Glossary

1. Early Access Programs

Also known as: early access programmes, pre-approval access, pre-authorisation access, unlicensed access, managed access, compassionate use, expanded access, named patient supply

Definition: Programs that allow patients to access medicines before full commercial availability or before local marketing authorization, usually where there is high unmet medical need.

Operational context: The term is used broadly, but the legal pathway varies by country. In practice, access depends on product status, local regulation, documentation, and the route by which supply is authorized.

Related terms: Compassionate Use, Expanded Access, Named Patient Programmes, Named Patient Programs, Specials, Individual Import, Unlicensed Supply

Related services: Managed Access Program / Market Access Solutions

2. Compassionate Use

Also known as: compassionate use programme, compassionate use program, early access, pre-approval access, managed access

Definition: A pathway that allows patients with serious or life-threatening conditions to receive a medicine before it is fully authorized, usually when no satisfactory alternatives are available.

Operational context: The meaning and formal pathway differ between jurisdictions. In some markets, compassionate use applies to groups of patients. In others, similar access may be handled under a different framework.

Related terms: Early Access Programmes, Early Access Programs, Expanded Access, Named Patient Programmes, Named Patient Programs

Related services: Managed Access Program / Treatment Access Solutions

3. Expanded Access

Also known as: expanded access program, expanded access programme, compassionate use, early access, pre-approval access

Definition: A term most commonly used in the United States for access to investigational medicines outside a clinical trial.

Operational context: Expanded Access is a defined regulatory concept in the US. Although often used interchangeably with compassionate use, the operational and regulatory requirements are market-specific.

Related terms: Compassionate Use, Early Access Programmes, Early Access Programs, Individual Import

Related services: Managed Access Program / Treatment Access Solutions /

4. Named Patient Programs

Also known as: named patient programmes, named patient supply, named patient access, patient-specific supply

Definition: A pathway under which a medicine is supplied for an individual patient on the request of an authorized treating physician, typically where the product is not locally available.

Operational context: This is one of the most important access models in international supply. Requirements vary by market, but execution is always patient-specific and documentation-driven.

Related terms: Early Access Programmes, Early Access Programs, Specials, Individual Import, Unlicensed Supply, Named Patient Supply

Related services: Managed Access Program / Treatment Access Solutions

5. Named Patient Supply

Also known as: named patient import, patient-specific supply, named patient access

Definition: A supply model in which an unlicensed or otherwise unavailable medicine is provided for an individual patient on the request of the treating physician.

Operational context: The term is often used interchangeably with Named Patient Programs, but in practice it usually refers more directly to the supply pathway than to the broader access framework.

Related terms: Named Patient Programmes, Named Patient Programs, Individual Import, Unlicensed Supply, Specials

Related services: Individual Import / Treatment Access Solutions

6. Specials

Also known as: unlicensed specials, special-order medicines, UK specials, specials manufacture

Definition: A term used primarily in the UK for unlicensed medicines supplied to meet the special clinical needs of an individual patient.

Operational context: In UK practice, Specials have a distinct legal and operational meaning. The term is not universal, so international users often search for it alongside named patient or unlicensed supply terminology.

Related terms: Named Patient Programmes, Named Patient Programs, Named Patient Supply, Unlicensed Supply, Individual Import

Related services: Individual Import / Treatment Access Solutions

7. Managed Access Programs

Also known as: managed access programmes, MAPs, controlled access programmes, controlled access programs

Definition: Structured programs designed to provide access to medicines outside routine commercial supply, often under defined medical, regulatory, or reimbursement conditions.

Operational context: The term can cover a range of models, from pre-approval access to post-approval controlled distribution. The key distinction is that supply is organized through a defined framework rather than standard commercial channels.

Related terms: Early Access Programmes, Early Access Programs, Compassionate Use, Patient Access, Market Access

Related services: Managed Access program / Market Access Solutions

8. Unlicensed Supply

Also known as: supply of unlicensed medicines, unlicensed medicines supply, non-commercial supply, pre-authorisation supply

Definition: The compliant supply of medicines that do not hold a local marketing authorization in the country where they are being used.

Operational context: Unlicensed supply is not a single pathway. It can sit behind named patient, compassionate use, specials, or individual import models depending on the market and product.

Related terms: Named Patient Programmes, Named Patient Programs, Named Patient Supply, Specials, Individual Import, Early Access Programs

Related services: Treatment Access / Individual Import

9. Unlicensed Medicines

Also known as: unregistered medicines, unauthorised medicines, non-authorised medicines

Definition: Medicines that do not hold a local marketing authorization in the country where they are supplied or used.

Operational context: The term describes product status rather than a specific access pathway. In practice, supply depends on the legal route available in the relevant market.

Related terms: Unlicensed Supply, Early Access Programs, Early Access Programmes, Named Patient Programs, Named Patient Programmes, Specials

Related services: Treatment Access Solutions

10. Individual Import

Also known as: patient-specific import, import for individual patient use, named patient import

Definition: The regulated import of a medicine for use by a specific patient, typically where no suitable locally available alternative exists.

Operational context: Individual import is usually highly documentation-dependent and linked to physician request, patient need, and local authority requirements. It is often one operational route within broader access models.

Related terms: Named Patient Programmes, Named Patient Programs, Named Patient Supply, Unlicensed Supply, Compassionate Use

Related services: Individual Import / treatment access

11. Patient Access

Also known as: access to treatment, access pathway, therapy access

Definition: The process by which eligible patients are able to receive a medicine or therapy within a given healthcare system.

Operational context: Patient access is broader than unlicensed supply. It may depend on regulatory status, reimbursement, supply routes, local infrastructure, funding mechanisms, and operational readiness.

