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Snapshot: What Is an Expanded Access Program?

When faced with a serious illness and no effective treatment, patients may look to experimental therapies as a last resort. Normally, patients can only access experimental drugs or other investigational medical products through clinical trials. Unfortunately, not everyone can access a clinical trial when they need it. Some live too far from research centres or are too unwell to travel. Others do not fully meet a trial’s eligibility requirements. Even after a product successfully completes clinical trials, it can take several months or even years for it to gain market approval and become available to patients. Expanded access programs are designed to address urgent patient needs in these situations.

What Is Expanded Access?

Expanded access (sometimes called “compassionate use” or “pre-approval access”) is a way for patients to access medical products before they are approved for a particular condition. These products may be new or existing drugs, biologics, vaccines, or medical devices that have not yet received full regulatory approval. Depending on the medical product and other factors, expanded access may apply to an individual patient or to a large group of patients.

How Expanded Access Works

Several conditions must be met for a patient to use a medical product through an expanded access program:

The patient must have a serious or immediately life-threatening condition, for which there is no satisfactory alternative therapy

The patient cannot enroll in a clinical trial testing the product or cannot wait for regulators to fully approve the product

The potential benefits of using the product outweigh the potential risks to the patient

Expanded access to the product will not interfere with its ongoing development, testing, or regulatory approval

These conditions are important, because regulatory agencies such as the U.S. Food and Drug Administration have not yet determined that the medical product is safe or effective for a particular condition. This means that the product may not help patients. It may even cause serious side effects.

The process of obtaining treatment under an expanded access program usually starts with a doctor determining that a patient has no alternative treatment options. This is either because there are no treatments available or because standard treatments have failed. If the doctor believes their patient is eligible to try a particular investigational product, they contact the company that developed the product to see if it can be made available through an expanded access program. If the company agrees, the request is reviewed by ethics boards and regulatory agencies to ensure the proposed use of the product is justified. Once approved, the patient begins treatment and is closely monitored by their doctor.

Expanded access programs vary by country, reflecting differences in regulatory frameworks. For example, Canadian patients have two primary pathways for accessing investigational medical products outside standard clinical trials. These include Expanded Access Clinical Trials, which are designed to be more flexible and inclusive than traditional trials, and the Special Access Programme, which allows individual patients to access investigational therapies in urgent situations. If you are considering an investigational therapy, your doctor can help find a suitable program and guide you through the process.

Expanded Access and Spinocerebellar Ataxia

There are currently no treatments that can stop or reverse the symptoms of spinocerebellar ataxia (SCA). As pharmaceutical companies develop new treatments for the disease, patients may be able to access them before they are approved by regulators. For example, one experimental treatment under development is Troriluzole. This drug helps normalize the way brain cells communicate by controlling levels of glutamate, one of the main signaling molecules in the brain. While we await full regulatory approval of Troriluzole, people with SCA may be able to access the drug through an expanded access program managed by the manufacturer, Biohaven Pharmaceuticals Inc. For more information about this program, visit: https://clinicaltrials.gov/study/NCT06034886.