Please prove me wrong

I want to challenge my perspective and broaden my understanding

As I look out the window of my apartment at the flag of Parliament House on the horizon, I contemplate the events that will take place tomorrow in that very building. I am both excited and nervous. My mind rushes. How will I feel tomorrow? Will I have enough energy or will I be putting all of my effort into keeping it together? How do I pace myself? Will that be enough? Should I wear a suit and tie? Where is my suit? Do I have a tie that matches? Should I go for a run after the event, that could be a good way to kick-off my Chapter Three running. Will my local senator David Pocock be there, I wonder whether he might come for a run? Probably should have reached out earlier…

Dear readers, you may be asking yourself at this point what has got Tim in such a tizz? 

Tomorrow I have the privilege to attend the The Rare Voices Australia (RVA) Parliamentary Event being held at our National Parliament House in my hometown of Canberra. This will be my first public event attending as an official RVA Ambassador. I will be joining five other RVA Ambassadors from around the country. Together we’ll be representing the estimated two million Australian’s living with a rare disease to our Federal Ministers, Parliamentary Advisors and Staff, Rare Disease Researchers, Pharmaceuticals Representatives and Patient Advocacy Bodies.

This is an annual event, but the first time it has been held in person since 2020. These events are critical for building and fostering relationships between Federal Government, stakeholders and the rare disease community. This year’s event is also special as it marks 10 years since RVA was founded.

Since its inception, RVA has helped the government to identify the specific needs of the rare disease community from the perspective of the rare disease community. This culminated in the co-development of the National Strategic Action Plan for Rare Diseases (the Action Plan) — the first of its kind in Australia. The Action Plan outlines the principles and actions required to improve health and wellbeing outcomes for Australians living with a rare disease. Now in its implementation phase, the Action Plan has the potential to radically change the landscape of rare disease care.

One area of particular interest to me is outlined in the Action Plan’s introduction, highlighted below

There are also cases where a medicine that will help a rare disease is only subsidised under the Pharmaceutical Benefits Scheme (PBS) for a common disease, meaning rare diseases patients have to pay more for the exact same medicine.

This is a challenge I can directly identify with as a patient. Tocilizumab, a biologic therapy, is widely considered one of the most effective treatment options for Still’s patients (1, 2, 3, 4). Over the past six years I’ve seen the profound difference that this treatment can have on disease control and progression. I’ve observed that those unable to access biologic therapies, like Tocilizumab, are more likely to experience severe chronic disability and poorer wellbeing. Contrastingly, those with access to biologic treatments have higher remission rates and better long-term outcomes. Unfortunately, from my limited understanding of the PBS, it is not available or easily accessible for Adult-Onset Still’s patients here in Australia. However, it is available for more common conditions such as Rheumatoid Arthritis and Juvenile Idiopathic Arthritis, both conditions that have similar pathologies.

But why, I ask myself?

I have a few ideas, but before discussing these, I must acknowledge my limitations. I am not an expert and I can at this point only comment on my interpretation from the perspective of a patient. This brings me to the title of this article Please Prove Me Wrong. I often catch myself looking for information that validates my perspective and then I stop there, maybe feeling like this confirms or justifies my position. In Chapter Three I want to do the opposite. To build my understanding of the barriers and mechanisms at play I want to find information that challenges, corrects or broadens my below understanding of the PBS. So please, if I’m off the mark, reach out and let me know. I want to learn.

1. Treatments for rare diseases are often not backed by large enough research studies or conducted on sufficient populations sizes to meet the PBS’ rigorous requirements for funding. For more common diseases these rigorous requirements are absolutely necessary to ensure the integrity of the system. However, the very nature of rare and ultra-rare diseases means that the data and studies required to get funding are either inadequate or simply don’t exist. Additionally, producing treatments or conducting studies on rare diseases often lacks the commercial viability to make things happen.

2. Price, these treatments are not cheap. Tocilizumab can costs upwards of $850 a month and patients may require ongoing treatment for the rest of their lives. When handling a finite budget these more expensive treatments may simply be cost-inhibitive without sufficient studies that back their efficacy (taking us back to problem 1).

3. Current legislation just can’t handle fringe cases, like rare diseases, very well as a consequence of the cycle between points 1 and 2.

With my limited understanding of the PBS I would say that it was fair. To over-simplify a complex system I would describe the current PBS as everyone needs to check the same boxes, and if you do then you get funding. This is fair. However, a fair system doesn’t acknowledge everyone’s starting point. For some patient groups checking the boxes is easy, for others it simply will never happen. So what we need is an equitable PBS. A system that acknowledges that all patient groups are not the same and has mechanisms in place to support them to access treatment if they don’t fall into the typical pathway. One example of this is the momentum that is building around new legislation that supports the development of orphan drugs (drugs that are not developed by the pharmaceutical industry for economic reasons but would meet a public health need) or the novel use of existing drugs (like Tocilizumab) for rare diseases (like Still’s).

There are holes in the system. But that is why tomorrow is so humbling, our federal government and the wider rare disease community are listening and want to facilitate change. It will take time, but they’re listening.

I am a complete novice when it comes to advocacy and knowing how to be an effective and active political citizen — I really am learning this as I go. The parliamentary event tomorrow is both exhilarating and totally petrifying. But my hope by attending this event is the same as my purpose for Running Rare, to encourage conversation and to try and humanise deeply complex issues. My specific condition may only affect 1 in a million, but tomorrow with the five other RVA ambassadors we stand up and provide a face for the two million Australians living with a rare disease.