GLOSSARY
Third-Party Logistics (3PL) refers to outsourcing logistics to a specialised provider. In pharmaceuticals, a 3PL manages warehousing, distribution, supply chain flows, and sometimes reverse logistics.
This solution enables companies to focus on core activities while relying on expert support and regulatory compliance.
Compassionate Access Authorisation (AAC) is a regulatory pathway in France enabling patients with serious or rare conditions, who have no satisfactory therapeutic alternative, to benefit from innovative medicines prior to marketing approval.
Supervised by the French Medicines Agency (ANSM), it ensures patient safety while facilitating early access to treatments addressing urgent unmet medical needs.
For Cevidra, supporting companies in obtaining and managing AACs is a key expertise, helping biopharmaceutical partners make their innovative therapies rapidly available in France.
Early access authorisation for innovative medicines prior to reimbursement
The Early Access Authorisation (AAP) applies to innovative medicines for severe or rare diseases addressing unmet medical needs. Managed by HAS and ANSM, it enables patients to access treatment before official reimbursement.
This framework ensures strict supervision, including a data collection protocol and continuous assessment of the benefit-risk balance.
It is designed to provide patients with faster access to innovative therapies while supporting the generation of robust evidence for subsequent market and reimbursement decisions.
Compassionate access to a drug without MA in exceptional cases
Compassionate Access (AC) refers to the exceptional use of a medicine that has not yet obtained marketing authorisation.
It is intended for patients with severe conditions and no alternative treatment options.
This procedure ensures timely medical response under the supervision of the health authority, with rigorous patient and treatment monitoring.
Its objective is to balance urgent therapeutic needs with safety requirements, offering access to innovative solutions when no other options are available.
General Agency for Health Products and Equipment (AP-HP)
The General Agency for Health Products and Equipment (AGEPS) is part of AP-HP. It manages procurement, production, and distribution of medicines and devices for Paris hospitals.
AGEPS also supports pharmaceutical innovation and ensures access to rare or specific treatments for hospital use.
Definition
French agency assessing, authorising and monitoring medicines and devices
The French National Agency for the Safety of Medicines and Health Products (ANSM) is the competent authority responsible for regulating medicines, vaccines, and medical devices in France.
It oversees evaluation, market authorisation, and post-market surveillance. Its mission is to ensure quality, efficacy, and safety of health products while enabling access to therapeutic innovations.
ANSM plays a central role in compassionate and early access decisions, as well as in pharmacovigilance and risk monitoring activities.
Mission and Responsibilities
ANSM’s mission is to protect public health by evaluating, monitoring, and regulating health products. Its responsibilities include:
1. Evaluation and Authorisation
- Assessing marketing authorisation applications (AMM) for national procedures
- Authorising clinical trials conducted in France
- Granting Early Access Authorisations (AAP/AAC) for innovative therapies
- Overseeing advertising and promotional materials for medicines
2. Pharmacovigilance and Safety Monitoring
- Monitoring adverse events and emerging safety signals
- Coordinating national pharmacovigilance networks
- Implementing risk-minimisation measures and product recalls when necessary
3. Market Surveillance
- Inspecting manufacturing, distribution, and import sites
- Ensuring compliance with GMP, GDP, and other quality standards
- Controlling the quality of products placed on the French market
4. Transparency and Information
- Publishing safety communications, recommendations, and regulatory decisions
- Providing scientific and regulatory guidance to healthcare professionals and industry stakeholders
ANSM also contributes scientific expertise to European procedures coordinated by the European Medicines Agency (EMA).
Role in the European Regulatory System
France is a key participant in EU pharmaceutical regulation, and ANSM experts contribute to:
- EMA committees and working groups
- EU pharmacovigilance activities (including EudraVigilance reporting)
- Inspections and quality oversight across Member States
- Regulatory development and harmonisation initiatives
Through this collaboration, ANSM helps shape European standards and supports consistent application of EU legislation.
Products Regulated by ANSM
ANSM supervises a broad range of products, including:
- Prescription and over-the-counter medicines
- Vaccines and biologicals
- Medical devices and in vitro diagnostics
- Blood products and tissues/cells
- Cannabis for medical use (pilot programme)
- Cosmetic and tattoo products
- Raw materials used in pharmaceutical manufacturing
Importance of ANSM in Public Health
ANSM plays a strategic role by:
- Ensuring rapid access to safe, high-quality medicines
- Responding to health emergencies and shortages
- Enforcing strict quality and safety standards
- Communicating transparently with the public and healthcare community
Its decisions directly influence patient safety, therapeutic availability, and regulatory oversight in France.
Early access to certain drugs before final reimbursement decision
Early Access (AP) is a mechanism that allows patients to obtain innovative treatments before reimbursement approval.
It applies to medicines addressing serious, unmet medical needs and helps reduce the gap between clinical development and effective patient access.
The framework is strictly regulated and requires real-world data collection, providing valuable evidence for future assessment and reimbursement decisions.
It ensures timely patient benefit while maintaining safety and regulatory oversight.
AP1 (Early Access pre-MA) is an authorisation granted by HAS in France allowing patients to access a promising medicine before marketing approval.
