicine from China, with Makary highlighting that the lag is not in technology but in institutional efficiency, particularly in the speed of early clinical trials [8]. - Makary is pushing for centralized IRB and standardized contract mechanisms to reduce bureaucratic delays in trial initiation [8][10]. Group 3: Drug Pricing and AI Regulation - Makary views GLP-1 drugs as important tools for managing metabolic diseases, advocating for lower drug prices in the U.S. compared to Europe, and promoting negotiations for better pricing [11]. - The FDA is working to expedite the approval of biosimilars and convert some prescription drugs to over-the-counter status to foster market competition [13]. - In the realm of AI in healthcare, Makary believes that traditional drug regulation methods should not apply, as this could stifle innovation; instead, a more flexible regulatory framework is needed [13]. Group 4: Vaccine Strategy - Makary is taking a measured approach to vaccines, aiming to restore public trust by prioritizing a core vaccine list rather than expanding recommendations indefinitely [16]. - He questions the one-size-fits-all policy for newborn hepatitis B vaccinations, advocating for a more nuanced approach based on risk assessment [16].