There is something profoundly wrong in this country that patients are now writing open letters pressuring companies to give their products away for free because decision-making is so cheap when it comes to new therapies.
It is understandable but how did we get to this point?
Vertex is under public pressure to extend enrolment to its program under which it provides patients with so-called 'compassionate' access to its cystic fibrosis therapy TRIKAFTA.
When did Australia, one of the richest countries in the world, get to the point where patients are forced into this situation?
It is tragic but why should we be surprised?
Maybe the federal government, which is the entity responsible for funding medicines in this country, could actually do its job and make a humane decision.
TRIKAFTA has been recommended for reimbursement through the PBS but it appears barely viable given it reads like a budget document prepared by the Department of Finance.
For context, Australia is now competing with New Zealand to be virtually the last developed country in the world to fund this innovative therapy.
Apparently, Australia and New Zealand are right and every other country is wrong.
Seriously?
How arrogant is decision-making in this part of the world that it believes it can secure the lowest pricing outcomes globally without any consequences for patients?
The federal government and its decision-making processes are prioritising budget considerations to the extent patients are now publicly pressuring a company to extend an access program.
The Pharmaceutical Benefits Advisory Committee cannot disassociate itself from the delay given it delivered the recommendation that has led to this situation.
These patients are just the latest to experience the direct consequences of this 'budget first' mindset. Think patients with any number of cancers, chronic migraine, dermatitis and spinal muscular atrophy.
Babies with what in many cases is a fatal disease are being forced to wait for funded access to a potentially curative gene therapy while the federal government negotiates a price. How well is Australia doing?
This is what should frustrate and even anger patients. They are being forced to wait so the federal government can achieve its budget objectives while hiding behind a process that has a minimal relationship with its stated intent.
Remember, the PBAC only recently prioritised the urgent recommendation of a COVID-19 treatment the federal government had already paid for and taken delivery of.
In the case of that treatment, the convenience of PBS funding for the supply chain was considered an 'urgent public health need'. Cystic fibrosis? Patients can wait while the federal government achieves its budget objectives.
It is entirely legitimate for patients to pressure a company. However, the company is not the villain in this case. It is a decision-making process that very simply prioritises budget outcomes over patient needs. In other words, it does not want to pay.
Of course, this can all be fixed tomorrow if the health minister actually chose to do his job.
Minister, do your job.
