The Use of Published Real-World Evidence In HTA: An Analysis Of The Evidence Base Of Company Submissions In 2016 Nice Appraisals

OBJECTIVES: The use of real-world evidence (RWE) to support HTA is of increasing interest to pharmaceutical companies. This study aims to quantify the use of published RWE in the evidence base provided by companies for the NICE appraisal process. METHODS: A retrospective, descriptive analysis of NICE technology appraisals in 2016 was performed. Of 56 identified records, only single-technology, first-time appraisals with available company-evidence submission documents were included (n=28). Information was collected from the references in the company-evidence submissions, including evidence type, peer-reviewed publication status, and document section referenced in. RESULTS: All company-evidence submissions in 2016 referenced RWE - as defined by ISPOR; however, only 18% (5/28) included RWE on the technology being appraised. The mean number of references in evidence submissions was 150 (median 132; range 66-303); RWE accounted for 15% (range 2-50%) of evidence, with the remainder being RCTs (27%), cost-effectiveness (10%), and other (48%). RWE was mainly used to describe health conditions (section 3,45%) and support cost-effectiveness analysis (section 5,33%), and less to support clinical effectiveness (section 4,16%) or other document sections (6%). Of all evidence, 30% was not published in peer-reviewed journals, 12% was published as congress abstracts, and 58% as full articles in peer-reviewed journals. Of RWE, 11% was unpublished; 78% was full-article published, mostly in journals with a clinical-specialist audience (76%), and less with a general-medical (9%), decision-making (8%) or health-economic (6%) focus. CONCLUSIONS: RWE is an integral part of company-evidence submissions to NICE for HTA. Currently, RWE is mainly used as background to describe health conditions, and sparingly to demonstrate real-world effectiveness of the technology. A substantial proportion of the total evidence is unpublished. There is future scope to enrich the total evidence base further with technology-specific RWE and to increase transparency and credibility by timely publication in peer-reviewed journals.