Public health preparedness: developing medical countermeasures for certain serious or life-threatening conditions

What GAO found

The Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) established the Animal Rule in 2002 to allow for the approval of medical countermeasures based on animal efficacy studies when human clinical trials are not ethical or feasible. Medical countermeasures are medical products that can be used to prevent, treat or mitigate the potential health effects of exposure to chemical, biological, radiological and nuclear (CBRN) agents. GAO found that the FDA has undertaken efforts to provide information and feedback to developers to support the development of animal rule medical countermeasures. For example, in 2015 the FDA published guidance that clarifies the types of studies and data needed to prove the product’s efficacy. The FDA has approved 16 medical countermeasures under the Animal Rule, 14 of which have been approved in the past decade.

Research of biological agents in a clinical laboratory

The FDA established the Animal Model Qualification Program in 2011 to provide publicly available animal models to support animal rule efficacy testing for multiple medical countermeasures for a given disease or condition. Researchers and developers can submit models to the program for qualification and, once qualified, a model can be used by other developers when appropriate. For example, an animal model for inhaled anthrax would include protocols, such as exposure and dosage times, to produce pathological manifestations that adequately reflect the manifestations of inhaled anthrax in humans. In April 2022, the FDA qualified an animal model under the program. FDA officials and many developers GAO spoke to attributed the limited number of qualified models to a lack of incentives to pursue the qualification. Notably, submitting a template for qualification is voluntary, and FDA officials said the qualification process is rigorous and resource-intensive, which could discourage applications. However, many developers reported that the limited number of skilled animal models did not prevent product development, citing other ways to identify animal models that can be used for product development. FDA officials and others GAO spoke to, including some developers and contract research organizations, said the program could still be beneficial. For example, FDA officials said the program could further aid the future development of medical countermeasures, particularly for CBRN agents who currently have no approved medical countermeasures.

Why GAO did this study

Past bioterrorist attacks, such as the 2001 anthrax attacks, highlight the threat of widespread disease and death posed by CBRN agents and the importance of medical countermeasures. GAO previously reported on the challenges of developing medical countermeasures. Medical countermeasures may need to be developed and approved under the FDA Animal Regulations.

The Pandemic and All-Hazards Preparedness and Advancing Innovation Act of 2019 included a provision for GAO to review the development of animal rule medical countermeasures. In this report GAO describes (1) the FDA’s efforts to support developers of medical countermeasures under the animal rule and (2) the extent of qualification of animal models under the FDA’s animal model qualification program and l effect of qualified models on the development of medical countermeasures.

GAO reviewed the FDA’s documentation, including the agency’s medical countermeasures development guide. GAO also interviewed or obtained written responses from FDA officials; other federal agencies involved in the development of medical countermeasures; and a non-generalizable selection of six developers, three contract research organizations, and four academic research and policy organizations. GAO selected respondents based on their involvement or knowledge of developing animal rule medical countermeasures.

HHS and the Department of Defense provided technical comments, which GAO incorporated as appropriate.

For more information, contact Mary Denigan-Macauley at (202) 512-7114 or [email protected]

Leave a Comment

Your email address will not be published.