Abstract
PHARMAC funds medicines in NZ to a fixed budget and according to a range of factors, including efficacy and cost effectiveness. This analysis compared real world outcomes with published clinical trial results for four recently funded cancer drugs: azacitadine for myelodysplastic syndromes, acute myeloid leukaemia and chronic myelomonocytic leukaemia; abiraterone for metastatic castration-resistant prostate cancer; gefitinib for non-small cell lung cancer; and lenalidomide for relapsed and refractory multiple myeloma. National databases provided by NZ’s Ministry of Health were used to identify all patients who had been funded on these medicines between January 2012 and December 2016. Details of time on treatment and survival were obtained from dispensing and registry data, respectively. Overall survival (OS) was calculated from these data and then compared with clinical trial data. Two drugs had shorter median OS than published trials. Azacitidine had a median OS of 41 weeks in myelodysplastic syndrome, 24 weeks in acute myeloid leukaemia, and 28 weeks in chronic myelomonocytic leukaemia, compared to 105 weeks in the key trials. Gefitinib had a median OS of 47 weeks, compared with 96.2-118.7 weeks in key trials. Lenalidomide and abiraterone did not reach median OS at the time of analysis, but both trended shorter than the clinical trials. In NZ, real world cancer survival outcomes were considerably worse than expected from clinical trial data for two newly funded cancer treatments. When published efficacy for cancer treatments is marginal, a poorer outcome from real world use could have a significant adverse effect on cost-benefit assumptions for funding.
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