4 things to know about Emergency Use Authorization in US and EU

Introduction

Emergency Use Authorization is a provision that allows usage of medical products or ‘medical countermeasures’ prior to approval, clearance, or licensing. It supports emergency preparations and fosters the availability of medical products for use in emergencies.

It is a time-consuming and complex process to get a new pharma product approved by the regulatory bodies. In the United States, it takes years for an experimental drug to reach the shelves of medical shops from the laboratory. However, there are situations in which a vaccine, drug or similar products need to be pushed into the market hastily for emergency usage. The Covid-19 pandemic, which was declared a public health emergency of international concern (PHEIC) by the WHO, also posed a situation where a vaccine for the disease became the need of the hour.

Global pharma giant Pfizer along with German biotechnology firm BioNTech used the provision of EUA for their Covid-19 vaccine against the SARS-CoV-2 virus, as they submitted a request to the U.S. Food and Drug Administration (FDA) for Emergency Use Authorization (EUA). The federal regulatory body issued the authority for emergency usage of the Pfizer-BioNTech vaccine mentioning the prevailing life-threatening conditions, no availability of the substitute for the vaccine, and the scientific evidence backing up the vaccine to be effective in preventing the Covid-19. This authorization process from the request submission to the approval took less than a month. During this process, the term ‘Emergency Use Authorization’ was heard in the pharma lobbies globally and remained a topic of discussion. 

Emergency Use Authorization is a provision that authorizes the introduction of a medical product in the U.S. state commerce which is not approved, licensed, or cleared under sections 505, 510(k), and 515 of the FD&C (Federal Food, Drug & Cosmetic) Act or section 351 of the PHS Act.

When the Health and Human Services (HHS) secretary recommends that an emergency use authorization is appropriate, FDA may authorize unapproved medical products to be used in an emergency. EUA allows FDA to authorize the use of unapproved medical countermeasures, in anticipation of a potential emergency, if certain criteria are met.

In European Union (EU), for the benefit of the public, a Conditional Marketing Authorization may be granted for emergency medicines on less comprehensive clinical data than normally required where the advantages of instant availability of the medicine outweigh the risk inherent. 

4 Aspects of Emergency Use Authorization

Here we will sneak peek into the four important aspects of the Emergency Use Authorization of medical and pharma products in the US and EU.

1. Issuance Criteria

• Serious or life-threatening situation: For an emergency authorization by FDA, the environment under which the medical product usage is proposed in the application declaration has to be serious or life-threatening.
• Product effectiveness: For an emergency authorization by FDA, there has to be sufficient scientific evidence to declare that the product 'may be' potentially effective in curbing the emergency at hand.
• Risk to benefit comparison: For an emergency authorization by both FDA and EMA, the known and the possible benefits of the product, should outperform the known and potential risks posed by the product.
• Unavailability of alternatives: Both the FDA and EMA require an absence of any potential approved alternative to the candidate product that can curb the situation at hand for issuing an emergency authorization.

EMA also expects the applicant to provide comprehensive data related to the product post its authorization.

2. Authorization condition

Set of all the elements that are part of the user authorization. It is where the roles and responsibilities are made explicit for the sponsors, manufacturers, etc. These conditions can be data collection and reporting, reporting of adverse effects, restrictions on advertisements, compliance with good manufacturing practices, providing specific information to recipients, etc.

3. Revision or termination of authorization

Both the FDA and EMA have provisions to revise or terminate the issued usage authority. if the situations justifying its issuance cease to exist for the benefit of public health.

A unique circumstance can also provide stimuli to revoke or revise the EUA, for e.g., adverse inspection findings at the manufacturing sites, reported inefficiency of the product, some negative effect of a drug, concerns regarding the purity of the product, etc.

The Conditional Marketing Authorization by EMA is valid for 1 year and needs renewal every year.

4. Pre Authorization Activities

• It is an early engagement of the FDA with industry sponsors or manufacturers. To effectively handle the approvals in an emergency, FDA recommends the sponsors of the product connect with one of its centers before making submissions for EUA. Here the product sponsor can discuss the aspects of a potential product.
• For a pre-EUA submission, the manufacturers need to prepare a file with information and data on the product's quality, safety, and efficacy. It has to be submitted in a relevant IND or cross-referenced MF before a EUA request is submitted. It also requires information on manufacturing, controls, and information about each manufacturing site and the current status of the manufacturing sites in light of the Current Good Manufacturing Practice (CGMP) requirements.
• In the EU, EMA advises applicants to discuss their development plans with the agency early in the development process.
• The applicants have to indicate a request for conditional marketing authorization in their application for marketing authorization.

Conclusion

The guidelines set for authorization of medical products in an emergency situation have many similarities with the principles of a successful regulatory process. Having clear-cut dialogues on the product-specific requirements with the FDA centers or with the EMA agents at a pre-authorization stage can be very helpful in navigating through the process. It will help to understand the needs and expectations of the regulatory bodies in terms of data, risk discussion, or any other specific requirements. The product sponsors, especially the new developers, may seek the support of a pharma regulatory service provider for preparing necessary documents and other submissions for a seamless process. There are premium consulting service providers who can provide end-to-end support in the whole process of navigating through an emergency authorization journey. 

Authored by: Prajeesh Pillai and Zafar Ibrahim

Note: The opinions that may be presented in the article are that of the authors

Also read: Pharmacovigilance in the Digital Era – Know Your Safety Reporting