We are committed to bringing mitochondria-targeted therapies to patients for the treatment of rare and life-threatening diseases. We conduct clinical trials to evaluate the safety and effectiveness of our investigational drugs in an effort to ultimately obtain regulatory approval to provide these therapies to the broadest group of patients. Regulatory authorities will permit us to provide patients with broad access to these investigational medicines only after we have demonstrated that they are safe and effective.
We believe the best way for patients to access medicines prior to approval is through participation in clinical trials. However, in some rare and very specific circumstances when enrollment into a clinical trial is not feasible, physicians caring for patients with a life-threatening or imminently debilitating disease or condition may seek special access to investigational medicines (referred to as compassionate use or also known as expanded access). Only in these rare and very specific circumstances patients may be able to receive investigational medicines outside the clinical trial process through an expanded access program. Stealth BioTherapeutics will consider Individual Patient Expanded Access requests to an investigational medicine outside of a clinical trial on a case-by-case basis for patients with a life-threatening or imminently debilitating disease or condition, provided the request initially meets the following criteria:
- The patient and a licensed physician are both willing to participate.
- The patient’s physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient’s disease or condition.
- That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition.
- There is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance.
- That providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval.
- The patient’s physician submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with FDA’s statute and applicable regulations for INDs, describing the use of the investigational product.
- The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial.
A qualified and licensed physician must submit the request for Individual Patient Expanded Access in writing. The request must contain, at a minimum, the following information:
- Demographics of the proposed Individual Patient Expanded Access patient
- Relevant medical history, including all therapies administered to date for the relevant indication
- Assessment of the patient’s prognosis
- Reason and rationale for requesting Individual Patient Expanded Access product
- Proposed treatment plan (inclusive of therapeutic and safety monitoring) under Individual Patient Expanded Access Program
- Statement that the physician (and the associated institution, if relevant) is able and willing to prepare and submit to the appropriate country and local regulatory authority the required documents to obtain a treatment IND for the patient (as applicable)
- Statement that the physician (and the associated institution, if relevant) will obtain informed consent from the patient and approval of the appropriate IRB and national regulatory authority prior to initiating treatment
Stealth BioTherapeutics will review the request in accordance with the criteria described in this Expanded Access Policy. A communication will be sent to the requesting physician as to Stealth BioTherapeutics’ denial or approval of the request for Individual Patient Expanded Access. There is no appeal process for a request that has been denied by Stealth BioTherapeutics.