The process of regulating, reviewing, and ultimately licensing a drug is critical to bringing safe and effective medicines to market. The guidelines that define these processes may vary depending on the state in which it is intended to operate, but the global trend is pushing toward defining processes that are increasingly similar to each other.

The Food and Drug Administration (FDA), which is responsible for drug evaluation and regulation in the United States, and the European Medicines Agency (EMA), which serves the European Union, on the other hand, are often treated as two substantially overlapping regional counterparts. Although this similarity is undeniable, there are differences between the two agencies that deserve to be analyzed.

FDA and EMA Jurisdiction

The FDA is the main government agency in the United States and is responsible for the regulation of:

Instead, the EMA is only responsible for the evaluation of human and veterinary medicines for countries within the European Union (plus Iceland, Norway, and Liechtenstein). Following Brexit, which led to the United Kingdom’s exit from the EU, drugs in the UK are no longer regulated by the EMA.

Unlike the FDA, which in addition to evaluating a drug can make regulatory decisions for a wide range of products, the EMA is responsible only for evaluating medicines based on efficacy and safety information. The body with the power to authorize or approve a drug is the European Commission (EC), to which the EMA offers suggestions that are useful in making the final regulatory decision.

Drug approval processes

In the United States and the EU, the processes are relatively similar. Although the complexities of each may differ, the general steps follow the same pattern:

  • Pre-authorization: the stage where it is confirmed that clinical research is necessary and rooted in scientific evidence. At this stage, attempts are made to minimize potentially adverse outcomes to safeguard the health of volunteers participating in clinical trials from unreasonable risks.
  • Clinical trials: the drug testing phase. In both countries, a scheme is followed consisting of phase 0 and 1, on a small number of healthy subjects, to test the safety of the drug in humans and clarify dosages. This is followed by phase 2, where several hundred patients with the condition to be treated are tested. Finally, in phase 3 the participants become thousands in order to show a large-scale analysis of the drug’s safety and efficacy.
  • Research review and evaluation: unless it is for orphan drugs, emergency situations, or one of the accelerated approval mechanisms in the U.S., we enter the drug approval phase. Here FDA and EMA follow two different paths.

The FDA oversees all drug approvals in the United States through New Drug Applications (NDAs), while biologics approvals occur through Biologics License Applications (BLAs). Both processes follow a centralized pathway exclusively.

The EMA, on the other hand, offers the option of four different routes: centralized, national, mutual recognition, and decentralized. In the centralized process, the EMA itself manages the process, issuing a single license that is valid in all EU member states. This approval pathway is mandatory for certain classes of drugs, such as treatments for HIV/AIDS, oncology, diabetes, neurodegenerative disorders, autoimmune diseases, and viral diseases.

The national process can be routed for all drugs that do not require the centralized process. In this case, it is the individual member state that uses its own approval pathways. Once a drug has been approved nationally, it can also gain approval in other states through mutual recognition.

Finally, there is the decentralized procedure of applying for approval in two or more states of the Union simultaneously. To date, this is the most traveled procedure for drugs not yet approved in any EU state. As with the others, this route is only pursuable for products that do not have to fall under the mandatory centralized process.

Today, the two bodies continue to work together to increasingly harmonize their processes to simplify the approval mechanisms for the world’s two largest pharmaceutical markets.