Expanded Access Policy
ACCESS TO INVESTIGATIONAL MEDICINES PRIOR TO REGULATORY APPROVAL
Retrotope, Inc. has created a new category of drug platform with the potential to treat a wide variety of mitochondrial myopathies and neurodegenerative diseases. Retrotope believes that clinical trials are necessary for (1) developing new medicines, (2) understanding effectiveness and safety, and (3) obtaining regulatory approval for a medicine. While patients in clinical trials are required to meet pre-defined inclusion criteria, some patients might not meet the requirements or are otherwise unable (e.g., geographically unable to access a study site) to enter ongoing clinical trials. These patients may be able to receive the drug under an expanded access protocol.
Retrotope may consider expanded access requests if the following factors are met:
- Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition,
- There is no comparable or satisfactory alternative therapy to treat the disease or condition,
- Patient enrollment in a clinical trial is not possible,
- Potential patient benefit justifies the potential risks of treatment, and
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
Retrotope requires that a health care provider (HCP), meeting the following requirements, makes the expanded access request on behalf of their patient. The HCP must:
- Be medically qualified and licensed
- Adhere to the treatment protocol
- Comply with safety monitoring and reporting requirements
- Obtain relevant health authority (e.g., U.S. Food and Drug Administration) and IRB approvals
- Comply with all applicable laws and regulations.
A licensed physician must submit the request for compassionate use in writing. The request must contain, at a minimum, the following information:
- Demographics of the proposed compassionate use patient
- Relevant medical history, including all therapies administered to date for the relevant indication
- Assessment of the patient’s prognosis
- Reason for requesting compassionate use
- Proposed treatment plan under compassionate use
- Statement that the physician (and the associated institution, if relevant) is able and willing to prepare and submit to the FDA the required regulatory documents to obtain a treatment IND for the patient
- Statement that the physician (and the associated institution, if relevant) will obtain informed consent from the patient and approval of the appropriate IRB prior to initiating treatment
Patients for whom a request is made must meet the following criteria:
- Provide informed consent to the known and unknown risks of the investigational drug
- Comply with the prescribed treatment and associated visits
- Comply with safety and monitoring requirements.
Retrotope will consider expanded access request from an HCP based on these criteria; however, Retrotope cannot guarantee that Expanded Access will be made available to a patient. All requests will be evaluated in a fair and unbiased manner. HCPs may request expanded access by contacting Retrotope through Sarah Endemann (firstname.lastname@example.org). Acknowledgement of the expanded access request will occur within 7 days. Additional information may be requested to assist with patient eligibility. Information submitted will be maintained according to HIPAA Privacy Rule and used solely for evaluating a patient’s eligibility for expanded access.