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ICER’s Rare Disease Problem

What’s rarer than a rare disease?  The controversial Institute for Clinical and Economic Review deciding that a drug to treat a rare disease meets the organization’s definition of value.

A recent report points out that ICER – the Boston-based drug price analysis group – did not give a “high value” rating to a single rare disease drug between 2014 and 2018.  Why? In, “Looming Challenges for ICER in Assessing the Value of Rare Disease Therapies,” the independent Pioneer Institute suggests that ICER’s approach has fundamental flaws.  

In particular, the think tank argues that:

  • ICER thresholds are not appropriate for rare disease drugs.  The report argues that ICER’s definition of cost effectiveness does not adequately account for the inherent challenges of orphan drugs.  Research for these targeted medicines is expensive, and clinical trials are difficult to enroll. And because so-called orphan drugs treat a small patient population, these treatments will naturally cost more per patient.
  • Clinical trials data are too limited to evaluate the value of rare disease drugs.  For these therapies, clinical trials are particularly small and data especially sparse.  Pioneer Institute notes that ICER feels free to use these shortcomings to undercut a drug’s value, but has no qualms about applying that same data to definitively announce the drug is overpriced.ICER seems less eager to base its conclusions on robust data than to signal [to] payers that rare disease drugs are not a good value,” the report argues.
  • ICER’s “one-size-fits-all” approach is out of step with precision medicine.  In a world where therapies’ value to individual patients may increasingly depend upon a variety of precise genetic factors, attempts to encapsulate a drug’s value for all patients into a single number will be less and less valid.
  • ICER’s thresholds and definitions for rare diseases are arbitrary.  ICER’s cost-effectiveness thresholds are “not ‘scientific’ in any economic sense,” the paper argues.  And the organization’s decision that “ultra-rare diseases” are those affecting 10,000 or fewer patients is likewise arbitrary, says Pioneer Institute.  Yet these numbers can nevertheless have a painful impact on patients’ lives when they are used to feign “expert” metrics that justify barriers to treatment.

The paper sets out to determine whether “European style cost-effectiveness reviews” like ICER’s make sense for orphan drugs in the United States.  The unequivocal answer, the paper concludes, is: No.

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