Expanded Access

Every approved medication has been tested to show that it is safe and effective. This testing can take a decade or more, which for many people living with a serious disease is too long to wait.

Regulators such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have taken steps to speed up parts of this process so that important drugs reach people sooner. One of these steps is expanded access, also called compassionate use. Expanded access gives people with a serious or life-threatening illness early access to a drug that is still being tested and has not yet been approved.

When expanded access is allowed

Drugs in expanded access are not yet proven to be safe and effective. For this reason, regulators generally prefer that people take part in a clinical trial first. They also take steps to make sure people are not exposed to undue risk.

In the United States, the FDA may allow expanded access when all of the following apply:

• The person has a serious or immediately life-threatening illness, and no comparable or satisfactory treatment is available to them.
• The person cannot enroll in a clinical trial, either because no trial is available or because they are not eligible for any trial.
• The potential benefit of the drug justifies the potential risks.
• Giving the drug will not interfere with the clinical trials that are needed to approve it.

In the European Union, each member state sets its own rules and runs its own compassionate use programs. The EMA gives recommendations on how a medicine should be used in these programs, but these recommendations do not create a legal framework. Rules in other countries differ. Ask your doctor or your national regulatory agency what is available where you live.

Forms of expanded access

Expanded access takes several forms:

• A program for a large group of people, to bridge the gap between the end of a clinical trial and marketing approval.
• A program for a smaller group of people who need a drug that is not being actively developed for clinical use.
• Permission for a doctor to give an individual person an investigational drug in an emergency.

Expanded access for people living with ALS/MND

ALS/MND is a serious, life-threatening disease, and current treatment options are limited. Some experimental ALS/MND drugs may therefore qualify for expanded access. Whether you are eligible depends on the drug, your situation, and the country where you live. Talk with your ALS/MND doctor, who can tell you whether an expanded access program exists for a drug you are interested in.

Risks of expanded access

Regulators support expanded access, but they prefer that people take part in clinical trials. Clinical research offers more protection if a treatment is unsafe or ineffective, and clinical trials are the best way to prove a medication’s safety and effectiveness.

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