OBJECTIVES: The analysis examines how often RWE is considered by HTA agencies and if it is a driver in HTA decision-making. RWE, providing results from outside of clinically-controlled environments, could improve the process of getting the right treatments to the appropriate patient populations.

METHODS: HTAs in 7 markets (IQWIG/G-BA, HAS, NICE, SMC, CADTH/pCODR, PBAC, and HIRA) from 2012-2017 were included. 3,800+ HTA’s were searched for mentions of observational studies, retrospective database analyses, and registry studies. Each instance was categorized and rationales for decision were used to determine if the RWE was influential. Stratification by agency, submission status, oncology/non-oncology, and orphan status were applied.

RESULTS: Of the 3823 reviews included, only 144 (4%) discussed RWE in their assessments. The range of the use of RWE varied from 0%-9% with HAS using RWE the most at 9%, followed by NICE at 5%. Across all agencies, RWE is used to support efficacy (44%)usage (17%), safety (17%), economic modelling (14%), long-term efficacy (7%), and validity of surrogate outcomes (2%). 33% of the reviews that reported RWE were for oncology products and 13% for orphan drugs. 54% of RWE mentions were in resubmissions/reassessments vs first submissions (46%). For HAS, 80% of the use of RWE was in resubmissions/reassessments. In decision rationales of cases found, 44% of reviews mentioned RWE in their decision-making, most being neutral or negative.

CONCLUSIONS: Reporting of RWE varies but remains low across HTA agencies; moreover, RWE being influential in decision making is relatively rare. In most cases, RWE is used to fill evidence gaps by supporting arguments for efficacy, usage, safety, and long-term efficacy. With the industry and stakeholders focusing on generating RWE, it is important to continue discussions with decision makers on how RWE can be utilized to help get the right therapies to the right patients faster and more effectively.