Related terms: Market Access, Early Access Programmes, Early Access Programs, Managed Access Programmes, Managed Access Programs, Named Patient Programmes, Named Patient Programs, Individual Import

Related services: Patient Support Program / Market Access Solutions

1. Investigational Medicinal Product (IMP)

Also known as: investigational medicine, investigational drug, study drug, investigational product, trial drug

Definition: A medicinal product being tested or used as a reference in a clinical trial. This includes newly developed products as well as approved medicines used in a different indication, formulation, packaging, or trial setting.

Operational context: IMP supply depends on protocol alignment, regulatory compliance, packaging and labeling requirements, and reliable delivery to sites across multiple jurisdictions.

Related terms: Comparator, Placebo, Clinical Supply, QP Release, Investigational Product

Related services: Clinical Trial Solutions / CT Consulting

2. Investigational Product

Also known as: IP, investigational medicinal product, study drug, trial product

Definition: A broader term used in clinical research for a product being investigated in a trial. Depending on the study, this may refer to a medicinal product, device, biologic, or combination product.

Operational context: The term is widely used in international clinical operations, but the regulatory meaning may differ depending on whether the study involves medicines, devices, or both.

Related terms: Investigational Medicinal Product (IMP), Diagnostic Medical Devices, Therapeutic Medical Devices

Related services: Clinical Trial Solutions / CT Consulting

3. Comparator

Also known as: comparator drug, reference product, active comparator, reference medicine, comparator product

Definition: A medicinal product used as a reference in a clinical trial to compare safety, efficacy, or other study endpoints against an investigational product.

Operational context: Comparator supply is often shaped by availability, documentation, batch consistency, and sourcing restrictions across different markets.

Related terms: IMP, Placebo, Comparator Sourcing, Clinical Supply

Related services: Comparator Sourcing / Clinical Trial Solutions

4. Comparator Sourcing

Also known as: reference product sourcing, comparator procurement, comparator supply

Definition: The process of identifying, procuring, and documenting comparator products for use in clinical trials.

Operational context: Successful sourcing depends on traceability, market availability, regulatory acceptability, and consistent product selection across countries and study sites.

Related terms: Comparator, Clinical Supply, Packaging and Labeling, Procurement

Related services: Comparator Sourcing

5. Placebo

Also known as: inactive control, placebo control, matched placebo

Definition: A substance or treatment designed to match an active investigational or comparator product in appearance and administration, but without therapeutic effect.

Operational context: Blinding integrity depends on exact matching in presentation, handling, and documentation. Even small inconsistencies can compromise trial validity.

Related terms: IMP, Comparator, Blinding, Packaging and Labeling

Related services: Packaging and Labeling / Clinical Trial Solutions

6. Ancillary Medication

Also known as: ancillary supplies, supportive medication, co-medication, concomitant support medication

Definition: Medicines or related products used alongside investigational treatment to support patient safety, protocol requirements, or study execution.

Operational context: Ancillary medication must align with protocol design, site-level clinical practice, and local regulatory expectations across participating countries.

Related terms: IMP, Clinical Supply, Diagnostic Medical Devices

Related services: Storage and Distribution / Clinical Trial Solutions

7. Clinical Supply

Also known as: clinical trial supply, study supply, trial materials supply, clinical materials supply

Definition: The planning, sourcing, packaging, labeling, storage, distribution, and return management of medicinal products and related materials used in a clinical trial.

Operational context: Clinical supply is shaped by protocol design, country-specific regulation, site requirements, and the need to maintain continuity throughout the study lifecycle.

Related terms: IMP, Comparator, Placebo, QP Release, Clinical Supply Chain, Clinical Trial Materials

Related services: Storage and Distribution / Clinical Trial Solutions

8. Clinical Trial Materials

Also known as: trial materials, study materials, clinical materials

Definition: The products and supporting materials required for the conduct of a clinical trial, including investigational products, comparators, placebos, ancillary items, and related packaging or labeling components.

Operational context: The term is often used broadly in trial operations and may extend beyond medicinal products alone, depending on the design and requirements of the study.

Related terms: Clinical Supply, IMP, Ancillary Medication, Packaging and Labeling

Related services: Storage and Distribution / Packaging and Labeling

9. Clinical Supply Chain

Also known as: trial supply chain, study supply chain, clinical trial logistics

Definition: The network of activities and stakeholders involved in moving clinical trial materials from manufacture or sourcing through to study sites and, where relevant, return or destruction.

Operational context: The clinical supply chain must remain aligned with timelines, temperature requirements, documentation standards, and changing protocol needs across markets.

Related terms: Clinical Supply, Cold Chain, Packaging and Labeling, Returns and Destruction

Related services: Storage and Distribution / CT Consulting

10. Blinding

Also known as: masking, blinded trial design, blinded supply

Definition: A trial design measure used to prevent participants, investigators, or study teams from knowing which treatment has been assigned, in order to reduce bias.

Operational context: Blinding depends not only on study design, but also on exact alignment in packaging, labeling, and presentation across all trial materials.

Related terms: Placebo, Comparator, Packaging and Labeling

Related services: Packaging and Labeling / Clinical Trial Solutions

11. Packaging and Labeling

Also known as: clinical packaging, trial labeling, trial labelling, study labeling, study labelling, clinical trial packaging

Definition: The processes used to prepare medicinal products for compliant identification, handling, and use in clinical trials.

Operational context: Requirements vary by country and study design. Errors in packaging or labeling can affect compliance, site usability, and blinding integrity.

