Sage is committed to discovering, developing and commercializing novel medicines to transform the lives of patients with life-altering brain health disorders. As part of this mission, we conduct clinical trials to determine if our investigational medicines are safe and effective for patients with a particular disease, disorder or condition which may allow us to seek the necessary regulatory approvals to provide wide access to these medicines.
We understand that, in some cases, patients with serious diseases or conditions may not be eligible for clinical trials and may not have other treatment options. Under very limited circumstances, we will consider making investigational medicines available for qualifying patients to treat a serious disease or condition outside of a clinical trial prior to regulatory approval. This type of access is often referred to as “early access”, “expanded access” or “compassionate use”. The following criteria must typically be met before we will consider making one of our investigational medicines available to individuals under an early access program or EAP:
- The patient has a serious disease or condition, has exhausted all available treatment options, and is not eligible for any appropriate clinical trials.
- The investigational medicine is currently being studied, or has been studied, in clinical trials for the treatment of a disease or condition that is similar in type and stage to the indication for which the expanded access is sought.
- The request is made by a licensed physician who is qualified and knowledgeable in the treatment of the relevant disease or condition. Patients who are interested in understanding access options should speak with the physician overseeing their care.
- There is sufficient evidence that the potential benefit to the patient, taking into account available safety and efficacy information, would likely outweigh the potential risks to the patient.
- Adequate clinical data exist to support the appropriate dose amount and frequency of the investigational medicine and duration of treatment in the relevant disease or condition.
- There are adequate supplies of the investigational medicine to meet the needs of the early access program without impairing ongoing development, including ongoing and planned clinical trials.
- The requested use will not have the potential for a negative impact on broader development programs, including ongoing or planned clinical trials or regulatory filings designed to make the therapy available to many more patients.
- The proposed use will occur in a country where we plan to seek approval of the investigational medicine.
- The requested use complies with applicable laws and regulations.
Even if these criteria are met, there is no guarantee one of investigational medicines would be made available through an EAP. Additional information regarding our clinical studies can be found at www.clinicaltrials.gov.
If a clinical trial may not be appropriate for your patient and an EAP for one of our investigational medicines is open, treating health care providers (HCPs) should submit early access requests on behalf of patients to Sage Medical Information. Please do not include a patient’s personally identifiable information in any request. Requests will be assessed, on a case-by-case basis, by a team at Sage that includes medical professionals who are familiar with the data collected on the investigational medicine. In evaluating a request for early access, we may consider all relevant factors not just those specified above, including other factors relevant to patient safety. We anticipate acknowledging receipt of requests within five business days. If required, the regulatory agency in the relevant country must also approve the proposed use of the investigational medicine. The requesting physician must agree to obtain appropriate regulatory and IRB and/or ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements.
If a request is granted, early access will be discontinued as soon as feasible when approval of the drug is achieved in the country, subject to any legal or regulatory requirements.
We may revise this policy at any time consistent with applicable law and regulations.