Expanded Access

Individual Access to Investigational Medicines Intended to Treat Serious Diseases

As it relates to individuals, expanded access, sometimes called “compassionate use”, is the individual patient use of an investigational medicinal product outside of a clinical trial that is intended to treat a serious or life-threatening condition. Wherever possible, use of an investigational medicinal product by a patient as part of a clinical trial is preferable because clinical trials generate data regarding the efficacy and safety of the investigational medicinal product. Those data help inform whether the benefits of the investigational medicinal products outweigh the risks, whether it should be approved by the regulatory authorities leading to wider availability and, if approved, when and how it should be used. When it is not possible for a patient to enroll in a clinical trial, Gilead will strive to provide access to the investigational medicinal product for individual patient use, or through an expanded access protocol when appropriate.

Various regulatory mechanisms exist in different countries to provide expanded access to investigational medicinal products and, as a result, regional variations in expanded access will occur.

It is important to remember that investigational medicinal products have not yet received regulatory approval; thus, their potential risks and benefits are not yet established. Physicians and patients should consider all possible benefits and risks when seeking access to an investigational medicinal product.

Expanded Access Criteria

Gilead uses the following criteria for considering requests to provide access to individual patients for expanded access: The patient is suffering from a serious/life-threatening condition.

• A strong biological rationale or clinical data that support the possibility that the potential benefits of administration of the investigational medicinal product to the patient for that serious/life-threatening condition could outweigh the potential risks.
• The patient’s physician has determined that treating the patient with the investigational product is in the patient’s best interests.
• The investigational medicinal product will be administered in accordance with applicable laws and regulatory requirements of the country where the patient is treated, including importation requirements.
• The patient is not eligible or able to participate in a clinical trial or similar sponsored access program.
• No therapeutic alternative is available.

Treating physicians submit expanded access requests on behalf of patients to Gilead at CompassionateAccess@gilead.com in addition to obtaining the local regulatory approval for the proposed use of the investigational medicinal product.

Medical professionals at Gilead who are familiar with the data collected on the investigational medicinal product evaluate each request and respond based on the scientific evidence available at the time of the request and the criteria listed above.

All individual patient use requests submitted by a treating physician must include the following information to be considered:

• Date of request
• Requesting physician’s name, contact information, address (including country), and professional designation (i.e., MD) or qualifications
• Name of the requested investigational medicinal product along with physician’s intended treatment plan, including therapeutic indication and expected duration of treatment
• Medical rationale for request including an explanation for why alternative therapy cannot be used, why the patient does not qualify for a clinical trial, and why use of the investigational drug is in the patient’s best interest.

A response for expanded access is typically received by the requesting physician within 30 days with either: approval, a request for additional information, or a rationale for non-approval.

Additional information regarding Gilead’s pipeline of investigational medicinal products can be found at www.gilead.com/research/pipeline and information regarding specific Gilead clinical studies can be found at www.clinicaltrials.gov.