Related terms: IMP, Placebo, Blinding, QP Release

Related services: Packaging and Labeling

12. Qualified Person (QP) Release

Also known as: Qualified Person Release, batch certification, QP certification

Definition: The certification of a medicinal product batch by a Qualified Person to confirm that it has been manufactured and checked in accordance with applicable regulatory requirements.

Operational context: QP Release is a critical control point in clinical supply and may determine whether material can legally move into or within certain markets.

Related terms: IMP, Clinical Supply, GMP, Batch Certification

Related services: Clinical Trial Solutions

13. Batch Certification

Also known as: batch release certification, QP batch certification

Definition: The formal confirmation that a medicinal product batch meets the required standards and can be released for use or supply in accordance with applicable regulations.

Operational context: In some markets or contexts, batch certification is used more broadly than QP Release, but both sit at the point where quality and legal supply readiness intersect.

Related terms: QP Release, GMP, IMP

Related services: Clinical Trial Solutions

14. Batch Collection

Also known as: batch sourcing, batch procurement, collection of required batches

Definition: The sourcing and procurement of specified medicinal product batches to meet clinical trial requirements.

Operational context: A study may require a particular batch, a limited number of batches, or multiple batches with defined quantities, expiration dates, and documentation. Planning must align these requirements with market availability and the study supply schedule.

Related terms: Comparator Sourcing, Comparator, Clinical Supply, Documentation

Related services: Comparator Sourcing / Clinical Trial Solutions

15. Local Sourcing

Also known as: in-country sourcing, local procurement, domestic sourcing, buy local and deliver local

Definition: The procurement of clinical trial medicines or materials within the country where they will be used.

Operational context: Local sourcing can support country-specific product presentations, documentation requirements, and domestic delivery. Its suitability depends on protocol requirements, product availability, supplier qualification, and the agreed supply model.

Related terms: Comparator Sourcing, Procurement, Clinical Supply, Documentation

Related services: Comparator Sourcing / CT Consulting

16. Kit Identifier (Kit ID)

Also known as: kit ID, kit number, clinical trial kit identifier, study kit number

Definition: A unique identifier assigned to a clinical trial kit so that it can be tracked and handled according to study requirements.

Operational context: Distribution requests may specify individual kit IDs rather than product quantities alone. Accurate selection, dispatch, and reconciliation help maintain traceability and, where applicable, protect blinding.

Related terms: Clinical Trial Materials, Randomized Trial Supply, Blinding, Packaging and Labeling

Related services: Storage and Distribution / Packaging and Labeling

17. Randomized Trial Supply

Also known as: randomised trial supply, randomized clinical trial supply, randomised clinical trial supply

Definition: The management and distribution of clinical trial materials in accordance with a study’s randomization and supply instructions.

Operational context: Randomization assigns participants to treatment groups. Supply operations must follow the resulting study instructions, including kit selection, allocation controls, and blinding requirements where applicable. Kit-ID-level distribution supports this process without making treatment allocation decisions.

Related terms: Kit Identifier, Clinical Trial Materials, Blinding, Clinical Supply Chain

Related services: Storage and Distribution / Packaging and Labeling

1. Good Distribution Practice (GDP)

Also known as: GDP compliance, GDP guidelines, GDP standards, wholesale distribution standards

Definition: The quality standard governing the proper distribution of medicinal products for human use.

Operational context: GDP is designed to protect product quality and integrity throughout storage, transport, and supply. In practice, compliance depends on controlled processes, documentation, trained personnel, and effective oversight across the distribution chain.

Related terms: Responsible Person, Pharmaceutical Wholesaler, Cold Chain, Documentation, Wholesale Distribution Authorisation

Related services: Storage and Distribution / Commercial Solutions

2. Good Manufacturing Practice (GMP)

Also known as: GMP compliance, GMP standards, cGMP, current Good Manufacturing Practice

Definition: The quality standard governing the manufacture of medicinal products to ensure they are consistently produced and controlled to the required quality level.

Operational context: GMP sits upstream of distribution, but it remains closely linked to release, documentation, and product quality throughout the supply chain.

Related terms: QP Release, Batch Certification, Documentation, Recall, GDP

Related services: Clinical Trial Solutions

3. Responsible Person

Also known as: RP, GDP Responsible Person, Responsible Person for GDP

Definition: The person designated under GDP requirements to ensure that pharmaceutical wholesale distribution activities comply with applicable regulations and license conditions.

Operational context: The Responsible Person plays a central oversight role across quality systems, documentation, training, deviations, and recall readiness within regulated distribution operations.

Related terms: GDP, Pharmaceutical Wholesaler, Recall, Documentation, Wholesale Distribution Authorisation

Related services: Commercial Solutions

4. Pharmaceutical Wholesaler

Also known as: wholesale distributor, licensed wholesaler, pharmaceutical distributor, medicine wholesaler

Definition: An entity authorized to procure, hold, supply, import, export, or distribute medicinal products within a regulated supply chain.

Operational context: Wholesale distribution is not only a logistics function. It operates within a licensed framework that requires traceability, product control, and compliance with GDP and national authority requirements.

Related terms: GDP, Responsible Person, Pre-Wholesale, Controlled Substances, Wholesale Distribution Authorisation

Related services: Commercial Solutions / Treatment Access Solutions

5. Wholesale Distribution Authorisation (WDA)

Also known as: wholesale licence, wholesale dealer authorisation, WDA(H) where applicable

Definition: The regulatory authorization required in many markets for the wholesale distribution of medicinal products.

Operational context: A WDA confirms that a company is permitted to carry out defined wholesale activities under applicable law. The scope and terminology vary by country, but the concept is central to regulated pharmaceutical distribution.

