ICER Recommending Eugenics for Kids With Duchenne Muscular Dystrophy

  • by: Robert Goldberg |
  • 07/25/2019
ICER claims :” Unfortunately, there is no persuasive evidence that [ Exondys and other treatments for Duchenne Muscular Dystrophy (DMD) ] improve outcomes that matter to patients, including functional status, quality of life, or length of life. Eteplirsen has been on the market for three years and yet we still found notably inadequate data on patient outcomes.”

But under the ICER model, no treatment for Duchenne Muscular Dystrophy would be considered valuable under that standard because none of the existing treatments had clinical trial data to support use.  ICER is engaged in hypocritical data manipulation:

1.    ICER claims there is no evidence that compared to ventilation that Exondys is improving outcomes.  Yet, there was never any prospective study of ventilation. So according to ICER’s methods, there is no persuasive evidence that ventilation improves outcomes that matter to patients. 
2.    Further, not one study demonstrated that ventilation meets ICER’s 100K per QALY threshold.  Indeed, one study found that mechanical ventilation users had 16.4 hrs per day of licensed practical nurse/ registered nurse care costing $269,370 per year.   Yet non-invasive ventilation, which has replaced mechanical or conventional non-variable ventilation (CNVS) which is less expensive would have been denied by ICER. 
3.    If the ICER standard has been applied to non-invasive ventilation, then tracheotomy would still be the preferred treatment.  Yet, without CNVS these patients would either still be in iron lungs around-the-clock or, more likely, would have undergone tracheotomies and by now be dead from complications related to the tube since four out of five patients with some NMDs using tracheostomy
4.    In fact, current treatments are also not cost-effective because they prolong life and add to the total cost of care.  Indeed, a recent study concludes that “DMD should be now considered an adulthood disease as well, and as a consequence, more public health interventions are needed to support these patients and their families as they pass from childhood into adult age.”
5.    That is, death is the preferred outcome if you only care about saving money:“(E)xtending life for a patient with DMD by 1 year would be associated with a QALY gain of 0.18 at a cost of $54,000 (not accounting for the cost of the evaluated intervention), resulting in an incremental cost-effectiveness ratio of $300,000, well above “ the ICER cut-off. 
6.    Further, the main cause of death in DMD in our population remains cardio-respiratory failure.  People with DMD are more likely to die if they have poor respiratory profiles and elevated cardiac biomarkers. Collectively, these factors highlight a high-risk cardiovascular population with a worse prognosis.  
7.    However, other technologies that would extend life that ICER compares to Exondys are also NOT cost-effective. For example, the incremental cost-effectiveness ratio (ICER) for destination ventricular assist device therapy (DT-VAD) was $179,086 per quality-adjusted life-year (QALY).

The documented delay in loss of lung and heart function from Exondys exceeds that of ventilation.  Moreover, the use of Exondys is likely to help reverse weight loss, also a risk factor for death, by allowing people to swallow food safely.  

Hence, every treatment for DMD between the 1980s and today has increased life expectancy and improve quality of life.  Yet NONE meet ICER thresholds.  ICER would have deprived tens of thousands of patients longer, better lives.  

This assumption, that people who, because of genetic conditions, are likely to be sicker and more likely to require medical care as they live longer is shaped by the same impulse justifying eugenics at the turn of the century. 

Here is the statement of ICER president Steve Pearson “In practice, however, a sickest-first principle might require allocation of resources even when only minor gains can be achieved and the cost is very high, which is obviously inefficient…coverage decisions must not only incorporate consideration of the benefits gained but the opportunity costs incurred when covering expensive orphan drugs.”

And here is the statement of Margaret Sanger, a eugenics proponent: 

"Every single case of inherited defect, every malformed child, every congenitally tainted human being brought into this world is of infinite importance to that poor individual; but it is of scarcely less importance to the rest of us and to all of our children who must pay in one way or another for these biological and racial mistakes."

Limiting the use of new medicines that prolong and improve the lives of people with DMD to save money is eugenics by another name.  And that name is ICER. 

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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