PBAC has recommended ruxolitinib be listed on the PBS for PV

We are thrilled to advise that the Pharmaceutical Benefits Committee (PBAC) at its March meeting has recommended that ruxolitinib (jakavi) be listed on the Pharmaceutical Benefits Scheme (PBS) for adult polycythemia vera patients who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).

This does not mean it’s on the PBS and available for us to be prescribed yet.
The approvals process will now continue. For example, cost still needs to be considered by government and so automatic approval and listing is not a given.  Also this may still take some time (eg, as in the case of momelotinib – Omjjara approval for anaemic myelofibrosis).

However it’s a really welcome step in the PBAC approval process and the MPNAA would like to thank those people who made a submission to the PBAC in support of its inclusion on the PBS. The MPN AA also made a submission.

Here is the excerpt from the PBAC report.

Patients may be wondering why the language ‘resistant to or intolerant of hydroxycarbamide (hydroxyurea)’ has been used when so many patients are in fact being treated with Pegasys. However this recommendation is couched in the words of the submission from the pharmaceutical company. That submission in turn was based on results of clinical trials that reported on hydroxyurea versus ruxolitinib.

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International review series on MPNs published

The April 2025 edition of Haematologica features an excellent review series on MPNs.

The authors of the introduction paper to the series, Australia’s Drs Steven Lane and Yin Yuan, explain that ‘the review series is not designed to be a definitive review of all aspects of MPN biology and treatment. Rather, we have selected a few key topics of particular interest to the field that have been informed by recent advances in basic or clinical research’.

In addition to the paper from Drs Steven Lane and Yin Yuan, paper no 4, ‘Pathogenesis and management of high molecular risk myeloproliferative neoplasms’ also features two Australian coauthors, haematologist and researcher Dr Victoria Ling and researcher Dr Megan Bywater.

The papers are all free to access and linked below:

  1. Prevention, diagnosis and management of myeloproliferative neoplasms: an introduction to a review series,
  2. Evolution of myeloproliferative neoplasms from normal blood stem cells,
  3. ‘Clinical and laboratory approaches to target and eradicate early disease-initiating stem cells’:
    Paper is titled New approaches to standard of care in early-phase myeloproliferative neoplasms: can interferon-a alter the natural history of the disease?,
  4. Pathogenesis and management of high molecular risk myeloproliferative neoplasms,
  5. ‘Strategies to prevent or treat the devastating clinical consequence of AML arising from antecedent MPN, also known as blast-phase MPN’. Paper is titled Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia.

 

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PV treatment ‘rusfertide’ trial results

Early results from the Phase 3 clinical trial of the hepciden mimetic ‘rusfertide’ have recently been published.

Rusfertide aims to overcome the need for phlebotomy in patients with polycythemia vera (PV), by keeping haematocrit below .45.

Early phase 3 trial results show that rusfertide was well tolerated and effective in keeping PV patients’ haematocrit below .45.

These results are extremely promising for PV patients.

The linked article provides more detail, including commentary from Australia’s Dr Cavan Bennett from the Walter and Eliza Hall Institute  HERE.

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February 2025 MPN Education Foundation patient conference videos

Every two years the MPN Education Foundation hosts a wonderful patient conference in Scottsdale Arizona, with some of the most distinguished MPN expert presenters. The conference is titled the Joyce Niblack Memorial Conference, and is hosted by the Mayo Clinic.

We are very fortunate to be able to view the conference video presentations and slides.

Here is a list of presenters and topics:

Jyoti Nangalia – MPN Molecular Biology

Rupali Bhave -Managing ET in 2025

Raajit Rampal – Managing PV in 2025

Naveen Pemmaraju – Managing ET in 2025

Ruben Mesa – Guidelines & Gaps in MPN

Jeanne Palmer – Transplants in MPN for 2025

Marina Kremyanskaya – New Drugs for PV

John Mascarenhas – New Drugs for MF

Angela Fleischman – Nutrition & Complementary Approaches for MPNs

Jean-Jacques Kiladjian – Long Term Outcomes of Interferon in MPNS

Steffen Koschmieder – Inflammation in MPNs

Susan Leclair – Learning about my Labs: An MPN Patient Guide

 

 

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2024 ASH MPN highlights with Dr Ruben Mesa

Courtesy of Ann Brazeau of MPN Advocacy and Education International, here is a wonderful summary video of the MPN highlights from the December 2024 American Society of Haematology (ASH) meeting provided by Dr Ruben Mesa.

Watch the video HERE

 

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Patients with CalR mutation – promising ASH update

The American Society of Hematology’s December 2024 conference included developments in treatments for MPN patients with the CalR mutation. A range of Australian and international immunotherapy research is underway and some treatments are already in clinical trials. It is too early to present results.

A fascinating video about CalR treatment research is linked below.
In the video, Drs Alex Rampotas and Zoë Wong explain that there are already specific immunotherapies being trialled against the CalR mutation, all of which may well be effective.  They specifically mention a ‘B specific T cell engager’ and ‘a blocking antibody against it’.

