New European Regulations Could Hinder Rare Disease Treatments, Pharma Industry Raises Concerns Over Stricter Clinical Trial Rules
New European Regulations Could Hinder Rare Disease Treatments, Pharma Industry Raises Concerns Over Stricter Clinical Trial Rules
Pharmaceutical companies, researchers, and patient groups are raising concerns about new European Union regulations that could hinder the development of treatments for rare diseases.
医药公司、研究人员和患者团体对新的欧盟法规提出了关切，认为这可能会妨碍罕见病的治疗方法的发展。
The new rules, set to take effect in January, impose stricter guidelines on medical trials, particularly limiting the use of single-arm trials, which are often essential for rare disease research.
这些新规则将于明年1月开始生效，对医学试验施加了更严格的指导方针，尤其是限制单臂试验的使用，而这些试验在罕见病研究中通常是必需的。
Nearly 40 organizations, including Cancer Patients Europe, the European Association of Urology, and the World Federation of Hemophilia, have urged Brussels to revise these regulations.
包括欧洲癌症患者、欧洲泌尿学会和世界血友病联合会在内的近40个组织敦促布鲁塞尔修改这些规定。
The Financial Times highlights that single-arm trials, in which all patients receive the experimental treatment without a control group, are vital for developing therapies for rare diseases, which typically affect few patients.
《金融时报》指出，单臂试验非常重要，因为所有患者都接受实验性治疗，没有对照组，这对于治疗罕见病通常影响较少的患者来说至关重要。
According to Paolo Morgese, Europe Vice-President of Public Affairs at the Alliance for Regenerative Medicine (ARM), randomizing control trials is often too expensive and unethical for rare diseases.
根据再生医学联盟公共事务欧洲副总裁Paolo Morgese表示，随机对照试验对于罕见病来说往往过于昂贵和不道德。
Morgese emphasized that the new guidelines could hinder the EU's ability to provide transformative therapies to patients quickly and may prevent access to these treatments in some member states.
Morgese强调，新指导方针可能会妨碍欧盟迅速为患者提供转化性疗法，并可能阻止某些成员国获得这些治疗方法。
The introduction of these guidelines comes amid growing interest in innovative cell and gene therapies, which have seen significant global growth.
这些指导方针的出现正值细胞和基因治疗领域的创新兴趣日益增长。
The FT report, citing data from IQVIA, notes that by the end of 2023, 76 cell and gene therapies had been launched globally, double the number from a decade earlier.
FT报告援引IQVIA的数据指出，到2023年底，全球已经推出了76种细胞和基因治疗方法，是10年前数字的两倍。
ARM's analysis revealed that about 80% of advanced therapies approved and available in the EU relied on single-arm studies, highlighting the importance of this trial method in the field.
ARM的分析显示，约80%在欧洲批准并提供的先进疗法依赖于单臂研究，突显了这种试验方法的重要性。
While the EU Commission argues that the guidelines will enable quicker and more coordinated authorization of medicines across member states, critics believe the restrictions on single-arm trials could stifle innovation in rare disease treatment.
尽管欧盟委员会认为这些指南将使授权药物在成员国之间更快、更协调地获得，但批评者认为单臂试验限制可能会扼杀罕见病治疗方面的创新。
The Regulation on Health Technology Assessment, which applies to cancer and advanced therapeutics from January 12 and to all medicines from 2030, does not prohibit single-arm trials, but the industry is concerned about the stringent implementation guidelines.
从1月12日起适用于癌症和先进疗法，2030年适用于所有药物的《健康技术评估法规》并未禁止单臂试验，但行业对其严格的实施指导方针表示担忧。
Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.
免责声明：本内容部分使用人工智能工具生成，并经Benzinga编辑审核发布。
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Image credit: Dall-E 3
图片来源:Dall-E 3