Rationing by Stealth—And Why It Should Matter to You

July 25, 2019
By Naomi Lopez

What is a child’s life worth? If they’re suffering from terminal illness, how much should be spent on their medication? Would you entrust a so-called “expert panel” to make that decision? Shouldn’t that choice be left up to that child’s own doctor?

This week, the Institute for Clinical and Economic Review (ICER) is meeting to review its most recent report on the treatments for Duchenne muscular dystrophy (DMD). DMD is a rare disease affecting mostly boys; almost all are wheelchair-bound by their teens and dead by their 20s.

This is a disease that involves enormous patient suffering—loss of ambulation, loss of respiratory function, and loss of ability to complete routine daily functions—and for which there are no cures and only limited treatments that may slow the progression of this cruel disease. That is why patient advocates are mobilizing to warn Americans that ICER’s approach of setting a dollar amount on the value of a patient’s life is not only immoral, but dangerous for all of us.

In the case of most patients, ICER uses a range of $100,000-150,000 per Quality-Adjusted Life Year (QALY). If you happen to suffer from chronic pain or have lost some ambulation, your QALY is lower. In fact, in its DMD report, ICER uses a QALY as low as $50,000 to evaluate the value of a treatment for these patients.

Let that sink in: ICER’s analysis to make recommendations about how much a treatment is worth is as low as $50,000 for a young boy born with a rare genetic disease for which there is no cure, who will be wheelchair-bound by the time he reaches his teens, and where treatments are few.

While most of us are fortunate to not have to face this cruel disease, we should all be alarmed that this “independent” analysis could be used to make decisions on insurance coverage and the availability of needed treatments. The state of Maryland recently became the first state in the nation to establish a Prescription Drug Affordability Board (PDAB) and no one should be surprised if this board takes a similar approach.

In a Goldwater Institute report released last year, Goldwater Visiting Fellow in Healthcare Policy Dr. Rafael Fonseca, who is also a renowned hematologist and oncologist, and his co-authors included a critique of the ICER approach as it related to multiple myeloma and wrote:

“Regarding myeloma, the conclusions reached by ICER’s evaluation are problematic and do not reflect a bona fide approach to understand best practices for the treatment of myeloma better. The ICER process was largely limited by the lack of myeloma experts in its panels, the lack of meaningful input by key stakeholders, the lack of consideration of biologic variability among myeloma cases, and the fact that by the time of this writing, its conclusions are already outdated given the rapid pace of clinical research in myeloma.”

The authors went on to point out that:

“ICER’s process is not peer-reviewed to a scientific standard, does not include disease experts as evaluators or authors, does not use patient-centered endpoints or definitions of value, does not reflect current standards of evaluation for evidence-based medicine, and lacks a mechanism for continuous review and revision.”

The current report on DMD faces the exact same shortcomings. There is no doubt that the cost of treatment is complex and deserves more attention, but is this the approach that we should allow, especially when lives hang in the balance?

Americans should know about and publicly debate whether their governments, insurers, and lawmakers can and should use these types of “expert” panels that could override patients’ own doctors’ medical expertise of the disease and knowledge of an individual patient’s condition.

Naomi Lopez is the director of healthcare policy at the Goldwater Institute.