In the summer of 2019, the Institute for Clinical and Economic Review (ICER) hosted a public meeting to discuss its value assessments of new treatments for Duchenne muscular dystrophy. For patient advocates and others who follow ICER, the events of that day have become rather infamous.
Duchenne is a rare degenerative neuromuscular disorder that leaves most patients unable to walk by they are 12 years old. The vast majority are left paralyzed from the neck down by age 21. The average life expectancy is 26. Of course, ICER’s value assessments rely heavily on the Quality Adjusted Life Year (QALY) standard, which places a monetary value on years lived in perfect health – not a benchmark Duchenne patients will be able to hit.
Parents of Duchenne patients raised concerns about this approach at ICER’s public meeting. One of them asked: “How am I supposed to make some comment on the value of my son’s life?”
The answer from ICER President Steve Pearson: “I’ll be honest, that’s why we don’t have you vote. We don’t think that’s fair, certainly.”
This callous response perfectly summed up a fundamental flaw in ICER’s methodology: It fails to account for – and, in some cases, is hostile to – patients’ perspectives on the value of new drugs and treatments.
Under the QALY standard, drugs that do not restore a patient to perfect health are inherently undervalued, even if – as is the case with Duchenne – a return to perfect health is not currently possible under any scenario. That probably explains why, from 2014 to 2018, none of ICER’s reviews of treatments for rare diseases resulted in a “high value” rating.
However, this isn’t just a problem when it comes to drugs that treat rare diseases.
As we’ve noted before, ICER makes cost-per-QALY estimates by constructing artificial simulations that track the progress of hypothetical patient populations through various stages of disease using either the drug being evaluated, or a chosen alternative. The progress in these models is measured by utility scores – usually extracted from literature – that are entirely focused on the functional status and clinical symptoms of patients. These measurements offer no information about the needs of patients or how a particular drug may meet those needs. And that is a serious problem.
In the case of Duchenne, an effective treatment might keep patients out of a wheelchair for a few more years, or continue to be able to feed, dress, and care for themselves.
For patients with rheumatoid arthritis, new drugs could mean the difference between living with minor soreness or unbearable pain.
For other conditions, the manner in which a drug is administered can have a serious impact on its value to a patient. For patients living in rural and underserved areas, drugs that can be self-administered – versus those that require travel to a doctor’s office or hospital – meet a whole different set of needs.
In all these instances, utility scores that focus on a limited number of clinical symptoms will not adequately reflect the value of a medication for a patient population. And, in recent years, ICER has made value assessments that failed to take the perspectives of patients into account in each of these areas. Instead, ICER has adopted an approach that largely reflects the health care provider’s perspective, focusing on broad standards to define symptoms and response levels.
Over the years, ICER has shown a passing interest in incorporating more perspectives in its value assessments by using patient focus groups to gather data. But, for the most part, those efforts have been extremely limited. For example, in a May 2019 assessment, ICER used a “focus group” that consisted of only three people. A few months later, another ICER assessment cited information from a single patient.
In other words, rather than performing real data collection to determine the needs and views of patients, ICER has often chosen to rely on minimal anecdotal evidence. As a result, its assessments rarely reflect the value of treatments from the perspective of patients.
ICER seemed to acknowledge this shortcoming in its methodology last year when it announced changes to its Value Assessment Framework that included changes to its patient engagement practices, but this new approach has yet to adequately address the glaring holes in ICER’s research by giving patients’ needs the careful attention and consideration they deserve.