Child patients will not receive cystic fibrosis drug due to cost

Study finds the use of the ‘wonder drug’ would cost €21m over a five-year period

Cystic fibrosis unit at St Vincent’s Hospital, Dublin. A ‘wonder drug’ used to treat adult cystic fibrosis patients has been rejected for use in a group of child patients.

Cystic fibrosis unit at St Vincent’s Hospital, Dublin. A ‘wonder drug’ used to treat adult cystic fibrosis patients has been rejected for use in a group of child patients.

 

A “wonder drug” used to treat adult cystic fibrosis patients has been rejected for use in a group of child patients after a study found it would cost more than €1 million per patient over a five-year period.

The National Centre for Pharmacoeconomics (NCPE) has recommended against approving the drug Kalydeco for use in the group of child patients aged two to five.

It said the use of the drug to treat the 18 children over a five-year period would cost €21 million.

The manufacturer, Vertex, had failed to demonstrate the drug’s cost-effectiveness at the price submitted, the centre said.

Cystic Fibrosis Ireland described the decision as very disappointing and frustrating for the families involved, who could benefit from “this groundbreaking therapy”.

“Accessing this life-changing treatment is essential as eligible patients stand to benefit so much at such a crucial time in their development, fighting a progressive, life-limiting disease,” said Philip Watt, chief executive of Cystic Fibrosis Ireland.

“We will be urging the government to enter into price negotiations as soon as possible. We would also urge Vertex to reduce the price of this important drug during these negotiations.”

Effects

The drug, which is currently available only to those over the age of six, increases lung function, slows the progression of lung disease and significantly reduces hospital admissions, according to Mr Watt.

In 2013, then minister for health James Reilly reversed a recommendation by the NCPE and approved Kalydeco for reimbursement under the State drugs schemes.

At the time, the estimated cost was €234,000 per patient per year.

Price talks between the HSE and Vertex over a separate cystic fibrosis drug, Orkambi, on a price under which the drug could be covered by the schemes, are continuing.

In June, the NCPE found against the reimbursement of Orkambi after calculating it would cost the health service €392 million over five years, or €160,000 per patient per year.