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.
醫藥公司、研究人員和患者團體對新的歐盟法規提出了關切，認爲這可能會妨礙罕見病的治療方法的發展。
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.
這些新規則將於明年1月開始生效，對醫學試驗施加了更嚴格的指導方針，尤其是限制單臂試驗的使用，而這些試驗在罕見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.
包括歐洲癌症患者、歐洲泌尿學會和世界血友病聯合會在內的近40個組織敦促布魯塞爾修改這些規定。
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.
《金融時報》指出，單臂試驗非常重要，因爲所有患者都接受實驗性治療，沒有對照組，這對於治療罕見病通常影響較少的患者來說至關重要。
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.
根據再生醫學聯盟公共事務歐洲副總裁Paolo Morgese表示，隨機對照試驗對於罕見病來說往往過於昂貴和不道德。
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強調，新指導方針可能會妨礙歐盟迅速爲患者提供轉化性療法，並可能阻止某些成員國獲得這些治療方法。
The introduction of these guidelines comes amid growing interest in innovative cell and gene therapies, which have seen significant global growth.
這些指導方針的出現正值細胞和基因治療領域的創新興趣日益增長。
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報告援引IQVIA的數據指出，到2023年底，全球已經推出了76種細胞和基因治療方法，是10年前數字的兩倍。
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的分析顯示，約80%在歐洲批准並提供的先進療法依賴於單臂研究，突顯了這種試驗方法的重要性。
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.
儘管歐盟委員會認爲這些指南將使授權藥物在成員國之間更快、更協調地獲得，但批評者認爲單臂試驗限制可能會扼殺罕見病治療方面的創新。
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.
從1月12日起適用於癌症和先進療法，2030年適用於所有藥物的《健康技術評估法規》並未禁止單臂試驗，但行業對其嚴格的實施指導方針表示擔憂。
Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.
免責聲明：本內容部分使用人工智能工具生成，並經Benzinga編輯審核發佈。
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Image credit: Dall-E 3
圖片來源:Dall-E 3