Global Data Access for Solving Rare Disease: A Health Economics Value Framework

Rare diseases have been an increasing area of focus as three waves have converged in recent years: the continuing innovation stemming from the genomic revolution, the regulatory financial incentives put in place by the US government for rare-disease therapies, and the increasingly mobilized, coordinated and sophisticated patient community. However, the very nature of rare diseases calls for scientific and societal collaboration on an unprecedented scale. Federated data systems are one such example of this scale. A federated data system is a type of meta-database made up of constituent databases that are transparently interconnected, but not merged – an important point for security and privacy concerns.