NIH, FDA, and VA Do Their Part to Advance Precision Medicine Initiative

The National Institutes of Health (NIH) is partnering with the US Department of Veterans Affairs (VA) to enroll veterans in the Precision Medicine Initiative (PMI) Cohort Program as participants in the largest ever study of its kind, the Obama Administration has announced.

The PMI, announced in January 2015, is a new research effort initiated by US President Barack Obama that aims to revolutionize the ways in which health is improved and disease is treated in the United States. The aim of precision medicine is to tailor prevention strategies and treatment decisions to an individual’s unique characteristics.

In early July, the Obama Administration announced a number of new investments, partnerships, and policies to support the goals of the PMI. This included $55 million in new awards from the NIH to health care provider organizations, technology developers, and community health centers to support their participation in the PMI Cohort Program. At least 1 million people are expected to be enrolled in the PMI Cohort Program. Participants are being recruited through partner health care organizations, including regional and national medical centers, community health centers, and VA health centers. For its part, the VA will build on its success with the Million Veteran Program, which included more than 490,000 participants, to help enroll patients in the PMI Cohort Program to provide health data and biological specimens that will be used to map patients’ genomes and to create a database of information to help guide treatment.

In a call with reporters on July 6, Francis Collins, MD, PhD, director of the NIH, shared some of the unique aspects of the research initiative: “First, participants in the program will be true partners—not subjects, not patients—and will be involved in every step… including what data to collect, what analyses to do in the lab, and what data should be returned to them. Second, the cohort will represent the rich diversity of America, engaging volunteers of all economic levels to produce meaningful health outcomes for all races, ethnicities, and communities. Third, data-sharing will be swift to both researchers and participants. Participants will have access to study information and data about themselves.”

On the same day, the FDA released two new draft guidances on next generation sequencing (NGS)–based tests. In a press release, the FDA states that the goal of the guidances is to “provide a flexible and streamlined approach to the oversight of tests that detect medically important differences in a person’s genomic makeup.” Said FDA Commissioner Robert Califf, MD, “Targeting the right treatments to the right patients at the right time is the goal of the President’s Precision Medicine Initiative… The FDA is preparing for this exciting approach at multiple levels.”

The first guidance provides recommendations for designing, developing, and validating NGS-based tests and addresses the potential for using FDA-recognized standards to demonstrate analytical validity, or how well the test predicts the presence or absence of a particular genomic change. The second guidance describes how test developers can use clinical evidence from FDA-recognized public genome databases to support clinical claims for their tests and provide assurance of accurate clinical interpretation of genomic test results, enabling an easier path for marketing clearance or approval.

Said Dr Collins, “NIH sees great value in these guidances and encourages test developers to adopt the best practices outlined in the guidances so that high quality tests can become available to the patients who need them.”—Kara Rosania