ICER reviewed new therapies for the treatment of hereditary transthyretin-related (hATTR) amyloidosis, a rare genetic condition characterized by nerve, heart, and eye damage that is currently treated through supportive measures alone.

The Council voted unanimously that, despite the net health benefit, both inotersen and patisiran represent a low long-term value for money. The votes were influenced heavily by the $450,000 annual list price of patisiran, and the assumption that inotersen would be priced similarly.

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Interventions of Interest

  • inotersen (Tegsedi®, Akcea Therapeutics)
  • patisiran (Onpattro®, Alnylam Pharmaceuticals)


Final Documents

Below you will find the final documents from the assessment review process:

“Hereditary amyloidosis is an insidious condition that gravely affects patients and their entire families over generations, and it is exciting to have two new treatment options that, based on short-term evidence, appear to provide important benefits. Unfortunately, the pricing vastly exceeds a level commensurate with our best estimates of the treatments’ ability to improve patients’ lives, and is likely to cause financial toxicity for patients and ultimately reduce access. Despite this concern, insurers crafting coverage policy to help maintain affordability of health insurance for all patients now and in the future should consider the treatments’ broader benefits to patients, caregivers, and society.”