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.

The MAA has partnered with the University of the Sunshine Coast to conduct research on Myasthenia Gravis and people’s ability to work. The research team is led by Dr. Michele Verdonck.

MG can significantly affect people’s ability to work. Little is understood about the factors that support or hinder people with Myasthenia wanting to work. We are seeking your valued input as a person living with MG. We are conducting research aimed at investigating the factors which impact someone who is living with MG and their ability to engage in and maintain paid work.

Participants are invited to complete an anonymous online survey, which will take approximately 30 minutes to complete. We are hoping to use the findings from this research to help improve the working lives of those diagnosed with MG through identifying the enablers and barriers commonly experienced.

For more detailed information and to access the survey, please use this link: https://uniofsunshinecoast.syd1.qualtrics.com/jfe/form/SV_b8zGLyrsXJWYEwC

Please also find attached the Research Project Information Sheet for more details. If you have any questions or concerns, please contact the Chief Investigator, Michele Verdonck, using michele.verdonck@usc.edu.au. If someone you know may also be interesting in participating in this study, please feel free to share this email.

Ethics Approval: S242004

The MAA is proudly celebrating ten years with all our keen supporters by sharing a few words from the three founding members who have been an integral part of this wonderful decade of the alliance.

Please do view the timeline included here. It reflects the key components of our history. We are sure that you will find the variety of content inspiring and diverse.

Thank you to all our MG Community for their continued support.

Together we are a strong and influencing voice!

“Who would have thought ……..” is my catchcry! It has been an amazing ten years of new, interesting and purposeful experiences where we are confident that we have made a difference for the Australian Myasthenia community.

It has also been a joy to work with such wonderful team members. A small but mighty group. Each volunteer offers so much to this organisation and they offer so much of themselves and their time.

There is great potential for the future endeavours of the MAA.

Susan

After a very detailed and concise planning process the Myasthenia Alliance Australia (MAA) was founded in 2014.  This group, which had a clear focus on the national agenda, was made up of the incorporated state associations.

As a foundation member of the MAA I have seen it grow from very humble beginnings to a highly strategic group with strong affiliations with government, pharmaceutical companies and other not-for-profit advocacy organizations.

The work of this alliance cannot be underestimated.  The work with the state associations to raise needed funds for the ‘build’ of MGBase, the ongoing advocacy with members of parliament in relation to the provision of services and products for people living with MG, the ever-increasing  recognition by pharmaceutical companies, the strong affiliation with both internationally and nationally recognised lead neurologists and the involvement in a wide breadth of research are just a broad indication of what the MAA has, and continues to, achieve.

I am very proud to have been a part of such a widely-recognised and proactive small group of individuals constantly working to improve the lives of people living with MG and their families.  I know that the MAA will gain further recognition into the future.

Carol

As a founding member, starting out I did not know what to expect, but very pleased we are still going strong and are supporting the MG community.

Glenda

Help Shape the Future of gMG Care – Market Research Closing Soon!

The MAA respectfully requests patient support in further telling the story of their experiences with all forms of Myasthenia. Please note, we are very pleased that this survey can be completed via a hardcopy option. Please call Libby on 0407 110 204 to arrange this.

Late last year Alexion asked several people living with MG to participate in an online insight gathering project that aimed to better understand the current patient and doctor dynamics so that they can help support people living with gMG by addressing unmet needs at different points in time along the patient journey.

Several members of our community put up their hands and shared their experience which was incredibly important in shaping a real world understanding of life with MG and has assisted Alexion’s efforts to bring access to new treatment options.

The main takeaways identified gaps in what patients may need or expect and their clinical experience:

• The disconnect between HCPs and people living with MG is very real
• All people living with MG interviewed recall difficulties and challenges with HCPs at some point in their journey – especially early on
• Some doctors can downplay the impact of MG which may cause them to appear ignorant or dismissive
• People living with MG can feel underwhelmed with their healthcare experiences, under-prepared and forced to advocate for themselves
• People living with MG who are not managed by MG specialists can face a more challenging journey
• The physical and emotional rollercoaster of MG can leave many people living with MG feeling isolated and socially outcast
• Managing side effects can prevent people living with MG from reaching their own personal goal of wanting to just feel “normal”
• It can take years to get to a point where people living with MG feel some level of satisfaction with treatment
• Doctors are focused on clinical markers to judge success more often than what a person living with MG might consider their own treatment or health goal

Whilst much of what was shared and learned from the exercise will not be new to many of you, the findings have helped Alexion to develop a more robust survey that will help more people living with MG validate the gaps raised in the initial phase of the project.  