Related terms: Pharmaceutical Wholesaler, GDP, Responsible Person, Regulatory Authorities

Related services: Commercial Solutions

6. Documentation

Also known as: controlled documentation, quality documentation, records, GMP documentation, GDP documentation

Definition: The system of records, procedures, instructions, and evidence used to support compliant pharmaceutical operations.

Operational context: Documentation underpins traceability, quality control, audit readiness, and regulatory compliance. In regulated supply, what is not documented cannot be reliably demonstrated.

Related terms: GDP, GMP, Recall, Returns and Destruction, QP Release, Quality Assurance

Related services: Clinical Trial Solutions / Commercial Solutions

7. Compliance

Also known as: regulatory compliance, quality compliance, pharmaceutical compliance

Definition: The state of operating in accordance with applicable laws, regulations, licenses, standards, and internal procedures.

Operational context: In pharmaceutical operations, compliance is not a single activity. It is the ongoing alignment of quality systems, documentation, people, and processes with regulatory expectations.

Related terms: GDP, GMP, Regulatory Authorities, Documentation, Quality Assurance

Related services: Commercial Solutions

8. Regulatory Authorities

Also known as: competent authorities, health authorities, regulators, medicines authorities

Definition: National or regional bodies responsible for authorizing, supervising, and enforcing the legal framework for medicinal products and related operations.

Operational context: Authority expectations vary by market. Effective execution requires understanding not only the regulation itself, but how specific authorities apply and interpret it in practice.

Related terms: Compliance, GDP, GMP, Recall, Controlled Substances, Wholesale Distribution Authorisation

Related services: Regulatory Landscape Guidance / Market Access Consulting

9. Recall

Also known as: product recall, market recall, drug recall, medicine recall

Definition: The removal of a medicinal product from the supply chain or market due to a quality defect, safety concern, or regulatory issue.

Operational context: A recall depends on speed, traceability, clear responsibilities, and effective communication across the supply chain. Readiness is as important as response.

Related terms: Documentation, Responsible Person, Returns and Destruction, Compliance, Quality Assurance

Related services: Commercial Solutions

10. Returns and Destruction

Also known as: compliant returns, product destruction, destruction of medicinal products, pharmaceutical returns

Definition: The controlled return and, where necessary, compliant destruction of medicinal products that are expired, damaged, recalled, or otherwise unsuitable for use.

Operational context: These processes require traceability, documentation, and alignment with both company procedures and local regulatory requirements.

Related terms: Recall, Documentation, GDP, Quality Assurance

Related services: Storage and Distribution / Commercial Solutions

11. Cold Chain

Also known as: temperature-controlled distribution, cold chain logistics, temperature-controlled supply, temperature-controlled logistics

Definition: A controlled supply chain in which products are stored and transported within a defined temperature range to maintain quality and usability.

Operational context: Cold chain control depends on validated packaging, monitoring, handling discipline, and escalation processes for temperature excursions or delays, with contingency planning across the full movement path.

Related terms: GDP, Pre-Wholesale, Documentation, Controlled Substances, Temperature Excursion

Related services: Storage and Distribution

12. Temperature Excursion

Also known as: temperature deviation, cold chain excursion, storage excursion

Definition: A deviation in which a product is exposed to temperatures outside its approved or validated storage or transport range.

Operational context: Temperature excursions require assessment, documentation, and decision-making based on product stability data, quality procedures, and the conditions of the deviation.

Related terms: Cold Chain, Documentation, Quality Assurance, GDP

Related services: Storage and Distribution

13. Quality Assurance (QA)

Also known as: QA, quality system oversight, pharmaceutical QA

Definition: The system of activities used to ensure that pharmaceutical products and operations meet defined quality requirements.

Operational context: Quality assurance links procedures, controls, documentation, training, and deviation handling into a structured system that supports consistent and compliant operations.

Related terms: GMP, GDP, Documentation, Recall, Compliance, Deviation

Related services: Clinical Trial Solutions / Commercial Solutions

14. Deviation

Also known as: quality deviation, process deviation, deviation management

Definition: A departure from an approved procedure, specification, process, or expected condition within a regulated pharmaceutical operation.

Operational context: Deviation handling is a core quality activity. It requires documentation, assessment of impact, root cause investigation where necessary, and defined corrective or preventive action.

Related terms: Quality Assurance, Documentation, Recall, Compliance

Related services: Commercial Solutions

15. Pharmacovigilance (PV)

Also known as: PV, drug safety, safety surveillance

Definition: The science and activities relating to the detection, assessment, understanding, and prevention of adverse effects or other medicine-related problems.

Operational context: Pharmacovigilance sits alongside quality and regulatory systems as a core part of compliant product oversight. In practice, it depends on timely reporting, clear responsibilities, documented processes, and alignment with local authority requirements.

Related terms: Adverse Event, Serious Adverse Event, Adverse Drug Reaction, Regulatory Authorities, Compliance

Related services: Patient Support Program

16. Adverse Event (AE)

Also known as: AE, adverse experience

Definition: Any untoward medical occurrence in a patient or clinical trial subject who has been administered a medicinal product, whether or not it is considered related to that product.

Operational context: Adverse event reporting requires clear capture, escalation, and documentation pathways. In regulated environments, speed and accuracy are critical.

Related terms: Pharmacovigilance, Serious Adverse Event, Adverse Drug Reaction

Related services: Patient Support Program

17. Serious Adverse Event (SAE)

Also known as: SAE, serious event

Definition: An adverse event that results in death, is life-threatening, requires hospitalisation or prolongation of hospitalisation, results in significant disability, or is otherwise medically important.

Operational context: SAEs require urgent assessment and reporting within defined timelines. The exact reporting pathway depends on study type, product status, and local regulatory requirements.

