Vertex cystic fibrosis drug Orkambi encounters hurdles

Vertex Pharmaceuticals Inc., the maker of the cystic fibrosis medication Orkambi want to negotiate with Canadian provinces a bulk deal for its drug. The provinces, however, have closed ranks in shooting down Vertex’s bid to negotiate the price for Orkambi which is around $250,000 for a one-year treatment per patient.

The Canadian Agency for Drug Technologies in Health (CADTH), which evaluates new medicines and determines if they should be publicly funded in the country, has on two occasions recommended against public funding for Orkambi. In October last year, the agency gave Orkambi a “do not reimburse” recommendation. CADTH said concluded that there is not enough evidence that the drug is effective.

However, supporters of the drug and some advocates for patients with CF maintain that the drug can be “life-altering” or a “lifesaver.”

Orkambi is the brand name for Lumacaftor/ivacaftor. It is a combination drug available as a single pill.  Ivacaftor increases the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein at the surface of the epithelial cell. Lumacaftor acts as a chaperone during protein folding and increases the number of CFTR proteins that are trafficked to the cell surface. It was approved by the United States Food and Drug Administration in July 2015.

CF stems from a defective gene that causes mucus to build up in the lungs. This makes breathing difficult for patients. There is no cure for the disease and treatment is currently focused on managing symptoms.

Two main studies involving the use of Orkambi on some 2,000 patients show that the drug might improve lung function by about three per cent, according to the Times Colonist News.

The health minister of British Columbia, Adrian Dix said he has pushed for changes in the review process so that Vertex can resubmit its application. Dix, however, told the Globe and Mail that the provinces will not bargain with Vertex until the drug is approved.

A spokesperson for Vertex said the company will resubmit its application if it is granted an expedited hearing and if the provinces will enter into price negotiations. The spokesperson argued that patients had already waited two years to access Orkambi and that there was no reason why “parallel negotiations” can transpire on the drug’s price.

Cystic Fibrosis Canada, a charitable organization which advocates for CF patients, has been lobbying for provincial governments to reopen negotiations on the price of Orkambi. Less than two per cent of CF Canada’s funding comes from drug companies such Vertex.

“When you purely evaluate drugs based on statistics without looking at the benefit to people’s lives, I think you are missing the boat. People aren’t statistics,” John Wallenburg, chief scientific officer for CF Canada, told the Globe and Mail.

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