Unapproved drugs among FDA's top research priorities

A greater understanding of how unapproved drugs are used by patients is one of the key science and research needs identified by the US Food and Drug Administration in a just-published report.

The document, prepared by the FDA's Center for Drug Evaluation and Research (CDER) and published in the Federal Register yesterday, is a preamble to a new strategic plan for regulatory science which is due for publication by the FDA shortly.

It is intended to identify the gaps in CDER's understanding which if addressed would "enhance CDER's ability to fulfil its regulatory mission."

The section on unapproved medicines highlights the need to understand the risks associated with the use of "compounded drugs, dietary supplements fraudulently containing active pharmaceutical ingredients, counterfeit versions of approved drugs, and marketed unapproved drugs that are not medically necessary."

The agency wants to move on from the current situation, in which it relies on adverse event reports and literature to determine the risk to the public of these products, to one where enforcement action is taken before public injury.

Knowledge gaps that need to be addressed include data on ingredients, products and manufacturing methods, patient and provider behaviour, supply chains and the impact of regulatory actions on patient outcomes.

CDER is also working on a screening strategy for contaminated, counterfeit, fraudulent and substandard drugs "to assess their occurrence and accessibility in the marketplace." Part of that effort involves the development of more sensitive analytical tools to screen for adulterants that may be present at low levels, and developing forensic signatures and/or fingerprinting profiles for drug components.

Other topics covered in the report include: improving access to post-market data sources; evaluating the effects of regulatory communications to the public and other stakeholders; assessing the link between product quality attributes, manufacturing processes and product performance; developing improved predictive models of safety and efficacy in humans; improving the design, analysis and conduct of clinical trials; and enhancing individualisation of patient treatment.