Clinical research has evolved significantly with the use of patient-centric technology, and the move from paper-based patient-reported outcomes (PROs) to electronic PROs (ePROs) is a prominent example.
This transition is not just a change in modality; it represents a shift in the paradigm of patient data collection. It is allowing sponsors to improve the quality of data collected while minimizing participant burden and mitigating data loss risks.
The Adoption of ePROs
As clinical research continues to evolve, digital advancements are becoming pivotal in pushing forward the adoption and use of ePROs in the clinical research community.
• Technological Advancements
Integrating cutting-edge technology to capture ePROs leads to more efficient, accurate, and user-friendly data collection methods. Innovations such as real-time data capture, reminder notifications, improved user interfaces, and integration with wearable devices are making ePROs more accessible and reliable than traditional paper-based methods. These advancements not only streamline the data collection process but also enhance the quality and availability of the data.
• Growing Preference for ePROs
There is a growing preference for electronic data collection in clinical settings. Convenience, efficiency, and improvement in data quality are driving this shift. Research shows over 86% of respondents in clinical trials preferred electronic methods over traditional paper-based approaches.1 This trend underscores the increasing acceptance and reliance on electronic data collection in clinical research.
As this shift continues to accelerate, researchers will have to be mindful of faithful migration best practices to ensure equivalency when migrating an existing paper PRO to an ePRO.
Faithful Migration and Equivalency
The migration from paper-based PROs to ePROs is more than a technical upgrade; it is based on the concept of 'faithful migration.' This process is extremely critical for preserving the original properties and intent of paper PROs while adapting them to an electronic format. Researchers should apply the appropriate level of scientific rigor to demonstrate that participants interpret and respond to the ePRO questionnaires in a manner consistent with the original paper format.
Demonstrating Equivalency
Demonstrating equivalency between paper-based and electronic data collection methods is essential as any significant alteration in a question, presentation or recorded response can inadvertently affect the quality and reliability of the overall data. This process is particularly important due to the inherent differences between paper and electronic formats.
For instance, the way a question appears on paper may be different than how it is presented on a digital screen, possibly due to factors like screen size, layout or the method of interaction (such as touchscreen vs. writing). Being able to demonstrate that these differences do not have an impact on equivalency may require further testing and validation.
Methodologies such as cognitive debriefing and usability testing can be vital to ensure that patients interpret and respond to ePROs in the same way as they would to paper-based PROs.3 These methods aid in identifying and rectifying discrepancies to ensure the electronic data's reliability and validity, and they may be applicable based on the level of modification made to the ePRO.
Defining the Level of Modification
The migration process typically encompasses three categories of modifications that potentially have varying impact on data integrity.
Minor modifications are generally straightforward and have minimal impact on the data's integrity, as they do not change the essence of the questions or the expected responses. An example would be changing the instruction from "please circle the answer" on a paper questionnaire to "please select the answer" electronically.
Moderate modifications involve more significant changes, such as altering the layout or presentation of questions or dividing a long question across multiple screens. While moderate modifications can slightly affect how participants interact with the questionnaire, they typically do not jeopardize how the question is interpreted.
Substantial modifications can significantly alter how questions are understood and answered. An example could be rephrasing a question for clarity in a digital format or changing the response options to suit a touchscreen interface. This type of modification requires extensive testing and validation to ensure that the data collected remains true to the original intent of the paper-based PROs.
Each type of modification requires careful consideration, and the appropriate level of scientific rigor should be applied to demonstrate equivalency so to as not compromise the quality and reliability of the data collected during the research.
Taking a Structured Approach to Address the Level of Modification
Best practices to address modification levels include a structured approach and a rigorous review process. It is vital to involve stakeholders, particularly regulatory bodies, in modifications that could affect data interpretation.
To achieve faithful migration and preserve data integrity, researchers must take copyright considerations into account and employ accepted modification methodologies. The process begins by contacting the copyright holder for permission and then reviewing the original version to identify necessary changes. The holder must approve these changes to maintain consistency with the original intent.
Resolving technological limitations, such as adapting content to fit digital platform constraints (like screen size and navigation) is also crucial. Usability testing with the target population helps assess how well participants understand, navigate, and submit responses through the ePRO process. Any significant issues identified during this testing must be rectified and reassessed to ensure the ePRO's functionality and user-friendliness.
Statistical validation is also critical for confirming the equivalency of paper and electronic versions. Minor modifications require qualitative assessments like a cognitive debriefing. A 2023 study highlighted that minor ePRO modifications are likely equivalent to original PROs post-testing.4 Moderate modifications necessitate quantitative comparability tests using statistical methods, and substantial changes demand full psychometric testing to ensure statistical robustness, including reliability, consistency, and validity checks.
Welcoming a New Era in Clinical Research
The faithful migration of paper-based patient-reported outcomes to their electronic counterparts is a significant aspect of modern clinical research.
The evolution of ePROs in clinical trials marks a shift towards more efficient, accurate, and patient-friendly data collection methods. With a noted preference for electronic data collection, ePROs are set to become increasingly integral in clinical research. Their ongoing development and refinement will undoubtedly continue to enhance the efficacy and precision of clinical trials.
References
- Applied Clinical Trials Online. eCOA and ePRO Provide Accuracy in Data Capture. Available at: https://www.appliedclinicaltrialsonline.com/view/ecoa-and-epro-provide-accuracy-data-capture?ref=guides.clarahealth.com. Accessed November 27, 2023.
- BMJ Open. Philipps, L., Foster, S., Gardiner, D., et al. (2023). Study within a trial of electronic versus paper-based Patient-Reported Outcomes Collection (SPRUCE): study protocol for a partially randomized patient preference study. Available at: https://bmjopen.bmj.com/ content/13/9/e073817. Accessed November 27, 2023.
- Alencar-Silva, T., Díaz-Martín, R. D., Zonari, A., et al. (2023). The combination of Synoeca-MP Antimicrobial Peptide with IDR-1018 Stimulates Proliferation, Migration, and the Expression of Pro-Regenerative Genes in Both Human Skin Cell Cultures and 3D Skin Equivalents. Biomolecules. May 2023. Available at: https://www.semanticscholar.org/ paper/ae807c153910b33e5890732b473e0f8d81ab6251). Accessed November 27, 2023.
- National Library of Medicine. McMullan, C., et al. (2023). Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10562321/. Accessed November 27, 2023.
Author Details
Melissa Mooney – Director, eCOA Solutions Engineering, IQVIA.
Melissa Mooney has over 19 years of experience in the development of eCOA solutions for use in clinical trials. Melissa’s area of expertise is eCOA solution design where she has supported clients and eCOA vendors in developing robust and usable eCOA software solutions that meet eCOA protocol requirements. She also brings a plethora of experience in eCOA requirement gathering, leading eCOA user acceptance testing, eCOA data management, and business development support.
Publication Detail
This article appeared in Pharmaceutical Outsourcing:Vol. 25, No. 1Jan/Feb/Mar 2024Pages: 22-23
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