Glossary

An abridged marketing authorization application (MAA) allows pharmaceutical companies to obtain regulatory approval without submitting full pharmacological, toxicological, or clinical trial data. Instead, the applicant can rely on existing data under specific conditions outlined in Article 10 of Directive 2001/83/EC.

The Active Pharmaceutical Ingredient (API) is the central substance in pharmaceutical products responsible for their therapeutic effect. In the EU context, the regulation and quality control of APIs are crucial to ensuring the safety, efficacy, and quality of medicines. Understanding the regulatory framework surrounding APIs is essential for manufacturers, regulatory bodies, and healthcare professionals.

The Active Substance Master File (ASMF), previously known as the European Drug Master File (EDMF), is a regulatory document submitted as part of a marketing authorization application (MAA) or variation request for a medicinal product. The ASMF provides detailed information about the active pharmaceutical ingredient (API) while protecting the manufacturer's confidential data.

Advanced Therapy Medicinal Products (ATMPs) are a class of innovative medicines that include gene therapies, somatic cell therapies, and tissue-engineered products. These products are used for regenerative medicine and personalized treatments, offering new therapeutic options for diseases with limited treatments.

An Adverse Drug Reaction (ADR) refers to a harmful or unintended response to a medicinal product. ADRs are central to pharmacovigilance activities in the EU, as outlined in Directive 2001/83/EC and Regulation (EC) No 726/2004. Monitoring and reporting ADRs helps ensure the ongoing safety of medicinal products post-authorisation across the European Economic Area (EEA).

The Agence nationale de sécurité du médicament et des produits de santé (ANSM) is the French national regulatory authority responsible for overseeing medicines, medical devices, and other healthcare products. It replaced the Agence française de sécurité sanitaire des produits de santé (Afssaps) in 2012 and operates under the French Ministry of Health.

API (Active Pharmaceutical Ingredient) or refers to the pharmacologically active component of a drug product that produces the intended therapeutic effect. API manufacturing is the production of these ingredients starting from chemical raw materials (and in some cases intermediates).

A Batch Record (Batch Manufacturing Record - BMR) is a critical document in pharmaceutical manufacturing that ensures compliance with Good Manufacturing Practice (GMP) regulations. It serves as a detailed written record of the entire production process for a specific drug batch, ensuring traceability, quality control, and regulatory compliance.

Before a batch of medicine can be released to the market, the batch record must undergo a thorough review process by Quality Assurance (QA) teams to verify that all manufacturing and quality control procedures were correctly followed.

The Centralised Procedure (CP) is a European Medicines Agency (EMA)-regulated approval process that allows pharmaceutical companies to obtain a single marketing authorisation, granted by the european commission,  valid in all EU Member States. This process leads to a Community authorisation, enabling the medicinal product to be marketed across the European Economic Area (EEA).

A Change of Ownership or Transfer of Marketing Authorisation (MAT) refers to the process of transferring an approved marketing authorisation from the current Marketing Authorisation Holder (MAH) (Transferor) to a new MAH (Transferee).

The transfer process differs based on whether the marketing authorisation (MA) was granted nationally  or via a centralised procedure. Regulatory authorities such as the European Medicines Agency (EMA) and national competent authorities (NCAs) oversee this process to ensure continuity of drug quality, safety, and pharmacovigilance obligations.

A Clinical Trial Application (CTA) is a formal request to obtain authorization for conducting a clinical trial in EU/ European Economic Area (EEA) Member States. The evaluation, authorization and supervision of clinical trials are the responsibilities of the EU Member States and EEA countries. Additionally, independent Ethics Committees are involved in the process by reviewing the risk-benefit ratio, trial design, and participant safety measures.

Before a clinical trial on a medicinal product for human use can begin in the European Union (EU), it must receive a favorable opinion from the concerned national members on Part II of the application. Part II includes details on how the protection of trial participants and the ethical integrity of the study is ensured. For evaluation of Part II, national members states appoint Ethics Committees who will assess the risk-benefit ratio, trial design, and participant safety measures before approving a study.

A Clinical Trial is a research study conducted to evaluate the safety, efficacy, and dosage of an Investigational Medicinal Product (IMP) in humans. It is a required step in obtaining marketing authorisation for new drugs.

