No update from HSE on agreement for CF drug, claims manufacturer

The manufacturer of a drug aimed at cystic fibrosis sufferers has said it has still not received an update from the HSE over whether or not funding for it will be recommended.

Vertex, which makes Orkambi, admitted it was “in the dark” after a weekend report indicated that the HSE’s drugs committee and the National Centre for Pharmacoeconomics (NCPE) were not going to recommend the drug for use due to its high price tag.

It sparked a furious reaction from Cystic Fibrosis Ireland and from Health Minister Simon Harris, who said negotiations over Orkambi had not yet concluded, and he moved to assure users of the drug on either clinical trials or compassionate access programmes that they would still be supplied with Orkambi until a deal was reached.

Yesterday, Vertex echoed that statement and said: “All people currently receiving Orkambi via clinical trials or compassionate use will continue to do so until an agreement is reached with the Government.”

However, a spokesperson for the pharmaceutical company said efforts had been made to contact the HSE regarding the weekend report and that it had not heard from the HSE since it submitted its most recent “solution” some two weeks ago.

The cost quoted at the beginning of negotiations 25 weeks ago was €159,000 per patient per year.

In a statement, the company said: “Vertex has not yet received any notification from the Health Service Executive as to whether it will recommend the funding of Orkambi, for people ages 12 and older who have cystic fibrosis with two copies of the F508del mutation.

“We have actively engaged with all parties throughout Orkambi’s assessment by the National Centre for Pharmacoeconomics and the HSE.

“Following the NCPE decision earlier this year, we immediately contacted the HSE and asked to enter discussions over how access could be achieved for the 500-plus people in Ireland in need of treatment. Over the past five months, we have met with HSE representatives six times and presented a range of solutions we believe could support access as soon as possible.

“We are currently awaiting a response from the HSE on our latest proposals.”

The company said it remained optimistic that a solution would be found.

Meanwhile, a medical device for CF sufferers which lessens their exposure to infections, resulting in fewer doctors’ visits, reduced antibiotic usage and shorter hospital stays, is expected to be market ready within two years.

SoloPEP was developed by researchers at the University of Limerick and is a disposable percussion-based chest physiotherapy device. Those behind the device have secured €500,000 in Enterprise Ireland funding for its development.