Development of a definition for real-world evidence using the Jandhyala method for observing consensus opinion among medical affairs pharmaceutical physicians

Abstract

Objective

Low rates of inclusion of real-world evidence (RWE) during regulation may arise from lack of clarity and consensus on its definition. A conceptually mature definition of RWE may have pragmatic utility, increasing its inclusion during regulation. The aim was to develop a definition of RWE to promote inclusion in regulatory submissions and assess its conceptual maturity.

Methods

Thirteen medical affairs pharmaceutical physicians completed two qualitative online surveys to generate items needed in a definition of RWE. Items that reached a consensus index of > 50% (CI > = 0.51) were retained in the final definition. The maturity of the definition was assessed using concept analysis.

Results

After attrition, 11 participants completed the study and generated 18 items to be included in a definition of RWE. All items reached the consensus threshold and were included. The definition was conceptually mature on three of the four dimensions: the potential for a consensual definition across stakeholders, a description of its characteristics and clear preconditions and outcomes. Further research is needed to delineate the boundaries of RWE.

Conclusions

A definition of RWE was generated that may increase its inclusion during medicines regulation, especially with further refinement from regulators and other stakeholders.

Keywords:

• Drug development
• pharmaceutical industry
• pharmaceutical research
• real-world evidence

Transparency

Declaration of funding

The author received no funding for this work.

Declaration of financial/other relationships

Dr Ravi Jandhyala is a visiting senior lecturer at the Centre for Pharmaceutical Medicine Research at King’s College London and is responsible for research into real-world evidence approaches and is the founder and CEO of Medialis Ltd, a medical affairs consultancy and contract research organization involved in the design and delivery of real-world evidence in the pharmaceutical industry. The Jandhyala method was developed by Dr Jandhyala but is free of commercial licensing restrictions and while used as part of proprietary methodology, is not a direct means of commercial gain for the author.

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

The author was involved in the conception, design, and analysis and interpretation of the data; the drafting of the paper and revising it critically for intellectual content; and the final approval of the version to be published. The author agrees to be accountable for all aspects of the work. The author affirms that the manuscript is an honest, accurate, and transparent account of the study being reported; that no important aspects of the study have been omitted, and any discrepancies from the study as planned (and, if relevant, registered) have been explained.

Acknowledgements

The author would like to thank members of the MAPP advisory board, Dr Andy Pain, Dr Lisa Moore-Ramdin, Dr William Spencer, Dr Daniel Franks, Dr Gerd Möller, Dr John Bolodeoku, Dr Daniel A Thomas, Dr Timir Patel, Dr Raj Rout and Professor Peter Stonier for participating in the study. Dr Omolade Femi-Ajao contributed to the study through the provision of study management and Mohammed Kabiri provided data analytics, both as part of their roles as employees of Medialis Ltd. Lauri Naylor contributed to the article through the provision of medical writing and editing services as part of her role as an employee of Medialis Ltd.

Data availability statement

The authors confirm that the data supporting the findings of this study are available within the article.

Ethical approval

This study was conducted in accordance with the principles stated in the Declaration of Helsinki. Favorable ethical opinion was granted by the King’s College London Research Ethics Committee (Reference number: MRA-21/22-26399). All participants gave written informed consent before taking part in the study.