Cystic fibrosis (CF) patients nationwide have welcomed the news that two groundbreaking drugs are to be reimbursed by the HSE.

Last year, the National Centre for Pharmacoeconomics (NCPE), which is responsible for assessing medicines to see whether they are cost effective, recommended that the drug, Orkambi, should not be funded by the HSE.

Orkambi is a drug that could benefit around 550 people with CF in Ireland, specifically those over the age of 12 with a specific genetic mutation.

Since the decision by the NCPE, the Department of Health had been in discussions with the drug's manufacturer, Vertex Pharmaceuticals, in the hopes of reaching an agreement on price.

The CF organisation, Cystic Fibrosis Ireland (CFI), had also been seeking the extension of Kalydeco drug therapy to children aged between two and five years. This drug also benefits patients with a specific mutation, but it had only been available for those aged six and older. It is also made by Vertex.

Last month, CFI had expressed concern about a lack of contact between the HSE and Vertex. However on April 11, the Minister for Health, Simon Harris, told the Dail that ‘agreement has been reached in principle on the commercial terms for the supply to patients of Orkambi (aged 12 and older) and for Kalydeco (for patients aged two to five).

"I wish to provide this update to the House to offer reassurance to patients on this important matter and to inform them that the HSE and Vertex expect the medicines to be available from May 1. I also wish to inform the Dail that both parties are now working to finalise the contractual arrangements and complete approval processes in advance of May 1," Minister Harris said.

He acknowledged that this has been ‘an extraordinarily difficult time for CF patients, their families and friends'.

"I am pleased that we are now in a position to reassure people we are now in the concluding stages of an agreement as I outlined earlier and expect the further work in the coming days will bring this matter to finality," the Minister added.

The news was ‘warmly welcomed' by CFI chief executive, Philip Watt

"The development is especially welcome, coming as it does during Cystic Fibrosis National Awareness Week. While we have yet to see the full details of the agreement that has been reached, CFI understands that it will be inclusive of all patients who have the potential to benefit from Orkambi and Kalydeco because of their particular CF genotype - almost 600 patients in total," Mr Watt commented.

He paid tribute to all of the CF community ‘who have spoken out so bravely in recent months'.

He also welcomed the part of the agreement which includes ‘pipeline drug therapies' from Vertex that are currently showing promise in clinical trials.

"We understand that these pipeline drugs are part of the agreement. Even with Orkambi and Kalydeco, there will be around 30% of the CF population that still has no drug that treats the underlying cause of their condition in Ireland. This is why a pipeline deal is so important. There also may be better drugs for those on existing Vertex drugs coming down the line," Mr Watt said.

CFI acknowledged the high cost of these drugs, however it believes that these costs will be significantly offset by other savings. For example, Orkambi slows the progression of CF, reduces the risk of exacerbations that can lead to hospitalisation, and reduces dependency on other drugs, such as expensive antibiotics. All of these can lead to major savings for the health service.

"In short, in addition to health gain, CFI contends that the approval of Orkambi and Kalydeco will result in significant other savings for the HSE and we call for independent monitoring to quantify these additional savings.

"CFI further calls on the HSE to come to a similar positive conclusion for patients with other conditions desperately seeking a drug therapy, including patients with Alpha-1, Duchenne muscular dystrophy and cystinosis," Mr Watt added.

For more information on CFI, click here