It targets innovative treatments addressing unmet medical needs, supported by robust clinical data. AP1 includes a monitoring protocol and real-world data collection.
AP2 (Early Access post-MA) enables patients to benefit from a medicine after European marketing authorisation but before reimbursement in France.
It ensures rapid access to medical innovations while gathering additional safety and efficacy data. AP2 is part of France’s broader early access framework.
Added therapeutic benefit of a new treatment compared with standard care
The Added Therapeutic Benefit (ASMR) is an evaluation issued by the HAS to measure the clinical improvement of a new drug compared with existing treatments.
ASMR is ranked from I to V, reflecting the level of innovation provided. This rating directly impacts price negotiations and reimbursement decisions.
A high ASMR (added therapeutic value) leads to stronger market positioning and favourable pricing, while a low ASMR may reduce economic impact even if the drug obtains marketing authorisation.
Advanced therapy medicinal products (cell, gene, tissue-based)
Advanced Therapy Medicinal Products (ATMPs) include gene therapies, cell-based therapies, and tissue-engineered products.
They represent a breakthrough in modern medicine by providing targeted solutions for rare and severe diseases.
Their development is strictly regulated under European law to ensure safety and quality. As these therapies emerge, there is increasing need for robust clinical data collection and post-marketing surveillance to confirm their long-term efficacy and safety in real-world settings.
Temporary authorisation for use of a non-approved medicine
The Temporary Authorisation for Use (ATU) was a French framework granting access to medicines before marketing authorisation, in cases of severe diseases without therapeutic alternatives.
It could be nominative or cohort-based, with close monitoring by ANSM. Since 2021, this scheme has been replaced by the Early Access (AAP) and Compassionate Access (AAC) frameworks. They now regulate early patient access to innovative therapies within a modernised regulatory system.
Temporary authorisation granted for a cohort of patients with the same condition
The Cohort ATU was an ANSM procedure that granted a group of patients with the same condition access to a medicine before marketing authorisation. It included a data collection protocol to assess the benefit-risk balance.
This system was replaced in 2021 by the Early Access Authorisation (AAP), which now regulates large-scale early access to innovative medicines in France.
Temporary authorisation granted for an individual patient on physician request
The Nominative ATU was an authorisation allowing a physician to request the use of a non-approved medicine for a specific patient.
Granted by ANSM in cases of severe disease and lack of alternatives, it responded to urgent individual needs. This scheme was replaced in 2021 by the Compassionate Access Authorisation (AAC), which now regulates such individual cases.
EMA’s Committee for Advanced Therapies
The Committee for Advanced Therapies (CAT) is a scientific body of the European Medicines Agency (EMA).
It evaluates advanced therapy medicinal products (ATMPs), including gene, cell, and tissue-based therapies.
Its scientific opinions provide input to the CHMP for the final marketing authorisation decision.
The CAT plays a critical role in assessing the quality, efficacy, and safety of next-generation treatments, supporting innovation in advanced therapies across the European Union.
Economic Committee for Health Products
The Economic Committee for Health Products (CEPS) is a French interministerial body. It negotiates reimbursed drug prices and regulates the economic conditions of medical devices. Its decisions rely on HAS evaluations (SMR, ASMR) and economic criteria, balancing therapeutic innovation with healthcare system sustainability.
EMA Committee for Medicinal Products for Human Use
The Committee for Medicinal Products for Human Use (CHMP) is a key body within the EMA responsible for evaluating centralised marketing authorisation applications.
It issues scientific opinions on the quality, efficacy, and safety of medicines for human use. Its recommendations form the basis for European Commission decisions to grant or refuse marketing authorisation.
The CHMP is one of the most influential committees in European pharmaceutical regulation.
Conditional Marketing Authorisation requiring further data
Conditional Marketing Authorisation (CMA) is a European mechanism that enables early access to a medicine based on incomplete data, when the potential benefit outweighs remaining uncertainties.
CMA is typically granted for serious, rare diseases or public health emergencies.
The marketing authorisation holder must commit to providing additional data within an agreed timeframe to confirm the product’s safety and efficacy profile.
Concerned Member State in an EU authorisation procedure.
In decentralised (DCP) and mutual recognition (MRP) procedures, a Concerned Member State (CMS) is an EU country participating in the evaluation of a marketing authorisation application, under the coordination of a Reference Member State (RMS).
The CMS must review, accept, or comment on the RMS’s scientific assessment, and its agreement is required to obtain market authorisation in that country.
EMA Committee for Orphan Medicinal Products
The Committee for Orphan Medicinal Products (COMP) is a scientific committee within the EMA responsible for reviewing orphan drug designation applications.
Orphan designation applies to medicines for rare diseases and provides regulatory and economic benefits, such as market exclusivity and reduced fees.
The COMP assesses the medical relevance and potential of these medicines before their development and market entry in Europe.
Centralised marketing authorisation procedure in the EU
The Centralised Procedure (CP) allows pharmaceutical companies to obtain a single marketing authorisation valid across all EU Member States and the EEA.
This procedure is mandatory for certain categories of innovative medicines, such as biotechnologies and advanced therapies.
Managed by the EMA, the process results in a final decision by the European Commission, granting or refusing the authorisation.
