Course Background

To protect our nation’s security, medical countermeasures (MCMs) for high consequence pathogens (HCPs) is essential to treat and prevent threats to global health. Sixteen 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 policymakers 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 311 registered attendees from 2013 - 2018.

The FDA and UTMB is currently expanding the education program curriculum to include a course addressing the conduct of HCP clinical trials and also to potentially 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, policymaking, funding, review, and inspections of studies necessary for the advancement of MCMs and protection of human health.

Course Description

This 2 1/2-day interactive pilot course allowed participants to enhance knowledge and integrate practical considerations for the planning and operation of clinical research protocols involving medical countermeasures for high consequence pathogens. Course participants acquired knowledge through innovative learning platforms and problem-based scenarios that help identify and mitigate barriers to data quality and integrity in domestic and international barrier nursing environments.  Course faculty included clinicians, nurses, biosafety officers, government leaders, and federal agency representatives with field experience. 

Overall Course Objectives

• Formulate solutions to regulatory, operational, and cultural challenges for the conduct of clinical trials of medical countermeasures for high consequence pathogens
• Identify and mitigate risks to data quality and integrity
• Resolve complexities of performing clinical trials in biocontainment / barrier nursing environment  under GCP 
• Experience conduct of clinical research tasks in a mock biocontainment environment while wearing high level PPE

Education Outcome

The goal of this unique educational course is to provide a learning environment that promotes collaboration of ideas, provides tools for clinical study conduct, enhances mutual understanding of clinical, 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 clinical, scientific and regulatory experts mutually engaged in, and committed to, the development, research, and approval of medical countermeasures for high consequence pathogens.

Target Audience

Physicians, Scientists, Principal Investigators, Physician Assistants, Nurse Practitioners, Nurses, Pharmacists, Clinical laboratorians, Clinical research staff, Regulators, Policymakers, Regulatory reviewers, Biosafety professionals, IRB professionals, and Sponsors.

Learning Outcomes

• Utilize best practices for the design and implementation of research clinical trials during an outbreak involving high consequence pathogens.
• Recognize and prevent potential risks to data quality and integrity.
• Apply the GCP principles to research clinical studies of medical countermeasures for high consequence pathogens.
• Mitigate data collection challenges in a biocontainment / barrier environment.