The purpose of this survey is to get to an even deeper understanding of the impact of gMG on the lives people living with MG and validate the gaps needs.

The self-completed online survey will take participants about 15 minutes. We have partnered with Elbow Insights and Strategy, a market research agency that specialises in this area, to help us develop and distribute the survey.

We are hoping we can have your help in sharing this research with community members so that those interested can participate in the survey.

If you are interested in helping us, you can use the link below to preview the survey or contact us to get your personalised link. Please note participants who use the link below needs to be completed in one sitting.

https://rebrand.ly/1816Pearl2

If you are unable to do the survey online, then please reach out to Libby @ Alexion on libby.noble@alexion.com or text her on 0407 110 204, and she will organize to send you a hard copy.

Dear MGNSW community

The MAA (Myasthenia Alliance Australia) respectfully requests patient support in further telling the story of their experiences with all forms of Myasthenia. Please note, we are very pleased that this survey can be completed via a hardcopy option. Please call Libby on 0407 110 204 to arrange this.

Late last year Alexion asked several people living with MG to participate in an online insight gathering project that aimed to better understand the current patient and doctor dynamics so that they can help support people living with gMG by addressing unmet needs at different points in time along the patient journey.

Several members of our community put up their hands and shared their experience, which was incredibly important in shaping a real world understanding of life with MG and has assisted Alexion’s efforts to bring access to new treatment options.

The main takeaways identified gaps in what patients may need or expect and their clinical experience:

• The disconnect between HCPs and people living with MG is very real
• All people living with MG interviewed recall difficulties and challenges with HCPs at some point in their journey – especially early on
• Some doctors can downplay the impact of MG which may cause them to appear ignorant or dismissive
• People living with MG can feel underwhelmed with their healthcare experiences, under-prepared and forced to advocate for themselves
• People living with MG who are not managed by MG specialists can face a more challenging journey
• The physical and emotional rollercoaster of MG can leave many people living with MG feeling isolated and socially outcast
• Managing side effects can prevent people living with MG from reaching their own personal goal of wanting to just feel “normal”
• It can take years to get to a point where people living with MG feel some level of satisfaction with treatment
• Doctors are focused on clinical markers to judge success more often than what a person living with MG might consider their own treatment or health goal

Whilst much of what was shared and learned from the exercise will not be new to many of you, the findings have helped Alexion to develop a more robust survey that will help more people living with MG validate the gaps raised in the initial phase of the project.

The purpose of this survey is to get to an even deeper understanding of the impact of gMG on the lives people living with MG and validate the gaps needs.

The self-completed online survey will take participants about 15 minutes. We have partnered with Elbow Insights and Strategy, a market research agency that specialises in this area, to help us develop and distribute the survey.

We are hoping we can have your help in sharing this research with community members so that those interested can participate in the survey.

If you are interested in helping us, you can use the link below to preview the survey or contact us to get your personalised link. Please note participants who use the link below needs to be completed in one sitting.

https://rebrand.ly/1816Pearl2

If you are unable to do the survey online, then please reach out to Libby @ Alexion on libby.noble@alexion.com or text her on 0407 110 204, and she will organize to send you a hard copy.

Recently the Pharmaceutical Benefits Advisory Committee (PBAC) announced the outcome of their July Meeting. The MG community were keenly waiting to hear the PBAC Outcomes as two new medications, Raviluzimab and Zilucoplan, had been reviewed. Regrettably both medications received a ”Not Recommended” verdict. The PBAC did recognise the high clinical need for effective therapies for gMG.

Whilst extremely disappointing it is not the end of the process. Alexion assures the MAA that they are committed to working with the PBAC alongside the MG Clinical and patient communities to bring access at the earliest opportunity to their treatment.

Both Pharmaceutical companies (Alexion and UCB) will resubmit in 2025.

