Expanded access

At Lilly, we understand that sometimes people may seek access to
investigational medicines before they are reviewed and approved by
a regulatory authority, such as the U.S. Food and Drug
Administration (FDA).

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This can be done in two ways - through clinical trials and

expanded access.

Our goal, whenever possible, is to encourage patients to participate
in clinical trials. Clinical trials are research studies designed to
determine if an investigational medicine is safe and effective for
patients. Data generated by these studies provide regulatory
authorities with essential information needed to evaluate a
medicine. Only after reviewing this information will regulators
approve a new medicine, allowing it to be widely available.

In rare cases, however, when people don’t qualify for clinical trials
and have exhausted all available medical options, Lilly may consider
providing an investigational medicine outside of a clinical trial. This
is called expanded access.

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Types of access to

investigational medicines

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Clinical trials

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Because clinical trials are well-controlled and usually require a large number of patients, they offer the safest and best opportunity for people to access an investigational medicine before it is approved by a regulatory agency. Clinical trial participants receive quality care as the health care providers managing the clinical trial closely monitor participants and all aspects of their disease. In addition, clinical trial participants contribute to greater scientific understanding of their disease and the investigational medicine being studied. This important information may lead to a new approved medicine, and also contribute to the development of other new medicines in the future.

To learn more about available clinical trials visit clinicaltrials.gov and search by company, disease
or medicine.

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Expanded access

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Expanded access, also known as compassionate use, may be an option for a limited number of individuals with serious or life-threatening conditions, who have exhausted all available medical options and do not qualify for
clinical trials.

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Lilly's approach to expanded access

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Making decisions about expanded access requests is always complex. We strive to make these decisions as comprehensively as possible, considering the patient’s case and the available medical and scientific information about the investigational medicine. Our primary responsibility is to ensure that expanded access can be done in a manner that upholds ethical principles, such as fairness, promoting good and minimizing the risk of harm for both current and future patients.

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Lilly will consider granting expanded access to an investigational medicine only if all of the following threshold criteria are met:

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• The patient has a serious or life-threatening illness with no comparable or satisfactory alternative therapies.
• The patient is ineligible for, or otherwise unable to, participate in a clinical trial.
• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational medicine and the benefit outweighs the known or anticipated risks.
• The investigational medicine is currently in clinical development - that is, it is currently being studied in humans.
• Providing the investigational medicine for the requested use will not interfere with the initiation, conduct, or completion of clinical trials.

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Once a regulatory agency approves a medicine for commercial use, existing expanded access programs will be phased out.

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Expanded access approaches

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On a limited basis, Lilly offers two different types of expanded access:

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Cohort (or ‘Group’) Expanded Access (also known as Expanded Access Programs)

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Over the course of developing a new investigational medicine for a serious or life-threatening condition, Lilly may obtain sufficient clinical information about that investigational medicine to identify a population of patients for whom expanded access may be appropriate. In these cases, Lilly may establish an expanded access program for a cohort, or group, of patients who meet specific eligibility criteria. These programs are managed by Lilly and follow a specific protocol, which is developed in consultation with a regulatory agency for the use of the
investigational medicine.

To find open cohort expanded access programs, search "expanded access programs" on clinicaltrials.gov.

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Individual Patient Expanded Access

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In the absence of a Cohort Expanded Access Program and in rare cases, Lilly may still decide - in response to a physician request - to provide investigational medicine access to an individual patient, where it is allowed by local regulations and when it meets our company’s expanded access criteria. Individual patient expanded access is managed by the patient’s physician.

Learn more from the FDA about expanded access in the U.S.

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Requesting Expanded Access

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We encourage patients to speak first with their physician about their eligibility to enroll in a clinical trial.

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If a treating physician believes expanded access may be the only option for a patient, the physician should contact Lilly to make a formal request on behalf of the patient. This will enable the physician to work with Lilly’s physicians to determine the best course of action.

The request for access to a Lilly investigational medicine can only be considered if the patient's treating physician is committed to, and supportive of, the requested treatment.

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In the U.S., physicians seeking help on behalf of their patients may contact Lilly at 1-800-Lilly-Rx (1-800-545-5979). Outside of the U.S., physicians may contact the local Lilly office within their country to speak with a member of our medical department. Once Lilly has received the request and required medical information, we will respond in no more than five business days. If the request is approved by Lilly, the country regulatory authority from where the request originated will need to grant final approval before Lilly can ship the investigational medicine to the requesting physician.

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