Access to Experimental Drugs for Single-Patient Use Shows Promise in Pediatrics

A recent study, “Use, Safety, and Efficacy of Single-Patient Use of the US Food and Drug Administration Expanded Access Program,” published in JAMA Oncology, has shown that a small but meaningful number of patients appeared to benefit upon accessing experimental therapies through compassionate use—pediatric patients, in particular. The Weill Cornell Medicine Children’s Brain Tumor Project is hopeful that single-patient use will work seamlessly in conjunction when designing patient-specific protocols based on genetic findings.

The federal “Right to Try” law now permits manufacturers to provide experimental therapies to terminally ill patients without FDA authorization. Prior to this bill, the FDA had traditionally granted significant flexibility to physicians pursuing access to experimental products through a mechanism called Expanded Access (otherwise known as “Compassionate Use”).

The Compassionate Use program has two major access routes: singlepatient use (SPU) and intermediate to wide patient population. Generally, the single-patient use mechanism is used to obtain products that may or may not have demonstrated safety or efficacy toward the particular indication requested.

Under the direction of Dr. Jeffrey Greenfield, Noah Feit, MD candidate in the Class of 2020 at Weill Cornell Medical College, dedicated countless hours toward the research effort that leveraged the FDA Expanded Access program to give access to investigational products to patients of all ages with a variety of cancer types. The team reviewed all investigational drugs that were accessed for SPU at Memorial Sloan Kettering Cancer Center over a six-year period. The aim of the project intended to: 1) describe the characteristics of patients for whom this tool is used, and 2) describe outcomes including safety and efficacy associated with use.

Although patients were heavily pretreated, the team observed benefit among the cohort of participants, of whom children represented 34.1% despite representing only ~2% of the patients seen at the center in 2017, suggesting SPUs may provide an important means of pediatric drug access.

SPU has the potential to be of special use to pediatric cancer patients, in whom access to experimental therapies has been particularly limited. The SPU mechanism provides a potentially useful outlet for flexibility for pediatric patients who have exhausted the standard of care and/or who do not qualify for clinical trials.

“My background in public policy and my family’s experience with cancer made this an especially meaningful endeavor for me,” said Noah. “In the era of precision medicine and enhanced connectivity, policy must work to connect people with cures, and the SPU mechanism provides clinicians with a tool to customize cancer care even when clinical trials are out of the question.”

This research was supported by the St. Baldrick’s Foundation Summer Fellowship