Active pharmaceutical ingredient means any substance that is intended for incorporation into a finished drug product and is intended to furnish pharmacological activity in the treatment or prevention of a disease.
Agence Nationale de securite du medicament et des produits de sante (ANSM)
French National Agency for the Safety of Medicines and Health Products
Agenzia Italiana del Farmaco (AIFA)
Agenzia Italiana del Farmaco (AIFA) is the Italian Agency for Medicines
Arzneimittel-Härtefall-Verordnung-AMHV
Hardship Case program for groups of patients, according to German laws and regulations.
Autorisation d’Accès Compassionnel (AAC)
Autorisation d’accès Compassionnel (AAC) is an authorization granted by the French Medicines Agency (ANSM) to allow early access to a patient suffering from severe or life-threatening disease access to an unlicensed product.
Autorisation d’Accès Precoce (AAP)
Autorisation d’Accès Precoce (AAP) is an authorization granted by the French Medicines Agency (ANSM) to allow early access to a patient suffering from severe or life-threatening disease access to an unlicensed product.
Chief Pharmaceutical Officer (CPO)
In France, the Chief Pharmaceutical Officer, also known as “Pharmacien Responsible”, ensures the safety and quality of pharmaceutical products sold within the country and for products in compassionate use protocols.
Cohort Protocols
Expanded access / compassionate use program for groups of patients.
Committee for Medicinal Products for Human Use
The Committee for Medicinal Products for Human Use (CHMP) is the European Medicines Agency’s (EMA) committee responsible for human medicines.
Compassionate Use Program (CUP) or Compassionate Use Programmes
Compassionate use is making a medicinal product available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life threatening, and who cannot be treated satisfactorily by an authorised medicinal product.
Early Access Care (EAC)
Our company, which provides comprehensive solutions to help pharmaceutical companies develop and manage expanded access / compassionate use programs.
Early Access Programs (EAPs)
An overarching term chosen by some pharmaceutical companies to describe the terms by which unlicensed or off-label medicines will be made accessible in a lawful and compliant manner.
Early Access System™ (EAS™)
The proprietary web-based platform owned and managed by Early Access Care that allows pharmaceutical companies to streamline expanded access / compassionate use request management.
Early Access to Medicines Scheme (EAMS)
The early access to medicines scheme (EAMS) gives access to patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation, in the United Kingdom.
Ethics Committee (EC)
A committee reviewing the ethical aspects of a request for an unlicensed medicine. The committee may be within an institution or at a national level.
European Clinical Trials Database (EudraCT)
EudraCT is a database of all interventional clinical trials in the European Union
European Medicines Agency (EMA)
The European Medicines Agency (EMA) is a decentralized agency of the European Union (EU) responsible for the scientific evaluation, supervision and medicines in the EU.
Expanded Access (EA)
Expanded Access refers to the use of an investigational drug when the primary purpose is to treat, monitor or diagnose a patient’s disease or condition. Expanded Access program (EAP), alternatively called compassionate use program provide a pathway for patients to access investigational medicines.
Food & Drug Administration (FDA)
The federal agency that regulates all approved medical substances and devices in the United States.
Health Authority (HA)
Terminology used to describe the Health Authority of a given country
Intermediate Expanded Access Protocol
A type of expanded access protocol (see Expanded Access Protocol term) for the treatment of multiple patients at one or more locations, where the pharmaceutical company, or other entity, is the Sponsor and holds the cross-reference IND.
Investigational Medicinal Product (IMP)
Investigational medical products include investigational new drugs and biologics. Investigational new drug means a new drug or biological drug that is used in a clinical investigation. The term also includes a biological product that is used in vitro for diagnostic purposes. The terms “investigational drug” and “investigational new drug” are deemed to be synonymous.
Investigational New Drug (IND)
An IND is a request from a Sponsor to obtain authorization from the U.S. Food and Drug Administration (FDA) to administer an investigational drug or biological product to humans. In the case of a single patient IND (SPIND) the physician is the Sponsor. In the case of intermediate Expanded Access protocols, the IND Sponsor can be the pharmaceutical company or physician.
Investigational Review Board (IRB)
Also referred to as Investigational Review Committee (IRC). This is an independent committee reviewing benefit risk balance of early access requests at individual patient (single patient; named patient) or protocol level.
Managed Access Program (MAPs)
An early access program once a manufacturer has secured a first regulatory approval, allowing the supply of the medicine to patients and physicians in countries where it is not yet approved
Marketing Authorisation (MA)
All medicines must apply for Marketing Authorisation (MA) to be commercially available in the European Economic Area.
Medicines and Healthcare Products Regulatory Agency (MHRA)
The Medicines and Healthcare Products Regulatory Agency (MHRA) regulates medicines in the United Kingdom, ensuring applicable standards of safety, quality and efficacy.
Named Patient Program (NPP)
An early access program wherein the requesting physician with direct responsibility for an individual patient under their care, makes a bona fide unsolicited request for unlicensed medicine.
Open Label Extension Study (OLE)
A registration study that provides clinical trial participants continued treatment to the respective trial drug after the clinical trial has been completed.
Pharmacien Responsible
In France, the Pharmacien Responsible is the legally responsible individual ensuring the safety and quality of pharmaceutical products sold within the country and for products in compassionate use protocols.
Post-Trial Access (PTA)
The provision of investigational medicinal product after successfully completing a phase 2 or 3 clinical study and requiring treatment for a prolonged interval beyond the end of the clinical study.
Pre-approval policy
Alternatively, an expanded access policy, implemented by a pharmaceutical company and posted on their website.
Real World Data (RWD)
Data relating to individual patient health status, or the delivery of health care routinely collected from a variety of sources.
Review Committee (RC)
An advisory and or decision making group consisting of experts in medicine, bioethics and a patient advocates who review an expanded access / compassionate use / named patient request and provide decision-making guidance to the sponsor pharmaceutical company.
Single Patient Investigational New Drug (SPIND)
The U.S. Food and Drug Administration term for an IND submitted for an individual patient under the direct care and supervision of a treating physician, where the physician is a Sponsor.
Special Access Program (SAP)
The mechanism by which a healthcare professional can request a drug not available in Canada.
Special Access Scheme (SAS)
The mechanism for arranging for drugs for import and supply of an unapproved therapeutic good for a single patient, on a case by case basis, in Australia.
Special Needs Program (Specials)
Specials are medicines which have not been assessed by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for safety, quality, and efficacy in the same way as licensed medicines.
SUSAR
The abbreviation for Suspected Unexpected Serious Adverse Reactions.
Treatment IND
One type of Expanded Access Protocol (EAP) in the United States, referring to an expanded access protocol for large numbers of patients when clinical trials are completed.
Clinical Trial
A research study to analyze the safety and effectiveness of new treatments in a group of patient participants.
ClinicalTrials.Gov
The web-based resource that provides patients, family members, healthcare professionals, researchers and the public with easy access to information on clinical studies, Expanded Access Programs, and Compassionate Use Programs, in the United States and in some cases global programs. Expanded Access or Compassionate Use programs listed on ClinicalTrials.gov are assigned an NCT number.
Investigational drug
An investigational drug, alternatively called an experimental drug, is unlicensed substance that has been tested in a laboratory and authorized by the local health authority, such as the U.S. Food and Drug Administration, for testing in humans with a specified condition.