I think the opportunity landscape for community/government partnerships is beyond phenomenal and where the pediatric cancer and rare disease communities are unmatched in dedication. These efforts can range from advocacy for access regulations associated with government-sponsored research data, to direct patient engagement in healthcare policymaking, to advocacy on behalf of research funding (this latter effort has been especially key in the success of efforts such as the Cancer Moonshot and the Gabriella Miller Kids First Pediatric Research Program https://commonfund.nih.gov/KidsFirst ).

However, one of the more fundamental shifts in understanding and culture that is poised for transformation under a government/community partnership relates to the empowerment of a patient's own data by the patients themselves, as informed and guided collaboratively by both the government and the specific disease community that supports and brings together their interconnected and engaged patient population. Currently, a patient's right to access and transmit their own clinical data remains in practice largely unaddressed with patients and hospital systems insufficiently informed of a patient's own rights to access, use, and contribute their data on behalf of whatever purpose they choose with very limited powers given to any entity to restrict such secondary access and use.

The FAQs found here provide some of the initial clarification and guidance on these topics by HHS:

https://www.hhs.gov/hipaa/for-professionals/privacy/guidance/access/#newlyreleasedfaqs

Relating specifically to the topics relevant to our discussion here and such platforms as the precisionFDA—I think a key portion on the above link of which we should take note is the following:

"An individual has a right under the HIPAA Privacy Rule to access, upon request, PHI about the individual in a designated record set maintained by or for a clinical laboratory that is a covered entity. The designated record set includes not only the laboratory test reports but also the underlying information generated as part of the test, as well as other information concerning tests a laboratory runs on an individual. For example, a clinical laboratory that is a HIPAA covered entity and that conducts next generation sequencing (NGS) of DNA on an individual must provide the individual, upon the individual’s request for PHI concerning the NGS, with a copy of the completed test report, the full gene variant information generated by the test, as well as any other information in the designated record set concerning the test."

Thus, the transformational opportunity on the very near horizon is the clear shift of robust molecular and genomic profiling of patients from academic research to clinical tests as virtually all hospital systems (and as supported by payers) engage the offering of precision medicine informed by clinical sequencing/profiling. If such rights are embraced by empowered patient communities, the data-driven-discovery landscape could be transformed--by patients, on behalf of patients, and justifiably unimpeded by “experts” who might think otherwise.