Aaron Carroll had a fabulous piece in yesterday’s New York Times entitled What if a Study Showed Opioids Weren’t Usually Needed?
Participants were randomly assigned to one of two arms. Both involved stepwise progression from less to more potent medications. One arm involved opioid medications (a progression from hydrocodone/acetaminophen to sustained release morphine to fentanyl patches, for example) and the other involved non-opioid medications (a progression from ibuprofen to nortriptyline to tramadol, for example).
The medications were adjusted based on patient preferences and responses. Providers could switch patients to different drugs at the same level; change the dose or frequency of doses; add other drugs to manage side effects; and move patients up or down levels of intensity. They were also allowed to use any nonpharmacological pain therapies they liked.
The results were unexpected:
[pullquote align=”right” cite=”” link=”” color=”” class=”” size=””]It doesn’t matter what works better in theory; it only matters what works better in practice.[/pullquote]
The study followed 240 patients for 12 months. Pain-related function, or how much pain affected their activity, was no different between the two groups. Pain intensity was actually better in the non-opioid group, and adverse symptoms were lower in that group as well.
How can that be? We know that opioids are “stronger” than non-opioids in theory; shouldn’t they perform better is practice?
Not necessarily because there is a difference between explanatory trials and pragmatic trials.
…[M]ost studies, even the gold standard of randomized controlled trials, focus squarely on causality. They are set up to see if a treatment will work in optimal conditions, what scientists call efficacy. They’re “explanatory.”
Efficacy is important. But what we also need are studies that test if a treatment will work in the real world — if they have effectiveness.
These … are called pragmatic trials …
It’s the difference between theory and practice. In theory opioids provide superior pain relief; in practice other medications can actually be more effective and have the additional, major benefit of avoiding opioid addiction.
This does NOT mean that the explanatory studies that showed opioids aren’t stronger than non-opioids were wrong. In the perfect conditions of the explanatory studies, opioids are more effective. But in real world conditions, they have no additional benefit and dramatically increased harms.
The take away message is this: it doesn’t matter what works better in theory; it only matters what works better in practice.
What does this have to do with childbirth and breastfeeding? Quite a lot as it turns out.
There are many explanatory studies of childbirth that claim to show that unmedicated vaginal birth is superior to C-sections. Natural childbirth advocates, midwives in particular, have seized upon these studies to rationalize their preference for unmedicated vaginal birth as an ideal toward which providers and hospitals should aim. The Royal College of Midwives in the UK used such studies to justify their “Campaign for Normal Birth.” The RCM predicted that their campaign would reduce intervention rates, save lives and save money.
That’s not what has happened. Indeed, the results have been disastrous. Maternal and infant health has not improved; preventable infant and maternal deaths have climbed; maternity liability payments have exploded.
Why? Partly this reflects the fact that many of the explanatory studies don’t correct for confounding variables so their results don’t show what their authors claimed. But mostly it reflects the fact that although unmedicated vaginal birth — like opioids — may be better in theory; it’s NOT better in practice.
Similarly, there are quite a few explanatory studies of breastfeeding that claim to show that breastfeeding is superior to formula. Lactation professionals have seized on these studies to rationalize their preference for breastfeeding over formula feeding. The Baby Friendly Hospital Initiative (BFHI) is a campaign to promote breastfeeding. Lactation professionals predicted it would increase breastfeeding rates, save lives and save healthcare dollars.
That’s not what happened. While the BFHI has increased initial breastfeeding rates, the fall off after leaving the hospital is quite dramatic. With the exception of extremely premature infants, it hasn’t been shown to save ANY lives in industrialized countries and certainly hasn’t saved any healthcare dollars on term infants. In fact, literally tens of thousands of babies are readmitted to the hospital each year because of breastfeeding problems (primarily insufficient breastmilk) at a cost of hundreds of millions of dollars
Why? Partly this reflects the fact that many of the explanatory studies of breastfeeding don’t correct for confounding variables. But mostly it reflects the fact that while breastfeeding — like opioids — may be better in theory; it’s not better in practice. Indeed, for some babies exclusive breastfeeding leads to serious health problems, permanent brain injuries and even death.
Where do we go from here?
No doubt drug companies will try to discredit the results of pragmatic trials of opioids and continue to bombard doctors with explanatory trials that show that opioids are stronger. Hopefully, doctors will no longer be swayed by the explanatory trials alone and will demand data demonstrating how opioids perform against non-opioids in the real world.
Similarly, midwives and other natural childbirth advocates completely dismiss the fact that campaigns for normal birth have utterly failed to produce the predicted results. They haven’t met a midwifery scandal resulting in preventable infant and maternal deaths that they don’t lie about, deny, hide and ignore. They comfort themselves and each other with “research” by which they mean explanatory trials. The only question remaining for the rest of us is how many more babies and mothers have to be harmed and die before obstetricians, government officials and public health authorities insist that midwives prove their claims are true in practice, not just in theory.
Lactation professionals behave in exactly the same fashion as midwives and opioid manufacturers. They dismiss the fact that the BFHI and other efforts to promote breastfeeding have utterly failed to produce the predicted results. When confronted with data that the benefits of breastfeeding in industrialized countries are trivial, that no term babies lives have been saved and no healthcare dollars have been saved, they wave explanatory studies that demonstrate the theoretical benefits of breastfeeding. The only question remaining for the rest of us is how many more babies and mothers have to be harmed or even die before pediatricians, obstetricians, government officials and public health authorities insist that lactation professionals prove their claims are true in practice, not just in theory.
As Carroll notes:
Randomized controlled trials are great for certain things. They absolutely have their place in determining efficacy and causality. But sometimes pragmatic trials are better. If we want to see improvements in care in the real world, not just the lab, we may need to push for more of them.
That applies to opioids and it applies equally to efforts to promote unmedicated vaginal birth and breastfeeding.
