The MAA has partnered with the Edith Cowan University to conduct research on exploring the lived experience of people living with Myasthenia Gravis. The research team is led by Associate Professor Christopher Kueh.

Our project focuses on understanding the experiences of individuals living with Myasthenia Gravis (MG) in Australia. We want to know about the journey of getting diagnosed with MG, living with the condition, and what a typical day looks like for those affected.

This information will help us create better support systems and improve overall health outcomes for people with MG.

Participants will be invited to attend short sessions on-line with researchers and then use an online whiteboard called Miro to record their experiences of living with MG.

Participants will be given assistance along with instructions from researchers on how to use Miro.

The use of Miro will allow researchers to understand the experiences of Australian people living with MG.

To express your interest, to participate or for more information on this research, please email A/Professor Christopher c.kueh@ecu.edu.au

The bold concept to have a physician captured, longitudinal data base reflecting real time evidence about treatment benefits for people with Myasthenia Gravis was first presented to the Myasthenia patient group in December 2016. The Myasthenia community committed to raising $40,000 to support this work. The project was known as eNID. At the 2017 Conference Dr Katherine Buzzard, who has been at the forefront of this work, explained the concept and by end of day the target had been met! Dr Buzzard and Dr Reddel continued to progress this project and by July 2020 additional funds had been raised inclusive of a total contribution of just over $100,000 from the Australian MG Community!

That feels like such a long time ago. Much patience was asked of our community but the outcome has been worth the wait. The eNID Project became MGBase. Please read on to understand more about how our Australian Specialists have taken the lead on this international opportunity!

MAA proudly supporting MGBase!

The MGBase Registry is the first global, observational registry dedicated to evaluating outcomes data in myasthenia gravis. ‘MGBase’ was launched in December 2021, addressing a critical gap in the collection of ‘real-world’ patient data for this rare disease. Owned and operated by the MSBase Foundation, the MGBase Registry is built on the same robust governance and IT infrastructure that underpins the highly successful MSBase Registry for multiple sclerosis.

MGBase is developed for clinicians and provides them with access to a comprehensive data entry tool and an online research platform, paving the way for collaborative, international myasthenia research that aims to enhance the quality of care and improve patient outcomes. Membership is free and open to all practising neurologists treating patients with Myasthenia.

As at February 2025, the international registry contained over 1050 patients and is growing daily. Over half of the total patient cohort is being contributed by Australian MGBase centres in Victoria, Western Australia, NSW, QLD and Tasmania. 154 members (Specialists) are engaged across 12 countries and 24 clinics. The data is already contributing to research projects.

Patient registries such as MGBase are powerful instruments for expanding our knowledge of diseases. They are particularly useful in rare diseases, such as Myasthenia, where they can provide the opportunity to pool ‘real world’ data to reach a sufficient sample size for epidemiological and clinical research. If you’re interested in having your clinical data contributed, please speak to your specialist about signing up to MGBase. A dedicated membership and IT team is available to support MGBase members. Visit the website for more information www.mgbase.org, or clinicians can contact info@mgbase.org directly for more information on how to join.

Today is our special day as the world recognises people with a rare disease such as Myasthenia. The MAA share the 2025 logo with you and perhaps you will share this email with others. For the rare disease sector there are challenges to gaining comprehensive and equitable care and to being understood. Celebrating today and telling others why you are celebrating is a step towards a better life for us all. Do embrace this day on behalf of everyone who travels life with a rare disease.

Rare Voices Australia again hosted a Parliamentary Luncheon to maintain a focus on rare conditions. It was held in Canberra on February 11th and the Myasthenia community were represented once again. We have become a familiar face to politicians, the RVA team, to other small support groups and to industry. Susan White supported by Donna Formosa proudly attended the 2025 event. It was a heartwarming day with multi-party engagement evident in all the speeches. The principals embraced by the National Strategic Action Plan for Rare Diseases (announced February 2020) are solidly cemented, and policy is reflecting the priorities captured in the action plan.

Please enjoy a selection of photos below from this significant day.

Visit:

• Rare Voices Australia
• The National Strategic Action Plan for Rare Diseases

Thank you to everyone who joined us for the webinar about new treatments and Pharmaceutical Benefits Advisory Committee (PBAC) submissions, and to all of you who made a submission and shared your lived experience and treatment preferences as part of the PBAC process. The PBAC meeting takes place in March, and we will know the outcomes of this meeting about 6 weeks later.

