An abbreviated history of innovation in regulatory science
The mission of the US Food and Drug Administration (FDA) makes the FDA, in many ways, unique among federal agencies. As both a regulatory and a science agency, the FDA is charged with both protecting public health through regulation and enforcement and advancing public health by catalysing innovations in product (eg drug) development. To that end, the FDA's history has been hallmarked with patient-centred programmes and policies meant to coalesce innovation and scientific rigour in a way that is translatable to individual patient populations.
The Center for Drug Evaluation and Research (CDER) is the wing of the FDA that regulates over-the-counter and prescription drugs to ensure that they are safe and effective. CDER's recent initiatives are indicative of its commitment to innovation and improved patient outcomes. These include such programmes as the Critical Path, Safety First, Safe Use and Sentinel Initiatives. In the early period after the completion of the Human Genome Project, CDER embarked on one of its most forward-thinking infrastructure building projects to date. As pharmaceutical companies began to conduct exploratory genomics and pharmacogenetics studies in the context of their drug development programmes, it became clear to leaders in CDER and the Office of Clinical Pharmacology (OCP) within CDER that the Center needed to: 1) encourage companies to continue to evolve the science, with the ultimate goal of enhancing drug product development; and 2) develop internal FDA expertise to deal with the myriad complexities of analysing and interpreting pharmacogenetic, genomic, proteomic and like data. To that end, the Voluntary Genomic Data Submissions (VGDS) programme was developed in the early- to mid-2000s, to allow drug companies, consortia, academic researchers and individuals to engage in scientific exchange with the FDA, and to allow FDA regulatory scientists to gain experience with data in this then nascent field .
In its early days, VGDS were largely focused on technical aspects of platforms used to generate genomic-era data. Since the first VGDS in 2004, however, the FDA has received over 40 submissions, which have become more applied in nature. That is, more recent VGDS have been concerned with the practical application of genetic or biomarker information to drug development. Over the past five years, there has been significant growth in the use of pharmacogenetic principles in drug development. This has established the need for regulatory scientists with a keen understanding of pharmacogenetics, clinical pharmacology, population science/epidemiology, clinical trial design and pharmacotherapy (ie clinical practice). The Genomics Group within the OCP in CDER is a prototype regulatory review group within which such skill sets are being integrated and further developed.