According to EU Regulation No. 536/2014, a clinical trial is a subset of a clinical study, specifically designed to assess the effects of an IMP on human health.

The Common Technical Document (CTD) is the internationally agreed format for organizing information in marketing authorization applications (MAA) for medicinal products. The CTD format is accepted by regulatory authorities in:

• United States (FDA)
• Europe (EMA, National Agencies)
• Japan (PMDA)

While the CTD structure is harmonized, content requirements may vary between these regions.

The Convention on Pharmaceutical Ingredients (CPhI) is an international trade show and networking event for the pharmaceutical industry. Founded in 1990, CPhI is held annually in rotating global locations and brings together professionals from pharmaceutical manufacturing, development, supply chain sectors and contract services.

CPhI serves as a platform for innovation, collaboration, and business development within the active pharmaceutical ingredient (API), excipient, biotech,  drug manufacturing industries and also Finished Dosage Formulation (FD) and Packaging & Drug Delivery for drug products, chemicals, herbals.

The Decentralised Procedure (DCP) is a regulatory pathway for obtaining marketing authorisation for medicinal products in multiple EU Member States simultaneously, without prior approval in any EU country.

It was introduced by Directive 2004/27/EC and can be used for all medicinal products for which the Centralised Procedure is not mandatory.

Drug Master File (DMF) is a confidential document submitted to regulatory authorities that A contains detailed information about the manufacturing process, facilities, and raw materials used in the production of a drug substance or excipient.

In the United States (U.S.), DMFs are submitted to the Food and Drug Administration (FDA), while in Europe (EU), the equivalent document is known as the Active Substance Master File (ASMF) and is submitted to the European Medicines Agency (EMA) or National Competent Authorities (NCAs).

Educational material refers to approved documents designed to enhance the safe and effective use of medicinal products. Within the EU regulatory framework, such materials are often part of Risk Management Plans (RMPs) as required by Directive 2001/83/EC and Regulation (EU) No 726/2004. They aim to inform healthcare professionals (HCPs), patients, or caregivers about specific risks associated with a medicinal product and how to mitigate them.

• handles tasks related to the implementation of the EU’s chemicals legislation and policy
• provides transparent, independent and high-quality scientific opinions and decisions
• provides tools, advice and support to industry, with a particular focus on small and medium-sized enterprises
• provides relevant, reliable, and objective information
• established in 2007 in Finland
• works for chemical safety

The European Medicines Agency (EMA) is the regulatory authority responsible for evaluating, supervising and safety monitoring of medicines in the European Union (EU). Established in 1995, the EMA ensures that medicinal products meet high safety, efficacy, and quality standards before entering the European market.

The agency is co-funded by the pharmaceutical industry and the European Union and is responsible for harmonizing regulatory requirements across EU Member States.

Also known as “Article 57 database” referring to Article 57(2) of Regulation 726/2004. The XEVMPD is a database with the aim of establishing a complete inventory of all medicines authorised (centrally and on national level) in the EU and EEA.

A food supplement is a concentrated source of nutrients or other substances designed to complement the normal diet. Food supplements are marketed in dose form such as capsules, tablets, powders, liquids, and ampoules.

In the European Union (EU), food supplements are regulated under Directive 2002/46/EC, which sets rules on composition, labeling, and marketing.

Good Manufacturing Practice (GMP) is a set of regulations and guidelines that ensure pharmaceutical products are consistently produced and controlled according to quality standards. GMP compliance is essential for regulatory approval, product safety, and market access in the pharmaceutical, biotechnology, and medical device industries.

GMP covers manufacturing processes, facility requirements, personnel training, documentation, quality control, and post-market surveillance to minimize risks such as contamination, mix-ups, and deviations from specifications.

These guidelines provide minimum requirements that a manufacturer must meet to assure that their products are consistently high in quality, from batch to batch, for their intended use.

A set of measures to facilitate safety monitoring of medicines in the European Union. 

The Health Products Regulatory Authority (HPRA) is Ireland’s national regulatory agency responsible for overseeing the safety, quality, and efficacy of human and veterinary medicines, medical devices, and other health products.

Founded in 1996 as the Irish Medicines Board (IMB), the HPRA ensures compliance with national and European Union (EU) regulations while advising the Irish government on health product safety and public health policies.

