IMPACT of De Novo Real-World Evidence (RWE) Generation in Health Technology Assessment (HTA): Case Study of the Institute for Clinical and Economic Review's (ICER) Sickle Cell Disease (SCD) Assessment
Author(s)
Jaksa A1, Bradt P2, Patrick A1, Schneeweiss S3, Spackman E4
1Aetion, Inc, Boston, MA, USA, 2Institute for Clinical and Economic Review, Boston, MA, USA, 3Vice-Chief, Brigham and Women's Hospital, Boston, MA, USA, 4University of York, Calgary, AB, Canada
OBJECTIVES: As HTAs and regulators explore expanding the role of RWE in decision-making, it is important to understand the direct impact RWE can have. We conducted a qualitative evaluation of the impact of de novo RWE generation on ICER’s 2020 SCD HTA. METHODS: ICER partnered with Brigham and Women's Hospital researchers to generate RWE to address gaps identified in the SCD evidence base during its assessment process. A protocol was developed prior to conducting the RWE analysis using IBM® MarketScan® commercial claims data analyzed in the Aetion Evidence Platform®. Following the completion of the SCD HTA, a qualitative assessment of the contribution of de novo RWE to both the clinical evaluation and the cost-effectiveness model was performed. RESULTS: The evidence gaps identified and addressed using RWE were age-stratified rates of chronic conditions, acute events associated with SCD, and the costs associated with both chronic conditions and acute events. The generation of these rates, stratified by age allowed for more accurate estimates of cost-effectiveness. RWE was also generated on the background rates of the recommended standard of care and compared to the placebo arms of the RCTs being reviewed. CONCLUSIONS: Four themes emerged on the utility of de novo RWE in ICER’s SCD assessment: 1) testing assumptions; 2) verifying other model inputs 3) improving the precision and timeliness of model inputs; and 4) altering the structure of the economic model. In cases where the drugs included in an assessment have been marketed for a period of time, RWE may play an even broader role by providing data on accumulated patient experience. This case study demonstrates the utility of de novo RWE analysis to inform the cost effectiveness of new therapies shortly after regulatory approval.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PRO106
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Reimbursement & Access Policy, Systems & Structure, Value of Information
Disease
Drugs, Rare and Orphan Diseases, Systemic Disorders/Conditions