Extended access, sometimes referred to as “compassionate use,” is the use of an investigational drug outside of a clinical trial. A patient may receive the product (if applicable) through extended access if enrollment in a clinical trial is not possible, for example if a patient is not eligible.B for ongoing clinical trials or if there are no ongoing clinical trials. Contrary to common usage, the terms “emergency use” and “compassionate use” are not synonymous. Be aware of specific and separate standards for emergency use and compassionate use/extended use to avoid violations of federal regulations and UCSF guidelines regarding the use of unapproved drugs, biologics, and devices. Whether the physician writes the consent or a sample of consent from another source (e.B. the sponsor/manufacturer of the test item), the emergency consent should contain the elements of informed consent that are found in the UCSF model, but does not have to follow the format and wording of the UCSF. It must be clearly stated that: 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 use by the patient. The requesting physician must comply with applicable regulatory law, obtain all necessary approvals from the regulatory authority or ethics committee, and comply with applicable regulatory requirements, including monitoring, reporting and record-keeping obligations. In addition, if Shionogi grants an extended access request, the physician must provide the patient with a legally effective declaration of consent describing, among other things, the risks and benefits of the investigational drug and alternative treatment options. However, in some cases, a patient with a serious or life-threatening disease or condition may not be eligible to enroll in a Shionogi-sponsored clinical trial, but the patient`s physician may believe that the patient could potentially benefit from access to the investigational drug before it is approved by a regulatory body.

The patient`s physician may attempt to obtain the investigational drug outside of the clinical trial through a process known as “extended access” or “compassionate use.” In these narrow circumstances, where no comparable or satisfactory alternative is available, regulatory authorities may allow companies to grant a doctor access to an investigational medicinal product outside of a clinical trial for treatment. Note: Emergency use is emergency clinical care and does not meet the DHHS research definition. The IRB`s agreement that a particular case meets the FDA`s emergency response criteria applies only to the treatment of a patient and is not the same as the IRB`s approval to conduct a research study. Sometimes referred to as “compassionate use,” extended access is a potential pathway for a patient with an immediately life-threatening disease or condition or a serious disease or condition to gain access to an investigational drug (drug, biologic or medical device) for treatment outside of clinical trials when comparable or satisfactory alternative treatment options are not available. 3. Once the Emergency IND has been received from the FDA, send an email to [email protected] with “Emergency Use Request” in the subject line: Please search clinicaltrials.gov publicly available information about Shionogi`s ongoing clinical trials. Exemption from registration of the inclusion/exclusion criteria of an approved protocol Learn more about extended access, including information about the extended access process, what the FDA considers, and the costs it may incur. For general questions or if you are not sure who to contact, contact patient affairs staff at 301-796-8460 or patientaffairs@fda.hhs.gov. 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 affiliates, is responsible for conducting clinical trials with research participants to determine whether investigational drugs are safe and effective for the treatment of a particular disease or condition before seeking regulatory approval to market these drugs. These clinical trials meet regulatory requirements to protect the rights, safety and well-being of research participants. Shionogi intends to use clinical data from these studies to support regulatory applications for marketing approval of its drugs. Extended access may be appropriate if all of the following apply: The FDA requires the attending physician to document emergency use in writing and submit the report to the IRB within five (5) business days of the start of emergency use. The doctor should use the emergency form after use. (1) the patient(s) to be treated suffer from a serious or immediately life-threatening disease or condition and there is no comparable or satisfactory alternative treatment for diagnosing, monitoring or treating the disease or condition; Learn how to complete and submit the required forms for each type of extended access request. If strict emergency requirements are not met, the physician and facility may face severe penalties. In 2017, the FDA began approving “Alternative IRB Review Procedures” to expand access to individual patients (sometimes referred to as single-patient INDs).

Prior to this FDA decision, requests for expanded access for individual patients had to be reviewed by a convened IRB committee. .