Pegasys® Update – Great News!!

Roche phoned me on 4th August to update me on the Pegasys for MPN issue. I am delighted to announce that Roche made a PBAC submission in late July, requesting UNRESTRICTED ACCESS of Pegasys on the PBS. This means that if approved, Pegasys will be available for prescription for any indictation, including MPN.

I was told Roche used my submission and added their own work on economic modeling, (which included close discussions with Professor Andrew Wilson, Chair of the PBAC) to prepare their PBAC submission. Interestingly, after Roche’s discussions with Prof. Wilson, they have made their submission directly to the PBAC, bypassing the Therapeutic Goods Administration (TGA)! My Roche contact told me this has only ever occured once before, for Rituximab, an oncology/haematology and rhuematoid arthritis drug.

Roche again thanked me for the work I did in my PBAC submission which included references to the scientific literature showing evidence of efficacy for IFN in MPN. I was told that Roche has also obtained feedback from haematologists around Australia on this issue too.

The PBAC have scheduled an out-of-session meeting (not their usual quarterly meeting) in late August, to consider Roche’s Pegasys submission. At this stage, (assuming the PBAC approve it) Roche do not know what the timeline is likely to be before Pegasys is listed on the PBS.

I have been invited to Canberra to attend the CanForum with Rare Cancers Australia on 9 August. I am looking forward to meeting with the Roche employees who prepared Roche’s Pegasys submission and also look forward to speaking with Professor Wilson.

I am keeping my fingers crossed that Roche’s PBAC submission is approved in favour of listing Pegasys for MPN on the PBS so those of you who wish to use this medication to treat your MPN can do so soon, cost effectively. I have been in contact with the Leukaemia Foundation on this too, and they plan to send out information to MPN patients and via the LF Facebook page soon, confirming these developments.

Warm wishes to all…Nathalie

Share to:

Jolanda Visser accepts $1,000 donation to the MPN fund

The MPN AA would like to thank the Big A Boutique Op Shop from the Colonel Light Gardens Church of Christ in South Australia for the generous $1,000 donation they issued the Leukaemia Foundation’s MPN Fund.

The Big A Boutique regularly donates part of their proceeds to charities and organisations that want to make a difference by supporting the community.

After accepting the generous donation, MPN AA member, Jolanda Visser, was able to present a talk on MPNs, how patients’ quality of life is affected and how the MPN AA is trying to make a difference through advocacy and support.

Share to:

European Haematology Association (EHA) Conference – Madrid

Haematologist Dr Cecily Forsyth recently attended the European Haematology Association (EHA) conference in Madrid and has kindly prepared a summary of the MPN studies presented at the conference, for MPN AA to share with the MPN community. Many thanks Dr Cecily!

View Dr Forsyth’s summary here.

Share to:

Medicinal Cannabis Consumer Update

On May 18, Ken Young attended the Medicinal Cannabis Consumer Update organised by the Consumer Health Forum on behalf of the Commonwealth Department of Health and the Australian Advisory Council for the Medicinal Use of Cannabis.

There has been considerable media attention to the decision by the Australian and State Governments to facilitate access to medicinal cannabis products to appropriate patients for medical conditions where there is evidence to support its use. Many people have questions about who is eligible, how they can access the product if eligible and range of other issues around supply, quality etc. The aim of the forum was to try to answer some of those questions by giving general information about medicinal cannabis, evidence around its use and the access pathways including clarification of who is eligible to use it and the process for doing so.

Medicinal cannabis became a controlled drug in the Poisons Standard on 1 November 2016. Medicinal cannabis is a controlled substance that is regulated to prevent diversion and illicit use. For this reason, demand and supply are linked in the regulatory process.

The current medicinal cannabis framework – agreed by the Australian and participating State Governments is:

  • medicinal cannabis is medicine to be available on prescription only
  • produced to quality standards
  • with patient access through the Therapeutic Goods Administration (TGA) protocol for unapproved medicines

Medicinal cannabis can be prescribed under the provisions of a controlled drug (Schedule 8) of the Poisons Standard. However, whether medicinal cannabis can be prescribed in a particular jurisdiction depends on whether the state or territory has adopted the change. So just as historically trains gauges changed at Albury there are difference between accessing medicinal cannabis in Victoria and New South Wales!

