Patients waiting for the reimbursement of Vertex's breakthrough cystic fibrosis therapy TRIKAFTA will be disappointed with the 'outcome' that has just been published on the PBS website.
Patients should also be disappointed about the wording of the published outcome given it contains at least one statement that is not correct.
The outcome claims the company did not submit a proposal consistent with its previous request seeking the PBS listing of TRIKAFTA for the more than 2,000 eligible patients currently on the Australian Cystic Fibrosis Data Registry (registry).
In fact, Vertex did submit such a proposal ahead of the July meeting of the Pharmaceutical Benefits Advisory Committee (PBAC).
It was just set aside with decision-makers essentially creating their own submission.
The published outcome has revealed a qualified 'recommendation' for just a small fraction of the more than 2,000 eligible patients.
It looks more like a rejection but it enables the government to claim the therapy is recommended. The vast majority of patients have been denied funded access.
The company did not request this limited listing.
It appears the PBAC has produced this qualified recommendation as a stop-gap for the around 300 patients currently unable to access any of the company's other reimbursed treatments. Approximately one-third of these patients are currently treated with TRIKAFTA via the company's compassionate access program.
In fairness, the committee might be trying to mediate an outcome between the company and whatever parameters the government has imposed on its consideration of this life-changing treatment.
The company may be unable to accept the stop-gap recommendation for several reasons. For a start, because it denies funded access for the vast majority of patients.
The published outcomes from July have also revealed the PBAC requested that Vertex provide more data on ORKAMBI to its meeting in December this year. The therapy was listed on the PBS in October 2018.
There is a reimbursement relationship between ORKAMBI (lumacaftor and ivacaftor).and TRIKAFTA because of the Managed Access Program.
It is no secret that companies do not trust the system to always fairly and transparently manage these relationships.
So, given the system's history of 'gaming' Managed Access Programs to its advantage, it would be understandable if Vertex chose not to accept the stop-gap recommendation for TRIKAFTA with the prevailing uncertainty on ORKAMBI.
Accepting the recommendation would simply mean the vast majority of patients continue to be denied access. It would also certainly make any future expansion to these patients more difficult because of the risk officials 'game' the reimbursement relationship with ORKAMBI.
The company's response
In a statement, the company said, "Vertex is very disappointed that the PBAC has not recommended government-funded access to TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) 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 said it was also disappointed the PBAC called into question the long-term benefit of ORKAMBI and SYMDEKO.
"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," it said.
Comment
At what point does a system that has veered so far from its founding principles and operating framework become irredeemable?
At what point will advisory committees like the PBAC tell the government and its officials that 'enough is enough'?
At what point do individual committee members see how their expertise is being co-opted by decision-makers to give credibility to and mask what are simply procurement outcomes?
The fact is that health minister Greg Hunt can take the first step to redeeming this system by accepting the PBAC's 'recommendation' on TRIKAFTA. In doing so, he can then set aside the 'qualified' advice and find a way forward to fund this therapy for all eligible patients.
The PBAC's 'qualified' advice has no legal standing and the minister can simply set it aside.
It would not undermine the PBAC. On the contrary, it would help ensure it is able to operate within its actual decision-making framework. Of course, that assumes it has not been directed to operate within certain parameters.
The government through the minister can impose parameters on the PBAC, directly and indirectly, but they should be in the public domain.
This issue on cystic fibrosis is seemingly complex but also very simple given it comes down to the system's reluctance to fund access for all eligible patients.
The processes and interdependence through Managed Access Programs are just the 'theatre' the system leverages to give some credence to the claim it is evidence-based.
It is important to remember that Australia is significantly alone on TRIKAFTA. Comparable countries around the world acted quickly to fund the therapy and without all the fuss of this 'negotiation by submission'.
This outcome simply gives a cynic the impression of a government trying to gain 'leverage' in an ongoing negotiation rather than ensure patient access to a new therapy.
In the end, Australians with cystic fibrosis will suffer the consequences of a system that now subordinates the need for timely access to new health technologies in favour of achieving certain procurement outcomes.
The issue of 'affordability' when it comes to PBS decision-making is a fine balance, at the best of times, and we are not in the best of times.
The irony is that it all started well for TRIKAFTA in Australia.
The Department of Health nominated the therapy to pilot its TGA-PBAC 'alignment' project. It lauded the ORKAMBI Managed Access Program in its submission to the parliamentary inquiry into approval processes for new medicines and novel medical technologies. The PBAC quickly accepted its benefit as a treatment for cystic fibrosis.
Yet here we are in a situation that must look familiar to so many Australian patients. Those suffering chronic migraine, atopic dermatitis and any number of other conditions. They have seen the emergence of new innovations but had to wait for funded access while the system twists itself inside out through health technology assessment processes that are becoming not much more than a 'penny-pinching' theatre.
There must be a better way.
