The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases.
The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases.
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DOI：
10.1186/1750-1172-6-49
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发表时间：
2011-07-06
影响因子：
3.7
通讯作者：
Kakkis ED
中科院分区：
文献类型：
--
作者：
Miyamoto BE;Kakkis ED
超过95%的罕见疾病缺乏治疗，尽管在动物模型中进行了许多成功的治疗研究。为了改善获得治疗的机会，实施了加速批准（AA）法规，允许使用替代终点来实现药物批准并加速救生疗法的开发。许多罕见病尚未使用AA，因为在未经治疗的罕见病中难以接受新的替代终点。为了评估改善AA可及性的潜在影响，我们使用15种罕见疾病治疗的拟定临床或替代终点设计了临床开发项目。我们证明，更好的AA准入可以将开发成本降低约60%，增加投资价值，并促进开发三倍于相同投资的罕见病药物。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关注，以允许在罕见疾病的临床试验中更多地获得AA批准途径。
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.
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