VP115 Practical Issues Of Using Real-World Data In Effectiveness Research

INTRODUCTION:The Innovative Medicines Initiative, IMI-GetReal project aimed to explore incorporation of robust methods for real-world data (RWD) collection and synthesis earlier in the medicines development process, both by pharmaceutical companies and healthcare decision makers. The focus was on the potential use of RWD, alone or in combination with randomized controlled trials (RCTs), to demonstrate effectiveness of new interventions. Four case studies were conducted in multiple disease areas to examine methods for predicting drug effectiveness and the perspectives of different stakeholders on these methods. This study aimed to identify practical obstacles in accessing and using RWD and RCT data for effectiveness research conducted as part of these case studies.METHODS:Qualitative content analysis was conducted to identify and characterize key issues relating to accessing and analysing study data from external sources, both RWD and RCTs.RESULTS:Accessing RWD from registries proved difficult due to multiple reasons, including: complex and non-transparent application procedures, resistance from registry owners to discuss applications and datasets not being research-ready within project timeframes. There were also issues with the RWD eventually accessed, including a lack of individual participant data (IPD) and incomplete data. Where access to IPD from RCTs was obtainable, there were restrictions imposed on how it could be used. For example, it could not be used to target analysis on an individual product, but rather explore methodologies for data synthesis in a product-anonymised setting. This condition encouraged additional data sharing by other stakeholders.CONCLUSIONS:Despite the collaborative, multi-stakeholder nature of IMI-GetReal and proper disclosures with data owners, access to data proved challenging. Such barriers to data accessibility can delay effectiveness research, restrict opportunities for the development of methods incorporating RWD and diminish the potential use of RWD in decision making. Where data is intended to be used for this purpose, sufficient attention should be paid to these potential barriers.

Download Full-text

Examination of Data, Analytical Issues and Proposed Methods for Conducting Comparative Effectiveness Research Using "Real-World Data"

Journal of Managed Care Pharmacy ◽

10.18553/jmcp.2011.17.s9-a.1 ◽

2011 ◽

Vol 17 (9 Supp A) ◽

pp. 1-37

Author(s):

Demissie Alemayehu ◽

Riaz Ali ◽

Jose Ma.J. Alvir ◽

Joseph C. Cappelleri ◽

Mark J. Cziraky ◽

...

Keyword(s):

Comparative Effectiveness Research ◽

Real World ◽

Comparative Effectiveness ◽

Effectiveness Research ◽

Real World Data ◽

World Data

Download Full-text

Do existing real-world data sources generate suitable evidence for the HTA of medical devices in Europe? Mapping and critical appraisal

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321000301 ◽

2021 ◽

Vol 37 (1) ◽

Author(s):

Benedetta Pongiglione ◽

Aleksandra Torbica ◽

Hedwig Blommestein ◽

Saskia de Groot ◽

Oriana Ciani ◽

...

Keyword(s):

Decision Making ◽

Medical Devices ◽

Real World ◽

Real World Data ◽

Data Accessibility ◽

World Data ◽

Transcatheter Aortic Valve ◽

Real World Evidence ◽

Hip And Knee Arthroplasty ◽

Valve Implantation

Abstract Aim Technological and computational advancements offer new tools for the collection and analysis of real-world data (RWD). Considering the substantial effort and resources devoted to collecting RWD, a greater return would be achieved if real-world evidence (RWE) was effectively used to support Health Technology Assessment (HTA) and decision making on medical technologies. A useful question is: To what extent are RWD suitable for generating RWE? Methods We mapped existing RWD sources in Europe for three case studies: hip and knee arthroplasty, transcatheter aortic valve implantation (TAVI) and mitral valve repair (TMVR), and robotic surgery procedures. We provided a comprehensive assessment of their content and appropriateness for conducting the HTA of medical devices. The identification of RWD sources was performed combining a systematic search on PubMed with gray literature scoping, covering fifteen European countries. Results We identified seventy-one RWD sources on arthroplasties; ninety-five on TAVI and TMVR; and seventy-seven on robotic procedures. The number, content, and integrity of the sources varied dramatically across countries. Most sources included at least one health outcome (97.5%), with mortality and rehospitalization/reoperation the most common; 80% of sources included resource outcomes, with length of stay the most common, and comparators were available in almost 70% of sources. Conclusions RWD sources bear the potential for the HTA of medical devices. The main challenges are data accessibility, a lack of standardization of health and economic outcomes, and inadequate comparators. These findings are crucial to enabling the incorporation of RWD into decision making and represent a readily available tool for getting acquainted with existing information sources.

Download Full-text

Regulatory-grade clinical trial design using real-world data

Clinical Trials ◽

10.1177/1740774520905576 ◽

2020 ◽

Vol 17 (4) ◽

pp. 377-382

Author(s):

Mark S Levenson

Keyword(s):

Real World ◽

Trial Design ◽

Clinical Trial Design ◽

Real World Data ◽

World Data ◽

Real World Evidence ◽

Study Results ◽

Potential Use ◽

The U.S

Real-world data and evidence provide the potential to address the effectiveness and safety of drugs. The U.S. Food & Drug Administration has initiated a program to evaluate the potential use of real-world evidence for regulatory uses. Whether a study is designed for regulatory purposes or for other purposes, existing regulation and guidance provide a reference for high-quality studies. Clarifying the study objectives and the role of real-world data in the study are important considerations. Robustness and transparency of the analysis allow for greater understanding and acceptance of the study results.

Download Full-text

"Ten Commandments" for Conducting Comparative Effectiveness Research Using "Real-World Data"

Journal of Managed Care Pharmacy ◽

10.18553/jmcp.2011.17.s9-a.s10 ◽

2011 ◽

Vol 17 (9 Supp A) ◽

pp. S10-S15 ◽

Cited By ~ 6

Author(s):

Richard J. Willke ◽

C. Daniel Mullins

Keyword(s):

Comparative Effectiveness Research ◽

Real World ◽

Comparative Effectiveness ◽

Effectiveness Research ◽

Real World Data ◽

World Data ◽

Ten Commandments

Download Full-text

YAMATO: Yet-another more advanced top-level ontology

Applied Ontology ◽

10.3233/ao-210257 ◽

2021 ◽

pp. 1-22

Author(s):

Riichiro Mizoguchi ◽

Stefano Borgo

Keyword(s):

Case Studies ◽

Real World ◽

Modern Society ◽

Real World Data ◽

World Data ◽

The World ◽

Philosophical Contribution

yamato sharply distinguishes itself from other existing upper ontologies in the following respects. (1) Most importantly, yamato is designed with both engineering and philosophical minds. (2) yamato is based on a sophisticated theory of roles, given that the world is full of roles. (3) yamato has a tenable theory of functions which helps to deal with artifacts effectively. (4) Information is a ‘content-bearing’ entity and it differs significantly from the entities that philosophers have traditionally discussed. Taking into account the modern society in which a flood of information occurs, yamato has a sophisticated theory of informational objects (representations). (5) Quality and quantity are carefully organized for the sake of greater interoperability of real-world data. (6) The philosophical contribution of yamato includes a theory of objects, processes, and events. Those features are illustrated with several case studies. These features lead to the intensive application of yamato in some domains such as biomedicine and learning engineering.

Download Full-text