From a MedTech perspective, ISPOR Europe 2024 was a little odd. There is no doubt that the number of attendees with a MedTech remit has increased. Attendance at the Devices and Diagnostics Special Interest Group was limited to standing room only, and post-meeting there has been a lot of interest in supporting upcoming projects. The ISPOR MedTech community is alive and well. And yet, if you look at the agenda, very few sessions took a MedTech-perspective. This was then highlighted in the plenary session on day 2, where the highest rated audience question asked what EU Joint Clinical Assessment (JCA) means for devices and diagnostics. Clearly plenty of interest in the topic, but sadly this question wasn’t addressed. Here’s hoping for change in 2025, and we will be submitting issue panel topics to help put MedTech higher on the agenda.
However, one session this year that did hit a hot topic for MedTech centred around the value of real-world evidence (RWE) to support regulatory and reimbursement activities for devices and diagnostics. The utilisation and acceptability of RWE in MedTech continues to draw focus, with discussions in this session highlighting both the opportunities and challenges associated with leveraging RWE in this context.
The global landscape for the evaluation of diagnostic tests and medical devices is marked by diversity in HTA frameworks and methodological approaches. Lavinia Ferrante di Ruffano, York Health Economics Consortium, took diagnostics as an example; there is consensus on the value of randomised controlled trials (RCTs), yet these are often uncommon. In their absence, evidence linkage—combining test accuracy with real-world management decisions and treatment efficacy to deduce health outcomes—is a common method. However, the disagreement between frameworks on how to link evidence is a challenge. This is symbolic of the broader challenge. There are numerous frameworks that consider the use and acceptability of RWE, be it for regulatory submissions or post-marketing authorisation requirements or HTA. The issue is that there’s little consistency between these frameworks. Global discrepancies in the acceptability and design requirements of RWE studies pose challenges for manufacturers developing evidence generation plans that are appropriate for multiple geographies; there is a real need for harmonisation of these frameworks to streamline assessment processes and improve the robustness of HTA outcomes. Ultimately this would widen patient access to new, effective technologies, and that’s what we are all here for.
Outlining RWE preferences and standards in a framework is one thing, but this then also needs to translate to acceptability when it comes to a submission itself. For example, NICE published a RWE framework in 2022, but this has not translated to acceptance of RWE across all programmes, especially if you consider their process for guideline updates, in which systematic reviews of clinical evidence prioritise RCT evidence still. A disconnect remains. Perhaps the framework is needed first, and this can lay the groundwork for greater utilisation of RWE within all programmes in time.
The acceptance of RWE in regulatory and reimbursement decision-making processes is, in general, expanding, as evidenced by the experiences shared by Chiara Capobianco and Colleen Longacre, who provided industry perspectives from Boston Scientific and Medtronic, respectively. In the United States, organisations like the FDA have increasingly relied on RWE to support post-approval requirements for medical devices, as demonstrated by Boston Scientific’s use of RWE for the Watchman device; Medicare claims data enabled the company to demonstrate long-term, 5-year outcomes, giving real-world context to clinical trials. In particular, success was underpinned by the explanation of why Medicare data were appropriate for this setting, representative of patients who typically undergo these procedures. This again echoes the need for frameworks or guidance that emphasise study considerations so that manufacturers can best shape study design to collect data that regulatory or HTA bodies need to see. Methodological considerations were also highlighted in a Medtronic case study that leveraged linked data from the Transcatheter Valve Therapy Registry and Medicare to present 10-year follow-up data. This was originally in collaboration with the FDA and other manufacturers to adequately define methods and outcome definitions, but has since been leveraged for post-market follow-up in the US, EU and China.
These examples highlight the growing acceptance of RWE, and the role of linked data in generating device-specific comparative evidence increasingly required by HTAs. RWE does not substitute for clinical trials, and no one at the conference claimed that it could. However, it does serve to complement and extend the evidence base, provide real-world insights for a greater diversity of patients, and ultimately can facilitate access to treatments. What was clear from this session is the need for harmonisation of evidence standards and use of RWE across regulators and HTAs going forward, which could really help ensure that these data are derived from appropriately designed studies that help address evidence gaps in ways that RCTs cannot always manage to do.
If you would like any further information on the themes presented above, please do not hesitate to contact Ben McNally, Head of MedTech (LinkedIn). Ben McNally is an employee at Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners.
