Real-World Evidence in Oncology Must Be Timely for Use in Payer Decisions

In a panel discussion at the Academy of Managed Care Pharmacy Nexus 2020 meeting, experts discussed driving factors of and barriers to the use of real-world evidence (RWE) by payers in oncology decision-making.

Daniel Malone, RPh, PhD, professor in the Department of Pharmacotherapy at the University of Utah, set the stage with a discussion of what RWE is and how it can be used to make decisions. RWE, which comes from sources outside the typical clinical trial setting, can be gathered from the electronic health record (EHR), pharmacy and medical claims, and disease registries. Unlike experimental studies, observational studies are not randomized, cannot prove causation, and may be influenced by selection bias, but they have the advantage of high generalizability to real-world settings.

The FDA is beginning to recognize the value of RWE, as it has released a framework for use of RWE to inform regulatory decisions that looks at whether the data are fit for use, the study design provides adequate evidence, and the conduct of the study meets FDA’s requirements on study monitoring and data collection, which Malone referred to as the toughest barrier to clear.

Beyond the FDA, some payers are starting to use RWE in their coverage decisions, Malone said, citing a study he coauthored that found that 77% of payers surveyed said that observational studies sometimes play a role in their organization’s medication use policies. These payers most commonly used RWE for utilization management decisions and individual appeals, and less often used it for formulary placement or tiering. The most common data source used in their internal analyses was pharmacy claims, but Malone predicted that use of the EHR will continue to evolve as these systems become more facile.

Perceived barriers to the use of RWE in decision-making included organizations’ lack of experience in interpreting and conducting such studies, as well as that the data are not always timely enough to be used in decisions. Still, he sees an important opportunity for RWE to help answer the most relevant questions to health care today.

“It reflects the fact that you have an ongoing firehose of information coming out and products you have to evaluate, and you don’t necessarily have the time to go out and collect real-world data within your own organization or to go look for those studies in the literature,” Malone said.

This challenge is especially relevant in oncology, according to Diana Brixner, RPh, PhD, executive director of the University of Utah’s Pharmacotherapy Outcomes Research Center, who explained that most new oncology therapies are approved through expedited review, making RWE more valuable for payers because limited clinical trial data are available. The high cost of oncology drugs may also contribute to a different role of RWE in this area.

Brixner highlighted key results from her report on a national survey on US payers’ use of RWE, including economic models and patient-reported outcomes (PROs), in oncology decision-making. The diverse sample of respondents reported high interest in RWE, which Brixner called encouraging but not surprising, and they perceived RWE as especially key for providing data on comparative effectiveness because there are usually no head-to-head clinical trials available when a product launches after expedited review. RWE is also used to inform and validate clinical pathways like the National Comprehensive Cancer Network guidelines, and 42% of payers said they were likely to make off-label formulary decisions based on RWE.

The survey respondents reported high utilization of economic models, especially cost-effectiveness models, which are important in oncology, where high-cost therapies are used in small subpopulations. However, some said they did not review economic models at all because the models were not available at the time of decision-making.

PROs are also considered more often in oncology, Brixner said, because of the need to understand patients’ quality of life, which can eventually drive outcomes like resource utilization and cost of care. PROs can be helpful in shared decision-making and encouraging patient-physician dialogues.

The main takeaways of the study include that “early, prelaunch information helps with the initial decision and was highly encouraged, and postlaunch evidence then helps validate decisions that have been made and how future decisions will go forward,” Brixner said. Payers welcome early involvement with manufacturers to ensure that RWE can be included in early decision-making and to develop and validate the economic models most relevant to their needs.

Christina Barrington, PharmD, vice president of pharmacy programs for Priority Health, contributed her experience with RWE within a payer organization. She noted that Priority Health most commonly uses data from pharmacy and medical claims due to limited access to EHR data. Challenges to acceptance of RWE include the relevance to the patient population and the timeliness of data that are available at the time of review.

Barrington recognized the importance of RWE on patient adherence, which may lead to personalized care if the payer can identify which patients are more likely to remain on a therapy. Patient adherence and treatment response are being evaluated in Priority Health’s outcomes-based contract in oncology, and the organization is looking to expand its involvement in such contracts because of the high growth of utilization and spending in oncology.

Like the other panelists, Barrington cited timeliness as a key determinant of the utility of RWE for payer decision-making. If these data are not available at the time of a pharmacy and therapeutics committee meeting, they can still be used later on to reassess utilization management criteria.

“As more real-world evidence becomes available, we’ll make changes as necessary, opening up the therapies to more patients and additional subpopulations and giving us the opportunity to look at the outcomes and drive better care in our members,” Barrington concluded.