Kakkis EveryLife Foundation For Rare Diseases, 77 Digital Drive, Suite 210, Novato, CA 94949, USA.
Orphanet J Rare Dis. 2011 Jul 6;6:49. doi: 10.1186/1750-1172-6-49.
Over 95% of rare diseases lack treatments despite many successful treatment studies in animal models. To improve access to treatments, the Accelerated Approval (AA) regulations were implemented allowing the use of surrogate endpoints to achieve drug approval and accelerate development of life-saving therapies. Many rare diseases have not utilized AA due to the difficulty in gaining acceptance of novel surrogate endpoints in untreated rare diseases.
To assess the potential impact of improved AA accessibility, we devised clinical development programs using proposed clinical or surrogate endpoints for fifteen rare disease treatments.
We demonstrate that better AA access could reduce development costs by approximately 60%, increase investment value, and foster development of three times as many rare disease drugs for the same investment.
Our research brings attention to the need for well-defined and practical qualification criteria for the use of surrogate endpoints to allow more access to the AA approval pathway in clinical trials for rare diseases.
尽管在动物模型中有许多成功的治疗研究，但仍有超过 95%的罕见病缺乏治疗方法。为了改善治疗方法的可及性，加速批准（AA）法规得以实施，允许使用替代终点来实现药物批准，并加速救命疗法的开发。由于在未治疗的罕见病中难以接受新的替代终点，许多罕见病尚未利用 AA。
为了评估改善 AA 可及性的潜在影响，我们设计了使用十五种罕见病治疗方法的拟议临床或替代终点的临床开发计划。
我们表明，更好的 AA 可及性可以将开发成本降低约 60%，提高投资价值，并促进相同投资下三倍的罕见病药物开发。
我们的研究引起了人们对使用替代终点的明确和实用的资格标准的需求，以允许更多的罕见病临床试验利用 AA 批准途径。
