Health technology assessment (HTA) bodies worldwide recognize the importance of real-world evidence (RWE) in addressing uncertainties around the effectiveness of new drugs at the time of launch and as part of resubmissions. We assessed the use and acceptability of RWE by analyzing HTA recommendations.

We analyzed 24,841 HTA reports, including original submissions, resubmissions, extensions of original indications, and renewals, published from January 2011 to October 2021 from more than 100 HTA agencies across 37 countries.

Our analysis showed that 3,820 (15%) reports mentioned RWE. Between 2011 and 2021 there was an eight-fold increase in the use of RWE, from 4 percent in 2011 to 34 percent in 2021. RWE was most commonly included in HTAs in oncology (26%) and endocrine and metabolic diseases (13%). The main areas supported were effectiveness (40%), safety (38%), and epidemiology (35%). RWE supplemented evidence on survival and quality of life as well as resource utilization, proxy comparators, and utility. Based on an analysis of the 1,474 reports that mentioned RWE, effectiveness was mainly supported by cohort (22%) and observational studies (13%), safety was mainly derived from pharmacovigilance data (9%), and epidemiology data were collected from registries (23%). The top five HTA bodies mentioning RWE in their reports were from France, Germany, Poland, and the United Kingdom (n=2). RWE was most accepted when it supported safety and epidemiological considerations, and to a lesser extent when it was used for effectiveness aspects or understanding management pathways.

The inclusion and acceptability of RWE in HTA recommendations varies between HTAs according to their data requirements and assessment methods. While it is not always specified how RWE was considered, there is a clear tendency for its increased use and acceptability, albeit not in all areas. Greater use of and transparency around RWE are likely to continue as multiple RWE initiatives emerge globally.