Is the US setting the bar too low for new CF drug? Canada says it’s not worth it

From The Globe and Mail:

Canadian governments should not pay for a $250,000-a-year cystic fibrosis medication because it is not clear the drug actually improves patients’ health, according to a fresh review that has devastated some with the potentially fatal lung disease….

Screen Shot 2018-10-15 at 9.06.47 AMIn a report released on Thursday, the CADTH’s expert panel said its review of the Orkambi clinical trials and other studies found the drug produced only slight improvements for about one in four patients.

The report also said Orkambi’s Boston-based maker, Vertex Pharmaceuticals, would have to slash the drug’s price by more than 98 per cent to satisfy the agency’s value-for-money analysis.

“[Orkambi] is better than nothing. It’s better than placebo,” said Trevor Richter, director of the CADTH’s Common Drug Review, which oversees the review process for non-cancer medications. “But the benefit is really small. Not only is it small, but there’s a huge amount of uncertainty about what the actual size of that benefit is.”…

Vertex called the agency’s recommendation “deeply disappointing.”

Patients, who say the drug benefited them, are suing.

Mr. Richter of the CADTH said it is difficult but necessary sometimes to disappoint patients.

“We don’t enjoy delivering recommendations that are seen as negative,” he said. “In the end, we are an evidence-based review process. It’s rigorous and it’s transparent.”

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