In the U.S., multiple legislative changes have been improving the information available for off-label use of medications (JPGN 2013; 56: 113-4).
The Pediatric Research Equity Act (PL 108-155) and the Best Pharmaceuticals for Children Act (PL 107-109) help the Food and Drug Administration (FDA) promote pediatric trials and better information.
- Pediatric data must be included in labeling whether results are positive, negative or inconclusive
- If pediatric trials are not completed because the drug would be ineffective or unsafe, this information must go into labeling
- Public internet posting of certain FDA pediatric reviews is required regardless of whether the trials led to an approval
Despite these improvements, the data may be difficult to interpret. The authors of this commentary note that only “48% of trials of products that had pediatric safety information added to the product’s labeling were reported in the peer-reviewed literature.” Furthermore, in about half of the published articles, the studies did not emphasize the same information as the FDA labeling.
Even when a fair amount of data is provided, it is frequently ignored. A specific example given by the authors: lansoprazole. Lansoprazole is not approved in patients younger than 1 year and labeling describes a negative trial in this age group. Yet, in the U.S., lansoprazole was dispensed off-label in this population approximately 358,000 times in 2010.
Pharmaceutical companies benefit whether the prescription is for an approved indication or for an off-label use. Even with approved uses, there have been substantial concerns about adverse effects. The likelihood of more risk and less benefit is much greater for off-label use.
So with the next prescription, it might be worth thinking about whether the medication works, whether there is sufficient data to support an approved indication, and how much risk is involved.
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