Families plea for children’s access to cystic fibrosis drug

Ashling Kelly was over the moon when her daughter Robyn (7), who has cystic fibrosis, was included in a cohort of children selected by the HSE for treatment with the new “life transforming” drug Kaftrio.

She attended a photocall with the Taoiseach in Government Buildings for the launch of national Cystic Fibrosis Day on April 8th. Micheál Martin and Minister for Health Stephen Donnelly posed for pictures with Robyn and her sister, Anna (9).

The Taoiseach brought Ashling and her daughters to see his office, while the conversation with Mr Donnelly centred on Robyn’s access to the new drug, Kaftrio, she said.

Now however, Robyn has been denied access to the drug because of a dispute over price between the HSE and the drug’s manufacturer, US firm Vertex.

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Robyn is one of about 35 children denied access to the drug, despite being told they would receive it this month. A further 140 children have been approved, a factor that the Kelly family say has devastated those approved for the life-changing treatment and those denied access to it.

The Kellys are supporting an online petition calling on the Government to make good on what they say was a commitment to supply Kaftrio.

Kaftrio has been used in adults and children around the world to tackle cystic fibrosis, which has severe effects on the lungs, the digestive system and other organs. The drug has been approved by the European Medicines Agency and Ashling said her daughter’s medical team described it as “the one we were waiting for”.

Phillip Watt, chief executive of Cystic Fibrosis Ireland, said Kaftrio was one of four new drugs that tackle the cause of cystic fibrosis rather than the symptoms, as other drugs have done.

The HSE deal had been warmly welcomed in 2017, he said, and while 140 children aged between six and 11 years are beginning treatment with the drug, “it seems there are 35 children in that age cohort whose specific genotypes were not listed in the original agreement, and they are currently being denied access to it”.

He called on the HSE and Vertex to settle the matter without further delay.

In a statement Vertex said its “long-term reimbursement agreement” with the HSE was composed in 2017 and “allowed Irish CF patients to be some of the first in Europe to gain rapid access to the latest approved Vertex medicines”.

But it said a small number of patients “in the six to 11-year-old patient population with specific genotypes who are eligible under the European Medicines Agency licence for Kaftrio, were not included in the original reimbursement agreement”.

Vertex said it is “looking to extend the agreement to include these patients and allow reimbursed access quickly, in a manner consistent with the existing contract”. The company said it stands “ready to resolve this as quickly as possible and remain committed to progressing these discussions with urgency”.

A spokeswoman for the HSE said it is committed to providing access to as many medicines as possible, in as timely a fashion as possible, from the resources available to it.

“HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds,” she said.

“There are formal processes which govern applications for the pricing and reimbursement of all new medicines, and new uses of existing medicines, to be funded and/or reimbursed.”

The spokeswoman said the application for pricing and reimbursement for this further subset of the licensed population for Kaftrio “remains under consideration with the HSE”.

“The HSE cannot make any additional comment on possible outcomes from the ongoing process at this time.”

The online petition is at https://chng.it/JjY87rNcqG