Getting Pegasys onto the Government’s Approved Drug Listing
By Nathalie Cook
MPN Patient and Advocate, Nathalie Cook, works as an Accredited Practicing Dietitian in Community Health in Melbourne
In Australia over the last 20 years, myeloproliferative neoplasm (MPN) consumer advocacy has been strong. This began with the tireless support that the late Ian Sweet offered fellow MPN sufferers via the US based MPN-NET email list from the late 1990s and continued when Ken Young established MPD-oz, the first Australian email list, which he still manages today. Together these men have responded to thousands of emails from people with MPN both in Australia and around the world with messages of knowledge, wisdom and reassurance. In recent years other Australian online MPN groups have been setup to provide peer support to the MPN community.
In 2004, Ian and Ken began advocating for Pegasys® (the slow release, better-tolerated formulation of interferon (IFN)), seeking the Government’s subsidised Pharmaceutical Benefits Scheme (PBS) for MPN, as an affordable treatment option. They approached Roche who suggested ‘off-label’ access. At that time, no international clinical trials had been done with Pegasys for MPN treatment, so evidence of efficacy was lacking. This made government approval of Pegasys for MPN seem bureaucratically insurmountable.
In 2015, MPN patient Lara Chapman encouraged people with MPN in Australia to write to the Government’s Senate Enquiry into ‘The Availability of new, innovative and specialist cancer drugs in Australia’. This resulted in 40 MPN consumer submissions expressing a desire for PBS access to Pegasys for MPN, out of a total of 205 senate enquiry submissions.
In 2015 Lara, together with five other MPN patient volunteers from around the country, (Jolanda Visser, Sue Taylor, Angela Willet, Ken Young and Nathalie Cook) formed the MPN Alliance Australia (MPN AA), with goals of increasing community awareness of MPN, educating and supporting the MPN community, raising funds for MPN research and advocating for Australians with MPN.
The MPN AA co-hosted Australia’s first MPN doctor/patient conference in 2017, in partnership with the US-based MPN Education and Advocacy International. This conference brought together 150 MPN patients from around Australia and New Zealand and 10 haematologists three US-based and seven Australian.
Thanks to generous donations from the community, in 2018 MPN AA donated $20,000.00 to support Brisbane based haematologists. Assoc. Professor Steven Lane’s contribution to the Chicago-based MPN Research Foundation’s Interferon Initiative. This international multi-center collaboration of experts seeks to deepen understanding of how IFN works to treat MPN.
In 2011, in addition to all the other Australian MPN advocacy, MPN patient Nathalie Cook began a seven year campaign to Roche Products and the government for PBS access to Pegasys for people with MPN. This led to Pegasys becoming available on the PBS for MPN from 1 August 2018, in the Australia’s first consumer-led PBS drug listing! This was also the first government approved Pegasys listing for MPN to occur worldwide.
After many years of phone calls, emails and letters to Roche and the government, the breakthrough started in 2016 when Nathalie was invited to the Rare Cancers Australia CanForum in Canberra, as a consumer representative with the Leukaemia Foundation. At the CanForum she spoke with Prof. Andrew Wilson, Chair of the Pharmaceutical Benefits Advisory Committee (PBAC), (whose role is to advise the government on new PBS drug listings) about the unmet need for Pegasys to treat MPN.
This discussion led to Nathalie writing a submission directly to Prof. Wilson, seeking the addition of Pegasys on the PBS for MPN. In her submission she included information on her own experience with both types of IFN, (Roferon and Pegasys) and her better tolerance of Pegasys compared with Roferon. She included extensive references to peer reviewed medical literature providing evidence of safety and efficacy of Pegasys in MPN.
The basis of her submission was that the older form of IFN (Roferon) was on the PBS for MPN, but was poorly tolerated, due to adverse side effects. She highlighted that Pegasys, the slow release better tolerated formulation of IFN should be on the PBS for MPN, because it is equally effective in controlling the disease, however the ‘off label’ price of about AUD$500.00 per weekly dose made it unaffordable for most patients.
When Nathalie’s submission was accepted by the PBAC for consideration in January 2017, she immediately contacted haematologists around Australia, the Leukaemia Foundation and Rare Cancers Australia, seeking support to get approval of Pegasys for MPN on the PBS. These groups subsequently sent the PBAC correspondence confirming a clinical need for Pegasys to treat MPN.
MPN-AA members Nathalie Cook and Ken Young attend the official announcement to put Pegasys on the government drug listing
The MPN AA used social media to encourage MPN patients around Australia to contribute comments via the PBS online consumer portal, stating why they wanted Pegasys on the PBS for MPN and what this change would mean to them in terms of quality of life.
Although the PBAC recognised a clinical need for Pegasys in MPN and knew it was safe long term with low toxicity, after a decade of experience with Hepatitis C treatment, the path to approval was not simple.
Firstly, Pegasys did not have Therapeutic Goods Administration (TGA) approval for MPN. TGA approval for a specific disease indication is usually required before a drug company can make a PBAC submission. Secondly, no Australian clinical trials had been conducted for Pegasys in MPN, another usual PBAC requirement for submissions.
Additionally, Roche had no plans to write a submission for PBS listing of Pegasys for MPN, because the drug was considered ‘old’ and was close to coming off patent. In this exceptional case, the PBAC requested Roche lodge a submission for PBS listing of Pegasys for MPN, in response to Nathalie’s consumer submission.
During 2017, the PBAC worked with Roche, Rare Cancers Australia, haematologists and the Leukaemia Foundation on the issue of access to Pegasys for MPN. In the meantime, Nathalie continued discussions with Roche on the matter. Just before the December 2017 holiday season, she received a phone call from Roche saying the PBAC had approved inclusion of Pegasys on the PBS for MPN.
The next step was for the government and Roche to finalise price negotiations, before doctors could prescribe Pegasys for their MPN patients at the PBS price of AUD$39.50 (or AUD$6.40 for concessional patients). The MPN AA was delighted to share this wonderful news with the MPN community!
In July 2018, Nathalie was invited and with Ken Young attended, the Health Minister’s announcement that Pegasy and also three other cancer drugs that would be available to patients on the PBS from 1 August 2018.
The achievement to list Pegasys on the PBS for MPN in Australia demonstrates the power of consumer advocacy and patient networking, and the importance of collaboration between consumers, the medical community, the pharmaceutical industry and government.
View Videos from the Australian MPN Patient/Caregiver Program
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