Immedica is committed to satisfying unmet medical needs and enabling access to safe and effective medicines to patients. Clinical development is a crucial part of our company’s mission to provide innovative medicines, particularly those that treat rare diseases, to patients with unmet medical needs. For medicines that are not approved by the authorities like the U.S. Food and Drug Administration, we believe that participating in a clinical trial is the best path for patients to access the medicine and ensure patient-minded guardrails are in place. However, we recognize this avenue may not be feasible for every patient and that patients and healthcare professionals may ask us to provide special access to investigational medicine. In the United States, these situations are typically referred to as expanded access.
As part of our commitment to supporting patients in need, we may provide unapproved medicines via expanded access in the United States. In general, we are not able to provide expanded access to approved or unapproved medicine if it is for a disease or condition that we have not studied or to an approved marketed medicine for an indication that has not been approved in the medicine’s label.
When providing expanded access, we will consider many factors, including:
- Seriousness of the Disease: The underlying disease or condition that the unapproved medicine treats is seriously debilitating or life threatening, and a comparable or satisfactory alternative does not exist for the patient.
- Safety and Efficacy of Medicine: The unapproved medicine must, in the opinion of the treating healthcare professional, be safe and effective for its intended use and the treatment of the patient. Specifically, there must be sufficient safety and efficacy data for the treating healthcare professional to determine that the benefits outweigh the risks of using an unapproved medicine.
- Available Clinical Data: Clinical data, preferably from Phase 3 clinical trials, must be available for the proposed use of the unapproved medicine.
- Regulatory Pathway and Supply: We will evaluate whether the program will interfere with the initiation, conduct or completion of clinical studies, including our ability to maintain supply for the clinical trials, as such interference could impact our ability to ultimately obtain approval for the medicine and provide a treatment option to a broader patient community in need.
In the event we decide to consider expanded access use in the future, we will evaluate and respond to each expanded access request on a case-by-case basis in a fair, impartial and equitable manner relying on the general criteria set forth below. Please refer to www.clinicaltrials.gov for more information.
- Treating Healthcare Professional’s Request: A licensed treating healthcare professional must submit the request for expanded access to the unapproved medicine on behalf of the patient. Please contact us directly at [email protected]
- Patient’s Health and Safety: A licensed treating healthcare professional must determine, based on the safety and efficacy data from clinical studies and the patient’s medical history, that the benefit of the unapproved medicine to the patient outweighs the risk.
- No Comparable or Satisfactory Alternative: The licensed treating healthcare professional submitting the request must have made a determination that the patient has no access to a comparable or satisfactory alternative therapy to diagnose, monitor or treat the underlying disease or condition.
While we endeavor to help patients in medical need, please know that we cannot guarantee access to our unapproved medicines, and even if we do decide to support expanded access, certain circumstances may arise that will prevent us from providing access to an unapproved medicine.
How do I learn more about Expanded Access to an investigational product?
- Patients should work directly with their treating healthcare professional
- Healthcare professionals can make a request on the patient’s behalf by contacting [email protected]
