Real-world evidence in lung and hematologic oncology health technology appraisals: a review of six assessment agencies

Abstract

Aim: To assess the use and acceptability of real-world evidence (RWE) in lung and hematologic cancer appraisals. Materials & methods: A review of appraisals published by National Institute for Health and Care Excellence (NICE) in the UK was conducted. A total of 20 case studies employing RWE were identified and compared across five additional health technology assessment agencies: Scottish Medicines Consortium (SMC) (Scotland), CADTH (Canada), INESSS (Quebec), HAS (France) and IQWiG (Germany). Results: Of 80 RWE references from 20 case studies from NICE, 67 were identified in the respective CADTH submissions, 46 in IQWiG, 37 in INESSS, 37 in HAS, and 33 in SMC. NICE had the highest RWE acceptance rate (90%), followed by HAS (88%), SMC (82%), INESSS (73%), IQWiG (68%) and CADTH (67%). Conclusion: RWE was generally accepted by respective committees, allowing improved access to innovative treatments.

Plain language summary

Use of real-world evidence for assessing the value of cancer treatments: Health technology assessment (HTA) is a process used to decide whether a drug works well enough to be worth paying for. Most drugs have data showing how well they work from special studies called clinical trials. Sometimes a manufacturer also has evidence of a drug or disease that is not from a clinical trial but from the real world. This review discusses how real-world evidence (RWE) is being used for HTAs of new lung and blood cancer therapies. We reviewed twenty HTA submissions for new therapies. All twenty were submitted to these agencies: National Institute for HealthCare and Excellence (NICE; UK), Scottish Medicines Consortium (SMC; Scotland), Canadian Agency for Drugs and Technologies in Health (CADTH; Canada), National Institute of Excellence in Health and Social Services (INESSS; Quebec), French National Authority for Health (HAS; France) and Institute for Quality and Efficiency in HealthCare (IQWiG; Germany). RWE was often used to describe the type of patient that needs the new therapy. RWE was also used to show the cost of the treatment and how well the treatment worked in relation to its cost. It was also used to show how well the new therapy works compared with other treatments. Most of the RWE was accepted by the agencies. High-quality RWE in relevant patients helped support access to new treatments.

Keywords::

• health technology assessment
• oncology
• real-world evidence

Financial & competing interests disclosure

This work was funded by Millennium Pharmaceuticals, MA, USA, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. E Curran, HM Lin and LJ Walton are employees of Takeda Pharmaceuticals. S Harricharan, K Gurjar, K Nguyen and A Forsythe are employees of Cytel, Inc., which acted as a consultant to Takeda Pharmaceuticals on this review. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Additional information

Funding

This work was funded by Millennium Pharmaceuticals, MA, USA, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. E Curran, HM Lin and LJ Walton are employees of Takeda Pharmaceuticals. S Harricharan, K Gurjar, K Nguyen and A Forsythe are employees of Cytel, Inc., which acted as a consultant to Takeda Pharmaceuticals on this review. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.