Having such data available would make it possible to narrow candidate drugs for a condition beyond the possibilities that can be identified without such data. It also would allow for the review of safety and efficacy data in repurposing drugs. These data are now available, but they belong to private companies, which constrains their release.
The FDA also has a tremendous amount of data developed as part of drug development projects, but it does not have the infrastructure, the resources, or the authority to analyze these data and make them available. Austin pointed out that having only summary data released by the FDA would not be acceptable since the analyses could not be verified. One option, said Pacanowski, might be for the FDA to draw together the parties that own the data to work out legal agreements so the data can be used. “That would potentially be very valuable.”
The allocation and protection of intellectual property are troublesome issues, however. For example, Trusheim observed that the developers of diagnostics can create tremendous value but are often poor at capturing that value. Instead, payers, patients, and drug developers collect much of that value. Only when diagnostic companies have strong intellectual property protection have they been able to force other partners to pay what a diagnostic is worth. Additionally, Austin said, many owners of the data believe that their release would be detrimental. For their part, private companies do not have an upside in releasing data that might be used to undercut the value of a compound. “Even when they would want to do it, they can’t.”
Companies need the exclusive use of data that they generate to receive returns on their investment. Otherwise, the development costs have to be paid up front. “It’s one or the other,” Austin said.
Hamburg said that a criticism sometimes made of personalized medicine is that it will serve only elites, but personalized medicine has at least the potential to do the opposite and help reduce the health disparities that exist among population groups today. By understanding more about the mechanisms and natural history of diseases, researchers can help uncover the reasons why groups differ in their susceptibility to disease and in their responses to therapy.
Hudson agreed, adding that intergroup differences emphasize the need for much more widespread participation in research. Minorities are still underrepresented in many clinical studies and NIH has many projects to increase participation, although the results have been “depressing.” The Clinical and Translational Science Awards have a specific focus on health disparities along with community engagement and implementa-