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 is a noxious and unintended response to a medicine, whereas an adverse event is an untoward medical occurrence after exposure to a medicine, which is not necessarily caused by that medicine.

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.

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.

A Drug Master File (DMF) is a confidential document submitted to regulatory authorities that 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 already approved in one EU country (Reference Member State - RMS) to be recognised in other EU countries (Concerned Member States - CMS).

MRP is commonly used for medicines that have been granted a National Marketing Authorisation (NMA) in one EU country and need approval 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 or in the UK. It is granted by the country’s National Competent Authority (NCA) and does not provide automatic approval in other EU countries.

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.

A New Drug Application (NDA) is the formal request submitted to the U.S. Food and Drug Administration (FDA) to gain approval for marketing a new pharmaceutical product in the United States. The NDA must provide comprehensive clinical, preclinical, and quality data demonstrating the drug’s safety, efficacy, and manufacturing consistency.

An approved NDA grants the manufacturer permission to market the drug in the U.S. under the Federal Food, Drug, and Cosmetic Act (FD&C Act).

An orphan drug is a pharmaceutical product developed for the treatment, prevention or diagnosis of rare diseases that are life-threatening or chronically debilitating. Due to the limited commercial potential, regulatory agencies provide incentives to encourage orphan drug research and development.

The definition of a rare disease differs by region:

• In the European Union (EU) – A disease is classified as rare if it affects fewer than 5 in 10,000 people.
• In the United States (U.S.) – A disease is considered rare if it affects fewer than 200,000 people per year (approximately 7.5 per 10,000 population).

Orphan Drug Designation (ODD) is a regulatory status granted to pharmaceutical products developed to treat rare diseases. Because rare diseases affect only a small percentage of the population, developing treatments is often financially unviable for pharmaceutical companies. To address this issue, regulatory agencies offer financial incentives, market exclusivity, and expedited review processes for orphan drug development.

Orphan drug regulations exist in multiple regions, with the US and the EU leading efforts to support rare disease treatments.

Over-the-Counter (OTC) products are medicinal products that can be purchased without a prescription. They are used for the self-treatment of common conditions such as pain, colds, allergies, digestive issues, and skin conditions.

OTC medicines must meet strict safety, efficacy, and labeling requirements set by regulatory agencies such as the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and national health authorities.

A Package Leaflet (PL), also known as a Patient Information Leaflet (PIL), is a mandatory document included in the packaging of all medicinal products authorized in the European Union (EU). It provides essential information about the medicine to ensure safe and effective use by patients.

The PIL must be clear, legible, and understandable, following the requirements of Directive 2001/83/EC, Articles 58 & 63. It must be consistent with the Summary of Product Characteristics (SmPC), which is intended for healthcare professionals.

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 marketed medicines 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).

A Pharmaceutical Consultant is a specialized expert who provides strategic guidance and regulatory support to pharmaceutical companies, biotech firms, and healthcare organizations. Their role involves ensuring compliance with regulatory requirements, optimizing drug development strategies, and enhancing operational efficiency across the pharmaceutical value chain.

Pharmaceutical consultants offer advisory services in areas such as regulatory affairs, clinical trials, quality assurance, drug safety, and market access. They work with small biotech startups, large pharmaceutical corporations, contract research organizations (CROs), and governmental agencies.

Pharmacovigilance (PV) is the science and practice of detecting, assessing, understanding, and preventing adverse effects or any other drug-related problems (adverse drug reactions) or special situation reports (pregnancy, misuse, quality issues, etc.). It plays a crucial role in ensuring the safety and efficacy of medicinal products throughout their lifecycle, from clinical trials to post-marketing surveillance.

Pharmacovigilance is a mandatory regulatory requirement for pharmaceutical companies, marketing authorization holders (MAHs), and healthcare professionals, enforced by agencies such as the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA), and the World Health Organization (WHO) and local competent authorities.

A Pharmacovigilance (PV) Post refers to the post-marketing surveillance activities and regulatory obligations required for monitoring the safety of medicinal products after they have been approved and made available to patients. This includes adverse drug reaction (ADR) reporting, risk management, periodic safety updates, and signal detection.

