The costs to patients are high when trials are carried out under the US Food and Drug Administration’s (FDA) pediatric exclusivity program, according to research results published in JAMA Internal Medicine.

Researchers identified 48 drugs that received the pediatric exclusivity incentive between 2007 and 2012. In their analysis, they considered labeling changes from the pediatric studies, labeling details, and industry estimates of trial costs per patient. They also looked at the net return and consumer cost accrued during the 6-month exclusivity period.

The 141 FDA pediatric exclusivity trials included in the study showed that 20,240 children had participated. There were 29 extended indications, 3 new indications, and new safety information for 16 drugs. The mean investment cost for these trials was $36.4 million. Of the 48 drugs, average return cost was $176 million with a median ratio of net return to cost of investment equaling 680%. Net return was positive for 42 of the 48 drugs included in the study.

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Researchers concluded, “Clinical trials conducted under the US Food and Drug Administration’s pediatric exclusivity program have provided important information about the effectiveness and safety of drugs used in children. The costs to consumers have been high, exceeding the estimated costs of investment for conducting the trials.” They suggested policymakers directly fund such studies to offset the costs to consumers.  

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Disclosures: One author reports affiliations with the FDA Office of Generic Drugs and Division of Health Communication.


Sinha MS, Najafzadeh M, Rajasingh EK, Love J, Kesselheim AS. Labeling changes and costs for clinical trials performed under the US Food and Drug Administration pediatric exclusivity extension, 2007 to 2012 [published online September 24, 2018]. JAMA Intern Med. doi: 10.1001/jamainternmed.2018.3933

This article originally appeared on Medical Bag