OBJECTIVES

: To review the National Institute for Health and Care Excellence (NICE) technology appraisals in oncology that used single-arm trial evidence in the economic model submitted in order to understand the methodologies applied, the types of data used and the criticism that these received.

METHODS

: IQVIA™ HTA Accelerator was used to identify the technology appraisals for drugs using single-arm trial evidence for the intervention arm in the economic model submitted to NICE. The NICE website was accessed to obtain the manufacturers’ submissions, the Evidence Review Group reports, the Appraisal Consultation Documents and the Final Appraisal Documents. Data extraction and analysis included details about the appraisal process, clinical effectiveness, cost effectiveness, and NICE critique and decision-making.

RESULTS

: Thirteen technology appraisals were identified. Seven of them were recommended by NICE, two were not, and four received negative preliminary recommendations. Leukaemia, lung cancer and lymphoma were the most common therapeutic areas. The average duration of the appraisals was 330 days. Matching-Adjusted Indirect Comparison and naïve comparison were the most common evidence synthesis methods used. Manufacturers used randomised clinical trials, SAT or non-randomised evidence to inform the comparator arm. Utility values were generally measured using EQ5D. Most of the base case Incremental Cost Effectiveness Ratios considered by the NICE Appraisal Committee were considerably higher than what is normally considered a cost-effective use of the National Health Service resources. When significant clinical uncertainty remained for drugs with a small eligible population with large unmet needs and/or poor prognosis, access schemes including discounts were implemented, which allowed for a switch to a positive recommendation.

CONCLUSIONS

: Despite the longer duration of SAT oncology appraisals, they are becoming more common recently. For drugs meeting end of life criteria, when significant clinical uncertainty remains, market access schemes seem key to obtaining NICE positive recommendations.