Regulatory authorities such as the European Medicines Agency (EMA), U.S. Food and Drug Administration (FDA), and the World Health Organization (WHO) enforce strict Good Pharmacovigilance Practices (GVP) to ensure continued drug safety and efficacy throughout the product lifecycle.

The PSMF is a legal requirement in the EU and is defined in Article 1(28e) of Directive 2001/83/EC: a detailed description of the pharmacovigilance system used by the marketing authorisation holder with respect to one or more authorised medicinal products.

More details about the structure of a PSMF can be found in Module II of the Guideline on good pharmacovigilance practices (GVP).

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.

Advertisement of medicinal products comprises activities designed to promote the prescription, supply, sale or consumption of medicinal products as regulated in 2001/83/EC.

The QP focuses on quality control and quality assurance during the manufacturing process.

A Qualified Person for Pharmacovigilance (QPPV) is a legally mandated role within the European Economic Area (EEA) and other international authorities (FDA, etc.) responsible for ensuring a company’s compliance with pharmacovigilance (PV) regulations. The QPPV oversees drug safety monitoring, adverse event reporting, and risk management activities for all medicinal products under a Marketing Authorisation Holder (MAH).

The position is required by law under Article 10 of Regulation (EC) No. 726/2004 and Article 104(3) of Directive 2001/83/EC.

A Quality Overall Summary (QOS) is a comprehensive summary of the quality-related data included in Module 3 of a Marketing Authorisation Application (MAA) or New Drug Application (NDA) dossier. The QOS provides a structured overview of the manufacturing process, drug substance, and drug product quality control, facilitating regulatory review by agencies such as the European Medicines Agency (EMA), U.S. Food and Drug Administration (FDA), and other national health authorities.

The QOS is part of the Common Technical Document (CTD) format, which standardizes the drug approval process across regulatory regions.

The QRD Working group is an EMA working group that provides templates for product information which can be used by applicants and MAH. Those templates are updated on a regular basis and can be downloaded in all European language on EMA’s website.

Templates differ slightly depending on the legal basis of the MAA.  Templates for centrally authorized products are available as well as templates for products for ATMPs. For mutual recognition, decentralized, referral and PSUR single assessment (PSUSA) procedures an adapted version is provided. Further appendices give standardized statements to be used in the product information e.g. in section 4.6 “pregnancy and lactation” or section 4.8 “undesirable effects”. But also terms and abbreviations for batch number and expiry for all European languages can be found in a QRD Appendix.

A Regulatory Strategy is a structured plan that guides the development, approval, and post-market management of a medicinal product, medical device, or biologic. 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, safety, and commercial considerations, enabling pharmaceutical and medical device companies to navigate complex approval pathways in various markets.

A Marketing Authorisation (MA) renewal is the regulatory process of extending the validity of an approved medicinal product in the European Union (EU) beyond the initial five-year period. The renewal is granted based on a reassessment of the benefit-risk profile of the medicinal product.

An RMP needs to be submitted with each marketing authorization application. It should include the following information:

• Safety profile of the medicine
• Risk prevention or minimization measures
• Plan for studies or further activities to gain more knowledge about the safety
• How effectiveness of the risk minimization measures can be measured
• RMPs need to be updated regularly
• Part of good pharmacovigilance practice and further described in module V

A Summary of Product Characteristics (SmPC) is an official regulatory document required for all authorised medicinal products in the European Union (EU). It contains scientific and medical information intended for healthcare professionals, including prescribing doctors, pharmacists, and regulatory authorities.

SmPCs are regulated under Directive 2001/83/EC, Article 11 and serve as the primary reference for the safe and effective use of medicines. The SmPCs have to be available in the official language(s) of the Member State where the product is marketed.

Annex I of the so called Product Information is the summary of Product Characteristics, which is a document comprising the basis information for healthcare professionals on how to use the medicine safely and effectively. Please refer to the guideline on summary of product characteristics for the full information to be included in the document.