Chief Pharmaceutical Officer (“Pharmacien Responsable”)
The Chief Pharmaceutical Officer (CPO), in France known as “Pharmacien Responsable,” is the legally accountable person for pharmaceutical compliance within a marketing authorisation holder.
The CPO ensures adherence to Good Manufacturing Practices (GMP), distribution rules, and regulatory obligations.
CPO is essential in safeguarding the quality and safety of medicines placed on the market or distributed to patients.
Clinically relevant comparator in clinical evaluation
The Clinically Relevant Comparator (CRC) is the reference treatment selected in clinical trials or health technology assessments to evaluate the efficacy of a new medicine.
The choice of CRC has a direct impact on demonstrating therapeutic benefit and on reimbursement decisions. The CRC must accurately reflect real-world medical practice within the studied indication.
Contract Research Organisation conducting trials and R&D
A Contract Research Organisation (CRO) is a service provider offering outsourced research and development activities, particularly clinical trial management.
Pharmaceutical companies and biotechs rely on CROs for scientific, regulatory, and operational expertise.
CROs play a crucial role in accelerating drug development by ensuring data quality, compliance with international regulations, and efficient trial execution.
The Transparency Commission (CT), under HAS, assesses the clinical efficacy and actual benefit of medicines. Its opinions determine reimbursement eligibility and coverage levels by national health insurance.
CT plays a key role in regulating access to innovative treatments in France.
Decentralised Procedure for EU marketing authorisation
The Decentralised Procedure (DCP) is a European route for obtaining marketing authorisation, allowing companies to apply simultaneously in multiple EU Member States without using the centralised procedure.
The DCP relies on a scientific assessment by a Reference Member State (RMS), which must then be recognised by the Concerned Member States (CMS). DCP is commonly used for generic medicines and certain well-established products.
ANSM digital platform for compassionate/early access
e-saturne is ANSM’s digital platform for submitting and tracking Compassionate Access (AAC) and Early Access (AAP) applications. It centralises communication between industry and health authorities, ensuring traceability and transparency.
The tool simplifies and harmonises access to innovative therapies.
UK Early Access to Medicines Scheme
The Early Access to Medicines Scheme (EAMS) is a UK framework enabling patients with serious diseases to access innovative medicines before marketing authorisation.
Supervised by the MHRA, it applies to treatments addressing unmet medical needs. The scheme provides early availability of promising therapies while ensuring structured collection of clinical and safety data to support future regulatory and reimbursement decisions.
Early Access Programs for innovative medicines
Early Access Programs (EAPs) are schemes established by health authorities or pharmaceutical companies to provide patients with innovative treatments before marketing authorisation.
EAPs typically target serious or rare diseases and address urgent unmet medical needs.
EAPs often include structured clinical follow-up and contribute to generating real-world evidence, which supports regulatory submissions and facilitates earlier patient access to promising therapies.
The European Commission (EC) is the executive body of the European Union.
It is responsible for implementing legislative and regulatory decisions, including those related to marketing authorisations of medicines.
Based on the scientific recommendations of the EMA’s CHMP, the Commission issues final centralised MA decisions, which are binding across all EU Member States and the European Economic Area.
European Economic Area (EU + EFTA countries)
The European Economic Area (EEA) includes EU Member States and three European Free Trade Association (EFTA) countries (Norway, Iceland, Liechtenstein).
It enables regulatory and commercial harmonisation, including for marketing authorisation of medicines.
A centralised MA granted by the European Commission is valid across all EU and EEA countries, ensuring uniform access to medicines within this extended market.
European Federation of Pharmaceutical Industries and Associations
The European Federation of Pharmaceutical Industries and Associations (EFPIA) is an organisation representing the leading pharmaceutical companies and national associations in Europe.
The European Federation of Pharmaceutical Industries and Associations (EFPIA) advocates for innovation, access to medicines, and the advancement of clinical research.
EFPIA plays a central role in shaping policy dialogue between regulators, patients, and industry stakeholders at the European level.
The European Medicines Agency (EMA) is the regulatory authority for medicines in the European Union.
Based in Amsterdam, it coordinates scientific evaluation, post-marketing surveillance, and pharmacovigilance.
EMA manages centralised marketing authorisation procedures and relies on several scientific committees (CHMP, CAT, COMP, PRAC). It plays a key role in harmonised access to medicines across the EU and EEA.
European Network of Paediatric Research at EMA
The European Network of Paediatric Research (EnprEMA) is an initiative coordinated by the EMA.
It brings together research networks, academic groups, and patient organisations to promote high-quality paediatric clinical research.
EnprEMA helps optimise Paediatric Investigation Plans (PIPs) and improve the availability of medicines adapted to children in Europe.
For more information, visit their website here.
European Public Assessment Report
The European Public Assessment Report (EPAR) is published by the EMA for every medicine granted a centralised marketing authorisation.
It provides a publicly accessible summary of the clinical data, efficacy, safety, and quality of the evaluated product.
EPARs are an important source of transparent information for healthcare professionals, patients, and researchers, helping build trust in regulatory decisions.
European Clinical Trials Database
EudraCT is the centralised database of clinical trials conducted in the European Union and European Economic Area.
It contains detailed information on protocols, objectives, and methodologies.
