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The long road to PBS listing for Orkambi

Orkambi (lumacaftor and ivacaftor), a treatment for cystic fibrosis (CF), will go before the PBAC for a fourth time at the upcoming July 2018 PBAC meeting. So far, the road to reimbursement has been filled with multiple rejections, a strong voice from patient advocacy groups, increasing media interest, and the apparent ‘impossible position’ in which both Vertex Pharmaceuticals (the sponsor) and the federal government find themselves.

The sponsor, and patient advocacy organisations, contend that Orkambi is an essential medicine for cystic fibrosis patients that would help limit hospitalisations associated with CF and enhance ability to participate in school, employment, and the community. This view is buoyed by remarks from Health Minister Greg Hunt, who has encouraged Vertex to “give it another go” with the PBAC following an earlier rejection. In commentary from previous submissions, the PBAC supports this view and yet has rejected Orkambi three times “on the basis of unacceptable cost-effectiveness at the requested price and uncertainty around the longer term impact of lumacaftor/ivacaftor on lung function and survival beyond two years of treatment”.

The clinical benefit of Orkambi to cystic fibrosis patients appears to be have been demonstrated and accepted in previous PBAC submissions, with the barrier to recommendation continuing to rest on price, despite repeated proposals for risk-share arrangements and price caps. It is however unclear whether Vertex is seeking a Special Pricing Arrangement.

Recent history has shown that innovative high cost medicines often require multiple trips to the PBAC before being recommended and PBS-listed. Examples include Spinraza (for spinal muscular atrophy), Ibrance and Kisqali (for breast cancer), all requiring multiple submissions prior to recommendation. Celgene’s Otezla (apremilast) has abandoned seeking reimbursement entirely, following seven PBAC rejections.

With its recent stint on Sunrise, Orkambi is a high-profile example of what can occur when both the PBAC and the sponsor are unable to reach agreement on the value of a medicine. Could this evolution of the reimbursement environment extend to current pipeline medicines? Upcoming treatments for migraine might face similar hurdles, as currently available treatments are older, cheaper medicines and the potential patient population is large (equating to high cost to government).

The outcome of July’s meeting may demonstrate that three or four PBAC submissions for innovative high cost drugs has become ‘the new normal’ for gaining reimbursement in Australia.

 

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