However their collaboration is about a third type of immunotherapy option, a novel second generation CAR-T cell therapy. They advise that CAR-T is the ‘strongest immunotherapy so potentially  ……. able to overcome some of the immune suppression of myelofibrosis and directly eliminate the malignant stell cells.’

The full video is 8 minutes long, unfortunately with background noise.  However if you have the CalR mutation, watching the video will give you a Christmas present of great promise!

https://www.vjhemonc.com/video/qsoln_mbhbk-development-and-evaluation-of-a-first-in-class-car-t-therapy-against-calreticulin-mutant-neoplasms/

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MPN Horizons meeting 6-8 September 2024 Warsaw – A personal reflection from Sharon MacIntyre

 

Well let’s just say it was an honour to attend the MPN Horizons conference this year.  There was representation from MPN specialists, many MPN advocacy groups, PV, ET & MF patients, pharmaceutical companies and more!  The title “Shifting the treatment paradigms of MPN” was very apt. Since I first attended in 2017, sponsors have grown from 3 to now 11! And patient advocacy groups now include Thailand, Korea, and other nations which previously were not represented.  It is such a growing global family to address MPN needs.

The almost 70 participants in Warsaw, Poland were from an amazingly geographically spread of 26 countries, including Australia, India, Chile, to Europe, America and Asia! All were very vocal in sharing new developments for MPN from research to trials, to advocacy and patient stories. All sharing a key purpose – to make life better for MPN patients.

Before we dived into a massive 3 day program, it was refreshing to attend a breakfast session where Pharma was the lead and seemed genuinely keen to collaborate and bridge the access barrier different countries and patients in need have. The session was called “Navigating the Path to Equal and Equitable Care” to discuss the most important barriers for MPN patients.  I learnt a lot, particularly helpful that there is a special consideration element to access which can be sought with the Pharma company directly for review/access to medications.

This was the third time I have attended Horizons and as a highlight for myself being diagnosed at 18 years of age. I was particularly impressed with the inclusion of a session solely for young MPN patients by Alice Watson from UK. Under the auspices of MPN Voice she has started a group for under 40’s MPN patients to ask questions, address issues such as study/work life balance, starting a family and pregnancy, long term MPN and progression.

The sessions from world leading specialists were very informative.  Dr Claire Harrison spoke on state-of-the-art news for Myelofibrosis – including the new prognostic model; how there are more options available for lower risk MF patients; and how the success of Haplo transpants as an option could be explored. A haematologist from Germany, Dr Susanne Isfort, talked about the 3 common MPNs and the drugs she typically uses to treat them, and about data showing that interferon in young patients may be looked at as an option to stop MF progression.

Jon Mattias from MPN Voice presented a session on a great new application “Health Unlocked” which is in development. This app tracks patient symptoms and data that hopefully could be useful for medical appointments and could be integrated into a wearable device.  This type of app could be rolled out globally – but more needs to happen on 3rd party permissions and how to protect personal data.

Dr David Ross from Adelaide Australia, spoke on the differences in an individual’s height and gender etc which often isn’t taken into consideration in treating patients. For example, spleen size, depth and volume in a female 5’2 and a man 6’2 can be completely different in what is considered large! He felt spleen volume is more important than just length – something I had never heard of before. There were pictures from Dr Wendy Erber’s lab in Western Australia on machine learning for precise fibrosis scoring which was very interesting.

Elena Greschner from Austria talked about fatigue and how around 80% of MPN patients suffer fatigue.  Yet only about 30% of haematologists ask their patients about fatigue. She stressed the fact that Quality of Life is important (as well as the blood cell numbers). Dr Patrick Harrington mentioned patient data in his MPN research that showed a fifth of MPN patients can only work reduced hours or need to stop work early before retirement age.  Emphasis was placed on being aware of increased clotting risk, infection and organ failure.

Another brilliant session I thought was valuable were the regional breakouts. Being an MPN AA advocate from Australia – ideas for more Asia/Pacific collaboration were bounced around and very positive. I’m looking forward to seeing some of these ideas come to fruition. Perhaps “Chai for Cancer” events at workplaces in Australia borrowed from advocacy group ‘Friends of Max’ in India’s success.

One of the advocates talked about their “March for Cancer” event and how citizen participation was important!  Perhaps we can bring back a lantern walk for MPN and other blood cancer advocacy.  The kids love this!

The importance of a healthy diet and exercise were again highlighted.  I for one will be trying to implement when my symptoms are ok, a more Mediterranean Diet of real foods (hopefully more than packaged and takeaway) and some gentle exercise for 30 mins a few times a week.

All in all the MPN Horizons conference was absolutely brilliant! A wonderfully informative and collaborative 3 days in Warsaw. I’m looking forward to seeing these developments turn into new application and drugs (some in clinical trials already) and much more positive patient outcomes for the future.