Since 2022, it has been integrated into the CTIS portal, but remains a historical reference for clinical trial registration and transparency.
Summary
European database on Manufacturing and GMP authorisations
EudraGMP is a public database managed by the EMA. It lists manufacturing and import authorisations as well as Good Manufacturing Practice (GMP) compliance certificates.
It is a key tool for transparency and quality control, enabling authorities and industry partners to verify the compliance of pharmaceutical manufacturing sites.
Definition
EudraGMP, part of the broader EudraGMDP database, is the official European database that lists Manufacturing and Import Authorisations (MIA/IMAs) and Good Manufacturing Practice (GMP) certificates issued by EU regulatory authorities.
It is maintained by the European Medicines Agency (EMA) and used by EU Member States, the European Commission, and other regulatory partners to ensure transparency, oversight, and harmonised GMP compliance across the European pharmaceutical system.
EudraGMDP is accessible to the public, although certain regulatory fields remain restricted for competent authorities.
Purpose of the Database
EudraGMP/EudraGMDP supports regulatory control and industry compliance by providing a centralised view of:
- Manufacturing and import authorisations for medicinal products and active substances
- GMP certificates issued after inspections
- Statements of non-compliance with GMP
- Registrations of active substance manufacturers and importers
This database improves transparency, facilitates inspections, and helps ensure the quality and safety of medicines circulating in the EU.
Key Components
EudraGMDP contains several interconnected modules:
- Manufacturing and Import Authorisations (MIA/IMA): records authorisations granted to companies that manufacture, import, or certify medicinal products for the EU market.
- GMP Certificates: lists sites that have been inspected and found compliant with EU GMP requirements. Certificates detail the scope of manufacturing activities (e.g., sterile products, biologics, APIs).
- Statements of Non-Compliance: published when an inspected facility fails to meet GMP standards. These notices inform regulators, industry, and supply chain partners of significant compliance concerns.
- Active Substance Registrations: includes information on manufacturers, importers, and distributors registered under EU obligations for active substances (API) used in medicinal products.
Who Uses EudraGMP?
The database is widely consulted by:
- Regulatory authorities conducting inspections or reviewing applications
- Pharmaceutical companies verifying supplier compliance
- Qualified Persons (QPs) assessing GMP status before batch certification
- Supply chain and quality teams ensuring traceability and regulatory conformity
Why EudraGMP Matters
EudraGMP plays a crucial role in safeguarding public health by:
- Ensuring that manufacturing sites meet EU GMP standards
- Providing transparency on compliance status for European and international partners
- Supporting regulatory decision-making for inspections, approvals, and variations
- Facilitating cooperation between Member States and third-country authorities
It is a foundational tool for maintaining the integrity of the EU’s pharmaceutical manufacturing and supply chain.
US Food and Drug Administration
The Food and Drug Administration (FDA) is the US federal authority responsible for regulating medicines, medical devices, vaccines, and food products.
It reviews marketing authorisation applications (NDA, BLA), supervises post-marketing safety, and sets quality standards.
The FDA plays a central role in granting access to therapeutic innovations in the US and significantly influences global pharmaceutical development strategies.
The Falsified Medicines Directive (FMD) is a European directive designed to protect the pharmaceutical supply chain against counterfeits.
It requires medicine packs to carry unique serialisation codes and safety features to verify authenticity.
This regulation strengthens traceability and reinforces patient confidence in the quality and safety of medicines distributed in Europe.
The Diagnosis Related Group (DRG) is the hospital financing unit in France (GHS “Groupe homogène de séjours”). Each hospital stay is classified according to medical and economic criteria to establish a fixed tariff.
This system supports fair resource allocation between hospitals and enhances healthcare cost management.
Good Pharmacovigilance Practices
Good Pharmacovigilance Practices (GVP) are the European guidelines governing the collection, evaluation, and prevention of adverse drug reactions. They aim to ensure patient safety after a product is marketed.
GVP define the responsibilities of marketing authorisation holders and health authorities, including the management of periodic reports and risk management plans.
A Health Authority (HA) is a public body responsible for evaluating, authorising, and monitoring medicines and medical devices.
It may be national (ANSM in France, FDA in the US) or international (EMA in Europe).
HAs set quality and safety standards, grant marketing authorisations, and oversee pharmacovigilance. Their role is central to ensuring patient access to safe and effective treatments.
French National Authority for Health – “Haute Autorité de Santé”
The French National Authority for Health (HAS) is an independent public body. It evaluates medicines, medical devices, and healthcare practices to ensure efficacy, safety, and public health value.
HAS issues opinions on reimbursement, care quality, and develops guidelines for healthcare professionals.
HealthCare Professionals (HCPs) include physicians, pharmacists, nurses, and other practitioners involved in patient care.
They are the main prescribers and users of medicines. Interactions with HCPs are strictly regulated to ensure transparency, appropriate use of treatments, and dissemination of validated medical information.
Their role is crucial in guaranteeing effective therapeutic monitoring and patient safety.
Health Technology Assessment (HTA) is a scientific and regulatory process that evaluates the medical, social, and economic value of a health technology, such as a medicine or medical device.
It supports reimbursement and pricing decisions by considering clinical effectiveness, cost, and healthcare system impact.