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) is a global initiative that develops harmonised regulatory guidelines for pharmaceutical quality, safety, and efficacy.

ICH was originally established as the International Conference on Harmonisation and now operates as an international association under Swiss law. Its goal is to align regulatory requirements across major pharmaceutical markets, ensuring that medicines meet high safety and quality standards worldwide.

An Individual Case Safety Report (ICSR) is a documented report of a suspected adverse drug reaction (ADR) in a single patient following the use of a specific medicine.

Medicines under additional monitoring have a black inverted triangle  displayed in their package leaflet, together with a short sentence that reads:      
▼ "This medicinal product is subject to additional monitoring." All medicines are carefully monitored after they are placed on the EU market. However, medicines with the black triangle are being monitored even more closely than others. This is generally because there is less information available about them compared with other medicines, for example because they are new on the market. Examples of medicines under additional monitoring include new medicines authorised since the start of 2011 and medicines for which regulators require more studies to be carried out, e.g. on long-term use or on rare side effects seen in clinical trials.

An Investigational Medicinal Product (IMP) is defined in Article 2(2)(5) of Regulation (EU) No 536/2014 as:
“a medicinal product which is being tested or used as a reference, including as a placebo, in a clinical trial”.

This includes:

• New medicinal products that have not yet received a marketing authorisation.
• Authorized medicines which are used outside the terms of their marketing authorisation (e.g., new indication, formulation or dose).

An Investigational Medicinal Product Dossier (IMPD) is a regulatory document required for clinical trial applications (CTAs) in the European Union (EU). It provides comprehensive data on the quality, safety, and efficacy of an Investigational Medicinal Product (IMP).

IMPD submission is mandated under Regulation (EU) 536/2014, which governs the approval of clinical trials in EU Member States.

Labelling refers to the mandatory information displayed on the immediate and outer packaging of medicinal products. The immediate packaging refers to the container or other form of packaging immediately in contact with the medicinal product. The outer packaging refers to the packaging into which is placed the immediate packaging. Labelling regulations ensure that patients, healthcare professionals, and regulators can access critical information about a medicine’s composition, usage, and safety.

Once a Marketing Authorisation (MA) is granted for a medicinal product, the Marketing Authorisation Holder (MAH) must ensure its continuous compliance with scientific, technical, and regulatory standards.

In Europe, under Article 23 of Directive 2001/83/EC, the MAH is required to update the product to reflect new scientific knowledge, quality improvements, and pharmacovigilance requirements. These updates often involve variation applications, renewal submissions, and periodic reporting obligations.

A Marketing Authorisation (MA) is a regulatory approval that allows a medicinal product to be marketed and sold in a specific country or region. It is granted to the Marketing Authorisation Holder (MAH) after an evaluation of the product’s safety, efficacy, and quality.

A variation is a change to the terms of a granted marketing authorisation (MA) for a medicinal product. Variations must be submitted by the Marketing Authorisation Holder (MAH) to ensure that medicines continue to comply with regulatory requirements, scientific advancements, and safety updates.

Variations may involve changes in manufacturing processes, safety and efficacy data, packaging, or quality control measures. The approval process for variations follows specific European Medicines Agency (EMA) and national competent authority (NCA) regulations.

The Medical Device Regulation (MDR) (Regulation (EU) 2017/745) is the legal framework governing the manufacturing, certification, and marketing of medical devices in the European Union (EU).

It replaces Directive 93/42/EEC (Medical Devices Directive - MDD) and Directive 90/385/EEC (Active Implantable Medical Devices Directive - AIMDD). The Medical Device Regulation became applicable on 26 May 2021.

<sub>The European Union extended the EU MDR transition periods for ‘legacy devices’ under certain conditions to: 

26 May 2026 for class III implantable custom-made devices. 
31 December 2027 for class III and implantable class IIb devices (excluding certain Well-Established Technology (WET) devices)  
31 Dec 2028 for devices covered by valid MDD/AIMDD Certificates (as of 2023/03/20) and that are other Class IIb devices (excluding implants , but including certain WET-devices), or Class IIa devices, or Class I sterile devices or Class I devices with a measuring function;
31 Dec 2028 for devices that did not require Notified Body certification under the MDD and for which the declaration of conformity was drawn up prior to 26 May 2021, but now require Notified Body certification under the MDR</sub>

The Medicines & Healthcare products Regulatory Agency (MHRA) is the UK regulatory authority responsible for the approval, monitoring, and safety of medicines, medical devices, and blood components for transfusion.

