Children with cystic fibrosis ‘are pawns’ in drug price row, say doctors

Young children with cystic fibrosis (CF) are being used by a pharmaceutical company as “pawns for greater profit” in a pricing row with the HSE, according to doctors who treat them.

Twenty-one respiratory consultants have accused Vertex Pharmaceuticals of a “lack of integrity” over its failure to make its drug Kaftrio available to children aged been six and 11 in the same way as it is provided to older children.

Earlier this year, 140 children with CF were given access to Kaftrio, which can greatly improve their quality of life. However, 35 other children, aged between six and 11 but with a different genotype, are still waiting for access due to the price dispute between the HSE and Vertex.

In 2017, the HSE and Vertex struck a €650 million deal to provide the company’s life-changing drugs to more than 1,000 CF patients. However, Vertex is seeking additional payment for the use of Kaftrio among younger children.

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The company told The Irish Times the 35 children were not included in the original agreement “because it was not known at that time that our medicines would work in these patients”.

The doctors, who treat all the CF patients in the State, say they find it difficult to understand why Vertex is supplying its drug to one group of children but not another.

“Discriminating against this specific group of children while seeking higher profits during a protracted series of meetings over many months is totally unacceptable, suggests a lack of integrity and is inconsistent with the stated aim of Vertex of ‘working collaboratively and flexibly with governments and stakeholders to enable access,” the letter signed by the 21 doctors states.

They note “astonishing progress” has been made in transforming CF from a universally fatal disease to one where the vast majority of children diagnosed at birth can live “a full and productive life, not defined by disease”. This has been achieved through a “close relationship” between patients, families, medical teams and “our industry partners”.

“However this partnership is under threat because of the approach of Vertex, who, we believe, are effectively using this group of vulnerable children as pawns in negotiations for greater profits.

“This unilateral fracturing of the very close and productive relationship between partners threatens to derail much of the progress we have made in Ireland, and we cannot see how we can continue the very positive and productive engagement if this situation is allowed to persist,” the doctors’ letter said.

A Vertex spokeswoman said the company is committed to seeking a rapid resolution to the issue, in conjunction with the HSE. “The discussions remain the highest priority for Vertex, and we recognise the importance of finding a solution. These talks with the HSE remain constructive and active to find a path forward.”

On Thursday Cystic Fibrosis Ireland met Minister for Health Stephen Donnelly to urge him to consider allowing immediate access to Kaftrio for the 35 children, “without prejudice to a final agreement on pricing”.

The charity has also called for international arbitration to resolve the impasse.