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


WEF Report: “…The genomic nature of rare disease suggests an opportunity. By bringing together genomic, phenotypic, and clinical data at a global scale, individual countries and hospitals carrying out genomics research could come up with more answers both to diagnose currently undiagnosed or misdiagnosed people with rare disease and to develop treatments.

With an estimated 15.2 million individuals expected to have clinical genomic testing for a rare condition within the next five years, it is time to evaluate the economic and societal benefits of developing a system that can share this data without compromising privacy.

The answer is a technical solution called a “federated data system”. This is a data superstructure that can aggregate remote data sets for querying while still allowing for localized, data control and security. The groupings are independent, but interoperable. They have their own governance and the information inside them is protected, but they offer evidence – conclusions gathered from the data – that can be used to feed a much larger, global data engine.

The result is a robust and well-annotated dataset that in the case of rare diseases can be added to and then used by different countries to enable global and country-specific solutions to diagnosis, treatment, patient trial recruitment and management of rare diseases. Developing federated data systems is one of the many investments countries are currently considering. Yet since its implementation can be costly (about half a million US dollars) is it the right solution?…(More)”.