OBJECTIVES

This research identified treatments recommended for use within the Cancer Drugs Fund (CDF), assessed trends in decision making and sought to better understand the use of the CDF as an option for decision makers in the presence of uncertainty.

METHODS

A targeted literature review was conducted to identify single technology appraisals (STAs) where the treatment under evaluation was recommended for use within the CDF. Data on the key elements of uncertainty described during evaluation were extracted and analysed to observe trends that emerged from the STAs and the data collection arrangements (DCAs) implemented.

RESULTS

The review identified 28 STAs conducted between June 2016 and May 2019 where the treatment evaluated was recommended for use within the CDF. Of these, only two have been reappraised following submission of new evidence. In all cases, overall survival (OS) was noted as a primary source of uncertainty, and 27/28 STAs had corresponding DCAs targeting OS as a primary outcome measure. The National Institute for Health and Care Excellence committees noted the submission of single-arm trial evidence as a key source of uncertainty in 13 appraisals; yet only three DCAs noted that new comparator data would be collected either via a new head-to-head study or using the Systemic Anti-Cancer Therapy dataset. Uncertainty in health-related quality of life (HRQL) data were considered a driver of uncertainty in 14 appraisals; however, further data on HRQL was only noted in four DCAs.

CONCLUSIONS

While the reasons for recommending a treatment for use on the CDF are relatively consistent, there can be discordance between these uncertainties and the data targeted for collection. There remain important questions regarding the extent to which the DCAs will resolve the uncertainties raised, which in many cases will not be known until the point of reappraisal and assessment of the new evidence.