Regulatory Frameworks for Precision Medicine at the Food & Drug Administration

The concept of precision medicine is not new; its promise and allure have a rich history in genetics and genomics, building squarely on international research efforts begun decades ago and culminating in the publication of the complete human genome sequence in 2004. Now, nearly 15 years following the conclusion of the Human Genome Project (HGP), the terminology describing the idea of channeling genomic information into more targeted medical products has evolved, to include pharmacogenomics, personalized medicine, targeted medicine, and, most recently, precision medicine. All envisage the ability to make medical care and medical products “precise” for the patient; that is, tailored to an individual’s genetic makeup in order to maximize safety, efficacy, mortality, and quality of life. In his unveiling of the Precision Medicine Initiative in 2015, President Obama noted this program is about “delivering the right treatments, at the right time, every time to the right person.” Similar to prior federal funding initiatives (such as the Human Genome Project, the National Nanotechnology Initiative, the BRAIN Initiative, and the Cancer Moonshot 2020), the Precision Medicine Initiative (PMI) has at its core a goal of fostering multidisciplinary approaches to research and development that support both scientific discovery and clinical medicine innovation. The PMI sets forth five core objectives: (1) discovery of cancer treatments; (2) development of a voluntary, national research cohort; (3) robust privacy protections and interoperability; (4) assessment of regulatory regimes; and (5) strong partnerships in research. While each objective deserves concerted examination and discussion, this article addresses the existing regulatory regimes that provide a framework for the Food and Drug Administration (FDA) as precision medicine products emerge. While many large-scale efforts to develop applications for precision medicine can be traced largely to the outcomes of the HGP and subsequent genomic advances, the structure of the PMI serves to set forth a principled plan for implementation and continuing robust funding. The original plan for a PMI Cohort Program was announced in September 2015, led by National Institutes of Health (NIH) Director Francis Collins. Subsequently renamed the All of Us Research Program, the goal is to recruit a million participants to gather blood samples for DNA analysis, collect clinical information, and access electronic health records as well as other health-related data. In December 2016, the 21st Century Cures Act further enhanced federal funding for precision medicine, appropriating an initial $4.8 billion in funding to the NIH for the PMI over the next decade. Along with targeted funding, the 21st Century Cures Act establishes an organizational framework for the PMI, including authority to the Secretary of the Department of Health and Human Services (HHS) to implement the initiative and foster rapid innovation. The Secretary is also expressly required to coordinate with the FDA to achieve the goals of the initiative, which will be crucial to the regulatory aspects. This article will explore precision medicine as related to the scope of products regulated by the FDA. First, it will analyze existing FDA regulatory frameworks for relevant products, including drugs, biologics, medical devices, and combination products. Second, it will explore FDA-approved products characterized as precision medicine and discuss FDA policy on use and labeling. Third, it will explore important recent legislative authority directed to precision medicine research and regulatory review and approval by the FDA. Last, it will identify several aspects of precision medicine that will pose both opportunities and challenges going forward for the FDA as the agency is faced with innovative diagnostics and therapeutics in this realm.