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Ruxolitinib available for some PV patients? PBAC call for submissions by 29 January 2025

In March 2025, Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) will consider an extension to the existing listing of Ruxolitinib (known as Jakavi®) onto the Pharmaceutical Benefits Scheme (PBS). The extension sought will be for the treatment of adult patients with PV who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).

NOTE: CLOSING DATE FOR PATIENT INPUT – 29 JANUARY 2025

To help the Government in its considerations, PBAC is taking consumer comments until 29 January 2025.
* See below for advice on making a submission.

The MPN AA is supportive of ruxolitinib being available on the PBS for PV patients. It would provide another treatment option when first line therapy fails. Thanks to the MAJIC-PV trial conducted in the UK, Ruxolitinib has now been established as a viable second line treatment option for PV.

For more information please see our News post about the Majic-PV trial.

*Next steps if you’d like to make a submission

Making a submission to the PBAC is a way for PV patients to have a voice by explaining how having access to this treatment could impact on their own quality of life.

If you follow the instructions below you will be able to make your comments via a simple process.

  1. To comment for the  2025 PBAC meeting, start by clicking on this LINK.
  2. On Page 2, enter your name and contact details and select the category that best describes your reason for input, ie:
    – Individual who would like to access the medicine to treat own health condition
    The drug you are commenting on is ‘Ruxolitinib Jakavi®.
    You will see that this is a ‘Resubmission to request a General Schedule Authority Required (STREAMLINED) listing for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).’ This is because the drug company unsuccessfully sought approval in 2019. However as more results are now available from the MAJIC study, the pharmaceutical company is resubmitting its application.
  3. On Page 3, provide your input by answering the questions in the free text boxes OR by attaching a PDF or Word file at the bottom of the page.
  4. On Page 4, please declare any conflicts of interest relevant to the responses provided in Pages 2 or 3.
  5. When you have completed the form, select ‘Submit Response’ on Page 5. You will be sent a confirmation receipt and link to a PDF copy of your response to the email address you provided on Page 2.

If you’d like any support in making your submission, you can contact the Department of Health at HTAconsumerengagement@health.gov.au or watch one of their facebook Q&A recordings HERE.

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Message for patients taking anagrelide

The MPN AA has become aware of a warning placed on the MPN treatment Anagrelide.

While haematologists would already know about the warning, and be managing patients’ treatment accordingly, the MPN AA wants to also inform any MPN patients taking anagrelide.  The warning states:

SPECIAL WARNING AND PRECAUTIONS FOR USE

Do not stop using anagrelide suddenly without checking first with your doctor.  Rather you may need to slowly decrease your dose before stopping it completely. Stopping suddenly will cause the platelet level in your blood to increase quickly. It should be noted that there is risk of thromboembolic events during this rebound phase which may lead to potentially fatal thrombotic complications, such as cerebral infarction. Platelet counts should be monitored closely when anagrelide is ceased.
We will update the other languages as promptly as possible, hopefully within a couple of weeks.

 

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Change to Pegasys access in Australia from 1 September

For anyone in Australia who is currently being prescribed Pegasys® (peginterferon alfa-2a) as a treatment for MPNs, the MPN AA wants to update you on some important changes to how you will be able to access this medicine moving forward.

While many of you will have been getting Pegasys® dispensed at a local pharmacy, this will start to change from 1 September 2024, with a new Pegasys® Home Delivery Program coming into effect.

This program is delivered by Echo Therapeutics (the sponsor of Pegasys® in Australia) through its EchoCare™ service, in partnership with JustMeds – a local specialist medicines delivery provider.

This means you will now have Pegasys® delivered directly to your home, workplace or another location, rather than having go into a pharmacy to pick up your medicine.  Importantly, 1 September is not a hard deadline to switch over to the new home delivery program. We know it may take some time for people to adjust to this new process, so there is flexibility built in for you to be able to transition across to the Pegasys® Home Delivery Program at the right time for you.

Echo Therapeutics has engaged with doctors and nurse practitioners who are involved in the management and care of people with MPNs, along with the Therapeutic Goods Administration (TGA). Pharmacists have been informed about these changes and will be aware of the new process.

This Pegasys® Home Delivery Program is a short- to medium-term solution.  It will be in place until Echo Therapeutics is satisfied the local supply chain for Pegasys® is more intact and there is a lower risk of unauthorised export.  Once confirmed, it is expected that Pegasys® will become available in retail pharmacies again.

You can find more information about this in the letter attached or visit https://www.echotherapeutics.com.au/echocarepegasyspatient for more information, and step by step guides on how to access Pegasys through the JustMeds partner provider. https://www.justmeds.com.au/pegasys

Alternatively, you can email rx@justmeds.com.au for more information.

As you know, MPN AA does not promote or recommend specific treatments for people living with MPNs. Decisions about any treatments should be made in careful consultation with your haematologist, after taking into account the potential benefits and side effects for each person’s individual circumstances.

Kind regards,
MPN AA Team

Patient Communication Letter from Echo pharmaceuticals re Pegasys

Patient instruction guides

 

 

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