Obama Announces $215m Precision Medicine Investment for NIH, FDA
By Allison Proffitt
January 30, 2015 | President Obama will outline this morning his proposed investments to improve health and treat disease through precision medicine. The president will set out a $215m plan, “to build the infrastructure we need to accelerate discovery in [precision medicine],” said Jo Handelsman, Associate Director for Science at the White House Office of Science and Technology Policy, yesterday.
Francis Collins, Director of the National Institutes of Health, called it an, “exciting, but somewhat general plan.”
The $215m will be part of the President’s proposed 2016 budget to be released in full next week, and will include $130m to the National Institutes of Health, $70m to the National Cancer Institute, $10m to FDA, and $5m to ONC to develop interoperability standards.
The broad goals of the initiative, Handelsman said, are, “catalyzing more and better treatments for cancer”, creating a national research cohort of Americans who are empowered to manage their own health, developing a new approach for the evaluation of next generation sequencing technologies, setting privacy standards, and building a personalized medicine infrastructure.
Infrastructure is the leitmotif of the initiative, with plans calling for meaningful privacy measures, more standardized electronic medical records, interoperability standards, and curated disease databases.
The bulk of the planned budget would go to NIH for the development of a national research cohort of a million or more volunteers to, “set the foundation for a new way of doing research through engaged participants and open, responsible data sharing,” said Collins.
Yet specifics were scarce. Collins said a “blue ribbon” panel would be created as soon as possible to get the very best ideas for how to develop and build the cohort, ensure privacy, and engage volunteers.
NIH isn’t starting from scratch, Collins stressed. Existing patient groups would be included. Collins estimated some 200 existing patient cohorts comprising thousands of patients, but noted that the types and amounts of data contained within those cohorts varied greatly. The initiative’s million-plus-member cohort would need to reflect diversity in terms of age, sex, ethnicity, health status, and socioeconomic status. Collins said he expects to need to “fill some holes” while still relying on existing patient groups.
But the power of the plan is not just the number of patients, Collins said.
“The precision medicine initiative is not just about scale,” Collins said. “It’s also meant to be a new model of research, one in which people who participate are partners—not subjects, not even patients, but partners.”
Cohort members will be followed longitudinally to give long term insights into health. Some sequencing is likely, of course, though Collins is concerned that whole genome sequencing is still too expensive. He suggested exome sequencing as an alternative, though said that ultimately he hoped whole genome sequencing was available to as many volunteers as were comfortable.
But at the initiative as presented is not a genome project, but instead focuses on underlying data frameworks.
$70 million of the initiatives funds are dedicated to the National Cancer Institute to, “scale up efforts to identify genomic drivers of cancer, and apply that knowledge to the development of more effective approaches to cancer treatment,” Handelsman said.
Collins said the short term focus on cancer belies a longer term goal: to generate knowledge that can be applied to a whole range of health issues.
Collins specifically called for more clinical trials with novel designs, non-invasive methods for tracking response to therapies, pharmacogenomics, and studies on drug combinations and drug resistance. And researchers need a “cancer knowledge network to store all of this data in a usable, digital format,” he said.
The initiative, “will require the involvement of many different sectors of science and society, including biologists, physicians, technology developers, data scientists, but especially the public. We hope to extend beyond our borders so we can forge collaborations on a global scale.”
FDA’s Role
The Initiative would grant FDA $10m to, “acquire additional expertise and advance the development of high quality, curated databases; develop the regulatory structure that’s needed to advance innovation and precision medicine while protecting public health,” Handlesman said.
For FDA, the initiative comes as the Agency has been shifting its view of drugs and market review approaches away from the one test-one drug paradigm to a new vision of genomic medicine, said Margaret Hamburg, Commissioner of the U.S. Food and Drug Administration.
Hamburg admitted that the advancing science has presented challenges for FDA’s review process, but she reiterated some of FDA’s earlier language, especially concerns it voiced when the Agency shut down 23andMe’s medical interpretation business.
“Simply having powerful sequencing platforms that are capable of identifying thousands and even millions of genetic variants isn’t enough. We must be confident that these platforms, and the tests that use these platforms are capable of producing reliable and accurate results, and that the test results can be interpreted in a way that’s meaningful for healthcare providers and their patients.”
Hamburg held up the Agency’s approval of Illumina’s MiSeqDx and cystic fibrosis tests for clinical use as a standard for the agency’s experience with next-generation sequencing and new technology. The key in this case, Hamburg said, was a “well-curated, shared database of cystic fibrosis mutations to demonstrate clinical validity.”
Hamburg called the database a model of patient participation, and said that such databases will be key to the Agency’s ability to, “accelerate development with a practical and nimble approach that will allow the medical advances in the field of genomics to be implemented as soon as possible.”
The White House fact sheet for the initiative, released on Friday, is available here.
