The panel discussed the various aspects of obtaining a drug such as uridine triacetate through the expanded access program.
Ms. Vogel: The expanded access program allows companies to provide patients access to their medicines while the drugs are still in preapproval process, but it is strictly regulated. Do you see uridine triacetate being a viable option for patients through this program?
Dr. Campen: Yes. This is the perfect example of a drug being available through an expanded access program.
Dr. Schwartzberg: There are some regulatory hoops to jump through, and I would presume that the patient would provide signed informed consent. Patients should be made to understand the risks and potential benefits of taking the drug.
Dr. Campen: That would also entail costs, including the manufacturer’s cost of preparing the drug, the institution’s costs of administering the drug, and any expenses surrounding that. So cost is a factor to consider. Some types of insurance, not all, will pay for the cost associated with treatment with uridine triacetate.
Ms. Vogel: I would imagine quick delivery of the drug would be vital in many cases.
Dr. Campen: Exactly. Rapid delivery is key.
Ms. Vogel: Can you elaborate on your institution’s protocol to obtain a drug via the expanded access program?
Dr. Schwartzberg: We are familiar with it; and to obtain the drug, we work closely with our research nurses, who participate in this process because they are used to the program’s regulations.
Dr. Campen: We have protocols set in place in our institution, especially because most of the time, we need quick access to the drug (that day or within the next 24 hours).
