Technology-Driven Innovation in Real World Evidence for Patient Registries

The demand for robust patient data and real-world evidence (RWE) continues to grow as regulators, payers, researchers, and physicians look to answer questions throughout the entire lifecycle of a drug. While patient registries are an important data source, traditional means of data collection greatly limit the types and amounts of information available. Advances in technology provide the necessary innovation to increase registry information and generate RWE to meet the increasing needs of diverse stakeholders.

Rising Demand for RWE

The use of RWE has grown to encompass the full patient journey and product lifecycle. Regulatory guidance from the Food & Drug Administration (FDA) and the European Medicines Agency (EMA) covers RWE use from pre-clinical planning to post-marketing settings. Payers, Health Technology Assessments (HTAs), and agencies emphasize evidence generation for market entry and post-launch evaluation of drugs, medical devices, and digital therapeutics. Additionally, physicians, patients, and patient foundations seek evidence to make informed decisions about therapy’s impact on care, outcomes, and quality of life.

The Advancement of Patient Registries

Developing patient or product registries helps researchers expand their understanding of diseases, patient outcomes, and treatment effectiveness and safety over longer periods in the real-world setting. Patient registries are pivotal in identifying potential treatment targets or patient subgroups and designing effective clinical trials. They provide representative subjects for monitoring drug safety and product effectiveness to inform healthcare decisions and policies that enhance patient care.

Registries address the growing demand for RWE through observational methodologies, offering holistic data on patient characteristics, clinical management, treatment, and outcomes. Longitudinal observational studies, conducted in real-world settings without influencing routine care, provide insights into the impact of therapeutics on health outcomes for diverse populations. These studies reveal how factors like age, comorbidities, medications, and lifestyle influence product effectiveness and physician decision-making, offering population-level trends in disease or clinical care changes. With robust data collection, researchers can detect rarer events or delayed effects and reflect real-world patient and caregiver experiences over years or decades.

Recognizing registries as adaptable larger-scale evidence platforms makes them strategic assets for organizations heavily focused on specific development areas, aligning data with specific needs while allowing for scalability. The increased collection of richer data both for clinical trial optimization and observational research helps to address the growing need across multiple stakeholders to enable them to launch substudies and other programs.

Benefits of Technology Advances

Advancements in data technologies and integrated research platforms can enable the efficient collection and integration of a mosaic of data sources for registries, enhancing patient and clinician health data insights. In addition to an expansion of the types of real-world data (RWD) available, technology helps integrate data sources into common databases and formats for analysis. This could include sources spanning inpatient and outpatient settings of care, patient-reported outcomes (PROs), disease-specific clinician assessments, administrative claims, electronic health records (EHRs) genetic biomarkers, and broader lab tests. RWD can also help identify eligible patients for a registry or study, enabling researchers to develop strategies to maximize recruitment and enrollment of their target patient populations.

Registries can be used to address multiple questions, generate insights over extended periods, and adapt to evolving treatment landscapes, to meet diverse stakeholder needs. Examples of large-scale registries run by patient foundations, such as Cure SMA, Cystic Fibrosis Foundation, Multiple Myeloma Research Foundation, Parent Project Muscular Dystrophy, and the Crohn’s & Colitis Foundation, demonstrate this unique potential and capability to flexibly support a range of evidence needs across stakeholders over time. Further information can be found in a recent white paper “Supporting Patients Through Research Collaboration.”

The adoption of digital and remote technology solutions and services into registry approaches was accelerated by the COVID-19 pandemic – but the trend has continued due to the proven ability to support high-quality data collection at a lower burden. This includes the greater use of telehealth, virtual nursing support, mobile phlebotomy, and remote data collection tools – which helps address the challenges associated with patient access, engagement, and follow-up. In addition, continued advancements in secure cloud-based data processing, hosting, and analytics tools mean registries can process and curate greater volumes of complex data types at lower costs and allow full auditability and traceability of the curation process to ensure research teams can accurately interpret findings.

