Article contributed by Molly Purser, PhD and Naoko Ronquest, PhD
ICER’s New Value Assessment Framework
On January 31, 2020, the Institute for Clinical and Economic Review (ICER) released a new value assessment framework document. Over 60 organizations—including patient advocacy groups, clinical societies, drug manufacturers, and payers—provided inputs during this update. In reviewing the changes introduced in this framework, we identified the following five changes as critical for stakeholders who are engaging with ICER in 2020.
- ICER will proactively seek real-world evidence to incorporate into its assessments.
- ICER will generate real-world evidence in-house via database analyses and surveys.
- Societal analyses may be considered as a co-base case in some therapeutic areas.
- ICER will evaluate the need for updating each evidence report 12 months after a final report is released.
- The threshold willingness-to-pay range for ICER’s cost-effectiveness analyses increased from $50,000–$150,000 to $50,000–$200,000.
How Will These Key Changes Affect Your Future Collaboration with ICER?
ICER Will Proactively Seek Real-World Evidence to Incorporate Into Its Assessments
Although ICER acknowledged potential issues involved in using data based on observational studies, the new framework highlighted ICER’s commitment to exploring how to use real-world observational evidence to assess new treatments more comprehensively. Since ICER will be more likely to review real-world, data-driven evidence that supports the value of new products going forward, we recommend stakeholders expecting an upcoming ICER review to:
- Consider the weaknesses of trial-based evidence in supporting product value and build a case for ICER to use real-world evidence when appropriate.
- Assess how best to fill gaps using prospective observational and retrospective database analyses.
- Submit study reports during open-input periods.
ICER Will Generate Real-World Evidence In-House Via Database Analyses and Surveys
ICER has traditionally relied on literature-based evidence in its reviews. However, in the newly released framework document, ICER stated it may generate new evidence to inform its economic evaluation. In a recent report, ICER conducted a de novo analysis using a MarketScan claims dataset to obtain clinical and economic inputs for the economic model. Furthermore, in the same assessment, ICER collaborated with a patient advocacy group to conduct an on-line patient survey to collect data on out-of-pocket costs, productivity, caregiver quality of life, and school attendance; those data were used to populate an economic model from a societal perspective. Based on these changes, we recommend stakeholders:
- Conduct patient-reported research to deepen the understanding of what is important to patients and be prepared to generate real-world evidence to address their concerns.
- Publish results from any real-world evidence studies such as database studies or patient surveys to address data gaps, thus providing a peer-reviewed source of information to be used in value assessments by ICER or other agencies.
Societal Analyses May Be Considered as a Co-Base Case in Some Therapeutic Areas
Previously, treatments’ potential ability to improve societal outcomes were not valued in ICER’s base case cost-effectiveness analyses. For example, the patients’ and their caregivers’ productivity, employment, and ability to stay out of nursing homes or residential care were only accounted for in scenario analyses. Thus, benchmark prices ICER provided never considered these societal benefits.
Under the new framework, when the societal benefits are deemed essential, ICER will use an economic analysis evaluated from the societal perspective as a co-base case. This means:
- ICER’s benchmark prices may incorporate the new product’s societal value.
- Manufacturers developing treatments expected to ease the societal economic burden will need to build a solid case for ICER to consider a model from the societal perspective, as a co-base case.
- Stakeholders’ real-world evidence generation strategy needs to account for not only studies identifying direct healthcare resources but also societal resources, such as patients’ and their caregivers’ lost wages and productivity, needs for nursing care, etc.
- To use societal resource data as inputs of economic models, manufacturers need to be prepared to submit study reports or published manuscripts in open-input periods.
ICER Will Check Needs for Updating Each Evidence Report At 12 Months After Releasing Each Final Report
Twelve months after ICER issues a final report, they will conduct a three-month process to determine if new information is available that may warrant an update. ICER will solicit input from manufacturers and the original round table participants. If no new evidence is available or if new evidence does not change the conclusion of the initial report, ICER will issue a statement and will mark the final report with this information. ICER may release a New Evidence Update as a stand-alone document posted to its website when new data on a small number of outcomes is available. For treatments approved by the FDA under accelerated pathways, ICER will also begin a pilot program that will generate real-world evidence 24 months after the final report’s initial release.
What does this mean to you?
- ICER may conduct an ad hoc New Evidence Update at any time after the release of a final report.
- Stakeholders can identify new information that could potentially impact results, and ICER will then update the literature search to determine if an update to the report is warranted.
- Manufacturers should be ready to generate or update real-world data for their product and share interim reports within the first-year post-launch with ICER. Analysis plans should be carefully designed to address the weaknesses of trial-based evidence.
New Willingness-to-Pay Threshold Range (50,000 To 200,000 USD)
Previously, ICER presented cost-effectiveness analysis results using the threshold willingness-to-pay ranges of $50,000–$150,000 for general therapeutic areas and $50,000–$500,000 for rare diseases. With this framework update, the upper limit of the cost-effectiveness threshold range will be updated to $200,000 regardless of the therapeutic area. For value-based pricing benchmark estimations, ICER’s willingness-to-pay thresholds will remain unchanged ($50,000, $100,000, $150,000 per QALY gained).