Advisory Group Recommends Against Vertex’s Orkambi

The Canadian Agency for Drugs and Technologies in Health rejected Vertex’s cystic fibrosis drug Orkambi (lumacaftor/ivacaftor).

The independent advisory group based its decision on uncertainty over clinical trials measuring the drug’s effect on patient health. It also cited lack of data on how the drug impacts survival or the necessity of lung transplants.

The group claimed Vertex would have to lower its nearly $249,000 price tag by up to 98.5 percent depending on the patient population to meet the threshold for a quality-adjusted life-year. The recommendation is non-binding but federal, provincial and territorial governments frequently follow the group’s advice.