Interventions of Interest:
- Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals)
- Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals)
- Ivacaftor (Kalydeco®, Vertex Pharmaceuticals)
- Elexacaftor/tezacaftor/ivacaftor (Trikafta®, 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 independent appraisal committee unanimously concluded that Trikafta delivers substantial benefits for patients, family members, and society.
Below you will find the final documents from the assessment review process:
“CF is a devastating disease that affects the lungs and other organ systems, typically requires substantial medical care, and robs patients and families of hours each day, before taking away full years of life. Trikafta is a remarkable advance for the majority of people living with CF, and we encourage public and private US payers to maintain a simple process for all eligible patients to access this important treatment. Unfortunately, the manufacturer has leveraged its monopoly to set a price — costing many millions of dollars over the lifetime of an average patient — that is far out of proportion to the treatment’s substantial benefits. Patients who receive Trikafta will benefit, but misaligned pricing causes harm to patients — some with CF and some with other diseases — who are forced to delay or forego care or even to drop health insurance entirely.”