Individual consumers cannot apply to obtain approval to import and access unapproved medicinal cannabis products. Patients will only be able to access medicinal cannabis if they:

live in a state or territory where it is not a prohibited substance
obtain a prescription from an appropriate medical practitioner, who obtains permission from:

  • the state or territory
  • the TGA for you to obtain a specific medicinal cannabis product

You can find out more about the process from the TGA website.

Currently in Victoria access to medicinal cannabis is restricted to children with severe intractable epilepsy. With other conditions being considered by an Independent Medical Advisory Committee. The Office of Medicinal Cannabis has been established within the Department of Health and Human Services and is responsible for establishing the Victorian medicinal cannabis framework. You can find out more information at their website.

Share to:

Queensland Coffee Cake and Chat

The Leukaemia Foundation hosted an informal coffee, cake and chat afternoon at the Jindalee Hotel on Saturday 13th May. It was well attended and provided a useful opportunity for those with an MPN to unwind and connect with others.

Share to:

MPN Clinicians and Researchers meeting – Melbourne 27 April 2017

The day prior to the MPNAE&I Patient Program in Melbourne, Australian MPN clinicians and researchers met with some US counterparts to share knowledge and ideas. In the photo below, Dr Ruben Mesa speaks  about the unmet needs of MPN patients, particularly those with Myelofibrosis. He discussed the role of Ruxolitinib, Interferon and clinical trials. He addressed the question of what would constitute failure of Ruxolitinib treatment, and when it might be appropriate to continue Ruxolitinib despite suboptimal symptom or spleen control, or hematological toxicity in this group of patients. Thanks again to Ann Brazeau and MPNAE&I for making this possible.

Share to:

MPN Patient Program – 28 April 2017

The Australian MPN community was given a wonderful gift on Friday 28 April by Ann Brazeau and her team from MPN Advocacy and Education International, together with Drs Ruben Mesa, Laura Michaelis, and Raajit Rampal from the US; and Drs Cecily Forsyth, Steven Lane, Kate Burbury, David Ross, and Wendy Erber, and Andrew Lim, (standing in for Prof Andrew Grigg), from Australia.

Months of planning culminated in an outstanding educational event for the group of over 150 Australian MPN patients, caregivers and haematologists.

We all expected great things from the US contingent, and they certainly delivered. We were also extremely impressed by the presentations from our home-grown haematologists. The level of passion, dedication and care for patients was very evident in each and every one of the presentations.

After the formal presentations, patients and caregivers were able to ask questions of the presenters in disease-specific break-out sessions.

I think for patients and caregivers ranging from the newly diagnosed to the old hands, everybody learned something, and valued the opportunity to share with patients and professionals during breakfast, lunch, and the wonderful Mix and Mingle event at the end of the day. If we didn’t finish all that wonderful food, it was only because we were so busy talking!

Given the amount of information which was presented on the day, I am looking forward to seeing the video of the presentations, to have the opportunity to revisit it.

Together with my MPNAA colleagues, I felt privileged to have had the opportunity to listen to the clinicians in their meeting on the Thursday afternoon, as we gained insights into the challenges of diagnosis and treatment of these complex blood cancers.

Thanks so much to Ann and Marina (as well as Kathleen behind the scenes) and the wonderful doctors who were willing to cross the vast Pacific to share their knowledge. Thanks also to all the wonderful clinicians and researchers who came from far and wide across Australia. Together you enlightened, empowered and entertained all those who were lucky enough to be present!

I feel so inspired.

Sue Taylor

Videos of the presentations are available here.

Share to:

Why MPN AEI came to Australia!

By Ann Brazeau, CEO, MPN Advocacy and Education International

MPN Advocacy and Education International hosts several patient/caregiver educational programs per year. Although most take place in the United States, their goal to expand to other countries where MPN groups are interested in partnering to benefit their MPN Communities has been a priority.

Through collaboration with the MPN Alliance Australia, the first patient/caregiver program was held in Melbourne on the 28th April, 2017. MPN specialists from Australia joined U.S. specialists to share up-to-date, relevant information to over 150 patients and caregivers.

