- The Institute for Clinical and Economic Review, a drug price watchdog, said Monday it has partnered with health data company Aetion to add so-called real-world evidence to its reviews of some new drugs as well as periodic re-analyses of marketed medicines.
- The announcement comes weeks after ICER said it would use such evidence, which pharma companies argue can show the value of their products beyond data from the randomized, placebo-controlled studies that are the gold standard for regulatory approval.
- Use of real-world evidence has stirred controversy in health policy circles, however, as many argue such data can exaggerate the benefit of a new treatment due to uncontrolled variables and lack of placebo comparisons.
ICER's value assessments of new drugs are frequently criticized by biopharma companies, which in many cases argue the group's reviews don't capture enough data about how medicines benefit patients. The non-profit organization has been making adjustments to how it prepares its assessments, and using real-world evidence represents a big step forward.
New York-based Aetion has both high-powered backers as well as big customers. Sanofi recently announced a partnership with Aetion, and New Jersey insurer Horizon is relying on Aetion's data platform to analyze healthcare utilization by patients with diabetes.
Meanwhile, former Food and Drug Administration Commissioner Scott Gottlieb recently joined the Aetion board, and the company has received financial backing from the venture capital firm founded by Andy Slavitt, the former acting administrator of the Centers for Medicare and Medicaid Services.
Aetion's analyses of real-world data would be used to review new drugs when data on comparator treatments or the natural course of a disease are needed to calculate the value of a new medicine, ICER spokesman David Whitrap said in an email.
For drugs receiving FDA accelerated approval based on single-arm trials and biological activity, re-analyzing an initial ICER assessment using real-world evidence can determine whether the benefit has been as good as expected at approval, he said.
"Randomized clinical trials remain the gold standard for developing unbiased evidence, but as the FDA is increasingly approving treatments based on thinner [trial] data, the entire health industry will need to fill larger knowledge gaps about how well a new drug actually works," he said.
"Real-world data reflects settings and populations not represented in clinical trials," Aetion CEO Carolyn Magill said in an interview with BioPharma Dive. By analyzing that data, regulators and insurers can make decisions about dosing or use in additional indications and patients.
ICER will continue to seek data from other partners, Whitrap said, although he commended Aetion for both the breadth of its data as well as a "mindset" that takes into account the strengths and weaknesses of real-world evidence.
Real-world evidence usually refers to information derived from analyses of data gathered from "real-world" sources like electronic health records or drug registries, rather than controlled clinical studies.
"We want to be as transparent as we can be as to when it is a no-brainer to use real-world data," Magill said, "as we are when the data is not there yet, or the endpoint isn't correct, or we can't adjust for confounding variables."
Real-world evidence's weaknesses were outlined in a recent New England Journal of Medicine article written by University of Oxford researchers.
"Because of the potential biases inherent in observational studies, such studies cannot generally be trusted when — as is often the case — the effects of the treatment of interest are actually null or only moderate," the researchers wrote.
"In those circumstances, large observational studies may yield misleading associations of a treatment with health outcomes that are statistically significant but noncausal, or that are mistakenly null when the treatment really does have clinically important effects," they added.