OBJECTIVES: In April 2016, reforms changed the Cancer Drugs Fund (CDF) to form a temporary reimbursement fund under the remit of the National Institute of Health and Care Excellence (NICE) to collect observational data to inform subsequent technology appraisals. Oncology therapies funded under the ‘old’ CDF were reappraised by NICE and this research aims to establish whether any real-world evidence (RWE) was used for these NICE re-appraisals.

METHODS: NICE Final Appraisal Determinations for Single Technology Appraisals of ‘old’ CDF therapies were identified (up to 12/01/2017) and key data extracted.

RESULTS: Of the 21 oncology drug:indication reappraisals identified, 15/21 (71%) had published guidance of which 14/15 (93%) were recommended, all contingent on patient access schemes. Observational data was provided in 3/14 (21%) drug:indication pairings: two from the CDF and one from an Early Access Programme (EAP). Observational data from a retrospective analysis of brentuximab vedotin treated patients in R/R cHL (mainly funded through CDF) provided additional survival data that was considered more relevant by NICE for inputs into the cost-effectiveness model than Phase IV data. In the second example, RWE on the median number of brentuximab vedotin cycles in patients with R/R ALCL collected from the CDF was provided but were not used in cost-effectiveness modelling. In the third example, HRQoL data from the cabazitaxel EAP provided inputs for the manufacturer’s economic model in the absence of utility values collected as part of a RCT.

CONCLUSIONS: NICE is increasingly considering the use of RWE as part of its decision-making in the absence of robust RCT data and in some cases observational data may be preferred over RCT data. However, manufacturers must carefully plan their observational data collection protocols to ensure outputs will be accepted by Health Technology Assessment bodies.