Health Technology Assessment (HTA) Case Studies: Comparing Acceptability of Real-World Evidence (RWE) in Appraisals for Oncology Medicines
Author(s)
Zong J1, Pan X2, Rojubally A3, Jiao X1, Bruno A1, Gdovin Bergeson J4
1Bayer Healthcare Pharmaceuticals, Inc., Whippany, NJ, USA, 2Bayer Healthcare Pharmaceuticals, Inc., Boston, MA, USA, 3Franklin Pharmaceutical Consulting, Surrey, BC, Canada, 4Franklin Pharmaceutical Consulting, Columbia, SC, USA
Presentation Documents
OBJECTIVES:
RWE is increasingly utilized in HTA appraisals. Clear guidance on RWE to be accepted by HTA bodies is lacking. This review compared RWE acceptability in HTA decision-making in recent appraisals for oncology medicines.METHODS: A search of
Technology Appraisals published by NICE, Benefit Assessments published by G-BA and Opinions on Medicinal Products reports published by HAS from January to December 2022 was conducted. Oncology medicine appraisals which included RWE were identified and RWE acceptability was classified as (primary evidence, supportive evidence, not adequate for decision making, not addressed, or other) by reviewer’s assessment. Medicines reviewed by more than one HTA body were selected for comparative assessment of RWE acceptability.RESULTS:
RWE was referenced in 16 reports by NICE, 14 by G-BA, and 10 by HAS. Six oncology medicines referencing RWE were selected as case studies: amivantamab, sotorasib and tepotinib (NICE and G-BA); avapritinib, axicabtagene-ciloleucel and tisagenlecleucel (HAS and G-BA). NICE acceptability of RWE was in alignment with G-BA's for amivantamab (RWE not adequate for decision-making) and divergent for tepotinib and sotorasib (NICE: RWE as supportive evidence and G-BA: RWE not adequate for decision-making). HAS and G-BA were in alignment for tisagenlecleucel and axicabtagene-ciloleucel (RWE not adequate for decision-making) and diverged for avapritinib (HAS: RWE as supportive evidence and G-BA: RWE not adequate for decision-making). RWE acceptability by NICE as supportive evidence is promising. HAS and G-BA’s evaluation of RWE suggests emphasis on data quality, selection biases, and confounding factors related to RWE use.CONCLUSIONS:
There is discrepancy in the assessment of RWE by the 3 HTAs in recent oncology appraisals. Demands for quick and uniform access of new oncology therapies warrant harmonization of RWE relevance across HTAs, and development of comprehensive consensus guidelines with all stakeholders to standardize best practices for RWE studies and assessment.Conference/Value in Health Info
2023-11, ISPOR Europe 2023, Copenhagen, Denmark
Value in Health, Volume 26, Issue 11, S2 (December 2023)
Code
HTA8
Topic
Health Technology Assessment, Real World Data & Information Systems, Study Approaches
Topic Subcategory
Decision & Deliberative Processes, Literature Review & Synthesis, Reproducibility & Replicability, Value Frameworks & Dossier Format
Disease
Drugs, No Additional Disease & Conditions/Specialized Treatment Areas, Oncology