HTA helps ensure fair allocation of healthcare resources and promotes evidence-based decision-making.
List of hospital drugs reimbursed outside DRG tariffs
The “Liste en sus” is a French scheme reimbursing certain innovative or high-cost hospital medicines outside DRG tariffs. It ensures rapid access to treatments deemed essential by HAS while providing hospitals with tailored financial coverage.
Marketing Authorisation (MA) is the regulatory act that allows a medicine to be marketed in a country or region.
It is granted after evaluation of quality, efficacy, and safety data. Without an MA, a medicine cannot be distributed outside of exceptional frameworks (early or compassionate access).
MA ensures that the product meets the required standards for safe use in patients.
Marketing Authorisation Application
A Marketing Authorisation Application (MAA) is the dossier submitted to regulatory authorities to obtain an MA.
It includes results from clinical trials, as well as manufacturing, quality, and safety data.
In Europe, it can be submitted through centralised, decentralised, mutual recognition, or national procedures.
The MAA content is standardised to ensure harmonised and transparent evaluation by regulators.
Marketing Authorisation Holder
The Marketing Authorisation Holder (MAH) is the legal entity or person holding a medicine’s MA.
As the legal responsible party, the MAH must ensure regulatory compliance, pharmacovigilance, quality, and product availability. While some activities (distribution, exploitation) can be delegated, the MAH remains legally accountable for the medicine’s safety and efficacy on the market.
The Mutual Recognition Procedure (MRP) allows a medicine already authorised in one EU Member State to have its MA recognised in other European countries.
The application relies on the evaluation by the Reference Member State (RMS), which must be accepted by the other states.
This procedure facilitates harmonised market access across the EU without requiring the centralised procedure.
A National Competent Authority (NCA) is the regulatory body of an EU Member State responsible for evaluating, authorising, and monitoring medicines. In France, the ANSM is the NCA.
These authorities participate in European procedures (centralised, decentralised, mutual recognition) and oversee post-marketing surveillance.
They ensure the safety and quality of health products within their jurisdiction.
The Economic Interest Note (NIE) is a French document used in health assessments. It analyses the economic impact of a medicine or health technology on the national system.
NIEs provide essential input for pricing decisions and reimbursement negotiations, complementing clinical and health-economic data.
The Therapeutic Interest Note (NIT) or “Note d’intérêt thérapeutique” is an opinion issued by HAS in France to guide prescribers and authorities on a medicine’s medical value.
It highlights clinical interest in a specific indication and may influence reimbursement and therapeutic strategy decisions.
The National Procedure (NP) is a marketing authorisation route valid in a single country.
It applies when a company does not seek immediate access to multiple EU Member States.
Although increasingly replaced by European procedures, NP is still used for certain generic medicines or specific products limited to national markets.
Orphan Drug Designation (ODD) is a status granted by the EMA or FDA to medicines intended for rare diseases.
It provides regulatory and financial incentives such as market exclusivity, fee reductions, and scientific guidance.
The scheme encourages the development of treatments for conditions with high unmet medical needs, supporting innovation in orphan drug research.
An Over The Counter (OTC) medicine is a health product available without prescription. It usually treats minor conditions and can be purchased directly at a pharmacy.
These OTC medicines must demonstrate a high safety profile and low risk of misuse. Their availability improves patient access to treatment by enabling direct management of common symptoms without medical consultation.
Price & Reimbursement (P&R) refers to the French process of negotiating between pharmaceutical companies and CEPS. It establishes a medicine’s economic value based on clinical efficacy, ASMR, and budget impact.
P&R ensures patient access to treatments while maintaining healthcare system sustainability.
Summary
The European Pharmacopoeia (Ph. Eur.) sets official quality standards for active substances, excipients, and finished medicines in Europe.
These standards ensure compliance, safety, and efficacy of health products. Ph. Eur. is regularly updated to reflect scientific and regulatory progress.
It serves as an essential reference for pharmaceutical companies, pharmacists, and health authorities.
Credits: European Directorate for the Quality of Medicines & HealthCare
Definition
The European Pharmacopoeia (Ph. Eur.) is an official reference work that sets uniform, legally binding quality standards for medicines and their components in Europe.
It is published and maintained by the European Directorate for the Quality of Medicines & HealthCare (EDQM), under the authority of the Council of Europe.
Ph. Eur. standards apply to active substances, excipients, finished dosage forms, biological products, and materials used in the manufacture of medicinal products.
Purpose of the European Pharmacopoeia
Ph. Eur. ensures that medicines circulating in Europe meet consistent, harmonised quality requirements, regardless of their origin or manufacturer.
Its objectives are to:
- Protect public health by defining essential quality attributes
- Harmonise pharmaceutical standards across Europe
- Facilitate regulatory evaluation of medicinal products
- Support manufacturers in complying with quality and GMP requirements
- Promote international alignment through collaboration with other pharmacopoeias
Ph. Eur. is legally binding in all countries that are signatories to the Convention on the Elaboration of a European Pharmacopoeia.
