Key Takeaways
- High‑quality patient registries have become indispensable public‑health assets, producing real‑world evidence (RWE) that complements trials and informs clinical care, regulatory reviews, reimbursement decisions and population‑health policy.
- Clinicians and patients benefit directly: registries benchmark outcomes, drive continuous quality‑improvement initiatives and provide long‑term follow‑up data that shape guideline updates and shared decision‑making.
- Regulators and payers increasingly rely on registry‑based studies for post‑authorization safety monitoring, label extensions and value‑based contracting—making “fit‑for‑purpose” data quality, governance, and stakeholder engagement critical success factors.
I. Introduction
Patient registries have matured from simple data warehouses into strategic public‑health assets. By systematically capturing outcomes in routine practice, registries generate real‑world evidence (RWE) that complements randomized clinical trials and informs clinical care, regulation, reimbursement, and policy. The European Medicines Agency (EMA) now recommends that sponsors consider registry‑based studies whenever long‑term or broad‑population data are needed, and the U.S. Food and Drug Administration (FDA) has issued guidance on assessing whether a registry is “fit‑for‑purpose” to support regulatory decisions.(1, 2) This article explores the external value of registries through the lenses of various external stakeholders.
II. Clinicians: Better Benchmarks and Shared Learning
For physicians, registries can be tools for improving patient care. Clinicians gain feedback on outcomes of their patients relative to peers, can identify gaps in care, and apply lessons learned to practice. In cardiology, the National Cardiovascular Data Registry helped drive the median door‑to‑balloon time for ST‑elevation myocardial infarction from 96 minutes to 64 minutes within a decade, a change associated with lower mortality.(3) Similar gains have been documented in stroke care, trauma and oncology. Strategies for clinician engagement include involving respected physician “champions” who advocate for the registry, integrating data entry into routine clinical documentation (to minimize duplicate work), and clearly communicating how the registry’s data can benefit practice (e.g., through periodic reports or dashboards of local vs. national outcomes). Continuous engagement via regular investigator meetings and newsletters can be useful for sustaining clinician participation over decades(4).
III. Patients and Caregivers: Voice, Visibility and Long‑Term Follow‑up
For patients, especially those with serious or rare conditions, registries represent an opportunity to contribute to knowledge that may improve care for themselves or others. The Cystic Fibrosis Foundation Patient Registry, for example, synthesizes annual outcome reports that families and care teams use to track survival, lung function and nutritional status.(5) These data empowered the CF community to advocate for earlier use of highly effective modulators and informed guideline updates that now call for universal newborn screening and triple‑therapy initiation once eligible.(4) Engaging patients involves building trust (assuring privacy, ethical use of data), minimizing burden (easy-to-complete forms, infrequent but meaningful contact), and often sharing results back in a lay-friendly way. Some registries include patient representatives on their advisory boards to ensure patient priorities are addressed. (6) It is also critical to be transparent with participants about the registry’s purpose and progress, which can be done through newsletters or patient portals. Sustained, interactive, and meaningful engagement with patients throughout the registry lifecycle strengthens the registry’s relevance and success. (7)
IV. Regulators and HTA Agencies: Robust Post‑Market Evidence
Regulators (like FDA and EMA) may use registry data to monitor post-market safety or evaluate effectiveness in broader populations. The EMA’s 2021 *Guideline on Registry‑Based Studies* sets methodological standards for using registries in marketing authorization dossiers and post‑approval obligations.(1) Likewise, the FDA’s 2023 guidance provides a stepwise framework for judging data relevance, reliability and transparency when sponsors submit registry analyses to support regulatory decisions.(2) Registry evidence has already contributed to expanded indications in oncology, rheumatology and rare metabolic diseases by documenting treatment outcomes across diverse real‑world populations.(2, 8-10). Close collaboration with regulatory experts during registry design (e.g., aligning data collection with what regulators consider robust evidence) can position a registry to have maximum impact.
V. Researchers and Academic Community: Rich Data Sources
Investigators look to registries as rich data sources to answer pressing clinical and epidemiological questions. By participating, researchers may gain access to a large, high-quality dataset for analyses and publications. To attract academic engagement, registries should uphold scientific rigor and offer fair opportunities for researchers to propose studies or ancillary analyses. Establishing a publication policy and a formal process for research proposals can help manage this. A thriving registry often has a pipeline of analyses led by various investigators, resulting in peer-reviewed publications that further enhance the registry’s credibility.
