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One woman’s remarkable resolve to help give better treatment options for people with rare blood cancers

Publish Date: 2/3/2017

A better treatment option for people with the blood cancer myeloproliferative neoplasms (MPN), is one step closer to being made available to Australians, with the new drug interferon alfa-2a (Pegasys®), being discussed at today’s Pharmaceutical Benefits Advisory Committee (PBAC) meeting.

Nathalie Cook

This is especially exciting news for Melbourne woman, Nathalie Cook who has worked tirelessly behind the scenes over the last six years campaigning for this drug to be made available to Australians.

Nathalie herself was diagnosed with the MPN called essential thrombocythaemia in 2008, which progressed to another MPN, polycythaemia vera, in 2010.

Seeking more information about her diagnosis and treatment, she attended the MPN doctor-patient conference at the Mayo Clinic in the U.S. in 2011 where she hoped to gain information to help her decide whether to continue with her current treatment, or try a different drug, interferon alfa-2a (Roferon-A®)*. 

Nathalie said the haematologists explained how interferon can cause MPN patients to go into deep molecular remission.

She also learned that the newer form of interferon – Pegasys – was easier to tolerate compared to Roferon, with fewer adverse side-effects.

However Pegasys was not yet listed on the Pharmaceutical Benefits Scheme (PBS).

“On the flight home, I made a decision – to try to get Pegasys on the PBS for people with MPN in Australia.”

Whilst taking Roferon Nathalie experienced a number of severe side-effects.

“I suffered from flu-like symptoms, fatigue, hair loss, nausea and fevers,” she said.

“I made several phone calls to the manufacturers of Roferon and Pegasys, to report the adverse side-effects I experienced.

“I asked if they had plans to request the government to add Pegasys to the PBS, but was told the company had no plans to make a submission.

“In early-2013, I had the opportunity to start treatment with Pegasys and the side-effects were much milder. Within weeks my hair loss stopped and it began to grow back, I had more energy, felt better and the flu-like symptoms diminished.” 

Nathalie attended the Mayo Clinic MPN conference again in 2013 and 2015 and in this time continued to send annual letters to the drug company Roche documenting her experience.

In 2015 Nathalie submitted a letter to the Senate Enquiry on the Availability of New, Innovative and Specialist Cancer Drugs in Australia, proposing the government include Pegasys on the PBS for the treatment of MPN.

She also met with her local MP who suggested she write to the Health Minister and also contacted Melbourne haematologist, Associate Professor Constantine Tam, to ask about clinical trials.

“He told me the Australasian Leukaemia & Lymphoma Group had previously requested drug company, Roche run a clinical trial of Pegasys treatment in MPN patients in Australia, however they showed no interest in doing so at that time,” she said.

Meeting with the PBAC

In October 2016, at the Disrupting Cancer Forum in Canberra, Nathalie met Professor Andrew Wilson, Chair of the PBAC.

“I spoke to him about the issue and he listened to my story with great interest. I also asked if I could write to him directly.”

In mid-January 2017, she received a response from the Department of Health to her letter to the Health Minister, advising that her submission to Prof. Wilson was being considered at the March 8 meeting of the PBAC.

“I was really pleased, delighted,” said Nathalie.

The Leukeamia Foundation also prepared a supporting PBAC submission.

 “Nathalie’s remarkable resolve has put Pegasys on the PBAC’s agenda. This is no small feat and our combined advocacy must continue,” said General Manager, Research, Advocacy & Services, Caroline Turnour.

Path to PBS listing

“The path to PBS listing isn’t straight forward in this instance. Pegasys isn’t registered by the Therapeutic Goods Authority (TGA) for MPN and this can only be done by a pharmaceutical company,” Caroline said.

Only a pharmaceutical sponsor (in this case, Roche) can seek TGA registration and PBS listing for any drug or particular indication.

“We keenly await the outcome of this meeting and are committed to working with the government and Roche to overcome any barriers that may stop it being listed.”

* Interferon alfa-2a* (Roferon-A®) is listed on the PBS.

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