Earlier this month, ICER released a white paper titled “The Next Generation of Rare Disease Drug Policy: Ensuring Both Innovation and Affordability.” If you have been monitoring ICER’s work over the years, the paper likely matches your expectations.

Ostensibly, the purpose of the white paper is to “examine potential reforms to current policies and practices related to orphan drug development, pricing and coverage.” It explores a handful of proposed changes to current policies and, true to its word, does not overtly recommend any one reform over the others. The problem is the paper looks at these ideas through ICER’s flawed lens, which inherently magnifies cost estimates without context and routinely discards factors that are essential to finding the true value of rare disease treatments.

On one hand, the paper does acknowledge the difficulties in developing treatments for rare diseases and the unmet needs that still exist for many of those conditions. It also recognizes the importance of Orphan Drug Act in the ongoing effort to meet more of those needs.

However, ICER being ICER, the report quickly pivots back to espousing a radical drug pricing ideology, one that is preoccupied with the interests of healthcare payers and public health agencies with little to no regard for the concerns of patients. Those suffering from rare diseases have distinct challenges that give them a unique perspective on the value of new medications when it comes to meeting their distinctive needs.  

A new treatment might be life-changing for a patient. It could mean the difference between going to work and staying home in bed or between excruciating chronic pain and being able to function without constant care. Yet, by ICER’s measures, such factors are often little more than blips on a cost-effectiveness scale.

In a similar vein, for patients suffering from rare diseases with very few available treatment options, the cost of an effective treatment may very well be lower than the costs of living without it, particularly if a new medication results in fewer hospitalizations, costly medical procedures, or reduced home care. Yet those kinds of comparisons rarely, if ever, find their way into an ICER value assessment.

Reading the white paper, it is clear ICER has no interest in shifting that perspective. None of the reform proposals include increased the use of disease-specific quality-of-life measures gathered from the perspective of patients and caregivers. In fact, the paper raises the views of patient advocates only once, and only then so the authors could write those views off as being too subjective and unworkable.

Keep in mind that ICER’s standard methodology inherently under values treatments for rare and chronic diseases. This is evidenced by the fact that ICER virtually always concludes that rare disease treatments are not cost-effective and recommends either steep pricing discounts or restrictions on access for patients.

Last, but certainly not least, is ICER’s continuing use of unscientific methods and measures in its value assessments. We’ve discussed these problems at great length in this space, but it is worth noting that ICER’s use of assumption-driven models and impossible value measures are particularly problematic when it comes to rare disease treatments, including some that, at the time of approval, are supported by limited evidence.

Information gaps – an inherent feature in data sets for rare diseases – are one of the reddest of red flags in treatment and technology assessments. It is only reasonable to assume that analysts hoping to assess the cost and clinical effectiveness of such treatment would tread lightly when drawing conclusions. However, ICER usually prefers to fill information gaps present at product launch with more assumption-driven simulations used to support its impossible, unscientific conclusions. Not surprisingly, none of the reforms outlined in ICER’s latest white paper would address this fundamental problem.

Rare diseases present unique challenges for policymakers, healthcare payers, and analysts looking to making pricing and coverage determinations for new treatments. To be clear, the Orphan Drug Act and the current system of for developing and approving rare disease treatments should be continually reexamined to uncover inefficiencies and consider potential improvements. However, it is disappointing that ICER’s latest contribution to this effort examines so many potential changes to public policy without also considering much needed changes to its own approach.