We’d like to share with you a bit about what the MAA submissions looked like. The key message of all submissions was how the new treatments offer a real opportunity to come closer to our goals of reducing the burden of living with Myasthenia Gravis (MG). This includes not having well-controlled symptoms or living with the fluctuations of MG. Other goals include improving the quality of life for Myasthenia patients and increasing the opportunities for Myasthenia patients to participate in work, to enjoy more family and community activities, to travel more freely, and to spend less time at medical appointments and more time out of hospital. Our vision is for each Myasthenia patient to have equitable and timely access to the best available treatment that most benefits them.

Our patient experience is diverse and varies among us and this is why the MAA supports a broad range of treatment options being available and accessible. The MAA also considered where there are gaps and unmet needs with current treatments and how the new treatments offer new targeted treatment pathways and flexible or convenient delivery options, compared to current treatments.

The growing evidence base from our research and surveys over the last few years was crucial for the MAA submissions. We thank you for contributing to this research, because this informed the MAA submissions on behalf of patients. Your input to each of these surveys made a tangible difference.

Some interesting statistics that represent our experience in Australia include the following:

• The MAA surveyed 194 MG patients in January 2024: 47% of those surveyed did NOT feel that their MG condition/symptoms are currently well-controlled and 75% felt that their MG, including side effects from treatments, is a burden.
• Research conducted by Janet Sansoni and colleagues in 2023 surveyed 280 MG patients and found that 58.3% of MG participants reported moderate to severe limitations in daily tasks and activities and 28.4% reported some limitations. Compared to the survey conducted in 2011, this survey also showed that workforce participation has not increased over time in Australia.
• More recently in January 2025, 64% of our webinar participants who answered a live poll question said they feel their MG should be better managed in terms of reducing the burden of living with the condition and/or improving their quality of life.
• 96% of the webinar participants feel that it is important to be offered new treatments (79% felt it is extremely important and a further 37% thought it is somewhat important).
• Finally, at our 2023 national patient MAA conference, we asked the patient audience in a live digital poll to share one key word that comes to mind when they think of MG. The most common words were challenging and frustrating.

For more details on our past and current research projects, please visit https://myastheniaalliance.org.au/research/.

Make sure you’re registered for MAA news and updates at https://myastheniaalliance.org.au/subscribe/. You can also contact us directly by calling 1800 802 568 or reach out to one of our state associations: MGNSW or MGAQ.

It is a busy time for Myasthenia and we thank you again for your contribution to all research and advocacy activities.

People may recall completing a survey way back in mid-2022 when researcher Dr Pakeeran Siriratnam was keen to understand more about our Covid experiences. He was delighted when 229 people successfully responded. After much time and effort Dr Sisriratnam’s paper was published in a prominent journal just prior to Christmas. He has responded to the MAA request for feedback.

The MAA thanks everyone who generously assisting with this project.

Doctor Siriratnam writes –

Our experience working with MAA:
We are extremely grateful to Susan and MAA members for their generosity in completing our survey. We found our MG community extremely responsive and motivated to help each other and advance scientific knowledge.

Impact of our work.
As the pandemic experience in Australia was vastly different to other countries (with very low rates of COVID infections initially and strict lockdowns), the experiences and voices of our patients was very important to share. The major findings of our study published in an international journal was that of the 229 patients who responded, our population were highly vaccinated and had lower rates of hospitalisation for COVID-19 than in other countries. However, COVID-19 infection during the period of the survey did pose risks and many of our patients experienced negative psychosocial impacts. We hope our paper helps clinicians be aware of the range of impacts from the pandemic for our community.

Public access to the paper can be found at (https://rdcu.be/d410h). The abstract is as follows-

Abstract

Introduction/aims: Previous studies have demonstrated high morbidity and mortality in patients with myasthenia gravis (MG) who acquired COVID-19. We aimed to identify the impact of the pandemic on MG disease control, treatment and quality of life.

Methods: A prospective observational cohort study was conducted to identify the impact of the COVID-19 pandemic on Australian patients with MG. We conducted an online survey through Myasthenia Alliance Australia from May 2022 to July 2022.

