Study Shows Variations in RWE Citation Frequency

Although clinical evidence plans seldom cite real world evidence (RWE), plans that do vary in frequency and types of RWE cited, according to study results presented at the Virtual ISPOR 2020, the annual meeting of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).

“We found that while all health plans reported reviewing some RWE, some plans have embraced RWE more than others,” said investigators in the report.

RWE is generated from data that is collected outside of controlled clinical trials; it typically comes from electronic health records and can cover case reports, open-label extension studies, cohort studies, and insurance claim analyses.

“There's a lot of variability in how people are defining [RWE]….For the most part, we were looking at retrospective and prospective studies that were performed in an observational format,” said presenting author Ari Panzer, a research associate at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center.

Frequency of RWE Citations

Of the 5227 clinical evidence plans observed, 16% cited RWE. Health plans also varied in frequency of RWE citations, ranging from 5% to 31% of the cited clinical evidence.

Variation in types of RWE cited were found as well, with 26% being from medical records, 18% from case studies, and 17% from case reports. Type frequency also varied from plan to plan.

Investigators found that RWE citations increased after the 7-year mark from FDA approval; the longer a drug is on the market, the more RWE is available. Plans cited 3.7 RWE studies for drugs after receiving FDA approval 10 years ago, compared to 0.1 cited RWE studies after 1 year.

“We're hoping to examine the evidence base of a product, and how it evolves throughout its lifecycle. So, as it gets further from approval and more research, real world evidence, and evidence synthesis is generated, how are health plans using this information and how does this base evolve,” said Panzer.

Variation in Disease Types

Plans cited RWE studies more often for certain diseases than others, with most frequent RWE citations applying to gastroenterological (35%) and infectious diseases (26%). Respiratory (11%) and oncological (12%) diseases had RWE studies cited the least.

Additionally, certain RWE types varied for disease categories. Medical records were more commonly cited for oncological, neurological, gastroenterological, infectious, and respiratory diseases. For ophthalmic and dermatologic diseases, case studies and case reports were more common, respectively.

“This may make sense given that certain types of studies may be easier to do with different diseases. So, it maybe conducive to producing certain types of [RWE] for different diseases and therefore, plans are citing those more because that's what's available, but it's still important I think, to note its difference across disease areas,” said Panzer.

RWE Influence on Decision Making

RWE gives insight into how a drug performs in a real world setting, however, how RWE influences payer decision making on specialty drug coverage policies is still unknown.

“That’s a question of the quality of the evidence and of what health plans value in terms of informing their decision making and some health plans may value 1 type of study versus another,” said Panzer.

“While some studies have surveyed and interviewed health plan officials to determine how they use RWE, research that illuminates how RWE can best inform decision making is necessary,” said investigators in the report.

Reference

Panzer A, Margaretos N, Lai R, Enright DE. Do health plans cite real-world evidence in their specialty drug coverage policies? An empirical analysis [published online March 25, 2020]. Wiley. doi: 10.1002/pds.4992.