Compassionate Use Glossary



Anatomic, therapeutic, chemical. International system for classification of medicines.


Biotechnology is used in two ways: first it describes the traditional biological methods which living organisms use to produce or modify chemical compounds (e.g. antibiotics and vitamins). Secondly, it includes gene technology, which is based on the ability to isolate, replicate, cut and recombine DNA and to transfer DNA from one cell to another. Organisms whose genetic material has been altered in a way that does not occur naturally are called genetically modified organisms (GMOs).

Centralised Procedure

Since 1995, medicinal products can be evaluated via the Centralised procedure. Medicinal products that have been approved via this procedure are issued a marketing authorisation that is valid throughout the EU. This marketing authorisation is granted by the European Commission. The use of this procedure is compulsory for medicinal products derived from a biotechnological process, for anticancer treatments, or for medicines to treat diabetes, AIDS/HIV infection, rare diseases, immune diseases, and neurological diseases. For other innovative products, such as products with a new active substance, a company can choose whether to follow this procedure or the Mutual recognition procedure. In the case of the Centralised procedure, a dossier must be submitted to the European Agency for the Evaluation of Medicinal Products (EMA) in London.

Clinical Trials

A clinical trial is a research study conducted in human participants to evaluate the safety and efficacy of a medicine to improve patient's health. Clinical trials conducted on new medicines and sponsored by pharmaceutical companies can only be started after a compound has survived rigorous pre-clinical development work, which involves laboratory testing (chemical/biological/pharmacological/toxicological). It is only when these tests show favourable and promising results that a company can proceed to assess the medicine in humans.

Committee for Medicinal Products for Human Use - CHMP

Within the European Medicines Agency (EMA), the Committee for Human Medicinal Products is the scientific committee responsible for preparing the Agency's opinions on questions relating to the evaluation of medicinal products for human use.

Compassionate Use

Refers to situations where a drug is provided to a patient prior to the drug's receiving regulatory approval.


A person who is not a healthcare professional such as a patient, lawyer, friend or relative / parent / child of a patient

Healthcare professional

For the purposes of reporting suspected adverse reactions, healthcare professionals are defined as medically qualified persons, such as physicians, dentists, pharmacists, nurses and coroners.

Informed consent

The concept of informed consent (required to participate in clinical trials and sometimes for compassionate use) is based on the principle that a physician/doctor has the duty to disclose information to the patient (e.g. potential risks, benefits and alternatives) that allows the patient to make reasonable decision regarding his or her treatment on a compassionate basis.

Name of the medicinal product

The name which may be either an invented name not liable to confusion with the common name, or a common or scientific name accompanied by a trade mark or the name of the marketing authorisation holder.

The common name is the international non-proprietary name (INN) recommended by the World Health Organization, or, if one does not exist, the usual common name.

The complete name of the medicinal product is the name of the medicinal product followed by the strength and pharmaceutical form.

Ultra-compassionate use

There are cases when a patient is at the end stage of the disease, suffering, or worsening. Aware that a new product is available, he or she is willing to take the risk, even if very little is known about the product, or even if the toxicity profile is not yet determined. This type of patient may prefer to rake the risk of dying from an adverse drug reaction if the product is finally not safe, because he or she will die soon anyway. And if there is the faintest chance that the treatment benefits him or her, at least he or she will have tried. In such dramatic situations, some regulators take the stand that it is not their role to intervene: it is up to the doctor and the patient to decide. Any regulatory intervention, even in an emergency context, would take time and from their position, regulators do not feel at ease to decide. It is a debate close to the debate on end-of-life  and euthanasia: when legal, the decision belongs to the patient and his/her doctor, there is no need to ask prior authorisation from any authority.

Page created: 30/10/2013
Page last updated: 23/03/2017
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