What the Ph. Eur. Contains
1. Monographs
Monographs can apply to active substances, excipients, medicinal products, or biologicals. Authoritative quality standards describing:
- Identity tests
- Purity requirements
- Assay methods
- Limits for impurities
- Specific manufacturing or storage considerations
2. General Chapters
Overarching requirements and analytical methods covering:
- Microbiology
- Biological assays
- Sterility
- Physical and chemical testing
- Reference standards
- Pharmaceutical technology
These chapters provide validated methodologies accepted throughout Europe.
3. Reference Standards
Official substances produced by EDQM that ensure analytical consistency when applying monographs or general chapters.
Regulatory Importance
Ph. Eur. standards are recognised by:
- EU and EEA regulatory authorities
- National competent authorities across Europe
- Manufacturers and quality control laboratories
- Qualified Persons (QPs) during batch certification
Compliance with Ph. Eur. requirements is essential for marketing authorisation applications (MAA) and ongoing GMP compliance.
Ph. Eur. and Global Harmonisation
The European Pharmacopoeia collaborates with international bodies, including the Pharmacopoeial Discussion Group (PDG), to align standards with the US and Japanese pharmacopoeias.
This harmonisation reduces duplication, facilitates global development, and improves regulatory consistency.
Why the European Pharmacopoeia Matters
Ph. Eur. plays a central role in the pharmaceutical supply chain by:
- Ensuring medicines meet strict quality expectations
- Supporting manufacturers with verified analytical methods
- Providing regulators with a common scientific basis for assessment
- Promoting patient safety through transparent, harmonised standards
The Patient Information Leaflet (PIL) is a mandatory document provided with all medicines.
Written in clear language, it explains indications, dosage, precautions, adverse effects, and storage conditions.
Approved by health authorities, the PIL supports patient understanding of treatment and promotes the safe and appropriate use of medicines.
The Paediatric Investigation Plan (PIP) is a document required by the EMA for all new medicines.
It defines the paediatric clinical development strategy, including planned studies and scientific justifications.
Its goal is to ensure that innovative medicines are also assessed for paediatric use, addressing children’s medical needs in a safe and evidence-based way.
French Hospital Data Exchange Platforms (PLAGE / PASREL)
PLAGE and PASREL are French hospital data exchange platforms for secure transmission of health information between industry, hospitals, and authorities.
They allow submission and tracking of medicine-related files while ensuring data traceability and confidentiality.
These tools support efficient access and reimbursement processes.
National Programme for Hospital Information Systems
The National Programme for Hospital Information Systems (PMSI) or “Programme de Médicalisation des Systèmes d’Information” collects and structures data from hospital stays in France. Used for financing, it groups medical acts and assesses costs through Diagnosis Related Groups (GHS).
PMSI is also a key tool for epidemiological analysis and health policy management.
Pharmacovigilance Risk Assessment Committee
The Pharmacovigilance Risk Assessment Committee (PRAC) is a scientific committee within the EMA. It assesses the risks of medicines, including safety signals and periodic pharmacovigilance reports.
PRAC issues recommendations that may result in marketing authorisation changes, usage restrictions, or product withdrawals.
It plays a central role in safeguarding public health across the EU.
The Periodic Safety Update Report (PSUR) is a regulatory document regularly submitted by marketing authorisation holders.
It provides an updated assessment of a medicine’s safety profile, including reported adverse events and corrective actions taken.
PSUR enables health authorities to maintain continuous surveillance and reassess the benefit-risk balance of treatments.
Periodic Safety Update Report Single Assessment
The Periodic Safety Update Report Single Assessment (PSUSA) is a European procedure coordinated by the EMA. It centralises the evaluation of PSURs submitted for the same medicine across multiple Member States.
Its goal is to harmonise safety profile assessments and streamline regulatory decisions. PSUSA strengthens the consistency of pharmacovigilance across Europe.
Paediatric Use Marketing Authorisation
The Paediatric Use Marketing Authorisation (PUMA) is a European regulatory pathway designed to promote the development of medicines specifically for children. It provides regulatory incentives, including 10 years of market exclusivity.
PUMA applies to products already authorised in adults but requiring tailored clinical data for paediatric populations.
Protocol for temporary use and data collection (AAP)
The PUT-RD (Protocol for Temporary Use and Data Collection) applies to early access (AAP) medicines. It defines treatment conditions and organises real-world data collection. The collected information helps confirm clinical benefit and informs reimbursement decisions.
Protocol for temporary use and data collection (AAC)
The PUT-SP applies to compassionate access (AAC) medicines. It sets out treatment conditions and clinical follow-up criteria. As with PUT-RD, the collected data contribute to building knowledge about the medicine prior to marketing authorisation or reimbursement.
Pharmacovigilance (PV) is the set of activities for detecting, assessing, and preventing adverse drug reactions.
It ensures patient safety after marketing and relies on reporting, monitoring, and analysis systems. PV may lead to adjustments in product use, restrictions, or even withdrawals if risks are identified.
The Qualified Person (QP) is a mandatory regulatory role for any pharmaceutical company holding a manufacturing or import authorisation in Europe.
The QP certifies that each medicine batch is manufactured and tested according to GMP standards before release.
This function is essential to ensure product quality and patient safety.
Qualified Person for Pharmacovigilance
The Qualified Person for Pharmacovigilance (QPPV) is a mandatory role for all marketing authorisation holders in Europe.
