culating the risk and benefit of an anthrax antitoxin today, for example, is very different from calculating the risk and benefit of the anthrax antitoxin once there has been a widespread anthrax release. Jesse Goodman of FDA said that the language around FDA’s EUA authority regarding the known and potential benefits outweighing the known and potential risks leaves a lot of room for different interpretations. Goodman, Parker, and others all noted that defining the emergency scenario up front would allow for different benefit-risk decisions than those that would be reached for use of common products by generally healthy people. Hatchett cautioned that the calculus done in anticipation of an emergency tends to be much more stringent than that which would actually be made during a real event. To aid benefit-risk decisions, Rose suggested, reviewers of MCMs ought to have the requisite security clearances to be allowed to read the associated confidential population threat assessments. In later discussion about safety assessment, Richard Forshee of the Office of Biostatistics and Epidemiology at CBER, mentioned current agency efforts to develop risk assessment models to support regulatory decision making. These probabilistic quantitative computer simulation models can help explore how different regulatory science options could ultimately have an impact on public health, and thereby improve decision making. FDA is also engaged in a number of computational toxicology computer simulations to help assess, for example, potential risk from vaccine adjuvants.

Key Messages: Enterprise Stakeholder Perspectives

  • The regulatory paradigm for MCM development needs to be supported by new regulatory science and evaluative tools that are product-independent. There is a need for a format to permit engagement between product developers and FDA outside the context of a specific product approval.
  • MCM development needs more clearly defined regulatory pathways. Priorities include:
    • Products approved under the Animal Rule, and
    • Diagnostics—prevalidated and pre-positioned in the field
  • The benefit-risk calculus may be different for MCMs to be used in low-probability/high-consequence events than for traditional products.
  • Repurposing of previously licensed products needs to be studied in a systematic and comprehensive manner.

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