Results: Among the 229 patients who responded to the survey, most patients had three (80; 34.9%) or four (116; 50.7%) doses of the COVID-19 vaccines, 65 (28.4%) had contracted COVID-19, with 6 patients (9.4%) requiring hospitalisation. A large proportion of patients responded that the pandemic had no impact on their MG disease control (123; 53.7%) or treatment (111; 48.5%). Most patients felt the pandemic had either a mild (111; 48.5%) or moderate (50; 21.8%) impact on their mood.

Conclusion: Our study provides a snapshot of the types of impacts experienced by myasthenic patients during the pandemic, in particular the psychosocial effects. As we continue to live with COVID-19, clinicians should consider its various impacts in the holistic management of their patients.

It is a new year and we have a once in a generation opportunity for new treatments for Myasthenia Gravis! The Pharmaceutical Benefits Advisory Committee (PBAC) will assess 4 new treatments for generalised Myasthenia Gravis (gMG) at its meeting in March 2025. You can make a personal submission by 29 January 2025 highlighting your lived experience as a Myasthenia patient to add to the submission by the Myasthenia Alliance Australia (MAA) as the peak representative body for all Myasthenia patients in Australia.

Not sure about this unique opportunity to support new treatments for Myasthenia Gravis? We appreciate that you may have some questions or need more information before making your submission…

Possibly you are wondering what a FcRn or complement inhibitor is? Do you want to know more about these new treatments, do you want to understand how they may fit with the existing treatments or how utilising them may reduce the burden of this condition.  Would you like to understand why your submission is important and how to make it count?

Then you must register for the MAA webinar so that you can hear directly from expert neurologists and patient advocates. Speakers include A/Prof Stephen Reddel, A/Prof Stefan Blum, Dr Katherine Buzzard and Ms Ann Single from Patient Voice Initiative.

When? 5:30pm (AEDT)/4.30pm (AEST) on Wednesday 15 January 2025

Why?  Learn about the types of treatments on offer, how to make a successful submission to PBAC for treatments of your choice, and understand why your voice will make a difference. The webinar will also include time for a Q&A session with our speakers and MAA board members.

Don’t worry if you can’t make this date or time because the webinar will be recorded. You do still need to register to ensure you can access the recording after the event. If you are not certain about how to participate please seek support from a friend or family member. Everyone is welcome to register.

Please also check out the MAA guidance on PBAC and the treatments via the MAA website, keep following updates by subscribing here, or contact your state association.

We look forward to you joining us for this educational webinar organised by the MAA and we thank Argenx for their support to make it happen.

Register now!

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:

1. In the first part, you tell your story about living with MG and the current treatments you take/receive.
2. 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. 

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 (MAThe 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 Vyvgart® (efgartigimod alfa) and PBAC 

The Pharmaceutical Benefits Advisory Committee (PBAC) will assess a new treatment Vyvgart® (efgartigimod alfa) 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 such as USA, Europe, Canada, Japan and China. This PBAC meeting further considers the funding and prescribing criteria for gMG patients.

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 experienceswith 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 patients (and their carers or families/friends) can make a consumer submission to contribute to the PBAC evaluation of Vyvgart® (efgartigimod alfa). 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. Vyvgart® (efgartigimod alfa) is a FcRn inhibitor treatment. Please read more details about this treatment below.

When: Make a submission by Wednesday, 29 January 2025. 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 efgartigimod alfa 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 email directly 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:

1. In the first part, you tell your story about living with MG and the current treatments you take/receive.
2. 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 – this is what you are the expert in! 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 your 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 ways to manage your MG?
• If appropriate, and guided by your doctor, how do you feel about the potential to reduce other medications or treatments you may be taking for MG? What impact would this have on your general health and wellbeing?

Efgartigimod alfa** 

Efgartigimod alfa 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. A FcRn inhibitor binds to and blocks a protein in the body called FcRn. By blocking FcRn, efgartigimod alfa decreases the levels of IgG autoantibodies in gMG patients.

Efgartigimod alfa represents a new pathway in treating MG as it targets the actual mechanism of neuromuscular impairment. It is also a manageable option that empowers Myasthenia patients: a 1-hour intravenous infusion in treatment cycles of one infusion per week for 4 weeks. Further treatment cycles are determined by the treating clinician and can go longer beyond 4 weeks, depending on the individual patient need. 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 efgartigimod alfa 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.

