PD20 Real-World Evidence To Support Single Arm Trials Of Oncology Interventions: Necessity or ‘NICE’ (National Institute for Health and Care Excellence) to Have?

Introduction The recent National Institute for Health and Care Excellence (NICE) consultation on methods for health technology evaluation (HTE) outlined plans to include guidance on the role of real-world evidence (RWE) in HTE. This is particularly pertinent for interventions where the evidence base consists of a single arm trial (SAT) design, which is more frequently observed in oncology. This study reviewed the influence of RWE submitted as part of the evidence base for NICE oncology appraisals. Methods A search for NICE HTEs was conducted for interventions supported by SATs from January 2017-November 2021. Evidence was stratified by submission packages with SAT evidence alone or in combination with randomized controlled trial (RCT) evidence, with or without RWE. Results Thirty-two decisions for interventions supported by SATs were made by NICE between 2017-2021, all in oncology indications. Fifty percent were supported by SAT evidence and fifty percent by RCT plus SAT evidence, both with or without RWE. A lower proportion of RCT/ SAT HTEs submitted RWE compared to SAT HTEs (fifty vs ninety four percent). Seventy five percent and nineteen percent of SAT HTEs received a positive recommendation, with and without restrictions, irrespective of submitting RWE. One negative decision was observed for SATs supported by RWE. Sixty three percent and thirty eight percent of RCT/ SAT HTEs received a positive recommendation, with and without restrictions. Overall, the proportion of positive recommendations were lower for HTEs submitting RWE (ninety six percent) compared to HTEs not submitting RWE (one hundred percent), which is in contrast to recent findings specific to orphan oncology HTEs (one hundred versus seventy eight percent). Conclusions RWE was more commonly submitted to support SAT HTEs, than RCT HTEs. The use of RWE seems to be established as a necessity to supplement a SAT evidence base, whereas RWE is more generally a nice to have in RCT HTEs. However, RWE appears to positively influence decision-making for orphan oncology indications with a more neutral influence for non-orphan indications.