Patient self-reported data has become increasingly important in today's clinical trials. Trials in some disease indications rely upon patient recorded diary data as the primary endpoint to demonstrate drug efficacy - including, for example, indications such as insomnia, migraine and pain. In addition, improvements in quality of life measured using patient questionnaires can now be included as claims on drug labelling. Traditionally these data have been collected using paper questionnaires and diaries issued to subjects. Regulators and the industry have become increasingly aware of the limitations of recording patient reported outcomes data on paper including data quality and integrity issues. As a result there is a growing interest in collection of patient reported outcomes data using electronic means (ePRO). Solutions include handheld PDAs, Interactive Voice Response (IVR) systems, and other site-based hardware such as touchscreen PCs. Recently, there has been much open debate with the regulators around the use of ePRO in clinical drug submissions.
US and European agencies have approved new drugs that have included ePRO data in the submission dossier, but there are many questions around the adoption of the technology that concern the community. These include: How should instruments developed on paper be adapted for electronic use, and what degree of validation should be done between paper and electronic forms? How can researchers ensure they are complying with regulatory requirements including the PRO guidance published by FDA in 2009 when using ePRO solutions? Can fewer patients be exposed in a clinical trial as a result of improved data quality obtained using electronic diaries? What type of solution should be used for certain patient populations and protocols, and how can ePRO solutions be designed optimally to increase patient acceptability and compliance? Bill Byrom and Brian Tiplady's "ePro" addresses all these issues, reviews the new FDA guidance, and provides a very contemporary view on this important subject.
Introduction, Bill Byrom and Brian Tiplady; Foreword, Stephen Raymond; Recall bias: understanding and reducing bias in PRO data collection, Alan Shields, Saul Shiffman and Arthur Stone; Cognitive interviewing: the use of cognitive interviews to evaluate ePRO instruments, Paul Beatty; Data quality and power in clinical trials: a comparison of ePRO and paper in a randomized trial, Allen Ganser, Stephen Raymond and Jay Pearson; Regulation and compliance: scientific and technical regulatory issues associated with electronic capture of patient-reported outcome data, Sonya Eremenco and Dennis Revicki; Selection of a suitable ePRO solution: benefit, cost and risk, Mikael Palmblad; Patient compliance in an ePRO environment: methods for consistent compliance management, measurement and reporting, Alan Shields, Saul Shiffman and Arthur Stone; Computerised clinical assessments: derived complex clinical endpoints from patient self report data, Bill Byrom, Keith Wenzel and James Pierce; Diary design considerations: interface issues and patient acceptability, Brian Tiplady; Equivalence testing: validation and supporting evidence when using modified PRO instruments, Damian McEntegart; ePRO applications and personal; mobile phone use: compliance documentation and patient support, Breffni Martin; Future developments and applications: emerging technologies and new approaches to patients, Brian Tiplady and Bill Byrom; Index.