Related terms: Adverse Event, Pharmacovigilance, Adverse Drug Reaction

Related services: Patient Support Program

18. Adverse Drug Reaction (ADR)

Also known as: ADR, adverse reaction

Definition: A harmful and unintended response to a medicinal product where a causal relationship between the product and the event is at least a reasonable possibility.

Operational context: ADR assessment sits at the intersection of medical review, product knowledge, and reporting obligations. Not every adverse event is an adverse drug reaction, but the distinction is operationally important.

Related terms: Adverse Event, Serious Adverse Event, Pharmacovigilance

Related services: Patient Support Program

1. Advanced Therapy Medicinal Products (ATMPs)

Also known as: advanced therapies, advanced therapy medicines, cell and gene therapy, CGT where commonly used

Definition: Medicinal products based on genes, cells, or engineered tissues, including gene therapies, somatic cell therapies, and tissue-engineered products.

Operational context: ATMPs operate under specialized regulatory frameworks and often require strict control over identity, timing, storage conditions, and site readiness.

Related terms: Cell Therapy, Gene Therapy, Cold Chain, Chain of Identity

Related services: Clinical Trial Solutions / Market Access Solutions

2. Cell Therapy

Also known as: cell-based therapy, somatic cell therapy, cell and gene therapy where used collectively

Definition: A therapeutic approach based on the administration of living cells to repair, replace, or modify biological function.

Operational context: Cell therapies often require tightly controlled handling, rapid execution, and precise coordination between manufacturing, logistics, and clinical administration.

Related terms: ATMPs, Gene Therapy, Cold Chain

Related services: Clinical Trial Solutions

3. Gene Therapy

Also known as: gene-based therapy, genetic therapy, cell and gene therapy where used collectively

Definition: A therapeutic approach that modifies, replaces, or introduces genetic material to treat or prevent disease.

Operational context: Gene therapies often involve complex manufacturing, strict handling requirements, and highly controlled supply pathways linked to specific treatment centers.

Related terms: ATMPs, Cell Therapy, Cold Chain

Related services: Clinical Trial Solutions

4. Biologics

Also known as: biological medicines, biologic therapies, biologic drugs, biopharmaceuticals

Definition: Medicinal products derived from living organisms or biological processes, including monoclonal antibodies, recombinant proteins, and other complex therapies.

Operational context: Biologics often require temperature-controlled handling, careful supply planning, and alignment with specialized administration settings.

Related terms: Biosimilars, Cold Chain, Orphan Drugs

Related services: Clinical Trial Solutions / Commercial Solutions

5. Biosimilars

Also known as: biosimilar medicines, biosimilar biologics, biosimilar products

Definition: Biological medicines shown to be highly similar to an already approved reference biologic, with no clinically meaningful differences in safety, purity, or potency.

Operational context: Biosimilar uptake depends not only on approval, but also on stakeholder confidence, reimbursement conditions, switching practices, and structured integration into clinical practice.

Related terms: Biologics, Market Access, Branded Drugs

Related services: Market Access Consulting / Market Access Solutions

6. Orphan Drugs

Also known as: orphan medicines, orphan medicinal products, rare disease drugs, ultra-orphan drugs

Definition: Medicinal products developed for the diagnosis, prevention, or treatment of rare diseases affecting small patient populations.

Operational context: Orphan drugs often involve fragmented access pathways, limited supply, and highly individualised patient journeys that require close coordination across stakeholders.

Related terms: Market Access, Patient Access, ATMPs

Related services: Managed Access Program / Market Access Solutions

7. Controlled Substances

Also known as: controlled drugs, controlled medicines, narcotics, psychotropics

Definition: Medicinal products subject to additional legal controls because of their risk of misuse, dependence, or diversion.

Operational context: These products require enhanced documentation, secure storage, restricted access, and close alignment with national authority requirements.

Related terms: Regulatory Authorities, GDP, Pharmaceutical Wholesaler, Compliance, Documentation

Related services: Storage and Distribution / Treatment Access Solutions

8. Diagnostic Medical Devices

Also known as: diagnostic devices, companion diagnostics, in vitro diagnostics where relevant, IVDs where applicable

Definition: Devices or tests used to identify, stratify, or monitor patients before or during treatment.

Operational context: Their role often extends beyond supply. Successful use depends on integration into clinical workflows, data processes, and treatment decision pathways.

Related terms: Therapeutic Medical Devices, Clinical Supply, Packaging and Labeling

Related services: Clinical Trial Solutions

9. Therapeutic Medical Devices

Also known as: treatment devices, therapeutic devices, treatment-enabling devices

Definition: Medical devices used to deliver treatment through mechanical, physical, or digital means.

Operational context: Implementation often requires coordination across logistics, training, site readiness, and patient-facing care pathways.

Related terms: Diagnostic Medical Devices, Clinical Supply, Market Access

Related services: Market Access Solutions / Clinical Trial Solutions

10. Nutraceuticals

Also known as: nutritional products, functional nutrition products, supplements where locally applicable, nutraceutical products

Definition: Products derived from food sources that provide nutritional or wellness-related value beyond basic dietary intake.

Operational context: Their classification and permitted use vary by market. In regulated settings, their role depends on local law, intended application, and how they fit within broader care or support pathways.

Related terms: Patient Access, Market Access, Commercial Distribution

Related services: Commercial Solutions

11. Veterinary Drugs

Also known as: veterinary medicines, animal health products, veterinary medicinal products

Definition: Medicinal products used for the prevention, diagnosis, or treatment of diseases in animals.

Operational context: Veterinary products are governed by separate regulatory frameworks and distribution channels from human medicines, which can significantly affect sourcing and supply models.

