Runge Carlisle Ford, Campbell James, Runge Carlisle P
Department of Applied Economics, University of Minnesota, St. Paul, MN, United States.
Division of Health Policy and Management, University of Minnesota School of Public Health, Minneapolis, MN, United States.
Front Pharmacol. 2024 Dec 10;15:1516725. doi: 10.3389/fphar.2024.1516725. eCollection 2024.
Rare diseases affect over three hundred million individuals globally. Investment in research and development remains incommensurate with the challenges rare diseases pose. Further investment in information sharing platforms to promote common and standardized network technologies for rare disease is needed. Rare disease R&D generates information and assets that spill over in other ways, providing benefits that may not be apparent to investors Analytical and computational methods recently applied at scale are promising. One important way of achieving efficiencies of scale in R&D is clustering rare diseases into groups with similar traits.
全球有超过3亿人受罕见病影响。对研发的投入与罕见病带来的挑战仍不相称。需要进一步投资于信息共享平台,以推广用于罕见病的通用和标准化网络技术。罕见病研发产生的信息和资产会以其他方式外溢,带来投资者可能并不明显的益处。最近大规模应用的分析和计算方法很有前景。在研发中实现规模效率的一个重要方法是将罕见病聚类为具有相似特征的组。