Clinical Scorecard: A Conceptual Model for Evaluating the Potential of Advanced Research, Development, and Regulatory Clearance of Medical Countermeasures: An Element of BARDA's Strategy for Emerging Infectious Diseases

At a Glance

Key Highlights

• Development of a conceptual framework to guide BARDA's portfolio decisions on MCM development for emerging viral pathogens.
• Identification of eight key factors central to assessing feasibility of advanced development and FDA approval of vaccines and therapeutics.
• Framework supports efficient resource allocation by prioritizing MCM candidates with highest potential for FDA approval within existing regulatory paradigms.

Guideline-Based Recommendations

Diagnosis

• Focus on generating pivotal clinical and nonclinical data to support FDA regulatory approval of vaccines and therapeutics.
• Early consultation with FDA to define appropriate regulatory strategies tailored to product and context.

Management

• Prioritize flexible platform technologies and invest through to FDA approval and stockpiling of priority MCM products.
• Utilize traditional and alternative FDA regulatory pathways (Traditional, Accelerated Approval, Animal Rule) based on feasibility and ethical considerations.

Monitoring & Follow-up

• Conduct post-marketing studies to verify clinical benefit and assess safety when required by Accelerated Approval or Animal Rule pathways.
• Monitor evolving epidemiology, technical capabilities, and infrastructure to reassess MCM development feasibility.

Risks

• Budgetary constraints limit development of MCMs for all possible emerging pathogens.
• Regulatory pathways remain product- and context-specific, requiring tailored approaches.
• Human efficacy trials may be infeasible or unethical for some pathogens, necessitating alternative approval pathways.

Patient & Prescribing Data

Individuals at risk of infection from prioritized emerging viral pathogens

Vaccine development currently appears more feasible than therapeutics for assessed pathogens; regulatory approval depends on product-specific data and pathway.

Clinical Best Practices

• Engage early and continuously with FDA to align on regulatory strategy and data requirements.
• Leverage a structured framework assessing key factors to inform portfolio management and investment decisions.
• Focus on generating robust safety data in humans across all regulatory pathways.
• Incorporate platform technologies to enhance flexibility and speed of MCM development.
• Plan for post-marketing studies when utilizing Accelerated Approval or Animal Rule pathways.

References

• BARDA mission and portfolio overview
• FDA Accelerated Approval Guidance
• FDA Animal Rule Guidance