CF group claims new data supports case for Orkambi approval

Cystic Fibrosis Ireland warns of ‘strong outcry’ if HSE fails to approve Orkambi on cost

CF Ireland says damage is being done to children with cystic fibrosis by the failure to authorise Kalydeco for  those aged two to five years

CF Ireland says damage is being done to children with cystic fibrosis by the failure to authorise Kalydeco for those aged two to five years

 

New evidence has emerged to support the case for approving the groundbreaking cystic fibrosis drug Orkambi, Minister for Health Simon Harris has been told.

Newly-published data shows Orkambi, by preventing a worsening of the condition of patients, will save the health system even more than was previously estimated, according to Cystic Fibrosis Ireland.

The percentage of cystic fibrosis patients that stands to benefit from the drug is among the highest in the world, the patient group says in a letter to Mr Harris.

With the HSE due to make a final decision shortly on whether to reimburse the cost of the drug, CF Ireland has warned the Minister of a “strong outcry” if no agreement is reached with the manufacturer Vertex Pharmaceuticals.

Orkambi was estimated to cost €159,000 per patient per year when price talks began last year but CF Ireland says it understands there has been a “considerable discount” on the original list price since then.

“This would indicate that the gap between what the HSE is reported to be willing to pay is not insurmountable, and with goodwill from both sides an agreement can be reached.”

The group says new data published in the 2015 report of the independent CF Registry of Ireland shows the high proportion of cystic fibrosis patients in Ireland who suffer pulmonary exacerbations or deteriorations in their condition.

In 2015, 41.1 per cent of adults and 27.2 per cent of children with cystic fibrosis experienced at least one exacerbation. The average time spent by all cystic fibrosis patients on antibiotics was 35.7 days that year.

Exacerbations

The data shows 56 per cent of Irish people with the disease has the gene alteration that is targeted by Orkambi. This compares with 50 per cent in the UK and 46 per cent in the US. Ireland continues to have the highest overall incidence of cystic fibrosis in the world.

The average lifespan of cystic fibrosis patients has increased from 13.4 years in the late 1980s to 27 years in 2014 and 30 years in 2015. The use of new and innovative drugs played a major role in this improvement, the letter says, adding: “The last thing we would need now would be the denial of new and proven drugs that would retard potential and more rapid development.”

CF Ireland also says damage is being done to young children with cystic fibrosis by the failure to authorise another drug, Kalydeco, for use by those aged two to five years.