Related terms: Regulatory Authorities, Pharmaceutical Wholesaler

Related services: Commercial Solutions

1. Marketing Authorisation Holder (MAH)

Also known as: MAH, licence holder, authorization holder, marketing authorization holder, marketing authorisation holder

Definition: The legal entity that holds the marketing authorization for a medicinal product in a given market.

Operational context: The MAH carries responsibility for the product’s legal status, compliance obligations, and lifecycle management in the relevant jurisdiction.

Related terms: Market Launch, Market Access, Pharmaceutical Wholesaler, Regulatory Authorities

Related services: Partnering / Commercial Solutions

2. Market Access

Also known as: reimbursement access, payer access, access strategy, pricing and reimbursement

Definition: The set of processes that determine how a medicine becomes available and funded within a healthcare system after regulatory approval or as part of a structured access pathway.

Operational context: Market access sits at the intersection of regulation, pricing, reimbursement, and implementation. Approval alone does not guarantee that a therapy will reach patients in practice.

Related terms: Patient Access, Managed Access Programs, Early Access Programs, Market Launch, MAH, Reimbursement, Pricing and Reimbursement

Related services: Market Access Consulting / Market Access Solutions

3. Pricing and Reimbursement

Also known as: P&R, reimbursement strategy, pricing strategy, payer reimbursement

Definition: The framework through which a medicine is priced and assessed for funding within a healthcare system.

Operational context: Pricing and reimbursement decisions often determine whether approved products can achieve practical uptake. They are shaped by national policy, payer expectations, clinical value, and market dynamics.

Related terms: Market Access, Reimbursement, Patient Access, Market Launch

Related services: Market Access Consulting

4. Market Launch

Also known as: product launch, commercial launch, market introduction, launch readiness

Definition: The first commercial introduction of a medicinal product into a market following approval or completion of required access preparations.

Operational context: Launch readiness depends on more than approval. Distribution setup, documentation, local regulatory requirements, and supply continuity all affect whether a product can enter the market effectively.

Related terms: MAH, Market Access, Commercial Distribution, Pre-Wholesale, Reimbursement

Related services: Fostering / Commercial Solutions

5. Commercial Distribution

Also known as: commercial supply, post-approval distribution, post-authorisation supply

Definition: The regulated supply of approved medicinal products through standard commercial channels after authorization.

Operational context: Commercial distribution must align with product status, market structure, customer channels, and local wholesale requirements to ensure continuity and compliance.

Related terms: Pharmaceutical Wholesaler, Pre-Wholesale, Market Launch, GDP

Related services: Commercial Solutions / Partnering

6. Pre-Wholesale

Also known as: pre-wholesaling, pre-wholesale services, pre-wholesale distribution

Definition: The storage and handling of medicinal products between manufacture and onward distribution into wholesale or commercial channels.

Operational context: Pre-wholesale is a controlled stage in the supply chain that supports product readiness, traceability, and compliant onward distribution.

Related terms: Commercial Distribution, GDP, Pharmaceutical Wholesaler, Cold Chain

Related services: Commercial Solutions

7. Branded Drugs

Also known as: branded medicines, originator medicines, innovator products, originator drugs

Definition: Medicinal products approved for clinical use and marketed under a proprietary brand, usually while protected by intellectual property rights.

Operational context: Commercial strategy for branded products often depends on market timing, reimbursement conditions, competition, lifecycle stage, and regional payer dynamics.

Related terms: Generics, Biosimilars, Market Launch, Market Access, MAH

Related services: Fostering / Commercial Solutions

8. Generics

Also known as: generic medicines, generic drugs, multisource products

Definition: Medicinal products containing the same active substance as a reference product and demonstrating bioequivalence after patent expiry.

Operational context: Generic market access is influenced by substitution rules, procurement models, pricing pressure, reimbursement logic, continuity requirements, and local prescribing dynamics.

Related terms: Branded Drugs, Market Access, Pharmaceutical Wholesaler, Procurement

Related services: Commercial Solutions

9. Reimbursement

Also known as: payer funding, reimbursement approval, coverage, formulary access where relevant

Definition: The mechanism by which a healthcare system, insurer, or payer agrees to fund the cost of a medicine or therapy.

Operational context: Reimbursement is often the key gate between approval and practical access. Delays or restrictions at this stage can limit uptake even after regulatory authorization.

Related terms: Market Access, Pricing and Reimbursement, Patient Access, Market Launch

Related services: Market Access Consulting

10. Consignment Stock

Also known as: consignment inventory, vendor-owned stock, consignment supply

Definition: A supply model in which products are stored at or near the point of use, while ownership remains with the supplier until the product is used or sold.

Operational context: In pharmaceutical settings, consignment stock can support responsiveness and continuity, but it must operate within clear quality, traceability, and contractual controls.

Related terms: Commercial Distribution, Pre-Wholesale, Pharmaceutical Wholesaler

Related services: Commercial Solutions

11. Procurement

Also known as: sourcing, purchasing, supply procurement, pharmaceutical procurement

Definition: The process of acquiring products or services in line with operational, quality, regulatory, and commercial requirements.

Operational context: In regulated pharmaceutical supply, procurement is shaped not only by price and availability, but also by product status, documentation, and market-specific restrictions.

Related terms: Comparator Sourcing, Pharmaceutical Wholesaler, Market Access, Generics

Related services: Comparator Sourcing / Commercial Solutions

1. Country-Specific Access Pathways

Also known as: local access pathways, national access mechanisms, country-specific medicine access

Definition: Legal and operational mechanisms that govern access to medicines outside routine local commercial availability in a particular jurisdiction.

