Use of "wonder drug" on cystic fibrosis patients could be reviewed

Tuesday 19 March 2024

The decision not to use a new "wonder drug" on some cystic fibrosis patients in Jersey could be reviewed.

Described as a "miracle" treatment by the NHS, and later by then-UK Health Secretary Sajid Javid is "life-changing", Kaftrio works by correcting a malfunctioning protein associated with a cystic fibrosis gene.

The NHS said it significantly improves lung function, helping people with the condition to breathe more easily, thereby "enhancing their overall quality of life".

In 2022, the MHRA made changes to its rules surrounding the use of the drug, allowing more than 1,300 children between six and 11 in England with cystic fibrosis to access the treatment for the first time.

But, the following year, the National Institute for Health and Care Excellent (NICE) said it was not value for money and would not recommend it for routine use on the NHS.

Health Minister Tom Binet was today questioned by Deputy Geoff Southern about whether he would agree that it was a "matter of urgency" to review whether cystic fibrosis patients carrying a specific gene mutation should be able to access the drug for a trial period of three months as an "exception" to NICE guidelines.

While Deputy Binet said that while it was not appropriate for him to intervene directly in clinical matters, he had asked the Medicines Optimisation Committee, which decides on the use of new medicines, to take "another serious look" at approving Kaftrio for patients carrying a specific gene mutation.

Pictured: Deputy Southern's question to Health Minister Binet.

The discussion in this morning's States Assembly meeting followed a written question from Deputy Southern earlier in the year, which revealed that the committee considered product licensing and NHS commissioning criteria for medicines and decided not to approve the use of a drug for patients with a specific gene mutation.

The Health Department follows the criteria for use set out in the NHS England commissioning statement for all cystic fibrosis drugs.

Deputy Binet said he hoped the Medicines Governance Committee would consider the matter at their next meeting.