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  • Leveraging Registry Data to Support HTA and Payer Decision-Making

    Leveraging Registry Data to Support HTA and Payer Decision-Making

    Registry

    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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    References

    1. Gliklich RE, Dreyer NA, Leavy MB, editors. Registries for Evaluating Patient Outcomes: A User’s Guide [Internet]. 3rd edition. Rockville (MD): Agency for Healthcare Research and Quality (US); 2014 Apr. 1, Patient Registries. Available at: https://www.ncbi.nlm.nih.gov/books/NBK208643/
    2. Murphy LA, Akehurst R, Cunningham D, et al. Real-world evidence to support health technology assessment and payer decision making: is it now or never? Int J Technol Assess Health Care. 2025 Mar 31;41(1):e20.
    3. MDIC HEPV Initiative. Payer and HTA Perspectives on Real-World Evidence for Medical Devices – Final Report. November 2021.
    4. Jaksa A, Arena PJ, Hanisch M, Marsico M. Use of Real-World Evidence in Health Technology Reassessments Across 6 Health Technology Assessment Agencies. Value Health. 2025; 28(6):898-906.
    5. Windfuhr F, de Vries ST, Melinder M, et al. Stakeholders’ Perspectives Toward the Use of Patient Registry Data for Decision-Making on Medicines: A Cross-Sectional Survey. Drug Saf. 2025 Feb 23.
    6. Evidera. Advancing the Use of Registry Data to Improve Health Technology Assessment and Payer Evidence. ISPOR 2024. Available at: https://www.ispor.org/docs/default-source/intl2024/120eviderav2.pdf?sfvrsn=a1d01ffe_0
    7. Rubinger L, Ekhtiari S, Gazendam A, Bhandari M. Registries: Big data, bigger problems? Injury. 2023; 54(3):S39-S42.
    8. Allen A, Patrick H, Ruof J, et al. Development and Pilot Test of the Registry Evaluation and Quality Standards Tool: An Information Technology–Based Tool to Support and Review Registries. Value Health. 2022; 25(8):1390-1398.
    9. Christian J, Dasgupta N, Jordan M, et al. Digital Health and Patient Registries: Today, Tomorrow, and the Future. In: Gliklich RE, Dreyer NA, Leavy MB, et al., editors. 21st Century Patient Registries: Registries for Evaluating Patient Outcomes: A User’s Guide: 3rd Edition, Addendum [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2018 Mar. 3. Available at: https://www.ncbi.nlm.nih.gov/books/NBK493822/
    10. Miller RS, Mitchell K, Myslinski R, et al. Health Information Technology (IT) and Patient Registries. In: Gliklich RE, Leavy MB, Dreyer NA, editors. Tools and Technologies for Registry Interoperability, Registries for Evaluating Patient Outcomes: A User’s Guide, 3rd Edition, Addendum 2 [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2019 Oct. Chapter 1. Available at: https://www.ncbi.nlm.nih.gov/books/NBK551883/
    11. European Commission. Implementation of the Regulation on health technology assessment: Regulation (EU) 2021/2282 of The European Parliament and of The Council of 15 December 2021 on Health Technology Assessment and Amending Directive 2011/24/EU. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32021R2282
    12. IRS. Inflation Reduction Act of 2022. Available at: https://www.irs.gov/inflation-reduction-act-of-2022

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  • Exploring Patient Registries: Managing Market Access With Real World Evidence

    Exploring Patient Registries: Managing Market Access With Real World Evidence
    Exploring Patient Registries Managing Market Access With Real World Evidence

    In the increasingly data-driven world of healthcare, patient registries have emerged as powerful tools for managing market access through the application of real-world evidence (RWE). These registries, essentially databases that collect information about patients’ demographics, clinical history, treatment outcomes, and other pertinent details, provide a treasure trove of insights that can bridge the gap between clinical trials and everyday medical practice. The use of patient registries in gathering RWE is transforming how pharmaceutical companies, healthcare providers, and policymakers evaluate and manage the accessibility and effectiveness of medical treatments and interventions.[1]

    Patient registries provide a comprehensive view of how treatments perform outside the controlled environment of clinical trials. Clinical trials, while essential for establishing the efficacy and safety of new therapies, often have limitations, such as restrictive inclusion criteria and relatively short follow-up periods. In contrast, patient registries encompass a broader patient population, including those with comorbidities and varying adherence levels, thus reflecting real-world scenarios more accurately. This inclusivity makes registries a valuable source of RWE, which is increasingly recognized by regulatory agencies and payers as critical for assessing the long-term value and impact of medical interventions. Regulators also view the use of registries as a key tool for generating RWE, supporting decision-making processes across areas such as post-market surveillance, submissions for regulatory approval, and health technology assessments (HTAs).[1]

