OBJECTIVES : Innovative therapies are increasingly securing expedited marketing authorization on immature data, but to justify the price tags of these technologies, payers and prescribers are demanding large amounts of evidence. One well-discussed option to bridge this gap is the collection of real-world evidence (RWE) to validate that the benefits a therapy shows in a controlled randomized clinical trial (RCT) translate into real-world treatment practice. This research evaluates their impact on reimbursement and prescribing decision-making.

METHODS: PAREXEL’s integrated panel of 14 reimbursement and medical affairs experts were surveyed in November 2018 on the importance of the following data types for prescribing and reimbursement across EU5 countries: clinical (RCT) efficacy, clinical (RCT) safety, quality of life (QoL), budget impact, cost-effectiveness, burden of illness, mechanism of action (MoA), real-world effectiveness data, real-world safety data. Each criterion was scored separately in terms of importance for payers and clinicians (scale:1 [low]—5 [high]).

RESULTS: For payers, highest scoring criteria were clinical (RCT) efficacy (4.4), QoL (3.6), and budget impact (3.2); the lowest were MoA (1.6), real-world safety (2.0) and real-world effectiveness (2.2). For clinicians, highest scoring criteria were clinical (RCT) efficacy (4.7), clinical (RCT) safety (4.0) real-word effectiveness (3.7), while the lowest were MoA (2.1), and cost-effectiveness (2.5).

CONCLUSIONS: Overall, both payers and clinicians place great emphasis on RCT data, but currently clinicians take greater account of RWE than payers. However, payer scores for real-world data are anticipated to increase in the future, driven by greater RWE collection and post-launch payer re-assessments. Nevertheless, there is alignment between key data criteria driving payer and prescriber decision-making. Early development and co-creation of payer messaging and scientific communication points can ensure alignment, facilitate engagement, and prospectively mitigate key evidence gaps in an efficient and integrated manner to reduce uncertainty for both physicians and payers.