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27.05.2026

What should you know about EU and US Drug Approval? Part 2

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The following Part 2 of this topic supplements and complements the previous Part 1.

Marketing authorisation routes in the EU

Centralised marketing authorisation procedure
The centralised procedure is the regulatory instrument for obtaining authorisation for the entire EEA through a single procedure. The legal basis was established by Regulation (EC) No 726/2004⁹. The same regulation also established the European Medicines Agency (EMA), which coordinates the evaluation of the marketing authorisation application during the procedure.

The centralised procedure is mandatory for certain medicinal products.

These include:

1. Medicines manufactured using specific biotechnological processes (e.g. recombinant DNA technology and monoclonal antibodies).
2. Advanced Therapy Medicinal Products (ATMPs), i.e. gene therapies, somatic cell therapies and tissue-engineered products.
3. Medicinal products containing a new active substance for the treatment of one of the following conditions:
a. acquired immunodeficiency syndrome
b. cancer
c. neurodegenerative diseases
d. diabetes
e. autoimmune diseases and other immunodeficiencies
f. viral diseases
4. Medicinal products developed for a rare condition (orphan disease).

For medicinal products that do not meet any of these conditions, the centralised procedure is optional. The applicant must then demonstrate that their medicinal product represents a significant innovation or that an EU-wide authorisation is in the interests of patient health.

As in the MRP and DCP, the assessment of the marketing authorisation dossier under the centralised procedure must be completed within 210 days, with clock stops not included in this timeframe. The evaluation is carried out by a rapporteur and a co-rapporteur, who draw up a report¹¹. This is then assessed by the Committee for Medicinal Products for Human Use (CHMP).

The centralised procedure is divided into three evaluation phases. In the first phase, which begins on Day 1 following the validation of the application, the rapporteur and co-rapporteur assess the marketing authorisation dossier and draft an opinion for the CHMP. On this basis, the CHMP draws up a List of Questions, which the applicant receives on Day 120. The procedure is paused; this 'clock stop' can last between three and six months and is intended to allow the questions to be answered.

Once the responses have been submitted, the second evaluation phase begins, which is considered Day 121. The rapporteur and co-rapporteur assess the responses and update their opinion. The CHMP checks whether all questions have been answered satisfactorily. If any issues remain unresolved, the CHMP draws up a List of Outstanding Issues and sends this to the applicant. Here too, the procedure is paused (Day 180); the pause may last between one and three months.

Once the responses have been submitted, the third evaluation phase begins on Day 181. The rapporteur and co-rapporteur assess the responses, update their opinion, and the CHMP reviews the matter again. In exceptional cases, where there are serious concerns, the applicant may be invited to an oral explanation. This meeting serves to clarify critical issues that would otherwise lead to rejection.

Once all issues have been satisfactorily resolved, the opinion is finalised and the CHMP issues its final opinion on the marketing authorisation application. The application is either rejected or receives a positive recommendation. It is important to note that the EMA itself does not grant marketing authorisations. Following a positive outcome of the centralised procedure, the European Commission issues an Implementing Decision within 67 days, which constitutes the formal authorisation and is subsequently published in the Official Journal of the EU¹².

Marketing authorisation process in the USA

The legal basis for drug authorisation in the USA is the FD&C Act, which is laid down in Title 21 of the Code of Federal Regulations (CFR)1,3. The FD&C Act was passed in 1938, has been revised several times, and today defines the powers of the Food and Drug Administration (FDA) as well as the conditions under which a medicinal product receives authorisation. The applicant submits a New Drug Application (NDA) to the FDA for chemically synthesised medicines or a Biologics Licence Application (BLA) for biologics.

An application for marketing authorisation in the USA can be divided into four phases¹³.

Technical validation of the application
Substantive review of the application and resource planning
Scientific evaluation of the application
Decision

On average, the FDA requires 12 months for the entire process, calculated from the date of submission to the final decision on the application.

A distinctive feature of the US procedure is the two validation phases. In the first technical validation, the FDA checks administrative aspects such as the payment of application fees. This phase is completed within 14 days. This is followed by the so-called Filing Review, in which the FDA assesses whether the application meets the minimum scientific requirements and contains all the documents necessary for evaluation. This second validation phase should be completed within 60 days of the first validation.

If all requirements are met, the applicant is informed that their application will undergo scientific evaluation in the next step. At this stage, the applicant often receives initial queries from the relevant departments. If the application does not meet regulatory requirements even at this validation stage, the FDA may issue a Refuse to File (RTF). This is a written statement setting out the grounds for the refusal and highlighting the deficiencies in the dossier to the applicant. It is important to note that the RTF is not a review decision. It is based exclusively on formal criteria and is issued before the scientific evaluation begins.

Following a successful second validation, the scientific evaluation begins. The FDA review teams assess the documentation and ask the applicant questions regarding the data collected during development. A key structural difference from the EU procedure should be highlighted here: in the US procedure, there are no clock stops for regulatory queries. The FDA may ask questions at any stage of the evaluation, and these must be answered during the evaluation. In addition to exchanging questions, the applicant also interacts with the FDA through various meeting formats.

In the Late Cycle Meeting, which takes place towards the end of the process, outstanding issues and their resolution are discussed. Another format is the Advisory Committee Meeting (AdCom).

It is convened primarily for new therapeutic approaches and serves as a decision-making aid for the FDA. The AdCom brings together scientific experts, industry representatives and patient organisations to discuss the efficacy and benefits of the drug under review. The AdCom's recommendations are not binding on the FDA. The entire scientific evaluation of the application takes around 8 months before the approval process enters its final phase.

During the decision-making phase, the FDA prepares its statement, through which the decision on approval is communicated to the applicant. If the application is deemed eligible for approval, marketing authorisation is granted and the applicant may place the medicinal product on the market. If, however, the FDA considers the application ineligible for approval, it issues a Complete Response Letter (CRL). The CRL sets out the reasons for the rejection and the steps the applicant can take to resubmit the application.

Summary

This article has provided an overview of the authorisation pathways in the EU/EEA and the USA. As the EU is a union of 27 member states, there are several options for obtaining authorisation there. With the centralised procedure, the European Commission has also created a mechanism for obtaining authorisation for the entire European Economic Area.

In the US, there is only one authorisation procedure, which may vary slightly depending on the type of medicinal product being assessed. This article has focused solely on the authorisation procedure for medicines containing a new active substance. Generic medicines undergo a similar procedure, but there are procedural specifics that would not be relevant for a general overview.

Beyond the procedures described here, a centralised authorisation in the EU or an FDA authorisation often forms the basis for further global expansion. Numerous regulatory authorities worldwide base their own assessments on the scientific opinions of the EMA or the FDA, which can significantly facilitate market access in additional regions. However, the strategic planning of such a global regulatory strategy would go beyond the scope of this article and forms the basis for a separate article.