A tough pill to swallow for the Cystic Fibrosis community

Pharma News: “Cystic Fibrosis doesn’t make the news a lot because our community is small, and our suffering is sometimes invisible,” wrote the Cystic Fibrosis (CF) community to the Hon Greg Hunt MP in April this year when the PBAC failed to recommend Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for Federal funding.

Cystic Fibrosis Australia has been running an advocacy campaign for Trikafta since 2020 with the aim of bringing hope to patients and their families. Trikafta pairs a CFTR modulator with Symdeko, which is itself a combination that includes another Vertex CF drug, Kalydeco. 

“Trikafta has a proven trial record and unmatched results for symptom management. We believe that the negotiations fell through on price alone, because the drug itself is a CF miracle…No one is saying that these are easy decisions, but surely the moral imperative here is undeniable. Trikafta is everything to us. It means survival and quality of life – the ability to leave our beds, to pursue a career and partake and contribute,” the community letter added.

Unfortunately the July 2021 PBAC meeting outcomes will continue to leave the majority of CF patients and their families disappointed.

According to a company statement “Vertex is very disappointed that the PBAC has not recommended government-funded access to Trikafta for all 2,200 eligible patients included in our PBAC submission. It has instead limited a recommendation to only a small subset of patients who are heterozygous for F508del in the CFTR gene with a minimal function (F/MF) mutation aged 12 years and older.

“This incredibly narrow recommendation would mean that 80% of eligible cystic fibrosis (CF) patients will be denied Government-funded access to this important medicine, which now has broad reimbursement in 19 other countries. Australians living with CF deserve the same opportunity for access.

“CFTR modulators have well-proven, long-term benefits for the patients that can access them. We disagree with restricting access based on PBAC’s hesitancy to recognise the long-term benefit of our reimbursed medicines. This only serves to delay access to Trikafta to the detriment of the CF community.

“In line with the deferral process, and in an effort to facilitate faster access, Vertex proposed a short-term arrangement that would provide immediate access to all eligible patients while further data are collected and other matters raised in the PBAC’s previous advice could be addressed. Unfortunately, this proposal was not considered by the PBAC.

“We remain committed to ensuring all eligible patients receive government-funded access to Trikafta as quickly as possible. We will continue our engagement with the Department of Health and the PBAC to find flexible and creative ways to achieve this.”

Vertex added that the company is also disappointed that the PBAC is calling into question the long-term benefit of CF medicines Orkambi and Symdeko (lumacaftor/ivacaftor and tezacaftor/ivacaftor).

“The long-term benefit of these medicines is well proven as evidenced by independent and Vertex clinical trial data and the support from the 29 other international Healthcare systems where patients have ongoing access to these medicines. This determination has also resulted in extremely restricted access to Trikafta.

“While the Department of Health’s submission to the Inquiry into approval processes for new drugs and novel medical technologies in Australia highlighted Managed Access Programs (with Orkambi as the example) as a potential solution for early access to innovative medicines; it is clearly not the case.”