Expanded access and compassionate use

Topic

It means giving a not-yet-approved product, outside a trial, to a seriously ill patient with no alternatives. It must balance hope and risk.

Updated Nov 13, 2026

Key points

Expanded access (compassionate use): using an investigational product outside a trial for a seriously ill patient with no alternatives.
Usual conditions: serious or life-threatening disease, no satisfactory alternative, no possibility of entering a trial, a potential benefit that justifies the risk, and no interference with trials.
Helsinki, para. 37: an unproven intervention may be used with expert advice, informed consent and, where possible, as the object of research.
Continued access: after a trial, the intervention is planned for participants who still need it (Helsinki, para. 34).
Colombia: Ley 23 de 1981, art. 12, par.: an experimental procedure may be used if it is the only chance of saving the patient, with authorization and, if possible, a medical board.
Decreto 481 de 2004 regulates something else: vital medicines unavailable in the country that are not under clinical investigation.

What it is

Expanded access, or compassionate use, allows an investigational medicine or device to be used outside a clinical trial to treat a patient with a serious disease who has no alternatives. It pits beneficence (the hope of someone with no other option) against nonmaleficence and justice: the risks may be unknown, the hope may be false, access goes to those with more contacts or resources and, if it becomes widespread, it can draw participants away from the trials that would give the answer. The Declaration of Helsinki (para. 37) accepts unproven interventions in an individual patient with expert advice, informed consent and, where possible, by making them the object of research. Continued access is different: going on giving the intervention to those who took part in a trial and still need it.

Key facts

Ethical risks: false hope, unknown harms, therapeutic misconception, inequity of access and fewer trial participants.
Consent must say that the benefit is unproven, that there may be unknown risks and what alternatives exist, including palliative care.
It is not research: its purpose is to treat one person; even so, adverse effects are recorded and reported.
Compassionate use programme: for a group of patients; named-patient use: for one specific patient.
Decreto 481 de 2004, art. 2: a vital unavailable medicine is one that is essential and irreplaceable to save life or relieve suffering and is not available in the country, or not in sufficient quantity.
Decreto 481 de 2004, art. 4: it must not be under clinical investigation, nor marketed in the country in sufficient quantity, nor have substitutes.

In clinical practice

A patient with advanced cancer asks for a drug in a trial that she saw online: check whether there is an open trial for her, explain the real evidence and do not stop palliative care.
If an unproven intervention is used, record the justification, the consent and the plan for monitoring adverse effects.
A medicine approved abroad for a rare disease and unavailable in Colombia follows the vital-unavailable-medicine route before Invima, not compassionate use.
Raise the justice question: could other patients in the same situation get the same access?

Legal framework in Colombia

Ley 23 de 1981, art. 12: only accepted means are used; parágrafo: in exceptionally grave circumstances, an experimental procedure that is the only chance of saving the patient is used with the authorization of the patient or their relatives and, if possible, by agreement of a medical board.
Decreto 481 de 2004, arts. 5 and 8: a vital unavailable medicine does not need a marketing authorization and may be imported for a specific patient, with an application to Invima, a prescription and a summary of the medical record.
Decreto 481 de 2004, art. 9: in a clinical emergency, import of non-commercial quantities may be authorized without the patient's documents, with the medical justification.
Ley 1751 de 2015, art. 15, lit. e): public resources do not fund services that are in an experimental phase.
Official texts checked on 2026-11-13 at minsalud.gov.co (original text of Decreto 481 de 2004 and Ley 23 de 1981) and normograma.supersalud.gov.co.

Test yourself

Think of the answer, then tap the question to see it.

What is expanded access or compassionate use?

Using an investigational medicine or device outside a clinical trial to treat a patient with a serious disease and no satisfactory alternatives.

What conditions does the Declaration of Helsinki set for using an unproven intervention in a patient?

Expert advice, informed consent and, where possible, making it the object of research (paragraph 37).

How does a vital unavailable medicine differ from one in compassionate use?

The vital unavailable medicine is not under clinical investigation: it is an essential medicine that cannot be obtained in the country (Decreto 481 de 2004, arts. 2 and 4).

What is a justice risk of compassionate use?

That only those with contacts or resources get access, and that participants are drawn away from the trials that would give the answer for everyone.

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Connections

← All of medical ethics

Educational content on ethics and health law; not legal advice. Laws and case law change: each page links the official text and says when it was last checked. For a real case, read the text in force and ask a lawyer or the medical ethics tribunal.

Sources: Expanded Access (U.S. Food and Drug Administration, 2026); WMA Declaration of Helsinki – Ethical Principles for Medical Research Involving Human Participants (World Medical Association, 2024); Ley 23 de 1981, por la cual se dictan normas en materia de ética médica (Ministerio de Salud y Protección Social, 1981); Decreto 481 de 2004, normas para incentivar la oferta de medicamentos vitales no disponibles en el país (Ministerio de Salud y Protección Social, 2004); Ley Estatutaria 1751 de 2015, que regula el derecho fundamental a la salud (Superintendencia Nacional de Salud, Normograma, 2015).

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