Why Senator Markey’s Message Hurts Children

Last week, Senator Edward Markey (D-MA) placed a hold on the Senate’s nomination of Robert Califf’s as head of the Food and Drug Administration (FDA). The move was less against Califf and more as political leverage against FDA’s approval of OxyContin. In August 2015, FDA approved OxyContin, a prescription painkiller, for pediatric patients ages 11 to 17. OxyContin is the painkiller most associated with United State’s prescription drug abuse epidemic, accounting for an increase in drug overdose and death over the last decade. FDA’s approval of OxyContin for children drew concern from Markey and others that the approval would lead to an increase in drug misuse for children and their family members. Markey, who has prioritized the fight against opioid addiction in his legislative agenda, hopes he can use the hold to convince FDA to reverse its August decision.

Senator Markey’s message is well intentioned, but may ultimately do more harm than good for children.

Before FDA approved OxyContin in August, children who suffered from severe, chronic pain due to cancer, extensive trauma, or serious surgeries had few drugs approved to treat their pain. Many physicians treating severe pain in children prescribed OxyContin off-label, without proof that OxyContin could safely and effectively treat children. These physicians often relied on their experience or intuition to not under or overprescribe the drug. FDA’s approval in August meant the pharmaceutical manufacturer finally provided physicians with instructions, backed by controlled studies, explaining how physicians could safely use OxyContin to treat children with severe pain.

Unfortunately, OxyContin was not the only drug used off-label in children. Studies show that 50% of drugs used by children in a hospital setting are prescribed off-label. Approximately 80% of children in pediatric hospitals receive at least one non-approved or off-label drug. The off-label problem also extends to adolescents who need to be treated for substance abuse disorders. As Senator Markey has stated, over 2 million children 12-17 have reported using an illicit substance. However, few studies have examined the safety and efficacy of substance abuse treatments for adolescents, and no substance abuse treatments have been approved for children under the age of 16.

These numbers are dangerous. Studies show that off-label drug use lacking strong scientific evidence produced two times higher adverse event rates than adverse event rates for on-label drug use. Off-label medicines used in children were more likely to be implicated in an adverse event than authorized medicines used in children.

Fortunately, the Best Pharmaceuticals for Children Act (BPCA) has helped to decrease the evidence gap for pediatric medicines by providing financial incentives to companies that study drugs in pediatric patients. Off-label use in pediatric settings remains high because historically, pharmaceutical companies have lacked incentives to test drugs in pediatric patients. Pediatric subjects can be difficult to identify, and raise additional ethical concerns not present for adults. Through the BPCA, drug companies have conducted clinical trials on over two hundred drugs, including OxyContin, to determine how these drugs can be used safely and effectively in children.

Markey’s call to withdraw approval of OxyContin for pediatric use will do little to prevent children from using OxyContin. Markey’s restriction would instead perpetuate the off-label prescription problem in children, depriving pediatric physicians of the evidence they need to ensure that children with severe, chronic pain can safely use OxyContin and other medications. If Markey wants to effectively fight opioid misuse in children, he has other options, including placing restrictions on how pharmaceutical companies can market OxyContin or by restricting how physicians can prescribe OxyContin to children. Markey’s call to withdraw FDA approval for OxyContin use in children would only exacerbate the evidence gap for pediatric drugs and ultimately, harm the children he intends to help.


2015-2016 Peter Barton Hutt Student Fellow During her fellowship year, Elizabeth Guo was a third year student in the JD/MPH Program at Harvard Law School and the Harvard School of Public Health. Her interests lie at the intersection of law, health care reimbursement, and biopharmaceutical regulation. Elizabeth graduated from Harvard University with a BA in social studies, focusing on the bioethics of assisted reproduction in China. Prior to law school, she was a senior associate at Avalere Health working with life science clients on a range of health policy topics, including Medicare coverage and reimbursement, biosimilar regulation, and healthcare quality programs. Elizabeth served as a student editor of the Journal of Law and Technology and a member of the Mississippi Delta Project’s Delta Project’s Food Policy Initiative. Elizabeth's fellowship paper was entitled: "Reputation and Judicial Deference: An Examination of FDA’s Reputation and Judicial Deference to Agency Action."

One thought to “Why Senator Markey’s Message Hurts Children”

  1. Thanks, Liz. This is an interesting example, though, because even if FDA now withdrew the approval for this population, the evidence that has been generated (incentivized by the BPCA) about how to use OxyContin safely and effectively in certain pediatric populations wouldn’t disappear. So wouldn’t doctors still have what they really need – making this different from other off-label uses where evidence is actually missing?

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