Human health: medicinal products, therapies and medical devices

Medicines for human use can be authorised in two ways in the EU:

• Centralised authorisation: This is granted by the European Commission, based on a scientific evaluation from the European Medicines Agency (EMA). This route is required for biotech-based medicines.
• National authorisation: This is managed by national authorities and can involve either a mutual recognition or a decentralised procedure (in both cases countries agree on an approval), or a national single-country procedure.

The requirements and procedures for marketing authorisation, as well as the rules for monitoring authorised products are laid down in the EU directive on rules for the authorisation and labelling of all medicines and the EU regulation on medicines authorisation at EU level. These requirements apply to all medicines to be placed on the EU market (including advanced therapy medicinal products, orphan medicines, and paediatric medicines).

Clinical trials

EU rules in clinical trials set out a unified process for submitting, validating, and approving clinical trial applications. While individual EU countries are responsible for assessing and overseeing trials, the same steps apply everywhere, making the process consistent.

To better understand national requirements, you can consult the Q&A document on the Clinical Trials Regulation, which you can find in Eudralex Volume 10.

See also

Contact points at the European Medicines Agency

Contact points of national authorities responsible for medicinal products for human use

Role of the European Medicines Agency (EMA)

The European Medicines Agency (EMA) is responsible for the scientific evaluation and supervision of medicines which are authorised by the Commission through the centralised procedure. The EMA provides guidance and evaluation at all stages of a medicine's development. This includes:

• Research and development: guidance and support to medicine developers
• Marketing authorisation: support in the run up to the submission and scientific evaluation of centralised marketing authorisation applications
• Post-authorisation: safety monitoring of medicines on the market, through pharmacovigilance, applications for variations to the market authorisation and reporting product defects or recalls.

There are also various EU incentives and rewards to encourage research and development of specific types of medicines:

• Medicinal products for rare diseases (‘orphan medicines’): EMA support for developers working on biological products intended for rare diseases.
• Medicinal products for children: EMA support for developers working on biological products intended for children.

Additionally, if your company qualifies as a micro, small or medium enterprise (SME), you will have access to dedicated support and incentives, including reduced fees for scientific advice, regulatory and procedural assistance, and early dialogue through the EMA SME Office.

See also

EU directive on rules for the authorisation and labelling of all medicines

EU regulation on medicines authorisation at EU level

EU regulation on orphan medicines

EU regulation on paediatric medicines

If your company is developing biological medicinal products such as vaccines or monoclonal antibodies, knowing EU rules is essential for bringing your product to the market. Here are the key things you need to know.

How to obtain a marketing authorisation for your product

• Centralised authorisation: if your product is biotech-derived or an advanced therapy medicinal product (ATMP) such as a gene therapy, tissue-engineered or somatic cell therapy medicinal product, you must submit an application to the European Medicines Agency (EMA). Based on the EMA's positive assessment, the European Commission will grant a marketing authorisation.
• National authorisation: for certain types of biologicals (naturally derived biologicals), you may need to work directly with national authorities in individual EU countries.

GMO assessment for biological products

If your product under clinical trials contains or consists of GMOs, it must undergo a GMO assessment under EU rules on deliberate release and contained used of GMOs.

EMA support and resources

EMA provides scientific guidelines on biological active substance and biological medicinal products to help medicine developers prepare marketing authorisation applications for human medicine.

EMA also offers support and services to help companies develop quality, safety, and efficacy data that meet regulatory standards. You can request such advice to clarify the design of pre-clinical studies and clinical trials, and the data requirements to support benefit-risk assessments.

• Scientific advice: you can benefit from advice on the most appropriate ways to generate evidence on your medicine’s benefits and risks or the qualification of innovative development methods for biological products by requesting EMA scientific advice.
• Special programmes for innovative biological products: if your product addresses an unmet medical need, you might qualify for the Priority Medicines (PRIME) scheme, which is an enhanced support programme for faster development and assessment.
• Early consultations for innovative biological products: you can engage with experts early by requesting assistance from the EMA’s Innovation Task Force (ITF), which can provide guidance on navigating regulatory requirements for innovative products or the Quality Innovation Group on innovative approaches of manufacturing and quality control of biological medicines.

See also

EMA services and support overview

EU directive on contained use of GMOs

EU directive on deliberate release of GMOs

Advanced therapy medicinal products (ATMPs) such as gene therapies, tissue-engineered products, and somatic cell therapy medicinal products must in addition to the general rules applicable to medicinal products meet specific EU standards before reaching the market.

EU rules on ATMPs

• Approval and quality standards: ATMPs require EU marketing authorisation, which demonstrates quality, safety, and efficacy. Applications go through the European Medicines Agency (EMA), which assesses products intended for EU-wide authorisation.
• Good manufacturing and good clinical practice standards: Good Manufacturing Practice (GMP) and Good Clinical Practice (GCP) guidelines apply to ATMPs.

GMO assessment for ATMPs

If your ATMP contains or consists of genetically modified organisms (GMOs), it will need a GMO safety assessment. Check the EMA guidance for more information.

Accessing EMA guidance

The EMA offers comprehensive support, and resources to guide companies through the development, application, and regulatory stages specific to ATMPs. You can also apply for consultations that will allow you to clarify technical and regulatory questions early in the development of your product.

