Integrating Real-World Evidence Into Oncology HTA Submissions: Recent EU Examples
Author(s)
Ivana Sestak, PhD1, Janneke Luijken, MSc2, Fang-Yi Lin, MSc, MD3, Sara Angleman, PhD4, Alice ROULEAU, MASc, MBA, MPH, PharmD5.
1Thermo Fisher Scientific, Milan, Italy, 2Thermo Fisher Scientific, Utrecht, Netherlands, 3Thermo Fisher Scientific, Los Gatos, CA, USA, 4Thermo Fisher Scientific, Sundbyberg, Sweden, 5Thermo Fisher Scientific, IVRY SUR SEINE, France.
1Thermo Fisher Scientific, Milan, Italy, 2Thermo Fisher Scientific, Utrecht, Netherlands, 3Thermo Fisher Scientific, Los Gatos, CA, USA, 4Thermo Fisher Scientific, Sundbyberg, Sweden, 5Thermo Fisher Scientific, IVRY SUR SEINE, France.
OBJECTIVES: The use of real-world evidence (RWE) in oncology health technology assessments (HTAs) is increasing in Europe to address limitations in traditional clinical trial data, particularly in single-arm studies, rare cancers, or accelerated approvals. The aim of this research is to review RWE for oncology HTA submissions in selected European countries, informed by recent case studies, and to identify barriers to its successful application.
METHODS: A targeted review of oncology HTA submissions from 2019 to 2024 in four European countries (UK, Germany, France, and Sweden) was conducted. Submissions incorporating RWE, such as external control arms, post-authorization studies, and real-world patient-reported outcomes, were extracted and reviewed. Methodological approaches and HTA agency feedback were summarized.
RESULTS: 25 oncology submissions using RWE were identified, with most submissions stemming from the UK (N=19 [76%])). France (N=1), Sweden (N=3), and Germany (N=2) continue to rely primarily on randomized clinical trial (RCT) data for oncology HTAs. Most frequent types of RWE used in applications were patient/disease registries, electronic health records, and patient reported outcomes. 85.7% of submissions including RWE were accepted by the respective HTA bodies. Acceptance was highest when RWE was used to complement trial data rather than replace it, when studies employed robust methodologies (e.g., propensity score matching, clear protocols, sensitivity analyses), and when RW data incorporated was mature. Of those that were rejected, lack of robust comparative analysis and statistical methodological issues were cited for not accepting RWE.
CONCLUSIONS: RWE can meaningfully enhance oncology HTA submissions when applied strategically and transparently. Alignment with agency expectations, data credibility, and methodological rigor are essential for successful integration. The UK (NICE) leads in its robust and growing use of RWE in oncology appraisals, while other countries evaluated continue to rely primarily on RCT data for oncology HTAs.
METHODS: A targeted review of oncology HTA submissions from 2019 to 2024 in four European countries (UK, Germany, France, and Sweden) was conducted. Submissions incorporating RWE, such as external control arms, post-authorization studies, and real-world patient-reported outcomes, were extracted and reviewed. Methodological approaches and HTA agency feedback were summarized.
RESULTS: 25 oncology submissions using RWE were identified, with most submissions stemming from the UK (N=19 [76%])). France (N=1), Sweden (N=3), and Germany (N=2) continue to rely primarily on randomized clinical trial (RCT) data for oncology HTAs. Most frequent types of RWE used in applications were patient/disease registries, electronic health records, and patient reported outcomes. 85.7% of submissions including RWE were accepted by the respective HTA bodies. Acceptance was highest when RWE was used to complement trial data rather than replace it, when studies employed robust methodologies (e.g., propensity score matching, clear protocols, sensitivity analyses), and when RW data incorporated was mature. Of those that were rejected, lack of robust comparative analysis and statistical methodological issues were cited for not accepting RWE.
CONCLUSIONS: RWE can meaningfully enhance oncology HTA submissions when applied strategically and transparently. Alignment with agency expectations, data credibility, and methodological rigor are essential for successful integration. The UK (NICE) leads in its robust and growing use of RWE in oncology appraisals, while other countries evaluated continue to rely primarily on RCT data for oncology HTAs.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA209
Topic
Health Policy & Regulatory, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
Oncology