(B30) Utility of Healthcare Professional (HCP) Surveys for Evaluating the Effectiveness of Additional Risk Minimization Measures (aRMMs): Experience from an HCP Survey Assessing the Effectiveness of aRMMs for Xeljanz® in Europe

Publication Number: 642

Background: Surveys are often used to evaluate the effectiveness of additional risk minimization measures (aRMMs), including the distribution, utilization and stakeholder knowledge about risks of an approved drug, to fulfill regulatory requirements in accordance with GVP module XVI.

Objectives: To describe operational challenges and methodological limitations associated with surveys based on recent experience with an HCP survey assessing the effectiveness of Xeljanz® aRMMs.

Methods: Two multimodal surveys were conducted in 10 European countries from 2021-2022 among specialty care HCPs who are intended recipients of aRMM materials: one survey among HCPs treating rheumatoid arthritis (RA) and psoriatic arthritis (PsA) (RA/PsA survey), and a second among HCPs treating ulcerative colitis (UC) (UC survey).

Results: 1) Regulatory processes delayed survey launch in all countries from an intended 6-12 months after aRMMs distribution to an average of 20 months. 2) Response rate was low: 164 of 18,764 (1.7%) invited HCPs and 81 of 12,777 (1.2%) completed the RA/PsA and UC surveys, respectively, despite multiple reminders. 3) Limitation with human recall is an inherent disadvantage with surveys: over 50% of HCPs did not remember receiving the aRMMs materials. 4) Social desirability bias was probable in responses to prescribing behaviors. For example, about 60% of HCPs answered “Yes” to the negative response question: “Check blood glucose level before initiating Xeljanz?” as they likely assumed that this was the expected answer. 5) Although HCPs’ overall responses to knowledge questions were below the study’s pre-set threshold for effectiveness (≥80% HCPs correctly answering ≥ 80% of all questions), variations in knowledge by key risk messages were observed, suggesting that the applicability of such threshold to measure effectiveness is subjective.

Conclusions: Classifying HCP surveys as post-authorization safety studies (PASS) in Europe poses operational burden due to stringent regulatory requirements, resulting in challenges with the timely assessment of aRMM effectiveness. Challenges in recruiting HCPs due to privacy regulations and HCPs’ low interest led to a low response rate, which has been noted in many prior European surveys targeting HCPs. Low response rate affects the precision of survey results, possibly resulting in selection bias, and making generalization difficult. Operational and methodological limitations of HCP surveys pose great challenges in the evaluation of aRMMs. Given the limitations within the confines of a PASS classification, additional methods leveraging secondary healthcare databases should be considered to evaluate the effectiveness of aRMMs by assessing both actual prescribing behaviors and outcome indicators.