Report on Status of Regulatory Science at FDA

Frank Torti, MA, MD, MPH, was appointed as the US Food and Drug Administration (FDA)’s first Chief Scientist and Principal Deputy Commissioner on 15 May 2008. Formerly, Dr Torti was an academic oncologist and epidemiologist, most recently as Professor of Medicine and Chair of the Department of Cancer Biology at Wake Forest Medical School. His earlier career was spent at Harvard and Stanford. Dr Torti’s report, with the same title as this Editorial, was issued at the end of February 2009. It clearly builds upon two reports that emanated prior to his appointment. Presuming that this report is intended to be taken seriously, some close review and comment upon it may be justified. Dr Torti builds his mission on five principles, none of which would be questioned by anybody wishing progressive change at FDA: 1. FDA must develop an overarching scientific strategy with an accompanying implementation plan, deliverables, timetables and budget; 2. FDA scientific strategy must be pre-emptive; 3. FDA must maintain and enhance its infrastructure, core expertise and human capital; 4. FDA must tell its story; 5. FDA cannot do it alone. It would be wrong to hold these five principles to the standards of other types of organizations.While the first four might be found in a typical research university or pharmaceutical company, the last (‘‘FDA cannot do it alone’’) distinguishes the special mission of a regulatory organization, and indeed represents a desirable situation (pace those who are deluded in thinking that the Agency conspires with its stakeholders). By analogy, judges often have the warring barristers before them do much of the legal research on points of law, without ceding any judicial ground. Evidently in consultation across the various centres within FDA, a variety of priority projects are then identified in the report and are sorted against these five principles. Here, the report is somewhat ‘retro’ and repetitive of what we have all known for a few years already: for example, overarching scientific priorities include rapid detection of pharmacovigilance signals, development of biomarkers, more efficient clinical designs and analyses, etc. Microbial contamination (a highvisibility issue for both foods and drugs in the US at the moment) appears more than once. An original idea is that an ‘omics’ czar is proposed, to deal with genomic, proteomic andmetabolomic issues across centres and reviewing divisions. This could well be seminal for the development of personalizedmedicine and is consonant with an FDA Science Board emphasis on differential drug response between racial groups. Combination product coordination also receives attention. However, its guru will only be an FDA fellow (essentially a 2-year post-graduate internship within the Agency). Outside interactions (‘‘FDA cannot do it alone’’) are thought to benefit especially the areas of drug-induced liver injury, nano-engineered products, and the development of centres of excellence in collaboration with academia. However, the report states: ‘‘Small biotechnology firms represent a flexible resource. They are operationally and scientifically nimble, and often have capability to devote a large proportion of human capital to a single project. Biotech firms that have an interest in partnering to resolve mission-critical FDA questions will be identified and engaged through contracts and other funding mechanisms.’’ Another opinion might be that the FDA seems oblivious of how small companies work. First, the present financial situation makes it unlikely that there will be any substantive venture capital for biotech for the next 5 years. Moreover, those few companies that survive might be expected to have their own mission-critical questions that will take priority over the FDA’s. It is also telling that this report refers to FDA staff as being ‘‘well-trained physicians and scientists’’, and even that FDA Fellows are ‘‘highly qualified and selected scientists’’; meanwhile, the biotech companies who are going to provide all this help evidently contain ‘‘human capital’’. EDITORIAL Pharm Med 2009; 23 (1): 1-2 1178-2595/09/0001-0001/$49.95/0