In his State of the Union address this year, President Obama announced that he’s launching the Precision Medicine Initiative — a bold new research effort to revolutionize how we improve health and treat disease. The goal of this initiative is “to enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized treatments.” It seeks to move our country away from a one-size-fits-all treatment methodology to one that focuses on each patient’s individualized needs. The White House is working with the Department of Health and Human Services (DHHS) and other federal agencies to solicit input from patient groups, bioethicists, privacy and civil liberties advocates, technologists, and other experts, to help identify and address any legal and technical issues related to the privacy and security of data in the context of Precision Medicine.
In order to help achieve the President’s goals, the US Food and Drug Administration (FDA) is developing new regulatory strategies for clinical tests based upon next generation sequencing (NGS) technologies. NGS technologies have significantly advanced the ability to derive more comprehensive genetic information on individuals in a relatively inexpensive and fast manner. To further the conversation on NGS technologies, the agency will hold two back-to-back public workshops on November 12 and 13. The first workshop, “Standards-Based Approach to Analytical Performance Evaluation of Next Generation Sequencing In Vitro Diagnostic Tests”, will focus on analytical performance evaluation standards, including potential ways to develop these standards, which can be used by test developers to ensure that their tests produce accurate and reliable results. The agency believes that stakeholder input about ongoing community standardization efforts is essential to construct flexible analytical evaluation approaches for a wide variety of NGS tests and modifications.
The second workshop, “Use of Databases for Establishing the Clinical Relevance of Human Genetic Variants”, will address current challenges in clinical validation of NGS tests. A single company, lab, or institution is unlikely to have enough information to definitively determine the clinical importance of test results. The aggregation of clinical information in curated databases will create a “data commons” that could serve as a reliable source of scientific evidence that test developers could use to demonstrate that NGS test results are relevant to a person’s disease or outcome. The workshop will highlight how scientists, patient groups, and private industry can work together to develop high-quality, curated clinical databases of genomic information that associate specific genetic changes with various diseases, such as cardiovascular disease or diabetes.
The public workshops will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (rm. 1503), Silver Spring, MD 20993-0002. Please register for the workshop(s) as space is limited. Comments and suggestions generated through these workshops will guide the agency’s development of best practices and/or regulatory standards, so they represent a great opportunity to voice your concerns, share your ideas, and guide decisionmaking.