The Australian Myasthenia community have a once in a generation opportunity to support new treatments for generalised Myasthenia Gravis (gMG) which are listed on PBAC’s March 2025 agenda!
The Myasthenia Alliance Australia (MAA) encourages Myasthenia patients and your carers or families/friends to consider making a personal submission highlighting your lived experience by 29 January 2025. Why? Because the patient voice matters.
Please also register for the MAA webinar on Wednesday 15 January 2025 so that you can hear directly from expert neurologists and patient advocates. You will learn about the types of treatments on offer, how to make a successful submission to PBAC for medications of your choice and understand why your voice will make a difference. It is ok if you cannot make this date because the webinar will be recorded, but you do need to register. We thank Argenx for supporting this webinar.
New treatment Rystiggo® (Rozanolixizumab) and PBAC
The Pharmaceutical Benefits Advisory Committee (PBAC) will assess a new treatment Rystiggo® (Rozanolixizumab)for acetylcholine receptor autoantibody positive (AChR+) gMG patients at its meeting in March 2025. This treatment is still under evaluation by the Therapeutic Goods Administration (TGA) but has been approved by health authorities in other markets including the USA. This PBAC meeting further considers the funding and prescribing criteria for gMG patients in Australia.
The MAA will make a submission on your behalf as the organisation representing all Myasthenia patients in Australia. Individual submissions add to this by demonstrating the diversity of our experiences with Myasthenia and the importance of having different treatment options to give us the best care and quality of life. No matter what form of Myasthenia you have, your feedback is valuable. We appreciate this may mean you have to think about some tough or difficult times and experiences, so please allow yourself space to do this or talk to someone about it.
It is important to note that after meetings in 2024 about other new treatments for gMG, PBAC has recognised the high clinical need for effective therapies for gMG. This is reinforced by 4 new treatments for gMG being on the March 2025 agenda. Please see MAA guidance on the other treatments via the MAA website.
Making a submission to PBAC
Who: Myasthenia Gravis patients (and their carers or families/friends)can make a consumer submission to contribute to the PBAC evaluation of Rystiggo® (Rozanolixizumab). PBAC is the regulatory body that recommends new medicines for listing on the Pharmaceutical Benefits Scheme (PBS) based on medical conditions, clinical effectiveness, safety, and cost-effectiveness.
What: This is a once in a generation opportunity for new treatments for MG. Rystiggo® (Rozanolixizumab) is a FcRn inhibitor treatment. Please read more details about this treatment below.
When: Make a submission by Wednesday, 29 January 2024. We suggest jotting down your thoughts for now, reading the guidance such as this provided by the MAA, and then attending our webinar on 15 January 2025. This still allows 2 weeks to finalise and lodge your submission before the closing date.
How: You can complete the online form here. Please note that you make a submission for each of the treatments separately, not all together. You can make a submission just for Rozanolixizumab or all 4 treatments and we encourage you to think about your preferences across them all.
If you have issues with using the online form, you can send an emaildirectly to commentsPBAC@health.gov.au. If you have difficulties accessing or using technology, please consider asking for help from family, friends or your local librarians.
Why: Because the patient voice matters. The MAA believes this is a once in a generation opportunity to share our voice and lived experience with national decision makers for new treatment options for MG. We want to ensure they are available, accessible, and affordable. This aligns with Priority 2.4 of the National Strategic Action Plan for Rare Diseases: Enable all Australians to have equitable access to the best available health technology.
What should you consider when making a submission?
Your submission to PBAC is guided by 5 questions and can be broken down into two parts:
- In the first part, you tell your story about living with MG and the current treatments you take/receive.
- In the second part, you focus on the advantages and disadvantages of the specific treatment.
This means you share your experience of living with MG. You do not have to have had or tried using the treatments to make a submission and you do not need to discuss the technical or scientific detail.
Talk about your unmet needs with current treatments, the desired benefits of the new treatment, and the outcomes that matter most to you as a patient… if you can imagine any treatment providing you with better management of your symptoms and better quality of life, this is where you can talk about it!
Have courage and clearly state your preferences when it comes to your treatment – this could be comparing benefits as well as highlighting any adverse events or side effects that are less acceptable to you than others. You should also explain how the burden of your disease can be reduced.
Some questions to think about (you don’t have to answer them all!):
- Does your current treatment come with a high burden and what does this look like?
- What are your symptoms and how do you manage them? Are they well-managed?
- How does MG impact your daily life: your ability to go to work or school or to have a social life? Are you isolated at home, unable to prepare meals or do housework, or did a family member have to give up work to care for you? How long does it take you to get ready to go out? How many days do you take off work for medical appointments?
- What adjustments have you had to make in your life since you were diagnosed with MG?
- Is your MG fluctuating, making you feel uncertain, or do you feel you do not have control of your MG?
- Are you seeking a more manageable treatment option in terms of less intrusive delivery or minimal side effects? Are you seeking innovative waysto manage your MG?
- If you use IVIg/PLEX to manage your gMG symptoms, how does this affect your daily activities? How easy do you find it to access these therapies?
- Are there any other experiences with current available treatments that you wish to highlight?
Rozanolixizumab**
Rozanolixizumab is a neonatal Fc receptor (FcRn) inhibitor treatment that is being proposed as an add-on to standard therapy (including immunosuppressants like corticosteroids, methotrexate, mycophenolate, and azathioprine) for AChR-positive generalised MG patients. FcRn inhibitors target the neonatal Fc receptor (FcRn) to reduce circulating levels of immunoglobulin G (IgG).
Rozanolixizumab represents a new pathway in treating MG by targeting the actual mechanism of neuromuscular impairment. It can also be a manageable option for MG patients: a 6-weekly subcutaneous infusion that can be administered at home with nursing support. A subcutaneous injection is an injection into the tissue between the skin and the underlying muscle. Not all patients respond in the same way to the same treatment, so options are important.
We encourage you to do your own research if you feel comfortable doing so. For example, you can search information available online for the markets that have approved Rozanolixizumab for Myasthenia patients.
Please also take a look at this previous MAA article on PBAC:
https://myastheniaalliance.org.au/hta-and-pbac-what-who-are-they-and-what-does-it-mean-for-us/
We emphasise that this is a once in a generation opportunity! Keep following updates via the MAA website (subscribe here) or your state association. State association members can also access the discussion about treatments from the 2023 MAA conference (please contact your state association if you need more information about this access). And don’t forget to register for our webinar on Wednesday 15 January 2025 to learn more about the types of new treatments and how to make a valuable submission so that your patient voice is heard.act your state association if you need more information about this access). And don’t forget to register for our webinar on Wednesday 15 January 2025 to learn more about the types of new treatments and how to make a valuable submission so that your patient voice is heard.
Kind regards,
Susan White (MAA Chairperson)
Natalie Windle (MAA Secretary & MGNSW President)
Carol Buchanan (MAA Board Member & MGAQ President)
More information and tips about making a PBAC submission can be found through the Rare Voices Australia (RVA) and Patient Voice Initiative (PVI) websites:
https://rarevoices.org.au
https://www.patientvoiceinitiative.org and
https://www.patientvoicehub.org/training/index.cfm?event=page.login
**Please note the MAA and state associations do NOT provide medical advice regarding medications or treatments for individuals. Decisions about whether Ravulizumab or Zilucoplan will be suitable for you as a patient must be discussed with your doctor or health professional. The focus of these submissions to PBAC is to describe our patient perspective of living with MG and where/how/why we feel we have unmet needs, uncertainty, and ongoing burden with current treatment options.