    One of the primary benefits of utilizing patient registries is the ability to generate evidence on long-term safety and effectiveness of treatments. This ongoing evidence collection improves patient trust in the intervention, thereby increasing patient access. For instance, post-marketing surveillance through patient registries can uncover adverse effects or benefits that may not be apparent during pre-approval clinical trials. This continuous monitoring helps in ensuring that the treatments remain safe and effective once they are in widespread use, supporting regulatory decisions and guiding clinical practice. Consequently, this fosters greater confidence and willingness among patients to adopt new treatments, enhancing their accessibility and uptake in the general population.[2]

    Patient registries also play a pivotal role in market access by demonstrating the value of new treatments to payers and health technology assessment (HTA) bodies. The data derived from these registries can support health economic evaluations, such as cost-effectiveness and budget impact analyses, which are essential for reimbursement decisions. By providing robust evidence on the real-world effectiveness and safety of treatments, registries help build a compelling case for their adoption and coverage. This is particularly important in an era where healthcare budgets are constrained, and there is increasing scrutiny over the allocation of resources.[3]

    Despite their benefits, patient registries face challenges that must be acknowledged to provide a balanced perspective. Issues such as data quality inconsistencies, difficulties in patient recruitment, and regulatory hurdles can hinder their utility. For example, inconsistent data collection processes may impact the reliability of insights, and recruitment barriers can limit the diversity of patient populations within registries. Addressing these challenges requires standardizing methodologies, fostering collaboration among stakeholders, and ensuring compliance with evolving regulatory frameworks. Additionally, emerging trends such as the integration of digital health technologies and artificial intelligence (AI) are redefining the capabilities of patient registries. AI, for instance, can analyze large-scale registry data to identify patterns, predict treatment outcomes, and optimize resource allocation, enhancing the potential of RWE to inform decision-making.[4]

    Collaboration among stakeholders is critical for the success of patient registry initiatives. Engaging a diverse group of participants, including patients, healthcare providers, researchers, and industry partners, ensures that registries are comprehensive and relevant. Leveraging technologies such as electronic health records (EHRs) and mobile health applications can streamline data collection, improve accuracy, and enhance data utility. These collaborative and technological advancements position registries as powerful tools for addressing unmet medical needs, optimizing healthcare delivery, and achieving equitable access to treatments.[4,5]

    In conclusion, patient registries stand as pillars in the contemporary healthcare landscape, offering a bridge between clinical trials and real-world practice. Their role in managing market access through the application of RWE is indispensable, shaping how pharmaceuticals are evaluated, accessed, and integrated into healthcare systems. With their ability to capture the intricacies of patient experiences over time, these registries empower stakeholders to make informed decisions, driving advancements in healthcare delivery and ensuring that groundbreaking therapies reach those in need. By addressing challenges, embracing innovation, and fostering collaboration, patient registries will continue to play a transformative role in optimizing patient care and healthcare resource utilization.

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    References:

    1. Trotter JP. Patient registries: a new gold standard for “real world” research. Ochsner Journal. 2002 Sep 21;4(4):211-4.
    2. Reid CM. The role of clinical registries in monitoring drug safety and efficacy. Heart, Lung and Circulation. 2015 Nov 1;24(11):1049-52.
    3. Blommestein HM, Franken MG, Uyl-de Groot CA. A practical guide for using registry data to inform decisions about the cost effectiveness of new cancer drugs: lessons learned from the PHAROS registry. Pharmacoeconomics. 2015 Jun;33:551-60.
    4. Gliklich RE, Dreyer NA, Leavy MB. Patient registries. InRegistries for Evaluating Patient Outcomes: A User’s Guide [Internet]. 3rd edition 2014 Apr. Agency for Healthcare Research and Quality (US).
    5. Daugherty SE, Lee SB, Nowell B, Peay H, Solomon D, Valbrun TG, Velentgas P, Whicher D. The increasing focus on the patient in patient registries. In21st Century Patient Registries: Registries for Evaluating Patient Outcomes: A User’s Guide: 3rd Edition, Addendum 2018 Mar. Agency for Healthcare Research and Quality (US).

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