Operational context: The appropriate route depends on the destination country, product status, clinical need, and whether access is patient-specific or organized through a broader program. Approval or lawful supply in one country does not automatically permit import or use in another.

Related terms: Individual Import, Named Patient Supply, Compassionate Use, Expanded Access, Specials, Unlicensed Supply

Related services: Treatment Access Solutions / Managed Access Program

2. Germany: Import under §73(3) AMG

Also known as: §73 Absatz 3 AMG, §73 Abs. 3 AMG, Section 73(3) AMG, Einzelimport

Definition: A German statutory provision permitting certain finished medicinal products without German authorization or registration to be brought into Germany through pharmacies under specified conditions.

Operational context: The provision includes requirements concerning pharmacy supply, quantities, lawful marketing in the source country, and the availability of comparable medicines in Germany. It also contains specific provisions for hospital supply. The applicable conditions must be checked for the particular request.

Related terms: Individual Import, Named Patient Supply, Unlicensed Medicines, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

3. United States: Expanded Access

Also known as: US expanded access, FDA expanded access, individual patient expanded access

Definition: A US regulatory pathway for treatment use of an investigational medical product outside a clinical trial when the applicable eligibility and regulatory requirements are met.

Operational context: Expanded Access can apply to individual patients or larger patient populations. It involves defined responsibilities for the treating physician, product provider, FDA, and institutional review board as applicable. It is distinct from importing a medicine approved in another country.

Related terms: Expanded Access, Compassionate Use, Early Access Programs, Country-Specific Access Pathways

Related services: Managed Access Program / Market Access Solutions

4. United Kingdom: Specials

Also known as: UK specials, unlicensed specials, special-order medicines

Definition: Unlicensed medicines supplied under the UK framework to meet an individual patient’s special clinical needs when available licensed medicines cannot meet those needs.

Operational context: Specials may be specially manufactured or imported. Supply depends on the prescriber’s assessment of clinical need and the applicable licensing, notification, and distribution requirements.

Related terms: Specials, Unlicensed Medicines, Named Patient Supply, Individual Import, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

5. Austria: Import under the AWEG

Also known as: Arzneiwareneinfuhrgesetz, AWEG 2010, Arzneiwareneinfuhr, Einfuhr und Verbringung von Arzneiwaren

Definition: The Austrian legal framework governing the import and movement of medicinal products into Austria, including requirements relevant to medicines without local authorization.

Operational context: Requirements depend on product status, source country, recipient, and the applicable authorization, notification, or exemption. Kleinimport is a specific provision for limited patient-specific supply through public pharmacies from the EEA. It should not be treated as the general route for all medicine imports.

Related terms: Individual Import, Named Patient Supply, Unlicensed Medicines, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

6. Switzerland: Professional Import under Article 49 MPLO

Also known as: Article 49 MPLO, Art. 49 AMBV, Einfuhr nicht zugelassener Arzneimittel

Definition: A Swiss provision allowing specified healthcare professionals to import small quantities of ready-to-use human medicines not authorized in Switzerland when the applicable conditions are met.

Operational context: Requirements depend on the professional’s authority, intended use, product status, and available treatment alternatives. Importers must verify and document eligibility and ensure GDP-compliant transport. This route is distinct from personal import and from sponsor-led temporary authorization for compassionate use.

Related terms: Individual Import, Unlicensed Medicines, Compassionate Use, Good Distribution Practice, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

7. Poland: Import Docelowy

Also known as: import docelowy, targeted import, Polish patient-specific import

Definition: A Polish mechanism for importing medicines without Polish marketing authorization that are necessary to protect a patient’s life or health, subject to statutory conditions.

Operational context: The process involves a medical request, specialist confirmation, and the applicable Ministry of Health procedure. Product authorization in the source country and the availability of equivalent medicines in Poland are relevant considerations. Permission to import and a decision on reimbursement are separate matters.

Related terms: Individual Import, Named Patient Supply, Unlicensed Medicines, Reimbursement, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

8. Czech Republic: Specific Therapeutic Programs

Also known as: specific therapeutic programmes, STP, specifický léčebný program, SpLP

Definition: Programs under Czech law allowing the use, distribution, and supply of medicines without Czech marketing authorization for defined therapeutic needs.

Operational context: These programs require written Ministry of Health approval, taking account of an opinion from SÚKL. Supply and use must follow the approved program. This is a structured national mechanism and should not be treated as interchangeable with individual patient supply.

Related terms: Managed Access Programs, Unlicensed Supply, Patient Access, Country-Specific Access Pathways

Related services: Managed Access Program / Market Access Consulting

9. Denmark: Compassionate Use Permits

Also known as: udleveringstilladelse, Danish compassionate use permit, dispensing permit for non-marketed medicines

Definition: Permission from the Danish Medicines Agency for the sale or dispensing of a medicine not marketed in Denmark in specified circumstances.

Operational context: The treating prescriber applies for the permit, which supports dispensing through a pharmacy. Denmark uses compassionate use terminology for this national dispensing mechanism. The term should not automatically be interpreted as a sponsor-led pre-approval program.

Related terms: Compassionate Use, Named Patient Supply, Unlicensed Supply, Country-Specific Access Pathways

Related services: Treatment Access Solutions / Individual Import

10. Sweden: License Medicines

Also known as: licensläkemedel, läkemedel på licens, licens, Swedish license medicines

Definition: Medicines not approved in Sweden that a pharmacy may sell under a license granted by the Swedish Medical Products Agency to meet a medical need that approved medicines cannot satisfy.

Operational context: The prescriber provides a medical justification and the pharmacy submits the license application. The license concerns a specific product. Regulatory permission and practical product availability are separate considerations.

