To protect our nation's security, medical countermeasures (MCMs) for high consequence pathogens (HCPs) must be developed to treat and prevent threats to global health. Fifteen years ago, the United States Department of Health and Human Services Food and Drug Administration (FDA) published the final rule for New Drug and Biological Drug Products; Evidence needed to Demonstrate Effectiveness of New Drugs When Human Efficacy Studies Are Not Ethical or Feasible (21 CFR Parts 314 and 601, Federal Register, May 31, 2002). The Animal Rule (AR), as often referenced, states that for drugs/biologics developed to ameliorate or prevent serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic substances, when human efficacy studies are not ethical and field trials are not feasible, FDA may grant marketing approval based on adequate and well-controlled animal efficacy studies when the results of those studies establish that the drug is reasonably likely to produce clinical benefit in humans. Because of the highly infectious nature of HCPs, these studies are often performed in high or maximum biocontainment (BSL-3/4). The logistics of assuring accurate and reliable data collected from these environments is challenging. These products are subsequently evaluated through clinical trials during a public emergency. Data derived from the nonclinical AR studies and the clinical studies for HCPs are reviewed by FDA and global authorities to draw a regulatory conclusion; therefore, data quality and integrity are imperative. In 2012, the FDA and the University of Texas Medical Branch at Galveston (UTMB) collaborated to design and implement an education program to cross educate sponsors, scientists, veterinarians, physicians, nurses, quality assurance personnel, regulators, reviewers, and policy- makers to enable the conduct of regulated studies supporting product approval via the FDA's AR. These studies require compliance with the FDA Good Laboratory Practice regulations (21 CFR Part 58) to the extent practicable in order to assure data quality and integrity. The result was a robust education program that delivered annual training to 269 registered attendees from 2013- 2017. The FDA and UTMB plan to expand the education program curriculum to include a course addressing the conduct of HCP clinical trials and also expand the catalog of on-line courses for distance education of individuals conducting HCP clinical trials at remote sites. Knowledge gained will address barriers to progress and increase communication between parties involved in the conduct, policy-making, funding, review, and inspections of studies necessary for the advancement of MCMs and protection of human health.
This work will fund the continuation and expansion of an education program to enable the conduct of nonclinical and clinical studies of high consequence pathogens for the approval of medical countermeasures (MCMs). The goal of this unique collaborative educational program is to provide a learning environment that promotes collaboration of ideas, provides tools for nonclinical and clinical study conduct, enhances mutual understanding of scientific and regulatory complexities, and promotes the data quality and integrity derived from these regulated studies. The result is a better prepared cohesive community of scientific and regulatory experts mutually engaged in, and committed to, the development and approval of MCMs.
