Compassionate use is a term used to describe the provision of an unauthorized medicine when it is given to a patient at no cost. This usually occurs when a drug company recognizes there is a serious need for its product. It, therefore, creates a program to provide it free of charge to an individual patient or a group of patients.
The drug provided through a compassionate use program can be considered unauthorized for the following reasons:
- It is an investigational medicine (IMP) that is still in clinical trials and not yet approved anywhere in the world – i.e. an unapproved drug
- It is an approved drug that that is not licensed locally in the patient’s country
This treatment option is for patients suffering from rare diseases, cancers or other life threatening conditions. Patients suffering from such a disease or condition will likely have no effective or approved treatment options available to them.
A compassionate use program is usually set up and managed by the drug companies, or a third-party provider (like WEP Clinical). However, in some cases a research or medical institution can also run a program.
The drug manufacturer must agree to provide the drug through compassionate use in order for patients to access it. Some drug manufacturers choose not to provide a compassionate use program, due to lack of resources. Others wish to focus on the clinical trial or commercial supply. However, there are many tangible benefits to providing a compassionate use program. For more information, check out the Sponsors section of our Expanded Access Programs and Named Patient Programs pages:
Patients who wish to be enrolled into an active compassionate use program must have a treating physician willing to prescribe the drug and complete the application process, on behalf of the patient. The physician must decide that the potential benefits of using the unauthorized drug outweigh the potential risks. The physician must also administer the drug and monitor the patient. When necessary, the physician will be expected to provide any safety and efficacy data to the drug company or applicable health agency. In the US, that would be the Food and Drug Administration (FDA) in the US.In the case that the product being provided is an investigational drug, the term compassionate use program is often used interchangeably with Expanded Access Program (EAP). An EAP refers to the use of an investigational drug to treat a patient outside of the clinical trial setting. Expanded access to an investigational drug is for patients who cannot be enrolled into a clinical trial. In the vast majority of EAP cases, the drug is provided at no cost, which is why an EAP is often referred to as a compassionate use program. More can be read about compassionate drug use through expanded access on the following websites:
Companies running phase 2 or 3 clinical trials in the US, must have an Expanded Access Policy published on their website. The policy should explain whether the company has chosen to provide its investigational product(s) to patients through compassionate use. The policy must include contact information which physicians can use to apply for a program or send questions. The policy must also explain how a company will review and respond to expanded access requests.
WEP Clinical provides template documents that companies can download and use to create their policy for their website: