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Australia: Orphan Drugs Kalydeco And Soliris Triumph

October 26, 2014



Australia’s Health Minister Peter Dutton announces on October 26th that the following 2 orphan drugs are to be listed on the Pharmaceutical Benefits Scheme (PBS) effective December 1, 2014:

•   Vertex Pharmaceuticals’ Kalydeco (Ivacaftor) for Cystic Fibrosis (CF)

•   Alexion Pharmaceuticals’ Soliris (Eculizumab) for Atypical Hemolytic Uremic Syndrome (aHUS).

I – Australia’s PBAC & PBS

Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) makes national funding decisions for the Australian public healthcare system.  The PBAC assesses orphan drugs separately from non-orphan drugs using very strict criteria. Australian approval and reimbursement of a new medicine is a multi-step process:

•   Therapeutic Goods Administration (TGA) approves the new medicine

•   Pharmaceutical Benefits Advisory Committee (PBAC) assesses the new medicine for effectiveness and cost-effectiveness

•   Listing on the Pharmaceutical Benefits Scheme (PBS) based on the PBAC assessment.

II – Kalydeco

 The addition of Kalydeco to the PBS follows over a year of discussion between patients, clinicians, Vertex Pharmaceuticals, the Australian government, and the Department of Health. The PBAC defers making a decision on Kalydeco at its July 2013 meeting, recommends Kalydeco in November 2013, and then recommends it for a second time in March 2014. According to Mr. Dutton’s October 26th Press Release:

“ .. The PBS subsidy of this medicine, which would otherwise cost approximately $300,000 a year per patient, will bring great relief to the patients and the families of people affected by this life threatening condition …Consistent with the advice of the Pharmaceutical Benefits Advisory Committee (PBAC) that this medicine could be listed on the PBS based on a pay-for-performance basis, all cystic fibrosis patients six years and older who have a G551D mutation in the CFTR gene will be treated with ivacaftor for as long as needed. I’m pleased to announce that the sponsor of ivacaftor, Vertex Pharmaceuticals, has agreed to proceed with the PBS listing as recommended by the PBAC

 The Australian government approves$174.5 million over the next 4 years to fund Kalydeco on the PBS. More than 250 Australians are affected by the CF G551D gene mutation.

Kalydeco is approved in Australia in July 2013 for patients with at least one copy of the G551D mutation.

III – Soliris & Other Medicines

Soliris (Eculizumab) for the treatment of the ultra-rare disease, Atypical Haemolytic Uraemic (aHUS), will also be added to the PBS effective December 1st of this year. This will help patients and their families meet the $500,000 annual cost per patient per year. The government approves $63 million over the next 4 years to provide Soliris on the PBS.

Nine other new and amended medicines are to be listed on the PBS starting December 1, 2014, including treatments for Hepatitis C and prostate cancer. All PBS listings are subject to final arrangements being met by the suppliers/sponsor companies of the medicines.


CF Australia’s Statement

Minister Dutton’s Statement

Vertex Pharmaceuticals’ Statement.

Please Note: “Chemlab2” by Tahoenathan (Own work) [CC-BY-SA-3.0] | via Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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