In August last year, Vertex was roundly criticised for indicating it would not accept the PBAC's July recommendation for cystic fibrosis therapy TRIKAFTA.
The company was publicly criticised by health minister Greg Hunt and one patient group that seemed oddly supportive of limiting funded patient access.
The company should have been thanked and congratulated.
If accepted, the recommendation would have provided reimbursed access to the breakthrough therapy for less than 15 per cent of patients included on the Australian Cystic Fibrosis Data Registry (registry).
The company said 'no' given the vast majority of the 2,200 patients on the registry would have been denied access.
It might have been easier for Vertex to just give in, accept the recommendation and avoid the public opprobrium, but the system would have been worse off as a result. Unless you think limiting patient access and less spending on medicines is a good outcome.
It is hardly unreasonable for a company to suggest Australia fund TRIKAFTA through the PBS in a way broadly consistent with other countries.
The company has been cautious and respectful in its public statements. It is not publicly campaigning, as it has in the past, in a way that seeks to proactively and directly pressure decision-makers.
Decision-makers and the system do appear to be feeling the company's stance as pressure. Is that because it is so unaccustomed to a company standing on principle?
In fact, it has been decision-makers and the government that have selectively released confidential information and sought to co-opt groups to pressure Vertex into accepting poor outcomes for patients.
The industry cannot criticise given its own predilections when it comes to information and patients. Yet, unlike decision-makers and the government, the industry makes no claim to purity.
The reality is that accepting the July recommendation would have just made it even more difficult to achieve the objective of securing reimbursed access for the vast majority of patients. This is because the system is now characterised by a dynamic that conceals a price negotiation behind a health technology assessment process.
Obviously, the pressure on decision-makers to recommend, act and properly reimburse a medicine is much diminished once it just appears on the PBS for a tiny subset of patients.
Vertex has taken a stand that reflects the interests of patients and the system.
People might scoff at this assertion but its decision obviously came at the expense of a rapid listing, albeit for a small number of patients, and revenue last year.
Not every medicine represents a life-changing innovation but they are all system-defining in how they navigate the process.
Companies act as proxies for patients in this system and they should consider themselves custodians. Vertex appears to understand this responsibility.
Accepting a system-defining outcome that only serves to entrench limits on patient access or parsimonious budget policy has wider and long-term implications. Concession might deliver a short term benefit but companies should always contemplate the extent to which accepting an outcome undermines the system.
The industry's competitive dynamic and commercial imperative makes it hard but more than ever we need leaders that act as custodians.
The minister's criticism of Vertex last year belied the fact he could have just exercised his lawful authority by accepting the PBAC's recommendation but reimbursed the therapy for all 2,200 patients.
This action would have ensured the government met its stated policy commitment of implementing all PBAC recommendations.
Unfortunately, the company was forced to resubmit to the December PBAC meeting for all patients.
At that meeting, the committee effectively amended its July recommendation to include all patients. Yet does anyone really believe that would have happened had the company succumbed to the pressure and public coercion by accepting the July recommendation?
The December outcome on TRIKAFTA might represent somewhat of a system back down from July but it remains an indictment of PBS decision-making given the continued refusal to make it simple.
The outcome's 'sting' on budget impact could be a serious barrier to implementing the recommendation.
In a statement, the company said it welcomes the recognition that all eligible patients should have reimbursed access to TRIKAFTA.
"However, it is disappointing that despite providing recent additional clinical data that supports the long-term effect of TRIKAFTA and applying health economics methodology consistent with prior PBAC advice, the outcome has come at the expense of recognising the value of the medicine and its impact on patients," it said.
"While there is still considerable work to do by all stakeholders in order to reach an agreement given the conditions related to the recommendation set out by the PBAC, we are committed to doing all we can to move the process along and have already initiated the next step.
"TRIKAFTA has been available internationally for up to two years. More than 25 countries, including countries with similar HTA systems to Australia, such as Canada, the UK, France, Germany, and the Netherlands, have recognised the clinical effect and value TRIKAFTA brings and funded the therapy for all eligible patients. Australian patients deserve the same," added the company.
Minister Hunt now has another opportunity to do his job and deliver patient access to this recommended medicine.
This might require a wider consideration than just PBAC advice but, in doing so, the minister could take the opportunity to contemplate what this entire process says about current PBS decision-making.
More companies can help trigger this contemplation by following the lead of Vertex and saying 'no'.
Patients and clinicians might think it counter-intuitive but it should never be forgotten that New Zealand's PHARMAC, which deplorably makes a public virtue out of achieving budget outcomes at the expense of patient need, managed to take hold and has existed for so many years because companies accept the way it operates.
The actions of more companies in not accepting increasingly budget-driven outcomes will be a far better driver of change than the much-hyped health technology assessment review that appears close to pointless in the current context of budget-driven outcomes.
More companies standing their ground, on principle and against terrible outcomes that only deny the real needs of patients, is the best vehicle to drive change. It would also help liberate the PBAC from this policy or ideological focus on budget impact.
It will not be easy and it will create conflict. Yet what is the alternative? How does accepting a bad system-defining outcome today make things better for patients tomorrow?
