The successes and challenges of open-source biopharmaceutical innovation

Figures & data

Table 1. The state of affairs for current open-source biopharmaceutical initiatives.

Open-source model	Sponsoring organization(s)	Focus
SGC	University of Oxford, University of Toronto	The SGC is engaged in precompetitive research to facilitate the discovery of new medicines. As part of its mission the SGC is generating reagents and knowledge related to human proteins and proteins from human parasites. The SGC believes that its output will have maximal benefit if released into the public domain without restriction on use, and thus has adopted an open-access policy.
Pistoia Alliance	AstraZeneca, GSK, Novartis, Pfizer	Pistoia Alliance members aim to lower barriers to innovation by improving the interoperability of R&D business processes through precompetitive collaboration. The organization focuses on bringing stakeholders to the table to define common, conceptual steps of the life science R&D workflow.
Open PHACTS	Innovative Medicines Initiative, Open PHACTS Foundation	The Open PHACTS Discovery Platform provides tools and services to interact with multiple integrated and publicly available data sources. To integrate these data, extensive cross-referencing of scientific concepts is needed across all databases.
Eli-Lilly PD2-collaborative compound analysis	Eli Lilly	Called the Lilly Phenotypic Drug Discovery Initiative, or PD2 (PD-squared), the portal employs Lilly’s disease-state assays to evaluate compounds synthesized at university and biotech labs around the globe. After biological testing is completed, Lilly provides the outside researchers a data report including a complete biological profile of the compound across four assay modules: Alzheimer’s disease, cancer, diabetes and osteoporosis. Lilly indicates that the in vitro model systems will provide researchers with broader assessments of a compound’s biological profile than what is generally available today in academic or government laboratories. In return for the data, Lilly gains first rights to exclusively negotiate a collaboration or licensing agreement with researchers who submit their compounds.
Eli-Lilly TargetD2 initiative-collaborative target analysis	Eli Lilly	With the implementation of the TargetD2 initiative, Lilly has expanded access to a panel of well-validated target-based assays plus relevant computational methods that will allow participants to engage in meaningful structure design cycles. The TargetD2 initiative – or Target Drug Discovery – focuses on evaluating a disease hypothesis through the process of discovery and clinical testing of a molecule designed to interact with a specific genomic target believed to be involved in disease pathogenesis.
Arch2POCM-Target-open compound and collaborative clinical analysis	Drs. Stephen Friend of Sage Bionetworks and Aled Edwards and Chas Bountra of the SGC	Arch2POCM focuses on novel targets that are judged as too risky to be pursued in industry, but that have significant scientific potential (e.g., cancer, immunology, autism and schizophrenia). Safe drug-like compounds that interact with the selected targets will be developed all the way through Phase IIb clinical trials in order to determine if modulation of Arch2POCM’s targets beneficially impact disease in patients. Market mechanisms are used instead of patent protection to incentivize downstream development.
GSK clinical trial data platform	GSK	The development of a broader system where researchers are able to access data from clinical trials conducted by multiple organizations. This system would be run by an independent third party who would be responsible for appointing and overseeing a review panel to assess research proposals. GSK is in discussions with interested parties to encourage the development of such a system.

GSK: GlaxoSmithKline; PD2: Phenotypic Drug Discovery; SGC: Structural genomics consortium; TD2: Target Drug Discovery.

Table 2. Addressing the challenges of open-source biopharmaceutical innovation.

The challenge	Solutions	Examples
Technological challenge	Data aggregation; data standardization; technologically driven access rights	Open PHACTS; Pistoia Alliance; GSK Clinical Trial Platform
Human capital development challenge	Platform-based collaboration opportunities; supportive training programs utilizing open data	Synapse platform; SGC Visitor Training Program
Data-material access challenge	Linkage between data and biological/chemical materials and models to support downstream value creation; collaborative downstream value creation	SGC; Arch2POCM; Lilly PD2 and Target2

GSK: GlaxoSmithKline; PD2: Phenotypic Drug Discovery; SGC: Structural genomics consortium.