ePro: Electronic Solutions for Patient-Reported Data

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.

Bill Byrom completed his PhD in disease control simulation at Strathclyde University in 1990 and this work took him to Africa to study the effects and spread of cattle diseases. This interest led him to join the Pharmaceutical industry in 1991 where he began as a clinical trial statistician. Bill transitioned to clinical development where he became a trial director working in a number of early phase therapy areas. Before joining ClinPhone in 2000, Bill took on responsibility for the scientific content of the European market launch of a new antipsychotic agent. Throughout his clinical trials experience Bill maintained an active interest in how new technologies and methodologies could be leveraged in clinical development programmes, and so his move to Perceptive Informatics (formerly ClinPhone) in 2000 was an ideal fit. In his role as Senior Director of Product Strategy he has helped to shape a number of the company's products and services including ePRO solutions, patient recruitment technologies and RTSM (Randomization and Trial Supply Management) solutions. Bill is currently responsible for the company's eClinical strategy. Bill has authored over 60 articles in international and trade journals, and is a regular speaker on the conference circuit. Brian Tiplady, B.A., Ph.D is Senior Clinical Scientist at Pro Consulting, a division of Invivodata Inc. Brian Tiplady started in the pharmaceutical industry in 1976 as a Neuroscientist carrying out clinical research in depression and senile dementia. He later became responsible for data management and statistics, and has worked on every stage of the clinical trial process. He started working with electronic patient reported outcomes (ePRO) in 1993. He has carried out and published evaluation studies on patient use of pen-based handheld diaries and questionnaires, supported in-house projects, and worked with strategic and logistic issues involved in effectively using ePRO, and in evaluating the benefits obtained. He currently works with PRO Consulting, a division of Invivodat Inc, providing scientific support for the development and deployment of ePRO methods in clinical trials.