In our view, the best way for patients to get access to investigational medicines is by taking part in clinical trials. In some cases, however, a patient with a serious or life-threatening disease or condition may not be eligible to enroll in a clinical trial sponsored by Shionogi, but the patient’s physician may believe the patient could potentially benefit from access to the investigational drug before it is approved by a regulatory agency.
Shionogi may consider individual expanded access requests when no comparable or satisfactory alternatives are available and there is sufficient evidence of the safety and effectiveness of the investigational product to support its use for a specific patient.
Expanded Access (Compassionate Use) Policy
Shionogi Inc. (“Shionogi”), a subsidiary of Shionogi & Co., Ltd., is committed to developing safe and effective medicines and treatments for patients worldwide. Shionogi, along with its corporate affiliates, is responsible for conducting clinical trials involving research participants to determine whether investigational drugs are safe and effective for treating a specific disease or condition, before seeking regulatory approval to market these drugs. These clinical trials are compliant with requirements imposed by regulatory authorities to protect the rights, safety and welfare of research participants. Shionogi intends to use the clinical data from these trials to support regulatory applications for permission to market its drugs.
Enrolling in a clinical trial is the most common way that people who seek access to an investigational drug may receive that drug, though enrolling in a clinical trial does not guarantee access to the investigational drug as the study design may dictate that a group of research participants receive other treatment options, or even a placebo.
In certain cases, however, a patient with a serious or life-threatening disease or condition may not be eligible to enroll in a clinical trial sponsored by Shionogi, but the patient’s physician may believe the patient could potentially benefit from access to the investigational drug before it is approved by a regulatory agency. The patient’s physician may seek to obtain the investigational product outside of the clinical trial through a process known as “expanded access” or “compassionate use.” In those narrow circumstances, when no comparable or satisfactory alternatives are available, regulatory agencies may grant companies permission to provide a physician with access to an investigational drug outside of a clinical trial for treatment.
In general, Shionogi will consider making available, for expanded access use for individual patients, an investigational drug intended to diagnose, monitor or treat one or more serious diseases or conditions, in response to an unsolicited request from a licensed physician (“Expanded Access Request”). Shionogi does not guarantee it can or will approve access to investigational drugs pursuant to an Expanded Access Request.
Expanded Access Requests directed to Shionogi must be in writing, must be signed and submitted by a licensed physician who is in a treatment relationship with the patient for whom the investigational drug is being requested, and must be sent to firstname.lastname@example.org. Shionogi may require up to five (5) business days to review and/or acknowledge receipt of the Expanded Access Request (“Review Period”).
Please note that Shionogi or the physician making the Expanded Access Request may be required to obtain permission from one or more regulatory authorities, including the United States Food and Drug Administration (FDA), before Shionogi may grant an Expanded Access Request, which may delay Shionogi’s decision. Shionogi intends to provide a written response to all Expanded Access Requests in a timely manner. Shionogi may contact the requesting physician during the Review Period if Shionogi requires additional information to consider the request.
In general, Shionogi will consider, among other factors, the following criteria when reviewing an Expanded Access Request:
- The nature of the disease or condition of the requesting patient;
- Whether clinical data are available to assess the potential benefits and risks in providing the investigational drug for treatment;
- Whether it is reasonable and appropriate, from a clinical and scientific perspective, to use the investigational drug for this particular patient given, among other things, the potential benefits and risks in providing the investigational drug for treatment;
- Whether there are alternative treatment options available and/or whether such treatment options have been used;
- Whether, with the possible granting of the request for the investigational drug, there would remain sufficient supply of the investigational drug for ongoing and planned clinical trials;
- Whether the use of the investigational drug will be monitored and controlled appropriately and adequately by a licensed physician; and
- Whether the Expanded Access Request, and Shionogi’s grant of the request, would be compliant with applicable local, state, and federal laws and regulations.
Please search clinicaltrials.gov for publicly available information related to Shionogi’s ongoing applicable clinical trials.
Shionogi may require the requesting physician to sign certain agreements, including a confidential disclosure agreement, before Shionogi can disclose confidential and proprietary information or ship the investigational drug for the patient’s use. The requesting physician must comply with applicable regulatory law, obtain any necessary regulatory authority or ethics committee approvals and comply with applicable regulatory requirements, including monitoring, reporting and recordkeeping obligations. In addition, if Shionogi does grant an Expanded Access Request, the physician will be required to provide the patient with a legally effective consent form that describes, among other information, the risks and benefits of the investigational drug, as well as alternative treatment options.