Randomized controlled trials (RCTs) are typically considered the gold standard source of clinical evidence for reimbursement submissions, but they can often be resource-intensive, expensive, and may not always be appropriate. For example, it may be unethical to assign patients to an untreated or undiagnosed control group, or blinding may not be feasible when assessing medical devices. Evidence for medical devices is therefore often limited to nonrandomized studies. We explored the use and value of real-world evidence (RWE) in the reimbursement of medical devices across several health technology assessment (HTA) agencies.
A narrative review was completed to compare the acceptability of RWE for the HTA evaluation of medical devices across a convenience sample of countries. English-language published guidance documents were reviewed, and study design preferences extracted.
In Australia, France, Germany, Ireland, Norway, and Scotland, HTA agencies prefer RCT evidence but accept RWE as supporting data. In England, there is no preferred study design, with directly observed clinical outcomes, evidence syntheses, nonclinical, and modelling studies accepted. Notably, methods and processes for HTA programs are being reviewed and are expected to place a greater emphasis on RWE. In Australia, pseudo-randomized trials, comparative cohort studies, case series, and other study designs are permitted. In France, nonrandomized or nonblinded trials, patient preference cohorts, prospective comparative observational studies, and propensity score matched cohorts are permitted, accompanied by justification. In Scotland, lived experiences, RWE, and systematic reviews are accepted. In Germany, nonrandomized studies are deemed to provide “minimum”, “very low” or “low” certainty of results. In Norway, RWE may be accepted if no RCT data are available, or to support RCTs.
In the assessment of medical devices, where RCTs are unsuitable, RWE can form a feasible alternative. Real-world evidence is increasingly being recognized as a valuable source of evidence for medical interventions and is accepted by a number of HTA agencies. No funding was received for this study.