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Scotland turns down Vertex's Orkambi

Published on 13/08/19 at 09:35am

The Scottish Medicines Consortium (SMC) has turned down Vertex’ cystic fibrosis drug Orkambi on the grounds that it is not good value for money.

Scotland’s cost-effectiveness body rejected the $272,000 a year drug as a treatment for cystic fibrosis in patients aged two and older due to “significant uncertainty around their overall health benefits in the long-term, in relation to their costs.”

Orkambi could benefit 330 people in Scotland, a region which has one of the highest incidences of cystic fibrosis in the UK.

The SMC said that while they had heard “powerful testimonies” from doctors and patients on the potential benefits of the drug, they could not fund it at the price on offer.

SMC chairman Dr Alan MacDonald, commented: "We understand the profound impact that cystic fibrosis has on patients and their families and we are aware of the need for effective treatments that target the underlying cause.”

“Patient groups and clinicians gave powerful testimonies about the impact of the condition and the potential benefits of these medicines.

“As these are orphan medicines, our committee members were able to apply additional flexibility in their decision-making but there remained significant uncertainty around their overall health benefits in the long-term, in relation to their costs.

“In order to be able to accept these medicines the committee will need to be satisfied of their cost-effectiveness and we continue to work with the company to achieve that.”

Louis Goss

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