Open Source Drug Discovery in Practice: A Case Study

BackgroundOpen source drug discovery offers potential for developing new and inexpensive drugs to combat diseases that disproportionally affect the poor. The concept borrows two principle aspects from open source computing (i.e., collaboration and open access) and applies them to pharmaceutical innovation. By opening a project to external contributors, its research capacity may increase significantly. To date there are only a handful of open source R&D projects focusing on neglected diseases. We wanted to learn from these first movers, their successes and failures, in order to generate a better understanding of how a much-discussed theoretical concept works in practice and may be implemented. Methodology/Principal FindingsA descriptive case study was performed, evaluating two specific R&D projects focused on neglected diseases. CSIR Team India Consortium's Open Source Drug Discovery project (CSIR OSDD) and The Synaptic Leap's Schistosomiasis project (TSLS). Data were gathered from four sources: interviews of participating members (n = 14), a survey of potential members (n = 61), an analysis of the websites and a literature review. Both cases have made significant achievements; however, they have done so in very different ways. CSIR OSDD encourages international collaboration, but its process facilitates contributions from mostly Indian researchers and students. Its processes are formal with each task being reviewed by a mentor (almost always offline) before a result is made public. TSLS, on the other hand, has attracted contributors internationally, albeit significantly fewer than CSIR OSDD. Both have obtained funding used to pay for access to facilities, physical resources and, at times, labor costs. TSLS releases its results into the public domain, whereas CSIR OSDD asserts ownership over its results. Conclusions/SignificanceTechnically TSLS is an open source project, whereas CSIR OSDD is a crowdsourced project. However, both have enabled high quality research at low cost. The critical success factors appear to be clearly defined entry points, transparency and funding to cover core material costs.

研究背景
开源药物发现（Open Source Drug Discovery）为开发新型廉价药物以对抗不成比例地影响贫困人群的疾病提供了可行路径。该理念借鉴了开源计算的两项核心要义——即协作与开放获取，并将其应用于药物研发创新。通过向外部贡献者开放项目，其研究能力可得到显著提升。截至目前，聚焦于被忽视疾病的开源研发项目仍寥寥无几。本研究旨在从这些先行者的成功与失败中汲取经验，以更深入地理解这一广受讨论的理论理念在实际中的运作模式与落地实施路径。

研究方法与主要发现
本研究采用描述性案例研究法，对两项聚焦被忽视疾病的特定研发项目进行评估：印度科学与工业研究理事会团队联盟的开源药物发现项目（CSIR Team India Consortium's Open Source Drug Discovery，简称CSIR OSDD）以及The Synaptic Leap的血吸虫病项目（The Synaptic Leap's Schistosomiasis project，简称TSLS）。研究数据来源于四个渠道：对14名项目参与者的访谈（n=14）、对61名潜在参与者的问卷调查（n=61）、项目网站内容分析以及文献综述。两项案例均取得了显著成果，但实现路径截然不同。CSIR OSDD鼓励国际协作，但其流程主要方便印度本土研究者与学生参与贡献。该项目流程较为规范，每项任务在成果公开前均需由导师（几乎均为线下开展）审核。相比之下，TSLS吸引了全球范围内的贡献者，但其总参与人数远少于CSIR OSDD。两项项目均已获得资助，用于支付设施使用、实体资源以及部分人力成本。TSLS将研究成果公开至公共领域，而CSIR OSDD则主张对其研究成果拥有所有权。

结论与意义
从技术层面而言，TSLS属于开源项目，而CSIR OSDD则属于众包项目。然而，二者均以低成本实现了高质量研究。关键成功因素似乎包括明确的参与入口、透明度以及可覆盖核心物料成本的资助。