A superb essay by Dr. Matthew Davis in JAMA Pediatrics (gated) addresses one of the great shortcomings of modern medical research: despite the attention, money, and glamour associated with curing adult disease, pediatric diseases get nothing even close. Focused on the messaging behind pediatric research, he proposes that pediatric researchers take a new tack—that child health is, ultimately, adult health:
“Children are not small adults” is not working as a rallying cry. A recent comprehensive assessment concluded that “lack of research, poor research, and poorly reported research” greatly limit child health research… Contrary to intentions, “children are not small adults” has led research priority setters to disconnect the needs of children from the predominant national focus on the health and health care needs of adults.
Children are indeed different from adults—but that does not imply that children are not connected to adults. Rather, the exquisitely meaningful links between children and their caregiving family members, and between children and their later adult selves, are keys to converting current child health research challenges into opportunities.
Davis’s paper is provocative and worth a read. Beyond its scope, however, are the systemic causes and consequences of our underinvestment in child health. Messaging plays a role, but there is more to the story:
- Knowledge: Medical education reinforces the myth that kids are simply little adults. At most schools, the only substantive education students get in pediatrics is a required 6-8 week clerkship in their third year. But in years 1-2, when we learn most of the science behind clinical medicine, little to no time is spent explaining how kids’ biology is different. The implication, yet again, is that they work just like adults, which time and time again we find not to be true. Yet it’s not just students with this knowledge gap—the entire establishment seems to suffer from it, too. A lack of research on drug dosages and safety in kids means that the vast majority of drugs prescribed to kids in outpatient offices are prescribed off-label (without an explicit pediatric indication from the FDA). Legislation from 1997 has increased the amount of clinical trials involving kids, but even with that law, pediatric research’s share of NIH funding is still on the decline:
- People: Yes, we’re low on doctors and nurses. But the workforce we do have is stilted away from what kids need, and it shouldn’t be surprising. Entering a pediatric subspecialty means additional training, which means more trouble paying debt, and likely less pay. As a result, pediatric subspecialists are missing in many geographic areas, have long wait times where they are present, and suffer high rates of burnout because of overwhelming demand for their services. The amount of pediatricians entering subspecialties is on the rise, but as with any discussion of the healthcare workforce, it’s all about location—are they entering areas where they’re needed?
- Incentives: The root cause beneath much of this is probably that children just don’t get sickthat often. The chart at right illustrates how kids’ hospitalizations pale in comparison to adults': despite making up almost a quarter of the population, they only use about 10-20% of hospitals’ resources—and when you cut out neonatal care, that drops to a measly 5% (more on that later). It’s certainly a good thing that kids are pretty healthy. But it creates a lack of incentive to develop resources specific to kids, from drugs, to care providers, to devices. I’ve mentioned drugs and providers here already, but the device story also deserves attention: because the market is smaller, companies have lagged in producing devices specific to kids. And many adult devices, like catheters and implants, simply don’t fit children. Recent policy will hopefully make it easier to research and approve new drugs and devices—but without modifying the underlying incentives, will they really close the gap?
Neonatology (intensive care of newborns, especially premature ones) is a well-known exception to most of the arguments I’ve been making–and with 7% of newborns admitted to a neonatal intensive care unit (NICU), the economics make sense. By way of comparison, only 2.7% of children aged 1-17 are hospitalized at all (let alone into intensive care). The promise of neonatal intensive care has improved dramatically—each decade the “limits of viability” allow one more week of prematurity—and with it, so have the economics. NICUs are known to be some of the most profitable components of pediatrics, so it shouldn’t be surprising that neonatology is one of the pediatric subspecialties least affected by shortage (though like the rest of medicine, it’s still maldistributed). There is one important exception to neonatology’s relatively privileged position: the unconscionable shortage of IV nutrition across the US. Since preemies need nutrition supplements more than any other age group, they’ve been most vulnerable to this crisis, and some have died as a result. The reasons for the shortage are complex, but most compelling to me is IV nutrition’s low profitability (since the mixtures are largely off-patent).
So what’s medicine to do?
The knowledge gap is probably one of the trickier problems to fix without taking resources away from other fields. Davis’s arguments on messaging will help with research, but they would do little to change the way doctors are taught. Individual schools modify the preclinical curriculum at their own risk: time not spent covering the topics in the USMLE Step 1 (the licensing exam required to enter years 3-4) is time that jeopardizes the future of those schools’ students—unless there’s a change in what Step 1 tests. Step 1 could change, if we had the will for it. Research funding is probably a similar zero-sum game: offering more funding to pediatric research usually comes with less funding for adult research. But extending patent protection for pediatric drugs, and making clinical trials easier to conduct—as current legislation does—seems to me a no-brainer.
“People policy” is moving in the right direction; Obamacare includes loan repayment programs for pediatric subspecialists who practice in underserved areas. But this policy has not been funded, which means it isn’t happening. We know that debt affects doctors’ practice choices, and that pediatric subspecialty fellowships are typically bad financial decisions—so these policies have a strong basis in evidence (if only they were implemented). Another way to spread the subspecialty workforce better would be to provide more attractive training opportunities where practitioners are needed most. Adult subspecialty fellowships are usually two years long, whereas those in pediatrics are three because they allow some time for research. Yet many say they’d prefer a two-year fellowship without research so that they could get into practice sooner (and presumably pay off those loans). Why not provide those opportunities, in the areas where those doctors are needed most?
Again, economics (specifically financial incentives) are at the heart of why we lack drugs for pediatric diseases. If we want to address this problem, we should address those incentives. A similar problem underlies the reality that we don’t have drugs for neglected diseases that affect the global poor—but many are calling for us to “delink” the development of those drugs from their profit margins. In short, delinkage boils down to the following (though you should really read Brian Till’s full article):
The use of cash prizes — rather than patents — to incentivize research; say, $2 billion for an effective therapeutic drug for Chagas disease. A cure, once developed, proven, and awarded a prize, would then exist as open-access intellectual property, with manufacturers around the world competing to produce the drug in the most cost effective manner. Implementing the idea… is effectively leveraging the power of the free market twice, once to produce the thing you want and then again to manufacture it as economically as possible.
The money’s already out there, in the form of research grants and the income streams that make it to the manufacturer of a successful drug. The idea of delinkage is to make those dollars flow toward where they’re needed most. If we want new agents for diseases in children, why wouldn’t this model wouldn’t work just as well?
As Davis notes, some of the most successful cures ever achieved—vaccines and sanitation—are essentially pediatric interventions that have improved the health of the entire population. It’s myopic to think there’s no more to be done.
Karan is a student at Robert Wood Johnson Medical School and Duke graduate who previously worked in strategic research for hospital executives.