They oversee compliance of the pharmacovigilance system, coordinate risk management, and ensure submission of safety reports. Based in the EU, the QPPV ensures that regulatory obligations on patient safety are fulfilled.
French hospital buyers’ network
The Hospital Buyers’ Network (RESAH) is a French public procurement organisation supporting hospitals in tendering processes. It covers medicines, medical devices, equipment, and services.
RESAH promotes economies of scale, efficient hospital procurement, and access to innovative healthcare solutions.
The Risk Management Plan (RMP) is a document required for all MA applications in Europe.
It describes identified and potential risks of a medicine, along with planned monitoring and minimisation measures. The RMP is regularly updated to include new real-world safety data.
It is a key tool for proactive pharmacovigilance.
The Reference Member State (RMS) is the national authority leading the scientific assessment of an MA dossier in mutual recognition (MRP) or decentralised (DCP) procedures. Other Member States (CMS) rely on its report to grant approval.
RMS plays a central role in harmonising regulatory evaluations across the EU.
Temporary Recommendation for Use
The Temporary Recommendation for Use (RTU) is a French framework regulating off-label use of an already marketed medicine.
Granted by ANSM, it defines prescription and monitoring conditions to ensure patient safety. RTUs are time-limited and address unmet needs while awaiting official new indications.
French digital platform for early/post-MA access applications
SÉSAME is the official HAS digital platform for submitting early (AP1) and post-MA (AP2) access applications.
It centralises exchanges between pharmaceutical companies and health authorities, streamlining submission tracking and validation. SÉSAME harmonises procedures and shortens review times for patient access to innovative medicines.
Small and Medium-sized Enterprises
The SME (Small and Medium-sized Enterprises) status is recognised by the EMA to support smaller companies in drug development. It provides financial (fee reductions), administrative, and regulatory incentives. This framework fosters innovation and helps emerging biotechs access European marketing authorisation procedures more easily.
Summary
Summary of Product Characteristics
The Summary of Product Characteristics (SmPC) is a regulatory document providing detailed information on a medicine: indications, dosage, contraindications, adverse effects, and precautions. Intended for healthcare professionals, it is the official reference for compliant and safe use.
SmPCs are validated during MA and regularly updated.
Definition
The SmPC (Summary of Product Characteristics) is the authoritative, regulatory document that describes how a medicinal product should be used in clinical practice.
Approved by regulatory authorities as part of the marketing authorisation, the SmPC provides essential information for healthcare professionals on the product’s indications, dosing, contraindications, pharmacology, safety profile, and handling requirements.
The SmPC serves as the legal basis for the safe and effective use of medicines in the EU and forms the foundation for patient-facing documents such as the Package Leaflet.
Purpose of the SmPC
The SmPC ensures that healthcare professionals have access to accurate, consistent, and up-to-date information about a medicinal product.
Its objectives are to:
- Provide clear instructions on posology, administration, and therapeutic indications
- Describe known risks, contraindications, and precautions
- Summarise pharmacological data supporting the product’s benefit–risk profile
- Guide prescribers, pharmacists, and other professionals in making informed decisions
- Establish the reference standard for promotional and educational material
The content of the SmPC is legally binding and must reflect the current scientific knowledge and post-authorisation data.
Structure of the SmPC
The SmPC follows a harmonised format defined in EU legislation and guidance. The main sections include:
1. Name, Composition, and Pharmaceutical Form
Identifies the medicinal product, active substances, excipients of known effect, and dosage form.
2. Clinical Information
Covers the key clinical aspects of use, including:
- Therapeutic indications
- Posology and method of administration
- Contraindications
- Special warnings and precautions
- Interaction with other medicinal products
- Use in pregnancy and lactation
- Effects on ability to drive or operate machinery
- Undesirable effects
- Overdose management
3. Pharmacological Properties
Describes:
- Pharmacodynamic properties
- Pharmacokinetic characteristics
- Preclinical safety data
4. Pharmaceutical Particulars
Includes:
- Shelf life and storage conditions
- Incompatibilities
- Container and packaging description
- Instructions for handling and disposal
5. Administrative Information
Lists marketing authorisation details, MAH contact information, and revision dates.
Regulatory Importance
The SmPC is a central document in the EU regulatory framework:
- It is approved by regulatory authorities and must be kept current.
- It informs pharmacovigilance activities, risk-management measures, and labelling updates.
- It is referenced by healthcare professionals for prescribing, dispensing, and counselling.
- It underpins the creation of the PL (Package Leaflet) and labelling texts.
The Actual Clinical Benefit (SMR) is an evaluation by HAS in France that measures clinical efficacy, safety, and public health relevance of a medicine. SMR level determines reimbursement eligibility and scope of coverage.
It is distinct from ASMR, which assesses improvement over existing treatments.
Summary
Suspected Unexpected Serious Adverse Reactions
Suspected Unexpected Serious Adverse Reactions (SUSARs) are serious, unexpected adverse events suspected to be related to a drug during a clinical trial. Prompt reporting to authorities and ethics committees is mandatory.
Managing SUSARs helps protect participants and may lead to protocol or dosage adjustments for the investigational medicine.
