ICER Report: Could a flawed approach to cost-effectiveness assessment put patients at risk?

Week in Review
Dr. Durie

Until earlier this year I was unfamiliar with ICER (Institute for Clinical and Economic Review). Then, I started to read reports about the methodological flaws and factual errors in the institute’s recently released report, “Treatment Options for Relapsed or Refractory Multiple Myeloma: Effectiveness and Value.”  Critics of the 138-page cost-effectiveness assessment called it “spurious,” “misleading,” and pointed to the report’s “invalid results.” Strong words indeed.

How could this be, since I also read that ICER recommendations were trusted and used by payers, and may be influencing CMS (Centers for Medicare & Medicaid Services) decision-making? Yet the depth of distrust in the myeloma community is almost palpable, despite the incorporation of what ICER characterizes as “prestigious stakeholder input” (and the caveat that “None of these stakeholders is responsible for the final contents of this report”).

The report’s authors are ICER staff members and a modeling group from the University of Washington. The modeling team has been most severely criticized for using old and irrelevant comparative data, underestimating QALYs (Quality of Life Adjusted Years), using low thresholds, overestimating budget impacts, and multiple technical errors (I could go on here, but will stop for the sake of brevity). Again, not a happy picture!

Serious flaws, serious implications

So what’s going on here? According to some public commentary, ICER’s pricing recommendations have serious implications and serious flaws.

On February 7, 2016, Dr. Rafael Fonseca wrote an eloquent commentary summarizing his concerns. “While an analysis like ICER’s is well intended, the content of its conclusions can be quickly used by policy makers and payers to limit treatment options for patients,” he writes. Dr. Fonseca points out that under the ICER recommendations, payers will be the beneficiaries (with reduction in costs for them) and patients could face significant limits in access to key new myeloma therapies that have demonstrated excellent efficacy and outcomes in published data.

Dr. Fonseca emphasizes the possibility of forcing patients to “fail first” using the ICER paradigm. This means that the rules could require, for example, using and failing on thalidomide first before using lenalidomide, or a variety of other therapies before using pomalidomide. The same could hold true for the proteasome inhibitor family of drugs.

Assessment of “value”

In another recent commentary, the “Value in Healthcare” by Anupam B. Jena & Tomas J. Philipson, the authors make strong points about how to approach assessment of “value.” They argue for the need to avoid “pseudo-science” and “voodoo economics.” This is an area of significant emotion and concern.

I suggest that stakeholders in the US read the ICER report and voice their concerns. More involvement of myeloma experts directly in the final report preparation can be helpful. I certainly encourage myeloma community opinion leaders to express their perspective. 

Patients must speak up, as they are currently doing, to add their personal voices of concern. These are tough financial times, and, faced with increasingly difficult medical choices, the last straw is to think that important choices can disappear because of an unchallenged, flawed process.

The elephant in the room in these discussions is the exorbitant cost of healthcare overall, and as part of that, the currently accepted costing structure for drugs. Until all parties come to the table to resolve these issues, it is important that both patients and physicians not get caught in the crossfire.


From the IMF Newsroom

1.       Phenotypic and genomic analysis of multiple myeloma minimal residual disease tumor cells: a new model to understand chemoresistance

Supported in part by a grant from the IMF’s Black Swan Research Initiative, this study, led by Black Swan Research investigator Dr. Bruno Paiva, shows very important initial observations regarding minimal residual disease (MRD). (Paywall; patient access.)

2.       The long read: The sugar conspiracy

A British scientist in 1972 sounded the alarm that sugar – and not fat – was the greatest danger to our health. But his findings were ridiculed and his reputation ruined. This fascinating article, published in the examines how the world’s top nutrition scientists got it so wrong for so long. The article, which appeared in the Guardian, cites the work of Dr. Robert Lustig, a pediatric endocrinologist at UC San Francisco, whose book “Fat Chance: Beating the Odds against Sugar, Processed Food, Obesity and Disease” I wrote about here.

Image of Dr. Brian G.M. DurieDr. Brian G.M. Durie serves as Chairman of the International Myeloma Foundation and serves on its Scientific Advisory Board. Additionally, he is Chairman of the IMF's International Myeloma Working Group, a consortium of nearly 200 myeloma experts from around the world. Dr. Durie also leads the IMF’s Black Swan Research Initiative®.

Source URL: