Leaders in Advocacy From the MPN Alliance Australia Share Their Story

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Joining Patient Power from Melbourne, Australia, myeloproliferative neoplasm (MPN) patients Nathalie Cook and Ken Young share their stories of being diagnosed and subsequent advocacy work in MPNs. Tune in to learn how advocating for approval of medications and forming the MPN Alliance Australia has benefitted those with MPNs in Australia and beyond.

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Andrew Schorr:                     

Hello and welcome to Patient Power. I’m Andrew Schorr, and we are just back from visiting New Zealand and Australia. And along the way, met some wonderful people living with one of the conditions I have, an MPN; for me, myelofibrosis. And we were very struck by the fact that people sometimes have to really go to bat to connect with a knowledgeable specialist and ideally get the treatment they need and deserve, and have it paid for by the government.

Well, joining me now are two Australians from Melbourne who’ve really done that battle, both living with polycythemia vera. So, there’s Ken Young. Ken, welcome to Patient Power.

Ken Young:                           

Hi and thank you, Andrew.

Andrew Schorr:                     

Thank you. And then also Nathalie Cook. Nathalie, welcome to Patient Power.

Nathalie Cook:                     

Thank you, Andrew. It’s great to be here.

Andrew Schorr:                       

So, Nathalie, let’s start with you. Tell me and us a little bit about your polycythemia vera story; how it started with ET, I think, and then how you have really fought for getting the treatment that would be right for you.

Nathalie Cook:                     

Thanks, Andrew. Yes, I was diagnosed with ET in 2008 and then by 2010, we realized that I actually had polycythemia vera. And so then I had to make treatment decisions. And the two treatments available were either hydroxyurea (Hydrea), or hydroxycarbamide, and the original form of interferon, interferon alfa-2a (Roferon). I read up on as many papers as I could get my hands on to help me make the decision on which treatment and I decided initially to start hydroxyurea to try to get my blood counts down, but long-term, I wanted to use interferon because I liked the research that had shown that it could improve long-term outcomes.

In Australia at that time, there was only the old form of interferon, the Roferon that was available on our Pharmaceutical Benefits Scheme, which is the government-funded medications scheme. And the newer form, Pegasys or pegylated interferon, was only available off-label and it was very expensive, so it was out of reach of most patients.

So, I started calling the drug company Roche, who are the producers of interferon, of peginterferon alfa-2a (Pegasys) and Roferon, to ask about their plans to apply for a PBS listing of Pegasys. And they told me they had no plans and they said there were no clinical trials being done on that for MPN patients in Australia and there was really no interest. And it was also not approved by our government agency called the Therapeutic Goods Administration. And that’s the first point in Australia that a drug has to be approved for a particular indication before the PBS can go through the approval process. So, there were several hurdles that no one was looking into.

So, I started Roferon, and I got all the typical side effects; flu-like symptoms, and aches and pains, and headaches, et cetera, and nausea, and even hair loss, which was very distressing. And I continued to call Roche and I told them about the side effects I was having. So, I started Roferon in early 2011 and regular calls and emails to Roche. And then in 2012 towards the end, they offered me Pegasys on compassionate grounds.

So, I called my hematologist and he arranged for me to get that. And initially, it was on for one year. And then during the course of that year, I continued to update Roche and told them about how better I was doing on Pegasys compared to Roferon and thanked them for it. And then they continued that access sort of on a year-by-year basis.

Andrew Schorr:                      

You were your own clinical trial.

Nathalie Cook:                     

Yes, exactly. Exactly. And then I started contacting my local politician, federal member, and I actually met with him in Canberra and asked him if he could help. And he asked me to write him a letter that he would give to the health minister. And then the health minister wrote back to me and basically said thanks for your interest, but it’s not possible.

Then I had the opportunity because I’d been doing a lot of voluntary work with the Leukaemia Foundation – I’m also a dietician, so I’ve presented as a dietician at patient conferences and write articles for the MPN News, et cetera. And I was invited to a rare cancers forum at Parliament House in 2016. And at that forum, I got to hear a lecture by Professor Andrew Wilson, who is the Chair of the Pharmaceutical Benefits Advisory Committee. And he spoke about the importance of hearing the patient’s perspective in making drug decisions.

And after his lecture, I actually got the opportunity to speak with him and I told him my story. And I asked him how can we get Pegasys on the PBS because I said Roferon, which is the same drug, is on the PBS, so why don’t you expand your listing to include the newer form of the same drug that you’ve already approved previously. And I asked if I could write directly to him. He said yes and he wished me luck.

So, I went home and I wrote a submission to him. I wrote it like a scientific report and had about two pages of references at the end. And I spoke about my personal story, but also the evidence had references to the evidence that supported use of interferon in MPN. And I got three physicians in Melbourne to review my submission before I sent it in; two hematologists and an infectious diseases physician who’s a friend of mine.

Then I sent my submission in and I got a letter back saying yes, the PBAC, the Pharmaceutical Benefits Advisory Committee, agreed that there was a need for interferon-based therapies in MPN, however, they hadn’t had a submission from Roche. And they asked me to continue lobbying to Roche, which I continued to do. Then the PBAC would get with Roche over the next year. And after it was tabled at three PBAC meetings, it was finally approved for listing.

And then the next step was for the health minister to sign off on it, which then happened. And then in July last year, I was asked to attend the announcement by the health minister at Peter MacCallum. Ken also came with me to that announcement, which was wonderful. And then 1st of August, 2018, it was on the PBS.

Andrew Schorr:    

Okay. So, let’s make sense of this for people. So, basically, you beating the band and connecting with the right people, some of it by happenstance at the conference where you met one of the head officials, and putting the evidence together, and lobbying the drug company while you were lobbying the government, you were able to get approval and now payment for a less toxic therapy that’s effective for MPN patients. Did I get it right?

Nathalie Cook:                     

Absolutely. That’s correct. Absolutely.

Andrew Schorr:                    

So, Ken.

Ken Young:                           


Andrew Schorr:                      

You’ve been around all this. Is this an example of the power of patients when we really start speaking out?

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Page last updated on September 4, 2019