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. 

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.

The Pharmaceutical Benefits Advisory Committee (PBAC) will assess resubmissions for treatments Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan) for acetylcholine receptor autoantibody positive (AChR+) gMG patients at its meeting in March 2025. Both treatments are already approved by the Therapeutic Goods Administration (TGA) for use and this PBAC meeting further considers the funding and prescribing criteria for gMG patients.

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 experienceswith 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.

What does the resubmission mean? 

Both treatments first went to PBAC for gMG in March and July 2024. They were not approved at the time as more information is required from the pharmaceutical companies sponsoring them. So, they are resubmissions for the March 2025 meeting.

Don’t worry if you made a submission already. Your submission for the previous PBAC meetings is still on the table and will again be considered at this next meeting. You may like to make a submission this time for either or both treatments for a variety of reasons: you may not have been diagnosed earlier this year, or you may have missed making a submission, or you may have some updated information or views you would like to share.

It is important to note that after these meetings in 2024, 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. More information will follow about the other 2 treatments.

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 Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan). 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. Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan) are complement C5 inhibitor treatments. Please read more details about these two treatments 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 for one 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 email directly 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:

1. In the first part, you tell your story about living with MG and the current treatments you take/receive.
2. 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.

Ravulizumab** 

Ravulizumab is a long-acting complement C5 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. Ravulizumab represents a new pathway in treating MG in a safe and fast-acting manner as demonstrated by its clinical trial. It is also a manageable option for MG patients; generally an 8-weekly treatment cycle of 45-minute infusions.

For more information, please see the Consumer Medicine Information, a TGA-approved document, and the following Australian Government link: https://www.healthdirect.gov.au/medicines/brand/amt,1405341000168107/ultomiris

Zilucoplan** 

Zilucoplan is a once daily, self-administered subcutaneous injection of a complement C5 inhibitor treatment. A subcutaneous injection is an injection into the tissue between the skin and the underlying muscle. Zilucoplan is being proposed as an add-on to standard therapy for AChR-positive generalised MG adult patients. Zilucoplan represents a new pathway to treat MG in a fast-acting and manageable manner. The daily self-administered injection can provide flexibility for MG patients by reducing hospital visits and for planning extended travel.

A key update since the July 2024 PBAC meeting is that Zilucoplan has been approved by the TGA, so please see the Consumer Medicine Information, a TGA-approved document.

The resubmission will focus on the treatment of REFRACTORY gMG (those of us who do not respond to current treatments adequately). The MAA strongly encourages this group of MG patients to make a submission for this treatment. 

Please also take a look at the previous MAA guidance on both treatments, including questions to think about and tips for writing a submission to PBAC from the Patient Voice Initiative (PVI).

• https://myastheniaalliance.org.au/information-about-making-a-pbac-submission-for-ravulizumab/
• https://myastheniaalliance.org.au/update-there-is-still-time-to-make-your-submission-to-pbac/
• https://myastheniaalliance.org.au/make-a-submission-to-pbac-about-a-new-complement-inhibitor-treatment-zilucoplan/

You can also view the public summary documents from the previous PBAC meetings here: https://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/psd/pbac-public-summary-documents-july-2024

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 mor 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. 

The Pharmaceutical Benefits Advisory Committee (PBAC) has published its agenda for March 2025 and there are 4 generalised Myasthenia Gravis (gMG) treatments for consideration. Two are resubmissions and the other two are new requests for PBS listings. You can see the full agenda here.

The closing date for consumer comments is Wednesday, 29th January 2025. The MAA will be making submissions on your behalf. The MAA is also here to help you make individual submissions by giving you relevant information and guidance, ensuring confident navigation of the process. We will be working hard throughout the Christmas-New Year break with a focus on telling a very strong story, hoping to secure new treatment options that are available as broadly as can be achieved.

We (the Australian Myasthenia community) are offered a unique situation with 4 products being listed. No new products have been offered for Myasthenia for more than 20 years. Gaining access to clinically trialled and targeted products is something worth speaking up for.

Make sure you are subscribed to receive MAA updates as soon as they are published.

To read more about PBAC and the Health Technology Assessment (HTA) process, see recent article here.