Leveraging Registry Data to Support HTA and Payer Decision-Making
Health systems worldwide are increasingly realizing the need for well-timed, relevant, and patient-centred evidence to supplement the adoption and reimbursement decisions of new health technologies. Conventional clinical trial data, while crucial for validating safety and efficacy, often fail to represent real-world populations, long-term outcomes, or challenges in the routine clinical practice. Patient registries, which systematically collect patient data on a particular disease or treatment, can be useful in this context. Registry data are being increasingly emphasized as their application expands beyond the traditional role in post-marketing safety surveillance to the foundation for depicting real-world value and efficacy of health technologies.(1)
Registries offer an exclusive, longitudinal view into patient outcomes, treatments, and healthcare resource utilization; making them a remarkably valuable source of real-world data (RWD) in the shift toward value-based care, where decisions made by healthcare systems must focus on the performance of health interventions in not just the randomized controlled trial (RCT) settings but across diverse and changing real-world populations.(2-4) For health technology assessment (HTA) agencies, registry data offer the ability to assess the efficacy of interventions in routine clinical practice, enable long-term outcome monitoring, and develop more rigorous economic models that account for real-life variables and healthcare resource utilization.(2, 5, 6)
European and several other HTA bodies are increasingly adopting registry data to make decisions on disease burden, treatment patterns, cost-effectiveness, and comparative effectiveness, especially in areas where conventional RCTs are limited, such as rare diseases or personalized medicine. Registries also facilitate integration of vital insights into long-term safety profiles and the advancing patient experiences over time, elements that are usually beyond the scope of pre-market studies. For payers, this facilitates more certain and customized decisions about coverage, pricing, and resource allocation, particularly when connected with cost and budget impact models.(2, 5, 6)
However, even with the growing significance of registry data, several structural and technical limitations restrict their widespread adoption in HTA and payer guidance. Data quality, standardization, and interoperability continue to pose challenges, while many registries still depend on internally specified criteria with few universally accepted standards, making cross-registry comparison difficult, which can even hamper the reliability of findings. Also, registries often struggle to keep up with the rapidly progressing health technologies, leaving evidence gaps when new interventions are introduced. These challenges highlight the growing need for standardized quality guidelines, transparent governance, and active data infrastructures that can adapt to the emerging evidence needs.(2, 5-8)
Having said that, recent digital health advancements, decentralized study models, and integration with electronic health records (EHRs) and claims data are transforming the collection and use of registry data. These innovations lessen the patient and clinician burden, enhance data granularity and relevance, and improve interoperability across systems. They also facilitate connection with refined datasets to address a variety of regulatory, clinical, and economic queries, making registry-based real-world evidence (RWE) more robust, receptive, and relevant to decisions.(9, 10)
With changes in regulatory landscapes, such as the introduction of European HTA Regulation (11) or the U.S. Inflation Reduction Act,(12) manufacturers, HTA agencies, and payers must rethink evidence types required for health technology submissions. A growing perception suggests that early and sustained collaboration between manufacturers, registries, regulators, and payers is crucial for aligning evidence generation approaches with decision-making requirements. This collective approach ensures the relevance of registries for future, while also supporting smarter, more unbiased, and impactful healthcare decisions.(2, 5, 6)
Strategically expanding the use of registry data can help the healthcare ecosystem progress towards a patient-centred model, where access, affordability, and outcomes are driven by RWE. Recognizing this potential will require collected effort and careful investment, but it can provide significant value to patients, providers, and payers alike.
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