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Rare Diseases and Orphan Products: Accelerating Research and Development
will alter the process of drug discovery and development (Zielinska, 2009); and
voluntary, open-source sharing of biological data through the Sage Commons, an initiative of Sage Bionetworks, a new, nonprofit medical research organization (http://sagebase.org).
The second example, Sage Bionetworks, uses data shared by pharmaceutical companies and others to develop computational models that predict potential drug targets as well as potential toxicities (Melese et al., 2009). The data shared with Sage will eventually be publicly available and could be particularly valuable for rare diseases research. For example, the organization has already provided a significant amount of clinical data to the Huntington disease research community. The data were generated in a clinical study of Alzheimer disease in which individuals with Huntington disease were used as controls. Without the Sage resource, these data would likely have remained unknown and unavailable to Huntington disease investigations (Marcus, 2010a).
Some rare diseases advocacy groups have made the sharing of research data by grant recipients a prerequisite for funding. For example, through its Accelerated Research Collaboration model, the Myelin Repair Foundation has insisted that those funded in its collaborations share their research findings with one another without awaiting scientific publication (MRF, 2010). To cite another example, in 2007 the Multiple Myeloma Research Consortium launched a Genomics Portal, through which researchers have unrestricted access to prepublication genomic and other molecular data (Kelley, 2009). This approach is not conventional in academic environments where researchers are rewarded for individual achievement, but by mandating data sharing, the consortium has succeeded in significantly expanding the therapies currently under development for multiple myeloma.
Sharing Compound Libraries
A related trend is the opening of what were once proprietary company libraries of chemical compounds to investigators interested in the potential of compounds to interact with drug targets across a wide range of diseases. Compounds and information on their structures have typically been generated and held tightly by pharmaceutical companies. In recent years, companies have begun sharing compound libraries with researchers working in neglected diseases areas. For example,