Please read below for a statement from Libby Noble at Alexion.

Patient Advocacy Director, Libby Noble told us: “We had hoped for a different outcome and we understand it’s both upsetting and frustrating to many who struggle with this condition and especially to those who took the time to share their views to the PBAC. We want to extend our sincere gratitude to MAA and the MG community for their invaluable input and support throughout this process. On a positive note, we are pleased that the PBAC recognised the high clinical need for effective therapies for MG, particularly in patients who are not responding to or are unable to use existing therapies, and for those with refractory disease. That recognition is testament to the lived experience shared by patients and caregivers that helped to shape that understanding, and sets a strong foundation for our continued efforts to bring access to much needed new treatment options”.

Alexion are working on their next steps and will share an update on timings with us as soon as possible. They would also like to invite the community to join a webinar on Friday 11 October to share the PBAC outcomes in greater detail, address community concerns, and update on their continued efforts to advocate for timely and equitable access in Mg.

More information will be shared regarding the webinar as it comes to hand.

The MAA first engaged with Amplo Biotechnologies at the 2019 Brisbane Conference. We are delighted to report that Patricio Sepulveda, CEO, is offering to share updated information on “what they are doing and where they are at”. Patricio reports that 35 different genes have now been identified to capture more people with Congenital Myasthenia Syndrome. This knowledge is important in providing the correct treatment. Patients, Clinicians and Carers are invited to attend the following information sessions. Australians can join at either 7am on August 14th (choose the US option) or 7pm on August 14th (choose the UK option). The session is expected to run for 45 minutes. Written questions will be answered on the day or by follow-up email.

Register utilising the QR code or link found in the flyer below. If you are sero-negative MG, please consider joining the webinar as some patients are being diagnosed quite late in life.

AMPLO IS DELIGHTED TO SHARE THE LATEST ADVANCEMENTS IN ITS GENE THERAPY FOR CONGENITAL MYASTHENIC SYNDROME. THIS IS A FREE WEBINAR FOR PATIENTS, CARERS AND CLINICIANS

Follow the link to register:

Tuesday 13th Aug, 5pm US EST
Wednesday 14th Aug, 10 am UK/GMT+1

To Australians with Myasthenia, the following is a message from Dr Carolina Barnette-Tapia MD, PhD of Canada.

The MAA thank Australians for their interest in supporting this project. Responses will be welcomed until early September 2024. Information about the outcomes resulting from this research has been requested.

Disclosure information is provided after logging in.

“We invite you to participate in this international study to assess how people with Myasthenia living in different countries make treatment decisions and which aspects of treatment are more important to them. We kindly thank the MAA for supporting our study, which will help understand if the preferences of Australians living with MG are different to those from other countries.

Carolina Barnett-Tapia, MD, PhD
Associate Professor of Medicine (Neurology), University of Toronto
Ellen and Martin Prosserman Centre for Neuromuscular Diseases
Elisabeth Raab Neurofibromatosis Clinic
Toronto General Hospital / UHN”

To participate, scan the QR code below or click here.

The Myasthenia Alliance Australia (MAA) supports and promotes research that is specific to the experience of Myasthenia Gravis (MG) patients in Australia. But we would not be able to do this without the essential and valuable contribution of Australians with MG.

One current project is VALUE-Ig, which aims to generate evidence to inform the optimal use of immunoglobulin (Ig) in four patient cohorts, including MG. This study is being conducted by a research team at Monash University Centre for Health Economics and is funded by the Australian Government’s Medical Research Future Fund.

VALUE-Ig includes a Discrete Choice Experiment (DCE) to explore the treatment preferences of MG patients in Australia. The first round of focus groups to help design this DCE study has been completed and we sincerely thank the MG patients who gave their time and insights to this important research. Please read below for an update from the VALUE-Ig research team. To find out more, you can contact the team directly at value.ig@monash.edu or reach out to the MAA by calling 1800 802 568 or emailing info@mgaq.org.au or info@myasthenia.org.au.

Monash University
Centre for Health Economics | Monash Business School
Level 5, Building H, Caulfield Campus
900 Dandenong Road
Caulfield East VIC 3145
Australia