2026-04-01 12:34 PM UTC / 路透社
路透社
2026年4月1日 12:34 UTC 更新于9分钟前
- 信件已送交特朗普总统、卫生与公众服务部部长肯尼迪、医疗保险局局长奥兹以及FDA局长马卡里
- 联盟指出FDA在罕见病药物审批方面灵活性下降
- 三分之二受访生物科技公司报告融资困难
4月1日（路透社）——周三，一个罕见病倡导联盟敦促特朗普政府恢复监管清晰度，目前美国食品药品监督管理局生物制品评估和研究中心（CBER）正考虑新的领导层人选。
该中心现任负责人维奈·普拉萨德博士将于4月底离任，其任期内围绕多款疫苗的审评引发了备受关注的争议，其中包括莫德纳的新冠疫苗、uniQure公司治疗亨廷顿舞蹈症的基因疗法以及其他罕见病药物。
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罕见病倡导、生物技术与投资者联盟已致信唐纳德·特朗普总统、美国卫生与公众服务部部长小罗伯特·F·肯尼迪、美国医疗保险局局长穆罕默德·奥兹以及FDA局长马蒂·马卡里。
该联盟包含近100家罕见病患者倡导团体、生物科技高管和投资者，他们表示，CBER在监管罕见病临床试验方面的灵活性已有所下降。
该联盟称，接受RDBI调查的生物科技投资者中，有84%因近期FDA监管的不确定性而缩减、暂停或退出了罕见病领域投资。约三分之二受访生物科技公司表示，在过去12个月里，这种不确定性使其融资难度加大。
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“我们认为，FDA选择一位理解罕见病研发独特挑战、尊重并重视患者与医生观点的负责人至关重要，”该联盟在信中表示。
2025年，CBER批准了5款孤儿药，同时发出了4封完整回应函，在上市前申请阶段也遭遇了一次类似挫折，否决了约一半的后期研发项目；而在此前两年的20个项目中，仅出现过1封完整回应函。
2026年第一季度，CBER仅批准了1款孤儿药并发出2封完整回应函，而同期FDA药物评估中心的获批数量为8款，完整回应函数量为2封。
如果FDA认为当前版本的申请无法获得批准，就会发出完整回应函。
斯里帕娜·罗伊在班加罗尔报道；塔西姆·扎希德编辑
我们的准则：汤森路透信托原则。
Rare disease advocacy group urges Trump administration to restore FDA clarity
2026-04-01 12:34 PM UTC / Reuters
By Reuters
April 1, 2026 12:34 PM UTC Updated 9 mins ago
Signage is seen outside of the Food and Drug Administration headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo
- Letter sent to President Trump, Health Secretary Kennedy, Medicare Chief Oz, and FDA Commissioner Makary
- Coalition cites FDA’s reduced flexibility in rare disease drug approvals
- Two-thirds of surveyed biotech firms report fundraising challenges
April 1 (Reuters) – A rare disease advocacy coalition on Wednesday urged the Trump administration to restore regulatory clarity as it considers new leadership at the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research.
Dr. Vinay Prasad, the center’s current incumbent, is set to leave the FDA at the end of April after a tenure marked by high-profile disputes over reviews for vaccines, including Moderna’s COVID shot, gene therapies such as uniQure’s therapy for Huntington’s disease and other rare disease drugs.
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The Rare Disease Advocacy, Biotechnology, and Investor Coalition sent the letter to President Donald Trump, U.S. Health Secretary Robert F. Kennedy Jr, Mehmet Oz, head of the U.S. Medicare agency and FDA Commissioner Marty Makary.
The coalition, which includes nearly 100 rare disease patient advocacy groups, biotech executives and investors, said CBER has become less flexible in overseeing rare disease clinical trials.
The group said 84% of biotech investors surveyed by RDBI had reduced, paused or exited rare disease investments because of recent FDA uncertainty. About two-thirds of biotech companies surveyed said the uncertainty had made it harder to raise capital over the past 12 months.
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“We believe it is of the utmost importance that the FDA chooses a leader who understands the unique challenges of rare disease development and respects and values the views of patients and physicians,” the coalition said in the letter.
CBER approved five orphan drugs in 2025 while issuing four Complete Response Letters and one comparable setback at the pre-marketing application stage, rejecting about half of late-stage programs, compared with one CRL among 20 programs over the prior two years.
In the first quarter of 2026, CBER approved one orphan drug and issued two CRLs, compared with eight approvals and two CRLs at the FDA’s drug evaluation center over the same period.
CRLs are sent by the FDA if the agency determines it will not approve the application in its current form.
Reporting by Sriparna Roy in Bengaluru; Editing by Tasim Zahid
Our Standards: The Thomson Reuters Trust Principles.
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