ATMP Regulation incentives and EMA support

• Fee reductions: the EMA offers reduced fees for scientific advice to help SMEs navigate the complex requirements for ATMP developments.
• Scientific classification recommendations: the EMA provides recommendations on ATMP classification, helping companies establish whether or not their product qualifies as an ATMP and, if so, under which specific category.
• Quality and non-clinical data certification for SMEs: the EMA can evaluate and certify quality and non-clinical data, which can facilitate future applications for clinical trials or marketing authorisations.

See also

EU regulation on advanced therapy medicinal products

EU directive amending the community code relating to medicinal products for human use as regards advanced therapy medicinal products

EMA advanced therapy medicinal products overview

Legal framework advanced therapies

EU Framework of advanced therapies

GMO legislation

In the EU, substances of human origin (SoHO), such as blood, tissues, cells, and organs, are essential for medical therapies like transfusions and transplants. They are also used as starting materials for certain medicinal products, including plasma-derived products and advanced therapy medicinal products (ATMPs).

To ensure safety and quality, all stages of handling SoHO must comply with specific EU rules and guidelines:

• Blood and blood components: EU rules set standards for the collection, processing, testing, and use of blood products, ensuring they are safe for patient use.
• Tissues and cells: EU rules govern the donation, collection, and use of tissues and cells, including those used in medicine, ensuring their quality and traceability.
• Organs: EU rules also apply to organ donation and transplantation, ensuring safety throughout the process.

The EU maintains a Tissue Establishment Compendium, which lists the establishments that are authorised to provide starting materials for medicinal products. You can use this registry to find approved suppliers and ensure that the materials you use are safe.

For additional regulatory queries or guidance on implementing SoHO rules, you can contact the EU’s SoHO coordination board at sante-soho@ec.europa.eu.

See also

EU guidance on SoHO

EU directive on quality and safety of substances of human origin

EU rules regarding medical devices aim to ensure safety, innovation, and efficient market access. Understanding EU rules can help you navigate the regulatory landscape and maximise opportunities on the EU market. There are strict rules regarding medical devices in the EU that ensure they are safe and effective for use.

Regulatory framework

The medical device sector in the EU is overseen by specific bodies tasked with assessing such products (known as “notified bodies”) and national authorities. These bodies work together to ensure that medical devices meet all the necessary safety and quality standards before they are sold. The European Commission helps coordinate the work of national authorities and other actors.

Certification and CE marking

To sell a medical device in the EU, it must undergo a conformity assessment which is done with the involvement a notified body for all except the lowest risk devices. This process ensures that the manufacturer has a quality management process, and the device is safe and effective for its intended purpose. The notified body will issue one or more certificates. After this the device can be labelled with the CE mark, showing that it meets all EU requirements.

Safety and performance requirements

The rules aim to provide a high level of protection for patients by ensuring that medical devices perform as expected. These requirements cover aspects like manufacturing processes, design and clinical performance including its follow-up after the device is placed on the market. Devices are also subject to ongoing surveillance and vigilance once they are on the market to detect any potential risks or issues.

In vitro diagnostic medical devices

If your product is an in vitro diagnostic medical device, such as a test kit or diagnostic instrument, which tests human specimens for medical purposes, it must comply with specific EU rules. These devices are subject to similarly rigorous safety and performance standards as medical devices, but with some specific differences related to their intended purpose.

Resources and support

The Medical Device Coordination Group provides guidance on various topics from device risk classification to safety reporting. These resources help ensure your device meets the necessary standards for the market.

If you have specific questions about carrying out a clinical study on a medical device or placing a device in the market in a particular EU country, contact the national competent authorities in that country. They can provide detailed guidance on any country-specific requirements.

See also

Medical devices overview

EU regulation on medical devices

EU regulation on in vitro diagnostic medical devices

Once a new medicine has received EU marketing authorisation, you can sell it in all EU countries. The decision to launch a medicine in a given EU country is an individual decision by the company. An application for pricing and reimbursement needs to be filed separately in each EU country.

What is an HTA?

A health technology assessment (HTA) summarises information about medical, economic, social and ethical issues related to the use of a health technology. Examples of health technologies include medicinal products, medical equipment for diagnostic and treatment and prevention methods. When HTA bodies evaluate a new health technology, they assess whether it works better, equally well, or worse than existing alternatives. For this, they assess the therapeutic effect of the drug, as well as potential side-effects and influence on quality of life.

EU-wide joint assessments

There is an EU HTA legal framework that offers advantages for biotech SMEs:

• Joint clinical assessment: a single harmonised assessment reduces duplication and saves time by replacing multiple national clinical assessments.
• Early joint scientific consultations: joint scientific consultations provide coordinated advice on study design for a given health technology, helping SMEs align with expectations across the EU to facilitate the generation of evidence that meets the likely evidence requirements of a subsequent joint clinical assessment on that health technology.
• Focus on clinical evidence: joint clinical assessments focus only on clinical effectiveness, creating a solid foundation for national pricing and reimbursement decisions.
• Faster market access: standardised procedures and methods developed within the EU HTA legal framework can make it easier to navigate HTA processes and can contribute to improved access to innovative technologies, such as medicines and certain medical devices, for EU patients.

The joint efforts to streamline clinical assessments makes it easier for you to focus on innovation and bringing your product to the market.

See also

EU HTA overview

EU regulation on health technology assessment