Compassionate Use Policy
Patient Access to Investigational Drugs
At Stealth BioTherapeutics, we are committed to bringing mitochondria-targeted therapies to patients for the treatment of both rare and common diseases. We are particularly sensitive to those patients and their families living with one of the more than 200 genetically-inherited mitochondrial diseases with no FDA-approved treatment. To obtain regulatory approval for these investigational drugs, we conduct clinical trials to evaluate their safety and effectiveness. 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.
At this time, we are providing access to investigational drugs only through clinical trials. We believe that participation in clinical trials is the best way for patients to access investigational drugs before they are approved. For more information about these trials, please visit our Clinical page or http://www.clinicaltrials.gov and search “Stealth BioTherapeutics.”
In rare situations when participation in a trial is not possible, patients with life-threatening diseases may seek special access to investigational drugs outside of a clinical trial. These situations are typically referred to as compassionate use, expanded access, or emergency use. Stealth will consider supporting individual expanded access to an investigational drug when all of the following criteria are satisfied:
- The patient suffers from a life-threatening disease, with no alternative therapies available including investigational therapies through clinical trials;
- Clinical data supporting the safety and efficacy of the compound in similar patients permit Stealth to assess the potential benefits and risks, as required by regulatory agencies, for an “individual expanded access” program;
- Sufficient data exist to allow Stealth’s clinical division to identify an appropriate dose and duration of dosing that is considered safe and has the potential of therapeutic benefit;
- Sufficient drug product supply exists to support Stealth’s clinical development plan and any approved expanded access until the drug is commercially available or clinical development has ended;
- The request for “individual expanded access” must be made by the patient’s treating physician and cannot be solicited by Stealth or any other individual or organization;
- Stealth’s clinical division and the patient’s treating physician agree that there is substantial evidence that the patient may reasonably expect to benefit from the investigational drug at the dose and for the duration proposed.
For patients who meet the requirements above, treating physicians can make a request via e-mail to email@example.com.
Stealth will acknowledge the request within three days of receipt and evaluate qualifying requests.
Stealth will consider the above criteria when evaluating “individual expanded access” requests. Stealth does not, however, guarantee that access will be provided and, even if expanded access is offered in some instances, Stealth does not guarantee that the investigational drug will be available for a particular patient. Patients with extraordinary safety risks that, in the judgment of Stealth have not been sufficiently studied, will be excluded. Any access to investigational drugs for expanded access is subject to local/national laws and regulations and must also comply with any applicable country-specific laws and regulations, including drug importation requirements. Approvals from applicable regulatory bodies and by an Institutional Review Board or Ethics Committee from the treating institution must be obtained. If approved, the patient (or his/her guardian) must provide written confirmation of informed consent and both the patient and his/her treating physician must agree to comply with Stealth’s safety and monitoring requirements, including provision of complete patient data prior to receiving approval for expanded access. If clinical trials fail to demonstrate a positive benefit or show unacceptable risk for particular indications or patients, Stealth may elect to end expanded access programs.