USING REAL-WORLD EVIDENCE FOR HEALTH TECHNOLOGY ASSESSMENT SUBMISSIONS: LESSONS AND INSIGHTS FROM REVIEW OF NICE’S TECHNOLOGY ASSESSMENT REPORTS (2016-2017) OBJECTIVES: Real-world evidence (RWE) is defined as evidence for product’s clinical effectiveness, safety, quality of life benefits or cost effectiveness in real-world setting. In many instances RWE provides valuable supplemental data to support product’s value proposition. Increasingly, health technology assessment (HTAs) agencies and even regulatory agencies are now requesting and accepting RWE. To understand latest trends in use of RWE, we conducted a systematic review of latest UK’s NICE technology assessments.
METHODS: A systematic review of all 2016-2017 UK’s NICE technology assessment (TA) reports was conducted. Reports were reviewed for all types of RWE (registries, observational studies, chart record studies, post-marketing surveillance, hospital audits etc). Reports were included if RWE was mentioned by NICE, ERG or Sponsor in the final recommendations or discussions text. For selected reports, we analyzed the context for RWE, NICE’s determination and any concerns. Lessons and insights were developed from selected case studies.
RESULTS: Nineteen HTA reports were found for which NICE requested, considered or accepted use of RWE. Twelve of the 19 reports were for cancer, 2 were for asthma, 2 for musculoskeletal disorders and other were for eye, HCV, rheumatoid arthritis and renal disease. In these 19 reports, we found 30 different references to RWE. In terms of context, 10 were for cost effectiveness, 6 for clinical effectiveness, 5 for clinical management, 3 for dosage, 4 for overall survival extrapolation and 2 for utility scores. In 21 of the 30 instances, NICE accepted the use of RWE. In 5 of 30 instances there were concerns and in 4 instances, NICE requested for RWE. The products with most instances of RWE discussion include
d Brentuximab vedotin (5), Ibrutinib (4) and Sorafenib (4). The accepted use examples included longitudinal studies (Sorafenib’s 3 studies) or chart reviews (e.g. Ibrutinib’s 10 year epidemiology study) or secondary reference for model inputs (e.g. Mepolizumab). NICE’s concerns were related to mismatch of patient population (e.g Brentuximab), uncertainty (e.g. Collagenase clostridium histolyticum) and small sample sizes (e.g. transplant registry for immunosuppressive therapy). The requests for RWE were for a new proposal for cancer drug funding to generate data for a specific subgroup (Ibrutinib), UK specific registry data (Mepolizumab) and US or French registry data in lieu of no UK RWE (Vismodegib).
CONCLUSIONS: To our knowledge this is the first report describing use of RWE for HTA submission. This analysis provides valuable lessons and insights for manufacturers to leverage RWE for HTA submission.
Source: NOVEL Health Strategies ISPOR Podium Presentation. Please contact for replays or questions or capabilities at info@novelhealthstrategies.com
www.NOVELHealthStrategies.com
Authors
Saurabh (Rob) Aggarwal
Ozlem Topaloglu
Sushil Kumar
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