OBJECTIVES: Advanced melanoma and ovarian cancer have historically had limited treatment options and poor survival with 1,800 and 1,000 Australians respectively predicted to die in 2017. Since 2013, six new treatments for melanoma and two for ovarian cancer have been listed under the Pharmaceutical Benefits Scheme (PBS). We examined the impact in terms of Australian deaths prevented and the cost effectiveness to achieve public funding.

METHODS: Using information in the Public Summary Documents (PSDs) we describe the pathway to reimbursement and estimate the number-needed-to-treat with the new treatment to avert one death at 2-years (NNT-2), based on overall survival (OS) for new treatment versus standard of care and online data sources.

RESULTS: The two new ovarian cancer treatments were recommended for listing at an ICER of AU$45-75k per QALY based on acceptance of superior OS (median OS increment [months]: bevacizumab 9.4; olaparib 7.6) with risk due to uncertainty in longer-term survival mitigated with Risk Share Agreements (RSA). We estimate for every 10 bevacizumab patients treated one additional person will survive to two years (NNT-2=10). Olaparib was recently PBS-listed (NNT-2=5). In contrast, the new melanoma treatments had less mature OS data available for PBAC decision making (median OS not reached for four treatments) and often a managed entry scheme (MES) was required in addition to RSA to obtain early listing with ICER $45-75k. Ipilimumab was listed on the basis of median OS increment of 3.6 months (NNT-2=10). Survival will further improve in Australia with PBS-listing of pembrolizumab (NNT-2=8 vs ipilimumab).

CONCLUSIONS: Despite the uncertainty surrounding the impact on OS of novel treatments for melanoma and to a lesser extent ovarian cancer, the Australian government and pharmaceutical companies have found ways to manage the risk. The availability of these products will extend survival for Australians.