Helping Patients Access Investigational Treatments

Cancer Commons, June 29, 2021

For some advanced cancer patients, the best course of action may lie beyond standard treatment options. Some of these patients can enroll in clinical trials that investigate promising new treatments. But others may find their best option is to apply for individual “expanded access” to an investigational treatment.

Here our Curious Dr. George asks two leaders from the Reagan-Udall Foundation for the Food and Drug Administration (FDA) how their organization supports the FDA by striving to facilitate patient access to investigational products:

Curious Dr. George: The goals and process of “Right to Try” legislation have blended with existing policies and practices of expanded access to investigational treatments for patients beyond standard of curative care—especially for those with cancer. How does the Reagan-Udall Foundation for the FDA help physicians better serve their patients’ needs by more easily navigating expanded access, including use of institutional review boards (IRBs) for Investigational New Drugs (IND) for individual patients?

Susan C. Winckler, RPh, Esq. and Lea Ann Browning-McNee, MS, of the Reagan-Udall Foundation for the FDA: Expanded access, sometimes called compassionate use, provides a pathway to investigational treatments not approved by the FDA for patients who have serious or life-threatening illnesses and cannot participate in clinical trials. Single-patient expanded access allows a treating physician to request an investigational product from a pharmaceutical company on behalf of an individual patient. Typically, FDA approves close to 99% of single-patient expanded access requests.