Friday 25 May 2018

Children with cystic fibrosis will be offered groundbreaking drug 'in weeks'

Health Minister Simon Harris. Photo: Damien Eagers
Health Minister Simon Harris. Photo: Damien Eagers
Eilish O'Regan

Eilish O'Regan

Children with cystic fibrosis who are aged from six to 11 years old should have access to the drug Orkambi in a matter of weeks, it has emerged.

It followed the decision of the European Medicines Agency to license the drug, which can improve lung function, for this age group.

Cystic Fibrosis Ireland called on Health Minister Simon Harris to ensure that children who have the potential to benefit from this extension of Orkambi are given the choice of accessing "this groundbreaking and innovative drug as soon as possible subject to the advice of their consultant".

A repeat of previous campaigns to force the HSE to reimburse the cost of the drug will be avoided this time following an agreement, which was made in April last year, with the pharmaceutical company Vertex, which makes Orkambi.

The long-awaited so-called pipeline deal meant that future extensions of Orkambi or another drug Kalydeco - or more new drugs that improve on Orkambi or Kalydeco from Vertex pharmaceuticals - will be made available to cystic fibrosis patients here.

Vertex said yesterday that 3,400 children across Europe between the ages of six and 11 had some form of cystic fibrosis.

In response, the minister said yesterday the "HSE will be putting in place the necessary reimbursement arrangements over the coming weeks for this age group".

A major goal in treating cystic fibrosis is slowing the progressive lung damage caused by the life-shortening genetic disease while improving health in the short term.

Studies of Orkambi in children ages six to 11 have shown improvements in clinically relevant outcomes such as lung function and weight gain.

Ireland not only has the highest incidence of cystic fibrosis in the world, but also the largest proportion of families with more than one child suffering from it.

As the population increases, the number of people with cystic fibrosis will rise.

Irish Independent

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