Researchers suggest that a recent pragmatic trial could have played a key role in curbing the crisis.
While opioids are effective for acute pain relief, the widespread addiction and dependence that have swept up the country have showed that the powerful pills have unintended consequences, even as studies suggest that opioids are less effective for long-term pain than over-the-counter options.
Most medications are approved after undergoing a randomized controlled trial, but a different type of scientific study could have showed the real-world problems with using opioids for chronic pain relief, according to Aaron E. Carroll, a professor of pediatrics at Indiana University School of Medicine who blogs on health research and policy at The Incidental Economist.
“These different kinds of studies actually exist. They are called pragmatic trials, and a recent one might have helped serve as a brake as the opioid epidemic accelerated,” Carroll writes in an essay for The New York Times.
Whereas randomized controlled studies evaluate whether a drug is effective in ideal circumstances, pragmatic studies measure a drug’s effectiveness in the real world.
“A pragmatic trial seeks to determine if, and how, an intervention might work in practice, where decisions are more complicated than in a strictly controlled clinical trial,” Carroll writes.
A randomized controlled study of opioids, for example, would compare whether people taking opioids get more pain relief than those taking a placebo. This is challenging, however, because people who are being treated for pain are desperate for relief, and often change treatments hoping to find one that will work.
“Under these conditions, it’s hard to get patients to participate, and the same with doctors,” Carroll writes.
The Strategies for Prescribing Analgesics Comparative Effectiveness study took a more pragmatic approach to analyzing the effectiveness of pain relief medications, comparing opioids to non-opioid treatment.
Whether a patient was receiving opioid or non-opioid treatment there were options to progress to stronger pain relief options, which helped people stick with the study long-term, rather than dropping out to try other pain relief. Doctors could also change doses and medications within the same class, tailoring treatment to the individual patients.
“That’s how actual care occurs,” Carroll writes. “This way, you can measure how treating someone with opioids might compare with treating someone without opioids for a sustained period.”
The study eventually showed that adverse symptoms were lower for patients treated without opioids, and those patients were also less likely to become dependent.
Although studies like this are important, Carroll writes that they’re unlikely to become mainstream because of their intricacies and expense.
“Although drug companies are willing and ready to pay for randomized controlled trials to prove efficacy, it’s not clear who is going to finance studies like these,” Carroll writes. “They use lots of different drugs—which is what happens in the real world—and no company wants to foot the bill for other companies’ products to be evaluated. Certainly no opioid-related companies would want to pay for this trial.”
