What is a Compassionate Use Program?
Compassionate use, also referred to as expanded access or early access, is a term used to describe access to a medicine that is not available through standard approval pathways. It typically applies when a patient has a serious or life-threatening condition and there are no suitable authorized treatment options.
A compassionate use program is usually established when a drug developer recognizes an urgent unmet medical need and makes its product available to an individual patient or a defined group of patients outside of a clinical trial.
A medicine accessed through compassionate use may be considered “unauthorized” for a few reasons:
- It is an investigational medicine still in clinical development and not yet approved anywhere (i.e., an unapproved drug).
- It is an approved medicine, but it is not licensed locally in the patient’s country.
Who Qualifies For Compassionate Use?
Compassionate use is generally intended for patients with rare diseases, cancers, or other serious or life-threatening conditions, and is utilized only when available approved treatments are ineffective, not tolerated, or do not exist.
Read More – Early Access Programs : What To Consider As a Patient
How Is A Compassionate Use Program Initiated?
A compassionate use program is usually set up and managed by the drug companies, or a third-party provider (like WEP Clinical). In some cases, a research or medical institution may also support the process.
For a patient to access a medicine through compassionate use, the manufacturer must agree to provide the product through that pathway. Companies often choose to implement a program when it aligns with their development plan and patient access strategy. Companies may decide not to offer compassionate use at a given time, due to operational constraints or because they need to prioritize clinical trial supply and development activities.
If you’d like more detail on sponsor considerations, visit our Expanded Access Programs and Post-Trial Access pages.
How Can Patients Access Compassionate Drug Use?
Patients who wish to be considered for an active compassionate use program generally need:
- A treating physician willing to prescribe the medicine and complete the required application steps on the patient’s behalf.
- A physician determination that the potential benefits outweigh the potential risks for that individual patient.
- Ongoing physician oversight, including administration (when required) and monitoring.
When appropriate, the treating physician may also be expected to share relevant safety and clinical outcomes with the drug company and/or the applicable health authority. In the US, this may involve the Food and Drug Administration (FDA).
In cases where the product is investigational, the term compassionate use is often used interchangeably with Expanded Access Program (EAP). Expanded access refers to the use of an investigational medicine to treat a patient outside of a clinical trial setting.
You can read more about expanded access from the FDA or The American Cancer Society.
Compassionate Use Policy Templates
Companies running phase 2 or 3 clinical trials in the US are required to publish an Expanded Access Policy on their website. This policy should explain whether the company will consider requests for expanded access (compassionate use) to its investigational product(s). It should also include:
- Contact information physicians can use to submit a request or ask questions
- How the company reviews requests
- Expected response process and timelines (as applicable)
WEP Clinical provides template documents that companies can download and use to create their policy: