#YesOrkambi – Cystic Fibrosis Advocates Campaign for Drug’s Approval in Ireland
A new social media campaign was launched in Ireland to advocate for the approval of Orkambi for cystic fibrosis (CF) patients in the country.
The drug combines the compounds ivacaftor and lumacaftor. Orkambi, which is a novel therapeutic approach designed to treat the root cause of cystic fibrosis instead of just the severity of symptoms, was approved in the U.S. by the FDA in July 2015, but has not been approved in Ireland.
The campaign, called Yes Orkambi (#YesOrkambi), was launched after the National Centre for Pharmacoeconomics in Ireland ruled against approval of the drug. The cystic fibrosis community decided to try to persuade the Irish government to approve Orkambi, a therapy that’s estimated to cost €160,000 per patient — the equivalent of $181,675.
Cystic fibrosis campaigners started the #YesOrkambi campaign with a video that shares an open letter to Taoiseach Enda Kenny and Minister for Health Simon Harris. “As you know, Orkambi is a drug that could change the lives of 600 people with cystic fibrosis in Ireland,” says the video, which features purple balloons, a symbol of the cystic fibrosis community.
People are also featured in the video, titled “Please don’t put a price on our lives.” They offer their thoughts about Orkambi, using words like future, hope, or more time at home with family and friends. The CF community shares its unhappiness with the Irish government for rejecting the funding that would give Irish patients access to Orkambi.
One of the main reasons why the National Centre for Pharmacoeconomics gave a negative assessment to the approval of Orkambi is the cost of the drug. Providing Orkambi to cystic fibrosis patients in Ireland would cost the health service €392 million over five years, the equivalent to $445 million in U.S. dollars. The center recommended the price for the drug be cut fivefold, instead of the current price of €160,000 per patient annually.
Advocates in the country believe the new campaign can open doors for price negotiation with the company that produces it, Vertex Pharmaceuticals. In the U.S., Orkambi’s FDA approval is indicated for cystic fibrosis patients age 12 and older with two copies of the F508del mutation in their CFTR gene. The FDA approval was based on two pivotal clinical trials conducted by Vertex (TRAFFIC and TRANSPORT) that revealed the effectiveness of the drug in improving patients’ lung function compared to a placebo.
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