Compassionate Use by Country
The requirements for government approval of compassionate use differ from country to country. Click on a country to learn about its compassionate use approval programs.
In the United States, the drug company must first agree to provide compassionate use of its experimental drug before the U.S. Food and Drug Administration (FDA) can consider a formal request for compassionate use under the Expanded Access program. If the drug company agrees, the patient’s doctor can then submit a formal approval request to FDA. The compassionate use request must meet the following criteria for FDA approval:
- There is no comparable or alternate treatment option available to the patient
- The patient cannot obtain the experimental drug through a clinical trial
- Risk to the patient from the experimental drug is no greater than the risk of not using the drug
- The safety and efficacy evidence warrant compassionate use of the experimental treatment
- Treatment plan (“clinical protocol”) is consistent with FDA rules and regulations
- Drug company agrees to give the patient(s) access to the experimental treatment
- The patient gives informed consent (in writing)
- Institutional Review Board (a medical ethics committee) reviews the treatment plan
- Compassionate use of the experimental treatment will not interfere or hinder clinical trials of the experimental drug
The Expanded Access program covers both individual patient compassionate use and compassionate use for groups of patients. See the corresponding FAQ to learn about the difference between individual and group compassionate use.
For more information, visit FDA Expanded Access Program.
The patient's doctor submits a request for the sale of an unauthorized drug to Health Canada's Special Access Programme (SAP). The request must be accompanied by supporting evidence for the patient's case. Health Canada considers the following factors in its decision:
- Seriousness of the disease
- Clinical status (prognosis) of the patient
- Other treatments tried and/or ruled out
- Any past experience the patient has with the requested drug
- Available data on the drug
- Availability of clinical trials for the patient
If Health Canada approves special access for the patient, the drug company then must determine if it is willing to provide compassionate use of the drug.
For more information, visit Health Canada’s SAP.
The European Medicines Agency (EMA) provides to EU Member States recommendations on how to administer, distribute, and use certain medicines for compassionate use. However, EMA’s recommendations are not legally binding. Rather, each Member State of the EU establishes and runs its own compassionate use laws and programs.
EURODIS provides a resource guide to seeking compassionate use in Europe, including a directory of national compassionate use agencies in each Member State.
In Australia, the Therapeutic Goods Administration (TGA) manages compassionate use under the Special Access Scheme (SAS). SAS classifies patients under two categories:
CATEGORY A: Patient is “seriously ill with a condition from which death is reasonably likely to occur within a matter of months, or from which premature death is reasonably likely to occur in the absence of early treatment.”
Doctors can provide special access (compassionate use) of unauthorized drugs to Category A patients without approval of the TGA. The doctor must notify the TGA of the treatment. The doctor must also send an Authority to Supply form to the drug company to provide the drug company legal authority to supply the drug. However, the drug company is under no obligation to supply the drug. The drug company must determine if will agree to provide compassionate use of the drug.
CATEGORY B: All other patients
For Category B patients, the TGA approves special access (compassionate use) to unauthorized drugs on a patient-by-patient basis. The doctor must submit an application to the TGA. If the TGA approves the request, the drug company must then determine if it is willing to provide compassionate use of the experimental drug.
This page is not intended to act as a complete guide to compassionate use approval processes. Rather, it provides program overviews and links for each below country. If you have information on additional countries that we can add to this page, please contact us.