Related terms: Unlicensed Medicines, Named Patient Supply, Individual Import, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

11. Singapore: Special Access Routes

Also known as: SAR, HSA Special Access Routes, named-patient special access

Definition: Singapore mechanisms permitting the import and supply of unregistered therapeutic products in specified circumstances under Health Sciences Authority requirements.

Operational context: The patient-use route includes named-patient and defined buffer-stock applications. It requires clinical justification, an eligible applicant, and approval for each consignment before import. Requirements depend on the product and application type; separate frameworks apply to other regulated product categories.

Related terms: Named Patient Supply, Unlicensed Medicines, Individual Import, Country-Specific Access Pathways

Related services: Treatment Access Solutions / Individual Import

12. Australia: Special Access Scheme

Also known as: SAS, TGA Special Access Scheme, SAS Category A, SAS Category B, SAS Category C

Definition: An Australian mechanism allowing registered health practitioners to access unapproved therapeutic goods for individual patients under specified conditions.

Operational context: The applicable SAS category determines whether prior approval or notification is required. Practitioners must consider appropriate approved treatment options and meet the relevant clinical and reporting obligations. The Authorised Prescriber Scheme is a separate pathway for treating a class of patients.

Related terms: Named Patient Supply, Unlicensed Medicines, Patient Access, Country-Specific Access Pathways

Related services: Treatment Access Solutions / Market Access Consulting

13. Brazil: Expanded Access and Compassionate Use Programs

Also known as: programas assistenciais, programa de acesso expandido, programa de uso compassivo

Definition: Anvisa-regulated programs enabling access to investigational medicines outside clinical trials under defined conditions.

Operational context: Expanded access, compassionate use, and post-study supply are distinct program models with different requirements. Execution involves program authorization, defined sponsor and physician responsibilities, and applicable import and reporting procedures. These programs should not be treated as interchangeable with routine patient-specific medicine imports.

Related terms: Expanded Access, Compassionate Use, Early Access Programs, Managed Access Programs, Country-Specific Access Pathways

Related services: Managed Access Program / Market Access Solutions

14. Spain: Foreign Medicines

Also known as: medicamentos extranjeros, medicación extranjera, foreign medicines access

Definition: A Spanish access mechanism for medicines authorized in other countries but not authorized in Spain, subject to AEMPS requirements.

Operational context: Requests are managed through the Medicines in Special Situations system, known as MSE. The process differs between hospital and outpatient supply and may involve an individual medical justification or a stock request linked to a supply shortage. Foreign medicine access is distinct from access to investigational medicines and off-label use, although all sit within Spain’s broader special-situations framework.

Related terms: Individual Import, Named Patient Supply, Unlicensed Medicines, Shortage Resolution, Country-Specific Access Pathways

Related services: Individual Import / Shortage Resolution

15. Portugal: Exceptional Use Authorization

Also known as: Autorização de Utilização Excecional, AUE, exceptional use authorisation

Definition: An INFARMED authorization permitting exceptional use of a medicine under specified Portuguese legal and regulatory conditions.

Operational context: The applicable process depends on the product and purpose of the request. Patient-specific exceptional use and authorizations for medicine batches during supply shortages are distinct mechanisms. The relevant application, clinical justification, and supply requirements must be established before execution.

Related terms: Named Patient Supply, Unlicensed Medicines, Individual Import, Shortage Resolution, Country-Specific Access Pathways

Related services: Treatment Access Solutions / Managed Access Program

16. Italy: Early Access Pathways

Also known as: accesso precoce, Italian early access, early access to medicines in Italy

Definition: Italian mechanisms allowing access to medicines before routine availability or for uses outside their authorized indications, subject to the requirements of the relevant pathway.

Operational context: Italy has several distinct mechanisms, including compassionate use, known as uso compassionevole, and access under Law 648/1996. They differ in eligibility, authorization, funding, and supply responsibilities. Compassionate use involves free supply by the manufacturer, while Law 648/1996 provides an AIFA listing mechanism for supply funded by the national health service.

Related terms: Early Access Programs, Compassionate Use, Uso Compassionevole, Legge 648/1996, Reimbursement, Country-Specific Access Pathways

Related services: Managed Access Program / Market Access Consulting

17. Ireland: Exempt Medicinal Products

Also known as: EMP, exempt medicines, Irish exempt medicinal products

Definition: Medicines without Irish authorization that may be supplied under a statutory exemption to meet the needs of patients under the direct responsibility of an authorized prescriber.

Operational context: The exemption applies where an authorized medicine is not available to meet the patient’s needs. Appropriately authorized manufacturers and wholesalers must meet HPRA notification requirements for the exempt products they receive. The exemption does not constitute a marketing authorization or establish product availability.

Related terms: Unlicensed Medicines, Named Patient Supply, Individual Import, Country-Specific Access Pathways

Related services: Individual Import / Treatment Access Solutions

18. France: Early and Compassionate Access

Also known as: accès dérogatoires, French early access, French compassionate access

Definition: French frameworks permitting access to medicines under defined conditions outside routine availability or authorized use.

Operational context: The frameworks include autorisation d’accès précoce (AAP), autorisation d’accès compassionnel (AAC), and cadre de prescription compassionnelle (CPC). These are distinct mechanisms with different eligibility, decision-making, and monitoring requirements. Legacy terminology such as ATU may appear in older materials and should be interpreted in its historical context.

Related terms: Early Access Programs, Compassionate Use, Managed Access Programs, AAP, AAC, CPC, ATU, Country-Specific Access Pathways

Related services: Managed Access Program / Market Access Consulting