Overview

The standard process for a population to gain access to a vaccine typically involves the issuing of a marketing authorization by the appropriate regulatory body in a country, followed by manufacturing and distribution. Once these steps are completed, the vaccine can be procured through public health programs, private healthcare providers or by individuals themselves. However, in cases of public health emergencies, alternative pathways for vaccine access may be implemented to expedite distribution and reach vulnerable and high-risk populations more quickly.

Flexible Policies for Public Health Emergencies

To effectively respond to outbreaks or pandemics, it is recommended that countries adapt flexible policies to enable vaccine deployment before full marketing authorization, where justified. These policies could include:

1. Clinical trials: use of an investigational new drug within the context of a clinical trial for the diagnosis, monitoring, or treatment of a serious disease or condition

2. Expanded access:  the use of an investigational new drug outside of a clinical trial in patients for the diagnosis, monitoring, or treatment of a serious disease or condition

3. Compassionate use:  making a new, unapproved drug available to treat a seriously ill patient when no other treatments are available

4. Emergency use authorization:  authorization of unapproved medical products or unapproved uses of approved medical products to be used in an emergency to diagnose, treat, or prevent serious or life-threatening diseases or conditions caused by CBRN (chemical, biological, radiological, nuclear) threat agents when certain criteria are met, and when there are no adequate, approved, and available alternatives. The speed of emergency use authorization may depend on several factors, such as:

   • Standardized benefit-risk assessment

   • Correlate or surrogate of protection

   • Platform readiness 

Implementation

Such measures can be implemented while final data is being generated, prepared, and submitted to regulators for review. The maturity level of the platform can influence how quickly regulators can assess safety and efficacy, potentially accelerating the authorization process during urgent situations.  Proposed mechanisms for providing access to developmental biological, pharmaceutical, vaccine and device products based on developmental phase and case fatality rates are outlined in Table 1. Real world evidence data should be evaluated in cases where efficacy trials are not conducted due to infeasibility and high mortality and transmissibility risks.

Note that the concepts presented in this document are recommendations made by CEPI based on experience with researchers, developers, funders and regulators and will require alignment with key stakeholders.

Table 1: Access to developmental products in a public health emergency

¹  After initial marketing application authorization, additional data may be needed to expand use to include special populations.

²  Logistically challenging for a large scale outbreak or pandemic

³  Requires that specific patients be identified for inclusion, therefore, it is infeasible to use this mechanism in a large-scale Public Health Emergency