Empowering patients with the knowledge they need to better understand the complexities of MPNs, is what MPN Advocacy and Educational International hopes to achieve at these events. Partnerships are essential to the success of these very important programs. Imparting valuable, reliable information to the MPN Communities around the world helps bridge the gap to understanding what is being done, and identifying unmet needs.

In the early days of my work in MPN little was being shared among researchers and academic institutions. Today, it is a common practice. Recognizing the importance and value in collaborating, researchers and clinicians are much more amenable and grateful for opportunities to share their experiences and findings. Thus, a physician/researcher meeting was held the afternoon before the patient event.

A half-day strategy meeting with the MPN Alliance Australia proved to be meaningful and productive. Their dedication and commitment to the patients and caregivers in Australia is evident in the many hours they volunteer each week.

We are proud to have been a part of this experience and were happy to pull this event together with our partners, MPN Alliance Australia.

Share to:

Follow Up – Pegasys® on the PBS for MPN

Dear MPN Community,

I received a letter from Professor Andrew Wilson, Chair of the PBAC, in response to my submission requesting Pegasys® (Interferon Alfa-2A) listing on the PBS for MPN.

Professor Wilson stated he and his PBAC colleagues agree there is a need for interferon based therapies in MPN treatment in Australia. He said however, the PBAC cannot recommend a drug to the Health Minister for PBS listing until the manufacturer/sponsor of that drug:
1) Applies for Therapeutic Goods Administration (TGA) approval for the drug for the particular indication (in this case MPN); and,
2) Puts in a submission to the PBAC for PBS listing of the drug (for that indication), with supporting clinical data.
To date, Roche has not applied for TGA listing of Pegasys® for MPN or lodged a submission to the PBAC asking to have Pegasys® listed on the PBS for MPN.

As recommended by Professor Wilson, I have contacted Roche regarding the two points above and the Department of Health has done the same. I have also contacted Rare Cancers Australia on this issue and they have offered to help follow up with Roche. I will keep you informed when I hear any further developments.

Best wishes to all,
Nathalie Cook

Share to:

MPN Education Foundation

I have just been lucky enough to attend the MPN Education Foundation biennial Conference at the Mayo Clinic in Scottsdale, Arizona, in a private capacity. I was one of two Australian MPN patients, accompanied by our partners.

It is impossible to summarise the whole conference in a few words. The tireless volunteer organisers from the MPN Education Foundation did a truly wonderful job, and provided an extremely hospitable, welcoming and professional atmosphere for all those who participated.

The 16 expert presentations, two panel discussions, and disease specific break-out sessions, were enough to answer most of my questions, and many more that I had never thought to ask. The expert presenters were incredibly generous with their knowledge and time, and joined patients during the breaks to answer individual questions, whilst trying to juggle their lunch and catch up with their colleagues. Exhausting doesn’t begin to describe it, even though we tried to have early nights, especially factoring in the jetlag and the 6.30am breakfast starts each morning, which we chose to reach by walking through the crisp 5 degree air from our desert hotel.

I think apart from the excitement of hearing of the research and developments in the pipeline, I was so inspired by the passion that was so evident in the presenters, and the thought that there are such wonderful people looking out for those of us with MPNs. They are incredibly knowledgeable, but the other message that came through loud and clear to me is that “its complicated” – a refrain used by many of the doctors when queried about particular scenarios. What I noticed is that they were not afraid to say if they didn’t know, or to admit that there IS a lot that they don’t know yet. During the question panels, it was also interesting that they didn’t always agree with each other, and they acknowledged that there may be trial and error, and a lot of careful judgment needed in both diagnosing and treating MPN patients. Treatment should be individually based on a holistic assessment of the patient including blood counts, but importantly symptoms and quality of life issues.

I was privileged to meet many of the wonderful presenters, including Prof Claire Harrison from the UK, Dr Richard Silver, Susan Leclair, and especially Drs Ruben Mesa and Laura Michaelis, who will be coming to Melbourne in April for the inaugural MPN Patient Conference in Australia, which will be co-hosted by MPNAA and MPN Advocacy and Education International.

Even with all those positives, probably the most special thing about attending the Conference in Scottsdale was meeting so many patients and their partners/ supporters, who were all willing and keen to share experiences, especially those whose posts and emails I have seen on various MPN fora.

For more details on the Scottsdale Conference, visit

Sue Taylor

Share to: