Authorisation of medicines
All medicines must be authorised before they can be marketed and made available to patients. In the European Union (EU), there are two main routes for authorising medicines: a centralised route and a national route.
From lab to patient: journey of a medicine
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Under the centralised authorisation procedure, pharmaceutical companies submit a single marketing-authorisation application to EMA.
This allows the marketing-authorisation holder to market the medicine and make it available to patients and healthcare professionals throughout the EU on the basis of a single marketing authorisation.
EMA's Committee for Medicinal products for Human Use (CHMP) or Committee for Medicinal products for Veterinary Use (CVMP) carry out a scientific assessment of the application and give a recommendation on whether the medicine should be marketed or not.
However, under EU law EMA has no authority to actually permit marketing in the different EU countries. The European Commission is the authorising body for all centrally authorised product, who takes a legally binding decision based on EMA's recommendation. This decision is issued within 67 days of receipt of EMA’s recommendation.
Once granted by the European Commission, the centralised marketing authorisation is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway.
Commission decisions are published in the Community Register of medicinal products for human use.
The centralised procedure is compulsory for:
- human medicines containing a new active substance to treat:
- human immunodeficiency virus (HIV) or acquired immune deficiency syndrome (AIDS);
- cancer;
- diabetes;
- neurodegenerative diseases;
- auto-immune and other immune dysfunctions;
- viral diseases.
- medicines derived from biotechnology processes, such as genetic engineering;
- advanced-therapy medicines, such as gene-therapy, somatic cell-therapy or tissue-engineered medicines;
- orphan medicines (medicines for rare diseases);
- veterinary medicines for use as growth or yield enhancers.
It is optional for other medicines:
- containing new active substances for indications other than those stated above;
- that are a significant therapeutic, scientific or technical innovation;
- whose authorisation would be in the interest of public or animal health at EU level.
Today, the great majority of new, innovative medicines pass through the centralised authorisation procedure in order to be marketed in the EU.
During their development, medicines are usually first tested in the laboratory to understand how they would work in the body.
They are then tested in humans, in studies called clinical trials, which help us understand how the medicines work and evaluate their benefits and side effects.
There are usually three successive phases of trials:
- Phase I trials involve in a small number of healthy volunteers or patients to confirm that the medicine behaves as expected from laboratory tests.
- Phase II trials involve a relatively small number of patients with the targeted condition to give more information about the best doses to use and also confirm that it works
- Phase III trials involve a larger number of patients with the targeted condition to compare the medicine with other treatments and to determine how well it works.
For rare diseases, because they affect only a small number of patients, phase III trials may not always be carried out or may only involve a very small number of patients.
Medicine developers who wish to conduct clinical trials in the EU need to submit applications to the national competent authorities of the countries where they want to conduct the trials.
EMA does not have a role in the authorisation of clinical trials in the EU; this is the responsibility of the national competent authorities.
However, EMA, in cooperation with the EU Member States, plays a key role in ensuring that medicine developers follow EU and international standards.
Whether they conduct these studies within or outside the EU, developers conducting studies to support the marketing authorisation of a medicine in the EU have to comply with strict rules. These rules, called good clinical practice, apply to the way they design the studies, how they record their results and how they report these results. These rules are in place to ensure that studies are scientifically sound and conducted in an ethical manner.
Medicines that are granted a marketing authorisation by the European Commission can be marketed throughout the EU.
However, before a medicine is made available to patients in a particular EU country, decisions about pricing and reimbursement take place at national and regional level in the context of the national health system of the country.
EMA has no role in decisions on pricing and reimbursement. However, to facilitate these processes, the Agency collaborates with health technology assessment (HTA) bodies, which assess the relative effectiveness of the new medicine in comparison with existing medicines, and EU healthcare payers, who look at the medicine’s cost effectiveness, its impact on healthcare budgets and the seriousness of the disease.
The aim of this collaboration is to find ways for developers to address the data needs of medicines regulators as well as those of HTA bodies and EU healthcare payers during the development of a medicine, rather than generating new data after its authorisation. If one set of evidence addressing the needs of all these groups can be generated early during the development of a medicine, it should make decisions on pricing and reimbursement at national level quicker and easier.
To achieve this, EMA and the European Network for Health Technology Assessment (EUnetHTA) offer medicine developers the possibility to receive simultaneous, coordinated advice on their development plans.
Patient representatives are involved in these consultations on a routine basis so that their views and experiences can be incorporated into the discussions.
 In 2019, simultaneous advice from EMA and HTA bodies was provided upon request during the development of 27 medicines. Patients were involved in two thirds of these cases. |
Once a medicine has been authorised for use in the EU, EMA and the EU Member States constantly monitor its safety and take action if new information indicates that the medicine is no longer as safe and effective as previously thought.
The safety monitoring of medicines involves a number of routine activities ranging from:
- assessing the way risks associated with a medicine will be managed and monitored once it is authorised;
- continuously monitoring suspected side effects reported by patients and healthcare professionals, identified in new clinical studies or reported in scientific publications;
- regularly assessing reports submitted by the company holding the marketing authorisation on the benefit-risk balance of a medicine in real life;
- assessing the design and results of post-authorisation safety studies which were required at the time of authorisation.
EMA can also carry out a review of a medicine or a class of medicines upon request of a Member State or the European Commission. These are called EU referral procedures; they are usually triggered by concerns in relation to a medicine’s safety, the effectiveness of risk minimisation measures or the benefit-risk balance of the medicine.
EMA has a dedicated committee responsible for assessing and monitoring the safety of medicines, the Pharmacovigilance Risk Assessment Committee (PRAC). This ensures that EMA and the EU Member States can move very quickly once an issue is detected and take any necessary action, such as amending the information available to patients and healthcare professionals, restricting use or suspending a medicine, in a timely manner in order to protect patients.
For more information, see Pharmacovigilance: Overview.
While the majority of new, innovative medicines are evaluated by EMA and authorised by the European Commission in order to be marketed in the EU, most generic medicines and medicines available without a prescription are assessed and authorised at national level in the EU.
In addition, many older medicines available today were authorised at national level because they were marketed before EMA was created.
Most Member States maintain National registers of authorised medicines.
Each EU Member State has its own national authorisation procedures. Information about these can normally be found on the websites of the national competent authorities:
If a company wishes to request marketing authorisation in several EU Member States for a medicine that is outside the scope of the centralised procedure, it may use one of the following routes:
- mutual-recognition procedure, whereby a marketing authorisation granted in one Member State can be recognised in other EU countries;
- decentralised procedure, whereby a medicine that has not yet been authorised in the EU can be simultaneously authorised in several EU Member States.
For more information see:
- Coordination Group for Mutual Recognition and Decentralised Procedures – Human
- Coordination Group for Mutual Recognition and Decentralised Procedures – Veterinary
The data requirements and standards governing the authorisation of medicines are the same in the EU, irrespective of the authorisation route.
The full text is available as a booklet:
