“…mathematical calculations known as “value frameworks” to justify the nonprofit’s preference to target certain drugs. But this is no unbiased test or dispassionate statistical measurement. It’s a Catch-22 in which patients can’t win because the ICER process determines they have a lesser quality of life than a healthy person – forever. “
Similarly, the Global Health Living Foundation had this to say about ICER’s zealous defense of the quality adjusted live year as a perfectly ethical and rigorous measure of what patient’s truly value from medical care:
“The QALY was ultimately found to be so offensive and contradictory to the beliefs surrounding patient care in the U.S. that its use in computations was disallowed (outlawed) by the Affordable Care Act. The ban on using cost-per-QALY thresholds also seems to reflect longstanding concerns that the approach would discriminate on the basis of age and disability. The worry is that the metric unfairly favors younger and healthier populations that have more potential QALYs to gain.”
Ouch. ICER continues to evade this important question. Apart from some soft promise to look at QALYs going forward, it continues to double down on their use in determine access to and price of medicines. And despite the legitimate ethical and methodological questions about ICER’s work, JAMA is willing to be ICER’s seal of medical approval.
Case in point: JAMA’s recent publication of “Cost-effectiveness of PCSK9 Inhibitor Therapy in Patients with Heterozygous Familial Hypercholesterolemia or Atherosclerotic Cardiovascular Disease.” This is a recycling of ICER’s blacklisting of the two PCSK9 inhibitors that reduce high cholesterol in people who – because of a specific mutation or side effects – don’t respond to statins.
I am not surprised that JAMA would publish a high speculative and poorly constructed comparative effectiveness article. That’s because in general most CER work winds up in medical journals where – to put it gently – the reviewers of articles have little or no expertise in econometrics or best practices for health economic studies. I even wonder if the reviewers even read the article or did more than check for typos. For instance, in the JAMA article on the authors conclude:
“The results of multiple scenario analyses suggest that reducing the price of PCSK9 inhibitors remains the primary approach to improving the value of these therapies.”
The article goes on to say that “If ongoing clinical trials demonstrate that the drugs do not improve clinical outcomes as predicted by their effect on LDL-C, this model will have overestimated their cost-effectiveness.”
The authors completely ignore the other possibility: that studies will show increased cost-effectiveness. This violates a cardinal principle of HTA research:
“All data are imperfect point estimates of underlying distributions that incorporate a variety of errors. All analytical methods are subject to biases and limitations. Thus, extensive sensitivity analyses are required to determine the robustness of HTA findings and conclusions. The limitations of the analysis should always be acknowledged.”
The JAMA/ICER study also violate best practice principles for cost effectiveness analysis proposed by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Drug Cost Task Force Report:
“The task force recommended discouraging studies from claiming that they are taking a true societal perspective when they are not. “
Yet ICER continually claims that their studies and recommendations consider ‘societal choices’ even though the costs and benefits of such choices are never formally modeled.
Further, ISPOR the task force suggests that studies state “that using some fraction (e.g., 40–60%) of net acquisition drug cost (i.e., cost net of discounts and rebates) would be an appropriate proxy for opportunity cost for a societal CEA for marketed products, but that a limited societal or a health systems perspective is more relevant and useful for current decision-makers.”
ICER ignores this modest proposal claiming it is too hard to estimate net drug costs (even as it has no problem estimating for patients how much their lives are worth.) This speaks to the fact that ICER gets most of its money from insurers and PBMs. That’s not a problem so long as all the exceptions to best practices are identified and limitations of ICER studies with regard to measure societal value are highlighted.
As a I have mentioned elsewhere, ICER uses list price to establish how much drug prices should be cut to be cost effective. It ignores the fact that the discounts and rebates to reach that price would go right to insurers, employers and PBMs, not patients whose QALY hangs in the balance. It fails to acknowledge that the choice of list price will affect it’s analysis or that it reflects the perspective of the health system.
ICER can get away with recycling it’s biased research in JAMA. The publication’s reviewers of HTA are about as effective in policing violations of basic HTA practices as the UN Peacekeeping Force in Lebanon is in preventing Hezbollah from threatening Israel.
But ICER’s day of reckoning is coming. Patients, researchers, real economists and others are beginning to challenge the role of ICER specifically (and pre-determined value frameworks in general) in making life changing decisions on behalf of everyone else.