The MHRA operates under the Department of Health and Social Care (DHSC) and ensures compliance with Good Manufacturing Practice (GMP), Good Distribution Practice (GDP), and pharmacovigilance regulations.

Following Brexit, the MHRA now operates independently from the European Medicines Agency (EMA) for medicines and devices approvals in the UK.

The Mutual Recognition Procedure (MRP) is a marketing authorisation pathway in the European Union (EU) that allows a medicinal product that is already approved in one or more EU countries to be recognised in other EU countries.

MRP is commonly used for medicinal products that have been granted a National Marketing Authorisation (NMA) in one EU country or authorisations in some countries via a Decentralised Procedure (DCP), but which are then sought to be approved in additional Member States.

A National Authorisation Procedure is a regulatory approval pathway that allows a medicinal product to be marketed in a single European Union (EU) Member State. It is granted by the country’s National Competent Authority (NCA) and does not provide automatic approval in other EU countries. Its equivalent in the UK is called National Assessment Procedure

This pathway is different from the Centralised and Mutual Recognition/Decentralised Procedures, which allow access to multiple EU Member States. Companies seeking approval in only one country can use the National Authorisation route.

The decision, whether a substance can be classified as new active substance is taken by CHMP. Guidance is given by the reflection paper (EMA/CHMP/QWP/104223/2015), published first in 2015 which describes the chemical structure and properties criteria taken into account as well as the required documents to be submitted by the applicant to support their claim.

An orphan drug is a pharmaceutical product developed for the treatment, prevention or diagnosis of rare diseases.
Due to the limited commercial potential, regulatory agencies provide incentives to encourage orphan drug research and development.
The requirements to receive Orphan Drug Designation (ODD) status as well as the related incentives differs by region.

A Periodic Safety Update Report (PSUR) is a mandatory pharmacovigilance document that provides an ongoing evaluation of the benefit-risk profile of an authorised medicinal product. It is required for all medicinal products in the European Union (EU) and other regulated markets to ensure their continued safety and effectiveness.

PSURs must be submitted by the Marketing Authorisation Holder (MAH) at defined intervals to regulatory authorities such as the European Medicines Agency (EMA), U.S. Food and Drug Administration (FDA), and national competent authorities (NCAs).

Post-marketing changes, also known as variations, refer to modifications made by the Marketing Authorisation Holder (MAH) to an approved medicinal product after its initial marketing authorisation (MA). These changes ensure that the medicine remains safe, effective, and of high quality throughout its lifecycle.

Regulatory agencies such as the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA), and national competent authorities (NCAs) have specific approval and notification procedures for different types of variations.

The Product Information (PI) is a mandatory component of the marketing authorisation for medicinal products in the European Union (EU). It consists of standardised documents that guide both healthcare professionals and patients on the correct and safe use of a medicine. The structure and requirements for PIs are defined under EU pharmaceutical legislation, including Directive 2001/83/EC. These documents include, among others, the summary of the product characteristics, the labelling and the package leaflet and they are assessed and approved as part of the authorisation process by national competent authorities (NCA) or the European Medicines Agency (EMA), depending on the regulatory route.

A Regulatory Affairs Consultancy provides expert guidance to pharmaceutical, biotechnology and medical device companies across all stages of product development, authorisation and post-marketing compliance. These consultancies help clients navigate complex and evolving regulatory frameworks, ensuring that products meet the legal, scientific and procedural requirements for successful market access.

Whether supporting early-stage biotech start-ups, mid-sized pharmaceutical companies without in-house regulatory teams, or global firms entering the European market, consultancies offer tailored, scalable solutions. Their services are particularly valuable during strategic transitions such as mergers and acquisitions, or the launch of novel therapies.

A Regulatory Strategy is a structured plan that guides the development, approval, and post-market management of a medicinal product or a medical device. It ensures compliance with global regulatory requirements while optimizing time-to-market, cost efficiency, and commercial success.

A strong regulatory strategy aligns scientific development with legal, regulatory, safety, and commercial considerations, enabling pharmaceutical and medical device companies to navigate complex approval pathways in various markets.