How to Incorporate Real-World Evidence into Oncology Care Decision-Making

Payers face significant and growing costs with oncology treatments. Real-world evidence (RWE), such as real-world observational studies, patient-reported outcomes (PROs), and economic models, can potentially help payers in their coverage and formulary decision-making.

During a session at AMCP Nexus 2020 Virtual, Diana Brixner, RPh, PhD, professor at the University of Utah and executive director of the Pharmacotherapy Outcomes Research Center at the University of Utah; Daniel Malone, RPh, PhD, professor at the University of Utah; and Christina Barrington, PharmD, vice president of pharmacy programs at Priority Health, discussed the use of RWE in oncology practice for decision-making.

Dr. Malone defined RWE as “information on health care that is derived from multiple sources outside typical clinical research settings.” RWE can be useful for safety, rare conditions, medical policy, adherence, utilization management, technology assessment, population management, financial considerations, and comparative evidence. “Does the study design provide the appropriate approach to answer the question? Does it meet regulatory requirements?” asked Dr. Malone, of the framework questions that should be answered with RWE studies.

Dr. Brixner described the results of a national survey on U.S. payer use of RWE, economic models, and PROs in oncology-based decision-making. “When we look at oncology, many of the products that are approved by the FDA are often approved through an expedited review process. That gives an opportunity for access to patients where the drugs can make a difference in their survival; however, it can also be very challenging for payers when there are limited data available to make formulary decisions,” said Dr. Brixner about why they chose to undertake the study.

In the study, most respondents (92%) were pharmacists, 56% worked as pharmacy administrators, and 69% had been in their role for five years or less. Most were from managed care organizations (36%) and pharmacy benefit managers (27%). There was good representation from various payer sizes in terms of lives covered, as well as geographic regions.

Payers were primarily focused on comparative effectiveness between products, but also valued burden of illness, adverse event profiles, total cost of care, subpopulations, treatment patterns, and off-label usage.

“What was encouraging to hear was that the interested in RWE in oncology is high,” said Dr. Brixner. The majority of U.S. payers (85%) said RWE informs formulary decision-making for oncology products in their organization. Almost half of payers (42%) said they were likely to make off-label oncology formulary decisions based on RWE results.

Barriers to conducting in-house RWE studies include lack of appropriate in-house data, lack of personal resources, lack of analytic capabilities, and lack of perceived value in conducting analyses.

High‐cost therapies for small‐population oncology drugs are driving payers’ increased demand for economic information in oncology, according to Dr. Brixner. She said AMCP Format for Formulary Submissions now includes a section on economic information.

In terms of economic models, payers reported more interest in cost-effectiveness models and budget impact models than simple cost calculators. Payers’ reasons for not reviewing economic models include lack of availability at decision time, potential bias, lack of personnel/training, lack of transparency, and complicated models.

More than half of payers believe PROs from clinical trials (60%) and real-world studies (55%) are useful for formulary decision-making, and 47% said a lack of PRO data would somewhat impact formulary review.

While payers typically look for more objective measures when making coverage decisions, PROs can be helpful in oncology because of the emphasis on quality of life, according to the study findings. Respondents said PROs are useful for developing/adjusting clinical pathways for quality of life, shared decision support, care management outreach and care delivery models, physician/patient dialogues, and characterizing overall and progression-free survival length and quality.

Dr. Barrington then discussed the use of RWE at her organization at Priority Health. “When we look at our trend data, oncology hits in the top one or two areas of high costs for us,” she said. “The rapid pace of drug development in oncology requires RWE to support drug safety and effectiveness. Only one-third of launched drugs actually meet their medical expectations,” she said of oncology products.

Decision-making bases clinical decisions on the strength of scientific evidence and

standards of practice on peer‐reviewed medical literature, randomized clinical trials, pharmacoeconomic studies, and outcomes research data; evidence‐based medicine processes; effectiveness as well as the type and frequency of side effects, adverse reactions,

and drug interactions; and impact of drug selection on patient compliance. Cost decisions are based on clinical effectiveness and patient safety; evaluation includes impact on total health care costs, and rebates or price concessions are only considered for an individual product not the entire product line of a manufacturer.

Barriers to outcomes-based contracting for oncology agents include complexity, definition of the outcome, niche patient population, ability to apply evidence to subpopulations, cost, resource, adjudication of claims, and lack of visibility into both medical and pharmacy data as well as total cost of care.

Presentation: R2 How is Real-World Evidence in Oncology Used by Payers in Decision-Making? AMCP Nexus 2020 Virtual.