Two FDA panels Thursday recommended that the US Food and Drug Administration approve Pfizer’s novel infant drug, Neprosyn for treatment of urinary tract infections. FDA staff had expressed concerns about the safety of the drug in children, even as it recommended it be approved for use by patients aged six months to two years.
In its report, the FDA staff cautioned that at the current dose, the drug could cause adverse effects for babies and very young infants (Neprosyn treats serious infections in the upper urinary tract). The report warned of a “real chance of serious adverse events that are more severe or permanent in infants”, and urged the FDA to consider a suspension of sales in babies and young children, pending additional safety data.
The FDA staff expressed similar concerns in their meeting briefing documents sent to all FDA advisory committees on Thursday, but from the moment they made their recommendations, experts from the two FDA advisory groups were in agreement on the drug’s safety. Both groups recommended approval for approval based on the same evidence: a New England Journal of Medicine study that included 362 children aged three months to two years. The study showed that Neprosyn was both safe and effective, with no significant side effects. The group of experts also included the chief medical officer for the Fenway Health Institute, which works on preventing and treating infections. According to the FDA’s approval action schedule for this issue, Pfizer has five business days to issue a letter to the FDA giving the go-ahead to use the drug for pediatric use.
All of this means that whatever other issues, however small, may arise with adult use, there is a path for parents to get information on this drug before they get their babies on it. And there’s even the argument that using the drug earlier may be more beneficial for children. A 2001 review found a quarter of the pediatric I.V. infections caused by fungal infections are complicated by pneumonia, which needs immediate medical attention. The researchers found that cephalosporin antibiotic treatment given to children was recommended for “immediate intervention”.
But with other babies and children not reported as having bacterial infections by the FDA in most reviews of drugs for infants and toddlers, parents will likely wonder how safe the children’s drug might be even before children have the disease.
Scientists are working to replace fear of the word “adverse” with gratitude for our knowledge. And companies are still trying to remove science from that word. Despite the need for objectivity to assess the validity of an intervention, most parents are simply more grateful for the routine tools and services that can ease the anxiety of a potentially fragile newborn, and for the relief that would come if more people could do what their bodies and genes would otherwise prevent. We all know parents who have faced a crowded emergency room after their babies stopped breathing while being sedated for surgery. We know the parent who needed to be restrained during childbirth because he couldn’t follow the procedure’s directions. We know the parents whose kids got pneumonia for which antibiotics were not an appropriate intervention.
Even doctors sometimes don’t fully appreciate the benefits of common devices or treatments for their patients. We have often joked that the best place for a doctor to ask a question is in their own bed. While doctors are reasonably happy to be looked upon as professionals, they tend to feel a fair amount of guilt when they realize their treatments affect others, even if it’s just children. There are undoubtedly plenty of times when physicians aren’t using their best judgment because they were afraid that other people would think of them as inadequate. But those times are rarer than we tend to acknowledge.
There is really no way to ensure that medicines are safe or effective for everyone, and there are still many medications that could cause harm to infants and toddlers if improperly used. But there are good reasons to think that newly approved and fully tested medicines for children are more likely to be safe and effective than their older cousins. Surely the knowledge that Pfizer can market a children’s medicine to parents at either end of the spectrum – hard-working parents desperate to help their sick children, or careless parents expecting to do so – is reassuring.