Expanded Access Policy
Ocugen approaches drug development with a sense of urgency, resolve, ingenuity, and boldness. We consider patients in everything we do. Ocugen believes courageous innovation drives science in new directions and breaking new ground in therapeutic areas encompassing serious medical conditions. Medical research is important to advance healthcare breakthroughs for diseases and conditions that are not addressed by current standards of care. Ocugen is responsible for conducting scientific studies and human clinical trials to evaluate whether investigational drug products (IDP) are safe and effective, with emphasis on safety. All IDPs must pass rigorous US Food and Drug Administration (FDA) standards before they can be tested in humans. The design and conduct of Ocugen’s clinical trials aim to generate valid and accurate scientific data with an overarching goal to protect the rights and welfare of all research participants. Participating in clinical trials is the most common way for individuals with serious medical conditions or life-threatening diseases to seek access to an IDP and may result in receiving the IDP. Whenever possible, an IDP should be used as part of a clinical trial.
ABOUT EXPANDED ACCESS PROGRAMS (EAP)
Mitigating circumstances and situations may exist when enrollment in a clinical trial is not possible (patient ineligibility) or feasible (distance to a trial is too far), that may preclude an individual with a serious condition or life-threatening illness access to an IDP. In cases where an individual is deemed ineligible to participate in a clinical trial, an expanded access program (EAP) (also referred to as compassionate use) presents another option for patients with serious medical conditions and/or life-threatening disease to gain access to IDPs outside of participating in a clinical trial. If both the patient and the patient’s treating physician believe that there is potential benefit(s) that could be derived from the IDP and there is no comparable or satisfactory alternative treatment available to manage that condition, the patient’s treating physician may assist the patient in gaining access to the EAP. IDPs available through EAP do not make claims or assertions that the IDP is safe or efficacious to diagnose, treat or monitor the patient’s serious medical conditions. And while an EAP is not considered a clinical trial, the program must operate within certain parameters because scientific data will be collected. In addition to FDA review of the EAP, an Investigational Review Board (IRB) should review the expanded access protocol and informed consent to ensure the patient is informed about the nature of the treatment.
Under FDA regulations, an IRB is an appropriately constituted group that has been formally designated to review and monitor biomedical research involving human subjects. In accordance with FDA regulations, an IRB has the authority to approve, require modifications in (to secure approval), or disapprove research. IRB review serves an important role in the protection of the rights and welfare of human subjects. The purpose of IRB review is to assure, both in advance and by periodic review, that appropriate steps are taken to protect the rights and welfare of humans participating as subjects in the research. To accomplish this purpose, IRBs use a group process to review research protocols and related materials (e.g., informed consent documents and investigator brochures) to ensure protection of the rights and welfare of human subjects.