Abstract

It is increasingly common for health technology assessment (HTA) submissions to be prepared based on evidence from single arm trials or in situations where comparisons cannot be made between randomised controlled trials (RCTs). Manufacturers are using increasingly complex statistical approaches to fulfil the requests of HTA bodies for robust comparisons between drugs of interest in these situations of data paucity. In this study we evaluate the use of two of the main approaches used in recent oncology HTA submissions, “Matching Adjusted Treatment Comparison” (MAIC) and “Simulated Treatment Comparison” (STC). HTA websites for England, France, Germany and Scotland were searched to identify oncology drug submissions that relied on the use of either MAIC or STC to compare between single arm trials or individual arms of RCTs. The methods, criticisms and response of the HTA agencies to both MAICs and STCs were assessed and summarised. Four oncology interventions for which HTA submissions have been submitted were selected as relevant: ibrutinib, axitinib, panobinostat and osimertinib. All of these interventions were assessed by NICE between 2015 and 2017. The use of both MAIC and STC was criticised in all cases due to their underlying assumptions. However, in all cases the treatments have been recommended by at least one HTA body. The use of MAIC and STC in the HTA submission process is still in its early stages and is still subject to some criticism. The different assessment criteria and data requirements of HTA bodies across these countries leads to differences in their willingness to accept MAIC and STC data. It is possible to receive positive recommendations based upon MAIC and STC data, this is especially the case in situations of high unmet need.

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