Reducing the Burden of Data Collection

Technology, when set up and designed into existing research workflows and systems, can also help streamline and in some cases automate data collection and integration across sources (EHR, secondary databases, digital health devices, electronic patient-reported outcomes [ePROs]) – significantly reducing time and resource burden across patient, site staff and operational teams. This can influence their willingness and ability to participate in research programs, especially those with prolonged timeframes and non-interventional designs. As an example, decentralized study portals available via web or smartphone applications can allow a registry participant to provide access to passive data on their sleep and activity from their smart devices to help gather useful information on their symptoms, experiences, and outcomes between regular healthcare visits. This approach enhances visibility, and credibility and reduces bias in meaningful real-world experiences and outcomes, and includes validated ePROs, connected devices/wearables, and survey-based mechanisms for data collection. These technologies also minimize repeated or time-consuming tasks, reducing barriers to frequent study participation and allowing less mature research sites to participate in registries.

Similarly, registry sponsors are adopting digital health technologies for efficient and accurate data collection, favoring decentralized study designs over traditional site-based case report form methods. Integrating available EHRs and existing secondary information enhances interoperability and data quality. Regulatory and interoperability advancements, such as the 21st Century Cures Act and United States Core Data for Interoperability (USCDI), pave the way for using technology to collect higher-quality, standardized information from clinical and third-party sources.

Technology Drives Patient Registry Growth and Generates RWE

Through advancements in technology, supported and guided by RWE experts and regulators, patient registry data becomes more comprehensive and able to fit multiple purposes and stakeholder needs. Four technology trends that are expected to impact the expansion of registries as an effective and efficient tool for RWE generation include:

Increasing use of integrated decentralized clinical trial platforms, supporting hybrid and direct-to-patient study designs with improved tools and services for site and patient-facing data collection and engagement modules within a unified platform.

1. Utilization of wearables and connected devices, such as smartphones, sleep and activity trackers, chipped devices, and scales, providing continuous data streams on individuals’ health, lifestyle, and patterns.
2. Ongoing advancements in interoperability standards and associated infrastructure for clinical and EHR data, facilitating better sharing and utilization of data for research while incorporating low-burden ways for patients to grant access.
3. Data linkage and integration, are driven by the growing use of tokenization and linkage pathways. This, coupled with the evolving governance and privacy models, will allow broader access to integrating with existing secondary databases and datasets. This shift breaks down silos, fostering better collaboration and supporting more robust evidence generation. The trend is expanding from the U.S. to encompassing other regions globally.

Evolving technologies continue to enhance patient registry data, enabling RWE generation to address regulatory, payer, physician, and patient inquiries. By taking a holistic, technology-driven approach to data collection that facilitates patient-centered approaches, minimizes burden, and gathers diverse data types from a broader patient pool, registries become an even more valuable resource for all healthcare stakeholders.

Author Details 

Emily Bratton- Category Lead of Safety Evidence for Regulators, Real World Solutions, IQVIA; Ian Bonzani- Senior Principal, Real World Technology Solutions, IQVIA

Emily Bratton has been in the pharmaceutical industry for 15 years, contributing to late-phase observational epidemiology research teams. As Category Lead of Safety Evidence for Regulators, Emily works across IQVIA’s Real World Solutions business to ensure our customers connect with the right people and design the right solution for their research needs -- particularly evidence generation for regulators. Before her current position, she led the scientific development, analysis, and interpretation of observational studies (both retrospective and prospective) and low interventional clinical trials. Emily received her PhD in Infectious Disease Epidemiology from the University of North Carolina at Chapel Hill.

Ian Bonzani is a Senior Leader in Global Real World Technology Solutions at IQVIA. In this role, Ian leverages his scientific background and healthcare consulting experience to design and implement large-scale real-world data and registry platforms that provide value across stakeholders by optimizing specialty care coordination, quality improvement, and research delivery. Ian has been with IQVIA for 15 years, managing large, global projects for pharmaceutical companies, providers, and other healthcare organizations. Prior roles include data and evidence strategy and implementation roles in Real World Analytics Solutions, Pricing & Market Access & Thought Leadership functions. He is a Regenerative Medicine PhD graduate of Imperial College London and has a BSc in Biomedical Engineering from WPI.

Publication Details 

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