Definition
A SUSAR (Suspected Unexpected Serious Adverse Reaction) is a safety event occurring during a clinical trial that meets four criteria:
- Suspected: It is reasonably believed to be related to the investigational medicinal product (IMP).
- Unexpected: The nature or severity is not consistent with the product information (e.g., Investigator’s Brochure or SmPC).
- Serious: It results in death, is life-threatening, requires or prolongs hospitalisation, leads to significant disability, or is a congenital anomaly.
- Adverse Reaction: There is a suspected causal relationship with the IMP.
SUSARs require expedited reporting to regulators, ethics committees, and investigators.
Why SUSARs Matter in Clinical Trials
SUSARs are critical safety signals. Because they are both serious and unexpected, they may reveal new risks about the investigational product and require immediate regulatory oversight.
Timely reporting protects participants and enables regulators to assess whether protocol amendments, risk-mitigation measures, or trial suspension are needed.
Examples of SUSARs
- A life-threatening arrhythmia in a trial participant when this risk is not described in the Investigator’s Brochure
- A severe hypersensitivity reaction occurring at a frequency or intensity greater than expected
- A congenital anomaly not previously associated with the IMP
These events exceed what is known about the product at the time of reporting.
Expedited Reporting Requirements
Regulatory timelines differ depending on severity:
- Fatal or life-threatening SUSARs: must be reported within 7 days, with follow-up information within 8 additional days.
- All other SUSARs: must be reported within 15 days.
Reporting obligations apply under both the EU Clinical Trials Directive and the EU Clinical Trials Regulation (CTR 536/2014).
In the EU, SUSARs are submitted through EudraVigilance.
Who Must Report a SUSAR?
The sponsor of the clinical trial holds primary responsibility. This includes:
- Assessing causality
- Determining whether the event is expected or unexpected
- Reporting within regulatory timelines
- Notifying all investigators participating in the trial
Investigators must promptly document and communicate any serious adverse events (SAEs) to the sponsor.
How SUSARs Differ from SAEs and SARs
| Term | Causality | Expected? | Serious? | Expedited Reporting? |
|---|---|---|---|---|
| SAE | Not required | Not relevant | Yes | No (unless also SAR/SUSAR) |
| SAR | Yes | Usually expected | Yes | No |
| SUSAR | Yes | Unexpected | Yes | Yes |
Main takeaways
Summary
French hospital procurement union
The Union of Hospitals for Procurement (UNIHA) is a French central purchasing body grouping public healthcare institutions. It pools procurement of medicines, medical devices, and services to secure better economic conditions.
UNIHA supports cost rationalisation and optimized hospital supply chains.
Definition
UniHA (Union des Hôpitaux pour les Achats) is a national cooperative purchasing network created by French public hospitals to centralise and optimise procurement across a wide range of healthcare products and services.
Established in 2005, UniHA brings together hundreds of public hospitals and university hospital centres (CHUs) to negotiate contracts, secure competitive pricing, and ensure access to high-quality medical goods, pharmaceuticals, technologies, and operational services.
UniHA is one of the largest public hospital purchasing groups in Europe.
Purpose and Objectives
The primary mission of UniHA is to strengthen the efficiency and performance of public hospital procurement by:
- Pooling purchasing power to obtain better prices and service conditions.
- Improving quality and safety of medical supplies and hospital services.
- Reducing procurement complexity through coordinated tendering and framework agreements.
- Supporting innovation by facilitating access to new technologies and healthcare solutions.
- Optimising hospital budgets, particularly in high-spend therapeutic and technological areas.
UniHA operates within French public procurement regulations and ensures transparency and compliance in all tendering processes.
How UniHA Works
1. Centralised Procurement
Members participate voluntarily in joint purchasing programmes covering pharmaceuticals, medical devices, biomedical equipment, digital health, energy, logistics, and more.
2. Thematic Purchasing “Segments”
Procurement is organised into thematic segments led by expert hospital teams. Examples include:
- Pharmacy & medicines
- Medical devices
- IT and digital systems
- Energy and utilities
- Laboratory and diagnostics
- Hospital catering and services
Each segment designs tenders, evaluates suppliers, and manages framework contracts.
3. Competitive Tendering
UniHA conducts public tenders on behalf of its members, ensuring compliance with French and EU public procurement law.
4. Contract Access for Member Hospitals
Once a supplier is selected, hospitals can join the framework agreement and benefit from negotiated pricing and service conditions.
Who Can Join UniHA?
Membership is open to French public hospitals — including CHUs, regional hospitals, specialised centres, and territorial hospital groups (GHTs).
Participation in each purchasing programme is optional but offers significant financial and operational benefits.
Why UniHA Matters
UniHA plays a crucial role in the French hospital ecosystem by:
- Strengthening procurement efficiency
- Reducing costs through large-scale negotiation
- Supporting sustainable and innovative purchasing strategies
- Harmonising procurement practices across public hospitals
Its actions contribute to better allocation of health resources and improved access to high-quality medical products for hospitals and patients.
The URSSAF (Social Security and Family Allowances Collection Office) is a French organisation responsible for collecting social contributions. It funds social protection (healthcare, unemployment, pensions, family benefits).
URSSAF also monitors social declarations and supports companies in meeting their regulatory obligations.
