Interventions of Interest:

  • Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals)
  • Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals)
  • Ivacaftor (Kalydeco®, Vertex Pharmaceuticals)

Cystic fibrosis is an autosomal recessive condition caused by mutations in the CFTR gene. It is relatively rare, occurring in approximately 1 in 2,500 to 3,000 livebirths, but is the most common, lethal genetic disease in Caucasian populations. CF is a progressive disease that affects many organ systems, but most of its morbidity and mortality are associated with its impact on the respiratory system.

The pricing overreach harms people with CF and threatens the health care system’s ability to support future innovation.

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Final Documents

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

ICER’s Chief Scientific Officer Dan Ollendorf, PhD stated:

“Discussions at our meeting highlighted that, while these therapies offer important benefits for those living with CF, the $300,000 annual price is far too high to pay year after year, harming patients and families today while threatening the health care system’s ability to maintain access to important future clinical advances. As the sole manufacturer of CFTR modulators, Vertex should be rewarded for its innovation; however, the company should also be willing to make their pricing decisions fully transparent, and fully engage in independent, multi-stakeholder assessments of the value of these medications.”