Cystic fibrosis patients lobby Vertex to drop drug prices

Healthcare professionals and patient groups have written to Vertex’s CEO Jeffrey Leiden in a bid to resolve a funding row over cystic fibrosis drugs, as UK MPs prepare to hold a public hearing investigating their price.

Vertex has a range of drugs that help to correct the defective protein that causes the debilitating symptoms of cystic fibrosis, including severe respiratory problems that are life-limiting.

Around 90% of CF patients are now covered, and the company is working on treatments for the remaining 10%.

But they are expensive, and in 2016 the UK’s NICE said that one of Vertex’s most recent CF drugs Orkambi (ivacaftor+lumacaftor) is not cost effective in patients with a specific kind of mutation.

Since then Vertex has been deadlocked in negotiations with NHS representatives in an increasingly bitter row over funding.

US pharma has refused to deal with NICE any more, saying that its assessments need to be revised.

As part of an ongoing campaign Christina Walker, whose eight-year-old son Luis has cystic fibrosis (CF) and is eligible to receive Orkambi, has written to Vertex’s CEO Leiden to try and resolve the matter.

Vertex, which has not published the price for the drug, maintains that the NHS is undervaluing its work to develop the drug and get it approved.

In the letter to Leiden, Walker and signatories from hospitals and patient organisations across the world, said: “As you discuss Vertex’s considerable fourth quarter earnings this week and look to the year ahead, please consider that both the company’s financial growth and altruistic aims are achieved in the same way – by getting your medicines to all patients.”

Leiden has already revealed that 18,000 of the 37,000 patients eligible for treatment with Vertex’s three licensed drugs are being treated, and the letter called for those patients missing out to receive treatment immediately.

“By extending the global reach of Vertex’s current medicines, not only can the company provide relief for and give hope to many more countries, it would also exclude fewer US patients, by lessening pressure on prices there, which are set so high because global coverage is so low.”

MPs sitting on the influential Health and Social Care Committee are due to meet on Thursday 7 March to discuss the price of Orkambi and other CF drugs from Vertex.

The session will “explore the current impasse” between Vertex, NHS England and NICE on the availability of the drugs as part of an ongoing investigation.

pharmaphorum has approached representatives of Vertex for comment.