Open Source Drug Discovery in Practice: A Case Study

Background: Open 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 Findings: A 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/Significance: Technically 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. Author Summary: Open source drug discovery can be an influential modelfor discovering and developing new medicines and diagnostics for neglected diseases. It offers the opportunityto accelerate the discovery progress while keeping expenditures to a minimum by encouraging incrementalcontributions from volunteer scientists. Publishing raw data and results in the public domain is positive within thecontext of neglected diseases since it facilitates open collaboration while obviating the ability to patent anyresults. In this way it effectively de-links the research and development costs from the sales price of the endproduct, the new medicine or diagnostic. This case study demonstrates that implementations of the open sourcemodel can differ while still achieving the ultimate goal of obtaining high quality research at reduced costs. However,the importance of clearly defined entry points, transparency and funding are shared success factors. Thesefindings present the practical challenges of implementing a theoretical concept and hopefully will assist otherscientists in organizing future open source drug discovery projects.