OBJECTIVES: On June 15, 2017, NICE announced the third Final Appraisal Determination (FAD) for the antibody-drug conjugate (ADC), trastuzumab-emtansine. This level of reconsideration is unprecedented and can be attributed to the simultaneous reforms of the Cancer Drugs Fund (CDF). This paper highlights the process by which trastuzumab-emtansine was assessed by NICE.

METHODS: Publically available CDF, EMA, NICE, and SMC data on trastuzumab-emtansine were screened up to June 27, 2017.

RESULTS: Trastuzumab-emtansine received marketing authorisation in Europe on November 15, 2013 for treating advanced/metastatic pre-treated breast cancer. It was approved for inclusion into the CDF with free-pricing in February 2014, costing £90,381 (€102,118) per patient. The first NICE FAD (August 2014), was not to recommend. The second FAD (November 2015), was again not to recommend. In September 2015, trastuzumab-emtansine was announced as to be axed from the CDF, as part of a cost-containment drive, but in November 2015 after significant discounting, this decision was reversed. Post NAO audit, trastuzumab-emtansine qualified for the CDF rapid reconsideration process in December 2016. As part of this, in its third FAD (June 2017), trastuzumab-emtansine was recommended for routine use on the NHS but with a patient access scheme. In Scotland (where the CDF does not apply), trastuzumab-emtansine was accepted by the SMC in April 2017 after an initial non-recommendation in October 2014.

CONCLUSIONS: Trastuzumab-emtansine received European market authorisation for over three years prior to it being approved by NICE or the SMC for public reimbursement. Nevertheless, in England, it was available for over two years as part of the CDF, including almost 18 months at list price. Once the CDF was closed, the manufacturer reached an agreement for reimbursement with NICE, suggesting that the existence of the CDF provided an alternative market access route that disincentivised the relevance of seeking a NICE recommendation.