VI. Payers: Evidence for Value‑Based Arrangements
Insurance payers, health plans, and health technology assessors have interest in real-world outcomes and cost-effectiveness. A registry that captures healthcare resource utilization, quality of care, and long-term outcomes can provide evidence to support reimbursement decisions or value-based contracting. For example, a payer might support or utilize a registry to track patient outcomes as part of a coverage with evidence development arrangement. Demonstrating the registry’s ability to answer questions about comparative effectiveness or patient sub-populations (like responders vs. non-responders) can engage these stakeholders. Moreover, if a registry shows improved outcomes (e.g., via feedback loops that drive quality improvement), health systems may be motivated to invest in it, since it aligns with their goals of improved care and lower costs.
VII. Public‑Health Surveillance and Guideline Development
Public‑health agencies rely on registry data to track incidence, prevalence, and outcome trends. Cancer registries underpin national survival statistics and have identified racial and socio‑economic disparities that shaped targeted screening programs. Professional societies mine registry outputs when drafting or revising clinical guidelines, ensuring that recommendations reflect contemporary, heterogeneous populations rather than the restricted cohorts of trials. When registry analyses revealed that active surveillance was safe for selected low‑risk prostate cancer patients, guidelines incorporated surveillance as a first‑line option, sparing thousands of men unnecessary surgery or radiation.
VIII. Stakeholder Engagement: The Glue That Makes Data Matter
The most influential registries maintain multidisciplinary governance boards comprising clinicians, data scientists, patient advocates and, where relevant, payer or regulatory representatives. Transparent policies on data access, authorship and publication foster trust and stimulate continuous analytic output. Regular stakeholder workshops—like those run by EMA’s Patient Registry Initiative—provide forums to harmonize data elements, share interim results, and decide on next‑generation questions. Such engagement ensures that registry findings are translated into practice guidelines, regulatory updates, and reimbursement models rather than languishing in academic journals.
Conclusion
External stakeholders depend on high‑quality registries to bridge the evidence gap between controlled trials and routine care. Whether improving frontline clinical practice, powering value‑based payment, informing policy, or safeguarding patients treated with cutting‑edge gene therapies, registries deliver real‑world insights that no other study design can match. Engaging all these stakeholders requires effective communication. Regular stakeholder meetings or workshops can be helpful – for instance, involving clinicians, patients, and payer decision-makers in discussing interim registry findings and how to act on them fosters a sense of shared purpose.
Stakeholder engagement is an ongoing process. As the registry evolves, continuing to seek input (What new questions are arising? What data might we add? How can we feedback results better?) will keep stakeholders invested. Medical Affairs professionals often play a pivotal role here, functioning as liaisons with these different stakeholders. They can champion the registry’s importance, ensure it remains aligned with scientific and medical needs, and help communicate findings in appropriate forums.
This article is a companion to the recent publication Implementing Registries for Evidence Generation
ACKNOWLEDGMENTS
Special thanks to:
Scott Flanders, PhD: Is an employee of ImmunityBio, Inc.; Is a stockholder with ImmunityBio, Inc; Johnson & Johnson; Pfizer, Inc; Kura Oncology Inc; Abbott Labs; Iovance Biotherapeutics Inc; Annexon Inc; Soleno Therapeutics Inc.
Phil Wakefield, DPhil: Is an employee of Inizio
Angela Williams, PhD: Is an employee of Kyowa Kirin
Cerise James, MD, MPH: Is the MAPS Evidence Generation Domain Lead and FAWG Co-Chair; Is an Owner of, Tilghman James Group LLC; Is a stockholder with Pfizer Inc., Merck Inc., and Aura Biosciences Inc.
References
- European Medicines Agency. Guideline on registry-based studies 2021 [Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-registry-based-studies_en.pdf.
- FDA. Real-World Data: Assessing Registries To Support Regulatory Decision-Making for Drug and Biological Products 2023 [Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/real-world-data-assessing-registries-support-regulatory-decision-making-drug-and-biological-products#:~:text=FDA%20www,making.
- Rathore SS, Curtis JP, Chen J, Wang Y, Nallamothu BK, Epstein AJ, et al. Association of door-to-balloon time and mortality in patients admitted to hospital with ST elevation myocardial infarction: national cohort study. BMJ. 2009;338:b1807.
- Porten SP, Cooperberg MR, Konety BR, Carroll PR. The example of CaPSURE: lessons learned from a national disease registry. World J Urol 2011;29(3):265-71
- Cystic Fibrosis Foundation. 2025 [Available from: https://www.cff.org/.
- Registries for Evaluating Patient Outcomes: A User’s Guide: 4th Edition. Secondary Registries for Evaluating Patient Outcomes: A User’s Guide: 4th Edition 2020. https://effectivehealthcare.ahrq.gov/sites/default/files/pdf/registries-evaluating-patient-outcomes-4th-edition.pdf
- Patient-Centeredness, Diversity, and Stakeholder Engagement. Secondary Patient-Centeredness, Diversity, and Stakeholder Engagement 2023. https://www.ahrq.gov/pcor/strategic-framework/stakeholder-engagement.
