New hope of Vertex drug for Irish children with cystic fibrosis as company comes under global pressure

HSE. Stock image: Frank McGrath

Eilish O Regan

February 07 2023 05:29 PM

Vertex, the pharmaceutical company which makes life-changing drugs for people with cystic fibrosis, confirmed today it has submitted a new dossier on Kaftrio to the health service arm here which assesses the cost and effectiveness of medicines.

The health technology assessment was submitted on December 21 and follows a long campaign by parents of children with CF aged six to 11 for access to Kaftrio with the HSE challenging the company on price.

A spokeswoman said Vertex had submitted the assessment to the National Centre for Pharmacoeconomics (NCPE) covering Kaftrio in combination with Ivacaftor for the cohort of children aged six to 11-year-old with certain mutations of the cystic fibrosis gene.

“We will work as quickly and collaboratively as possible with both the NCPE and HSE to support a timely review, as sustainable access for these 35 children remains a high priority.”

It comes as a global coalition of patients and their families, led by the grassroots cystic fibrosis community group Vertex Save Us, and the patient-led organisation Just Treatment, launched what they are calling a “fight back” against the drug company. They accuse the company of “pursuing a cynical and profit-hungry strategy that sacrifices the lives of tens of thousands of patients around the world in order to enrich corporate executives and investors”.

But Vertex’s monopoly rights are not absolute – World Trade Organisation agreements and national laws include a series of flexibilities designed to ensure intellectual property (IP) rights do not threaten citizens’ right to life, said the patients’ group.

In response a spokeswoman for Vertex said it has worked for over 20 years and invested billions of dollars to design, discover and develop medicines to treat the underlying cause of cystic fibrosis.

"We also continue to invest billions of dollars to discover and develop even better medicines for cystic fibrosis as well as medicines for those who don’t have disease modifying treatments today. We believe that intellectual property rights are critical to encourage and protect innovation,” she said. 

“We aim to provide our medicines to as many cystic fibrosis patients around the world as possible. We have formal reimbursement agreements in more than 40 countries outside of the US and our medicines have been used by patients in more than 50 countries.

"We continue to work actively to expand access including in lower income countries, recognising the complexities and access challenges in these markets,” she added.

She said that in In low-income countries which often have more limited healthcare infrastructures “we are looking at a variety of different approaches to access, including a product donation programme which we began piloting last year”.