Efficacy vs. effectiveness: Registry data key to which drugs are useful in real world
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The key to better understanding the efficacy vs. effectiveness gap in drug therapies could lie in real-world data and patient registries, according to a speaker at the FDA & American College of Rheumatology Virtual Summit.
“I think when people are either establishing registries, or they have ongoing registries, exploring ways to build in biobanks, it is going to be increasingly critical, as there is likely not going to be any single better treatment option for all patients, so we need further information to help us,” Kimme Hyrich, MD, PhD, a professor at the University of Manchester, in the United Kingdom, told attendees.
Physicians in general have a habit of overestimating the real-world effectiveness of drugs, Hyrich added.
“I remember when biologics were first introduced, and I was told that we might not even have a need for rheumatologists in 5, 10 years,” she said.
Despite the differences between randomized-clinical-trial and real-world data, the goal is always for patients to respond well to treatment, according to Hyrich. Some of the factors that may account for the differences between real-world data and trials include different patient demographics and disease indications.
“Where I feel registry data have a strong role, is they can help us understand the efficacy-effectiveness gap, and through doing this, and understanding where there are differences, they can teach us more about the drugs we are using and they can improve the way we use them,” Hyrich said.
Although trials answer questions of efficacy for a single or group of indications, registries can ask more specific questions, like asking which type of drug will be more effective in a given patient population, she added.
“What do we do with these patients with highly refractory disease, for which there is less and less clinical trial data?” Hyrich said. “These are situations where we may have to only rely on registry data to help us have more information.”
According to Hyrich, whereas clinical trials show how a treatment performs ideally, registries are able to offer a glimpse at the more realistic performances that can be expected, including examinations of the drug across different disease states and in combination with different therapies.
“When we look at registries, I think these do offer a lot further insight into what can be the best choice of therapy, and in some cases, what may be the only source of data for certain treatment comparisons,” she said.
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Hyrich K. What can we learn from registries about treatment efficacy and effectiveness? Presented at: The FDA & American College of Rheumatology Virtual Summit; May 17-18, 2022.
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