Are Our Babies Guinea Pigs? Unapproved Drugs And Newborns

As the mother of a premature baby born at just 31 weeks, I’m incredibly thankful for modern medicine.

From the neonatologists to the pharmacists, and obviously the nurses, so many had a hand in ensuring my daughter not only survived, but thrived.

Her first few days were filled with oxygen, fluids and medications. The last, medication, brings a feeling of thankfulness they were available and also thankfulness that she didn’t suffer any ill effects from them.

Are Our Babies Guinea Pigs? Unapproved Drugs And Newborns

It may come as a surprise, but an estimated 90% of medications administered to neonates aren’t FDA approved for use in neonates. Neonates meaning premature babies and full-term babies less than 28 days old.

Hearing such a statistic can make a parent wonder if their vulnerable child is being treated as guinea pig.

It’s important to note, though, that not being FDA approved for use in neonates doesn’t necessarily mean a drug is unsafe. It means there haven’t been adequate studies showing appropriate dosing, safety and effectiveness. Nevertheless, it’s still worrisome.

Are Newborns Really Guinea Pigs?

When we think of medical studies, we often think of scary testing. The reality is that some testing is necessary to prove the safety and effectiveness of medications. However, it’s nothing like the dramatised mad scientist lab we might see on TV.

In actuality, newborns are rarely, if ever, involved in medical trials. A parent’s worry about their medically fragile child being a guinea pig is the same worry that’s behind few drugs being tested and approved for use in neonates.

Few parents are comfortable with their baby being involved in a drug trial. And few medical providers and pharmaceutical companies are comfortable with the ethics and liability involved in trialling medicine in such young infants.

This creates a bit of a dilemma where few medications are approved but medically fragile infants require medications.

Is Anything Being Done To Get Drugs FDA Approved For Neonates?

Dr. Jonathan Davis, Tufts Medical Center’s chief of newborn medicine, says some infants admitted to the NICU can receive as many as 60 medications in their first month of life.

While off-label medication use in general isn’t uncommon, no other area of medicine seems to have such a lack of medication research available as neonatology.

Dr. Davis heads one of two global initiatives started in 2015 aimed at reducing this lack of medication research for neonates.

He heads the International Neonatal Consortium which recently published a contribution to neonatal medicine encouraging adequate drug trials for neonates.

Is It Safe To Conduct Trials On Neonates?

In a perfect world, newborns would never be subjected to medications. In reality, around 10% of babies in the US are born prematurely, there’s a general NICU admission rate of over 8% of full-term babies, and there are also neonates born with a number congenital conditions which require treatment. Many of these babies will require at least some medication.

For these medically fragile infants, the benefit of medications generally outweighs potential risks as they’re used for lifesaving treatment.

While nothing is without risk, Dr. Davis points out that our current system isn’t safer than not conducting approved trials. He said: “We can’t be certain which drugs, in which doses, to use when.” As it stands, each neonate receiving medication is essentially involved in an uncontrolled, unapproved drug trial of one without collecting the data to improve the treatment of other neonates.

The director of pediatric oncology drug development at Swiss pharmaceutical company Roche, Dr. Raphaël Rousseau, said: “Children are protected through research.”

Avoiding research in children, especially neonates, likely has more risk than avoiding trials all together.

Are Babies Receiving Unsafe Medication In The NICU?

Chances are, if you had a baby in the NICU, no one explained to you that 90% of their potential medication is unapproved for use in your baby’s situation. Why? Because in nearly every situation where medication is being used in a neonate, other options aren’t likely to exist.

While most medications aren’t FDA approved for use in neonates, doctors are still making informed and educated decisions. The reason there’s a push for more clinical trials and FDA approval is to increase the amount of information and evidence available to neonatologists so they don’t have to rely solely on anecdotes and intuition.

Neonatologist Dr. Matthew Laughon of the University of North Carolina at Chapel Hill said: “When you’re sitting there at the bedside, and you’re looking the parents in the eyes, and the baby is dying or really sick, to just stand there and say, ‘We don’t have anything to do because it hasn’t been proven’ —well, that’s challenging. If we never gave drugs because they were off-label, we wouldn’t have any drugs.”

Having no drugs available for neonates would be devastating for many.

When it comes to dosing, while clinical trials are rarely available for doctors to decide doses, they do their best to make informed decisions. Dr. Davis said: “We take it right out of the vial of an adult drug, dilute it down, and give it to the babies.” He acknowledges there were situations where this method led to unfortunate outcomes, and that’s precisely the reason more research is needed.

Catherine Sherwin, division chief of pediatric clinical pharmacology at the University of Utah School of Medicine, studies pharmacology in neonates and shared why the above method carries risks. We know that infants metabolise drugs differently than adults. She said: “Yet we haven’t done the studies to know exactly what those differences are. We just know they’re different.”

What Does This Mean For My NICU Baby?

Having a baby in the NICU is an incredibly stressful and scary experience. Hearing about the lack of evidence available for neonatologists can be a scary thing to hear.

It’s important to remember that when a doctor prescribes something for such a fragile infant, they truly believes the benefit of the medication outweighs possible risks. They are using their experience and any available information, as well as working with a pharmacist.

It’s also important to remember that lack of approval doesn’t necessarily mean lack of safety. It means a lack of evidence. There’s hope that the two initiatives will lead to more research.

In the meantime, remember that using off-label medication in the NICU has saved countless lives and could eventually lead to drug approval.

Surfactant, one of only two drugs approved for neonates in the last 25 years, was a major advancement in helping neonates with respiratory distress. It was one of the first medications my daughter received after birth and contributed to her not only surviving, but thriving.

See just how amazing premature babies can be in this Photo Series That Celebrates The Strength Of Premature Babies.