Patients who are seriously ill and have exhausted their treatment options often turn to drugs that have yet to be approved by the U.S. Food and Drug Administration (FDA). These investigational drugs can offer alternatives to patients who have physician approval. However, in the past, the process of providing a patient access to investigational drugs has been long, difficult, and complicated. The FDA has introduced a way to streamline the process in hopes of expanding access to those who qualify.

ONS has worked with the Maryland state legislature to be better understand this process, helping decision-makers recognize the plight of the patient and the role of the FDA has as a regulating agency in healthcare. With this in mind, the FDA has made changes to its own procedural manual and is seeking a streamlined approach to handling these cases. 

Richard A. Moscicki, MD, the FDA’s Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, wrote about the new process. “To make the expanded access process more efficient, we’ve just introduced a much simpler application form, which doctors will fill out when they want to provide an investigational drug for a patient through expanded access,” he said. 

“As part of our commitment to streamlining the expanded access process, the FDA met with stakeholders to explore additional options that might help patients and their physicians understand the process to request access to unapproved drugs. A common theme of the meeting was that navigating the expanded access process really does take a village,” Moscicki noted. “The physician, the drug company, FDA, and the institutional review board (IRB) all have important roles and must work together for the expanded access process to succeed.”

The FDA asserts that “expanded access is designed for seriously ill patients who have exhausted other options. The last thing a patient suffering from a serious or life-threatening condition needs is red tape.”