Medlior was delighted to have the opportunity to be an exhibitor at ISPOR EU in lovely Copenhagen last week. The event brought together experts, researchers, and industry leaders in the field of HEOR to discuss the latest developments and trends in our industry. This year’s theme was “HEOR at the Nexus of Policy and Science” and, as with ISPOR in Boston in May, the real-world evidence (RWE) discussions largely focused on the use and acceptance of real-world data (RWD) in regulatory/reimbursement submissions.
RWD in External Control Arms
Among the various topics explored, the integration of RWD in external control arms (ECAs) emerged as a key focal point, particularly as rare diseases and orphan drugs continue to be a focus at ISPOR. ECAs use data external to a clinical trial to emulate the characteristics of a control arm in single-arm trials where having a control arm would not be ethical or feasible. ECAs can involve patient-level data or summary-level data as the basis for understanding treatment effects in the absence of a trial control arm.
RWE Challenges and Solutions
The use of ECA has its own drawbacks and challenges, including uncertainty around the RWD quality, context specificity (i.e., fit for purpose), and the statistical methodology and study design. Further, the research questions that can be addressed using RWD may be limited by the availability of the data and data elements captured. For example, RWD for novel therapeutics may not exist or populations may be too heterogenous to provide insights on treatment effects. Methodological concerns, such as bias and confounding, were also acknowledged. However, ISPOR EU 2023 also showcased ongoing efforts to develop robust methodologies in RWD generation and statistical techniques to address these challenges.
Regulatory Recognition and Guidance
A highlight from ISPOR EU 2023 was the growing recognition of RWE and ECAs by regulatory and reimbursement bodies, with the development of draft guidance on ECAs by the US Food and Drug Administration, the National Institute for Health and Care Excellence RWE framework from 2021, and evolving guidance from the European Medicines Agency. However, there are still differences in what is accepted as evidence across regulatory/reimbursement agencies, and there is a need for standardization on data quality. As the field continues to evolve, there was a call for the evaluation of submissions with ECAs using RWD to follow a more principled or pragmatic approach rather than a specific set of rules for evaluation.
Overall, ISPOR EU 2023 stirred up great discussion on RWE, particularly in the context of ECAs. The integration of RWD in ECAs not only enhances the relevance and assessment of treatment efficacy from single-arm clinical trials, but also aligns with the ethical imperative of patient-centric approaches. As the industry continues to navigate the challenges and opportunities associated with the use of RWE, it serves as a promising avenue shaping the landscape of evidence generation in healthcare.
How Can Medlior Help?
Medlior was delighted by the continued interest in Canadian RWD sources and Medlior’s capabilities to access and analyze it for industry-sponsored research studies. The advantages of Canadian data over other datasets include the data provenance and quality of health system data. The data are population-based with long-term follow-up and include linkages between comprehensive structured lab data, vital statistics, inpatient, outpatient, and drug claims datasets. Contact us at to learn more about our services and how we can support your RWE Strategy as we enter 2024.
We are grateful for our opportunity to participate in such a thought-provoking conference and look forward to next year!