The U.S. Department of Health and Human Services (HHS) recently released a report on its examination of the federal government's medical countermeasures (MCMs) enterprise, The Public Health Emergency Countermeasures Enterprise Review: Transforming the Enterprise to Meet Long-Range National Needs. HHS initiated this review in December 2009 as a result of the challenges it encountered with respect to the 2009 H1N1 influenza pandemic and, in particular, the pandemic flu vaccine. The review focused on the development of countermeasures (i.e., products critical to protecting or treating the population against naturally occurring or intentionally delivered chemical, biological, radiological, or nuclear threats), from the identification of a national requirement and the resulting product concept all the way through to regulatory approval, manufacturing, procurement and stockpiling. In the course of its review, HHS developed a strategy and vision that the nation "must have the nimble, flexible capacity to produce MCMs rapidly in the face of any attack or threat, known or unknown, including a novel, previously unrecognized, naturally occurring emerging infectious disease."

Notably, some of the key findings of the report--as well as its major initiatives--relate to FDA's regulation of MCMs. The report found that the current regulatory framework and the need for advances and development of regulatory science create both perceived and real barriers to entry. The report lays out several initiatives aimed at the development of a "21st century regulatory science" and the provision of greater regulatory assistance to MCM developers, often small biotechnology companies with limited experience navigating the regulatory process.

In particular, the report indicates that, in FY2011, FDA will undertake a new initiative, "Advancing Regulatory Science for Public Health," that is "designed to focus on augmenting the tools used to assess the safety, efficacy, and quality of medical products, with a particular focus on MCMs, and to get them from concept through the approval process efficiently." It will focus on building up FDA's scientific infrastructure and capacity; support the identification and qualification of animal models and surrogate efficacy measures; expand capability to pre-qualify certain manufacturing facilities; and improve assays for potency, sterility and stability.

Second, FDA has committed to creating focused "Action Teams" to work with other components of the MCM enterprise program management team on high-priority MCM projects. Together, they will help sponsors to, among other things, identify and resolve scientific issues earlier and facilitate faster evaluation/review of such products, including through assistance with the development of a "Regulatory Science Plan" (i.e., a plan that "specif[ies] known scientific gaps or opportunities for improvement, and identif[ies] priority areas and the required strategies and resources to appropriately address them as needed, before or after project initiation"). If the teams are successful, FDA may consider broader adoption of the approach for routine use throughout the regulatory process.

Finally, in collaboration with other members of the MCM enterprise, FDA will begin a project to examine and optimize the legal and policy framework for MCM oversight and approval, "to better ensure that laws and regulations support preparedness and response." It will examine mechanisms such as "restricted or conditional licenses" for products to be stockpiled for emergency use, but not otherwise marketed; identify ways to collect data during emergencies that could support FDA approval and/or post-market surveillance requirements; and make recommendations regarding any statutory changes needed to achieve improved emergency preparedness and response.