Real-World Evidence (RWE) in Action: A Review of Australia's Health Technology Assessment (HTA) of Non-Drug Health Technologies

OBJECTIVES: Compared to drugs, clinical evidence on non-drug health technologies tends to be limited due to reasons such as challenges in implementing a double-blind procedure and a lack of infrastructure to conduct randomized controlled trials (RCTs). As such, there is a growing interest in leveraging real-world evidence (RWE) to demonstrate the value of non-drug health technologies in health technology assessment (HTA). We explored how RWE has been used to support clinical claims in applications assessed by Australia’s Medical Services Advisory Committee (MSAC).

METHODS: MSAC applications published between March 2019 and April 2024 were identified, from which RWE and assessment outcomes were extracted.

RESULTS: Forty-nine of the 66 applications identified (74%) used RWE to support clinical claims, with 31 (63%) of the technologies being recommended for public funding. Applications using RWE spanned various therapeutic areas, with 29 (59%) pertaining to high-risk medical devices. Similar to drug assessments, RWE informed a range of outcomes (e.g., safety, effectiveness, diagnostic accuracy, change in patient management) and supported clinical claims in multiple ways, such as confirming RCT evidence, providing a control arm for treatment comparisons where the assessment was based on a single-arm trial, and addressing evidence gaps when trial evidence was unavailable or infeasible. MSAC’s concerns around RWE included a lack of comparators, risk of bias, and small sample size due to the rarity of the condition; however, such concerns may be superseded by factors such high unmet need, equity to access, and ethical considerations.

CONCLUSIONS: RWE is used in diverse ways to support clinical claims in the HTA of non-drug health technologies in Australia. Consistent with current MSAC guideline recommendations, RWE is often acknowledged as a valid source of evidence in MSAC applications, particularly when high quality RCTs or comparative non-RCTs are limited. The relative impact of RWE compared with other factors warrants further exploration.