'Repurposing' Adult Drugs for Kids Usually Evidence- Based

Pharmacy Professor Presents Research on Medications Used in Children That Have Not Been FDA-Approved for Pediatric Use at Touro College Research Day

April 28, 2015

Drugs approved by the US Food and Drug Administration (FDA) for treating adults are frequently prescribed for different indications in children, but a poster being presented at Touro College Research Day shows that most of the time there's scientific evidence supporting this medication “repurposing.”

Most medications used in children have not been FDA-approved for pediatric use, according to study author Martha Rumore, PharmD, JD, MS, LLM, an associate professor at Touro College of Pharmacy and Fellow of the American Pharmacists Association, so the findings were reassuring. “That was good news, in other words they're not being used experimentally, they're being used because there's some strong or intermediate evidence out there to suggest their use,” she said.

Organized by the Touro Research Collaborative, Touro College Research Day is being held from 11:30 a.m. to 5 p.m. on Tuesday, April 28, at the Touro College of Osteopathic Medicine and the Touro College of Pharmacy campus at 230 West 125th Street in Harlem.

Dr. Rumore decided to conduct the study when she observed, while working at a hospital pharmacy, that clinicians often had questions about off-label medication use in children. “Our clinicians were constantly asking us drug information questions about these strange uses for drugs,” she said.

After sifting through more than 2,000 studies, Dr. Rumore and her colleagues identified 101 medications that were being “repurposed” for children and adolescents. For example, doctors were prescribing the back-pain medication baclofen to treat gastroesophageal reflux disease in kids. Physicians were also treating infantile hemangiomas—the benign growths in babies known as “strawberry marks”--with the hypertension drug propanolol.

Dr. Rumore and her team found strong evidence for the use of 39.6% of the medications, intermediate evidence for 40.6%, and weak evidence for 19.8%. Repurposed medications were rarely used in babies.

Less than 5% of the repurposed medications were first marketed after 2011, Dr. Rumore noted, which is also good news, because there is more information available on potential adverse effects of older medications.

The findings underscore the need for better incentives from the FDA to encourage pharmaceutical companies to conduct studies of medications in children, Dr. Rumore added.

The Touro Research Collaborative, a dedicated group of faculty who pursue research in the medical and health sciences, founded the Research Day—now in its fourth year—to foster collaborations among faculty and students. 

Most medications used in children have not been FDA-approved for pediatric use, according to study author Martha Rumore, PharmD, JD, MS, LLM,  an associate professor at Touro College of Pharmacy and Fellow of the American Pharmacists Association, so the findings were reassuring. “That was good news, in other words they're not being used experimentally, they're being used because there's some strong or intermediate evidence out there to suggest their use,” she said.

Organized by the Touro Research Collaborative, Touro College Research Day is being held from 11:30 a.m. to 5 p.m. on Tuesday, April 28, at the Touro College of Osteopathic Medicine and the Touro College of Pharmacy campus at 230 West 125th Street in Harlem.

Dr. Rumore decided to conduct the study when she observed, while working at a hospital pharmacy, that clinicians often had questions about off-label medication use in children. “Our clinicians were constantly asking us drug information questions about these strange uses for drugs,” she said.

After sifting through more than 2,000 studies, Dr. Rumore and her colleagues identified 101 medications that were being “repurposed” for children and adolescents. For example, doctors were prescribing the back-pain medication baclofen to treat gastroesophageal reflux disease in kids. Physicians were also treating infantile hemangiomas—the benign growths in babies known as “strawberry marks”--with the hypertension drug propanolol.

Dr. Rumore and her team found strong evidence for the use of 39.6% of the medications, intermediate evidence for 40.6%, and weak evidence for 19.8%. Repurposed medications were rarely used in babies.

Less than 5% of the repurposed medications were first marketed after 2011, Dr. Rumore noted, which is also good news, because there is more information available on potential adverse effects of older medications.

The findings underscore the need for better incentives from the FDA to encourage pharmaceutical companies to conduct studies of medications in children, Dr. Rumore added.

The Touro Research Collaborative, a dedicated group of faculty who pursue research in the medical and health sciences, founded the Research Day—now in its fourth year—to foster collaborations among faculty and students.