real world evidence
RWE
RCT
randomized controlled trials
payer
perceptions
FDA
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Payer perceptions of the use of real-world evidence in oncology-based decision making
Summary
- US payers find real-world evidence (RWE) useful for improving costs and outcomes in oncology, and for making formulary decisions.
- Payers prioritize comparative effectiveness evidence but also value other RWE types such as total cost of care, burden of illness, treatment patterns, and economic data.
- RWE generation has become more robust, supported by guidelines from the FDA and NCCN.
- Advancing technology is making real-world data more accessible for meaningful studies.
- Payers use RWE at all phases of a product’s life cycle and access it through literature searches and manufacturer data pre- and post-launch.
- Pre-launch RWE is beneficial for P&T committees, but comparative effectiveness RWE is often unavailable at launch.
- Post-launch RWE can be used to reevaluate decisions, highlighting the importance of ongoing RWE generation.
- Payers conducting their own RWE studies often use medical and pharmacy claims data but acknowledge its limitations, leading to increased use of electronic health records (EHRs).
- RWE can support off-label use of medications, with CMS considering reimbursement if included in a compendia.
- There is an opportunity for collaboration among payers, manufacturers, and researchers to target research resources towards impactful evidence for coverage or contracting decisions.
- Payers and manufacturers should develop best practices for incorporating RWE into decision-making processes.
- Outcomes-based contracts are expanding, with an opportunity for refining measurable definitions and best practices.
Innovative therapies for cancers are being introduced with growing frequency. Some of these therapies, especially those in areas with high unmet needs and limited patient populations, receive accelerated regulatory approvals, sometimes based on Phase II studies only or single-arm trials. In 2012, the US Food and Drug Administration Safety and Innovation Act was enacted to expedite the development and review of certain new drugs for serious or life-threatening diseases (sometimes designated as breakthrough drugs).
his act allows promising drugs to receive accelerated approval based on surrogate markers of disease, while requiring that efficacy testing continues post-launch to confirm the initial results. A 2018 study showed that of drugs released in the US from 2012-2017, those designated as breakthrough drugs achieved first FDA approval in 5.2 years vs those designated as non-breakthrough drugs, which received first FDA approval in 7.1 years. As a result of accelerated approvals based on limited clinical trial data, however, payers are often forced to make oncology coverage decisions with limited information...
real world evidence, RWE, RCT, randomized controlled trials, payer, perceptions, FDA
