Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 117 6♦ B. Top Five Most Referenced Appraisals TA391 Cabazitaxel for hormone-relapsed metastatic prostate cancer treated with docetaxel In 2016, NICE assessed the cost-effectiveness of anticancer taxane therapy cabazitaxel for the treatment of metastatic prostate cancer that relapsed after it was treated with docetaxel. Sanofi was the submitting company and the School of Health and Related Research (ScHARR) produced the evidence review group (ERG) report. TA391 is an updated appraisal of TA255. In this appraisal, the company did not collect data on health-related quality of life in the main trial that investigated the use of cabazitaxel, so it took utility values from the expanded access programme in the United Kingdom. The ERG found several issues with data from this program: the open-label nature, generalizability (patients were potentially more fit than in the trial), the analysis was performed at interim and had not yet been subject to peer review. The Committee partly shared the vision of the ERG: ‘the Committee was concerned about the uncertainty around the utility value and whether the utility value as calculated from the early access programme could be applicable to the wider population with hormone-refractory metastatic prostate cancer refractory to docetaxel treatment’. On the other hand, the committee also appreciated the efforts of the company: ‘The committee acknowledged the limitations of using data from the UK early access programme but, in the absence of more robust evidence on health-related quality of life, it concluded that the company had used the best available data to estimate utility values.’ The initial Final Appraisal Determination did not recommend the use of cabazitaxel, leading the company to appeal to the Appeal Panel, focusing in part on the interpretation of the EAP trial. (‘the context of the EAP trial was misinterpreted, data from the EAP trial were incorrectly interpreted, and the nature of interim data was misunderstood by the committee’). The Appeal Panel ‘understood both sides’ positions and regarded them both as reasonable’ and as such dismissed all the grounds of appeal. After a new confidential discount to the price of cabazitaxel was arranged, its use has been recommended within the NHS.
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