Kaftrio Campaign update
Following on from the exclusion of 35 children for the Kaftrio age 6-11 extension (those with minimal or unclassified plus F508del gene mutations) CFI has sought meetings/statements from both the Department and Health and from the manufacturers of Kaftrio. Following our request, VERTEX has agreed to meet CFI at the European CF Conference in Rotterdam on Friday 10 June and they have issued a statement (below). We remain hopeful of a meeting with the Minister for Health Stephen Donnelly TD, but we await confirmation of a date for this meeting.
CFI understands that there is still no breakthrough in the pricing row that has resulted in the exclusion of the 35 children. We have urged the Minister and VERTEX to resolve the issue before the issue is referred to the NCPE for a full Health Technology Assessment (HTA) as this will likely delay access. The letter from VERTEX indicates that this referral to the NCPE will now happen.
For general information, CFI publishes recent statements by the Minister for Health and VERTEX with the ‘health warning’ that a) the negotiations are in camera/confidential and CFI is not party to those negotiations and b) sensitive information is often omitted from public statements by both parties, in part related to point (a). In short, we may still not yet have the full detailed picture behind this dispute. CFI once again urges that the exclusion of 35 children from Kaftrio is resolved as a matter of urgency by both VERTEX and the HSE/Department of Health.
Statement by Minister Donnelly TD 24 May 2022 Following PQ form Deputy Jennifer Whitmore
Deputy Whitmore:
"I have a question for the Minister for Health. Some 35 children with cystic fibrosis, CF, between the ages of six and 11 have been excluded from accessing the life-changing CF drug therapy, Kaftrio. There was no expectation that these children would be excluded from this drug therapy and the news has come as a crushing blow for the parents and children. Will the Minister confirm if it is indeed a pricing dispute between the HSE and the pharmaceutical company, Vertex, that has resulted in this? What measures is he taking to resolve this impasse for these children?"
Deputy Stephen Donnelly
"The issue is very much of concern for the 35 children involved and their families. I am engaging with the Department and the HSE on this. As the Deputy will be aware, the HSE has statutory responsibility for decisions on pricing and reimbursement on medicines under the community drug scheme and for administration of this scheme. Reimbursement is for the licensed indications which have been granted market authorisation. What is important to me and, no doubt, to the Deputy is that the children get access to the drug they need. Intensive work is going on between the Department, the HSE and the companies involved to that end."
26th May 2022 Open Letter to the Cystic Fibrosis Community in Ireland
We wanted to share an update with the CF patient community in response to several questions that we have been receiving regarding access to Kaftrio® for a number of children with CF in Ireland.
We want to reassure the community that enabling fast access to our medicines for these children is our top priority and our teams are focused on finding a way forward to this as quickly as possible.
Since 2017, when the innovative long-term reimbursement agreement between Vertex and the Health Service Executive (HSE) was completed, approximately 1000 individuals living with CF in Ireland have gained access to the latest approved CF medicines.
The long-term agreement took into account everything we knew about our medicines and our pipeline for CF at the time, however, over the years that followed, since the agreement was signed, the knowledge and data regarding how our medicines demonstrate clinical value to CF patients has evolved.
During the past five years, we have included additional patient populations into the agreement following discussion and alignment with the HSE which enabled people with CF to get quick access. For example, the Kaftrio® 12 years and older population including those patients with a minimal (F508del CFTR mutation along with a minimal function genotype) or unclassified gene (F508del CFTR mutation along with an unclassified mutation) were added in as part of an update to the agreement in December 2019.
Since the beginning of this year, we have been working with the HSE to update the reimbursement agreement to ensure the broadest possible access for our medicines today and in the future. Our focus has been to provide the authorities with all the information required and we have put forward several solutions that would allow rapid access for these 35 children and other patient populations who are not explicitly currently included in the agreement, who may become eligible for our medicines in the future. This has included extending the agreement in a manner consistent with the existing terms.
May 2022 IE-32-2200053
Vertex Pharmaceuticals (Ireland) Limited Unit 49, Block F2, Northwood Court, Santry,
Dublin 9
DO9 T665 Ireland
The HSE now requires that Vertex follow the NCPE recommendation of a full health technology appraisal process for these 35 children. This could mean a delay.
Vertex remains committed to finding a quick solution for access for the 35 children currently impacted and other populations of CF patients who may be eligible in the future. We are ready to continue discussions with the HSE again as soon as possible, in the hope that they will reconsider expanding the current agreement to allow for the broadest possible access to our medicines today and in the future.
The Vertex Team