When collecting patient data gets electronic 4
Promises and challenges of deploying an ePRO system in clinical trials and observational studies ISPOR 16th Annual European Congress Workshop #12, Tuesday 5 November 2013
Dublin, Ireland Laure Delbecque, PhD
First challenge : Demonstrate the added value of PROs
Industry
Academic Patients
Clinicians
Policy makers, regulators
Second challenge: Improve the quality of PROs 44.8% of the new drugs approved by the FDA between 2006 and 2010 included PROs in their clinical trials ‐ only 24.1% received a PRO label (Carla DeMuro et al., 2012) No treatment benefit 14% Admin issues 10%
Fit for purpose 38 %
Statistical issues 11% Study design, data quality, interpretatio n 27 %
Solution: Electronic PRO (ePRO)
Data Quality
Compliance
Efficiency
ePRO – Powerful tool to gather very different types of data, behind efficacy data Engage the patient
ePRO – Powerful tool to gather very different types of data, behind efficacy data
Population level
• Current disease knowledge • Current patient behaviours • Health survey
Individual level
• Patient‐clinician interaction
ePRO – Well accepted by patients Patient burden of completing large numbers PROs is often a concern, particularly when PROs must be completed daily, or at multiple timepoints over long studies – ePRO completion is quick, easy, not burdensome and could be even less burdensome if ePROs are carefully designed, using skip‐patterns and event driven items (Arbuckle et al. ISPOR 2013 – PRM164) – Works also with elderly : Subject and site ePRO acceptance was high ‐ average age of study subjects exceeded 71 years. (Kuca et al. 2002; Krystal et al. (2003)
– ePRO was preferred in 88.2% of the articles vs. paper PRO (example of reasons: up‐to‐date/novel, portability, faster to complete, easy to use, more fun, like appearance, green, ...) (Ross et al. 2010)
ePRO are largely used
Meacham et al. (2008) – Data set of 150 studies
ePRO are largely used
Meacham et al. (2008) – Data set of 150 studies
Still reservations : why?
Paper
ePRO
What does an ePRO study involve? 1. Selection of technology 2. Questionnaire – –
Development? Migration?
3. Logistic
Choice of technology (I)
Choice of technology (II) The best mode is study‐specific and depends on: – Characteristics of the instrument (e.g., long questionnaire, body diagram : avoid IVRS; long items: avoid small handled device) – Characteristics of the patient population (e.g. functional limitations; sight issues) – Characteristics of the study (e.g., where the questionnaire will be completed & how often; how long is the study; internet connectivity; geographical reach of the study; population without computers) – Budget? • Patient’s own phone/computer: reduce the cost related to shipping devices, training patients on devices, device itself
ePRO – Development: additional steps compared to paper questionnaire
Select technology
Develop and validate the ePRO
Cognitive debriefing for usability testing
Adapt items & instructions wording, items length & front according to technology; Electronic implementation assessment (C‐path)
ePRO – migration
Select technology
Develop and validate the ePRO
Cognitive debriefing – usability testing
Revise instructions & items wording, length, format
(Equivalence /full psychometric testing)
Logistic (I) Patient and site training
Standardized training should be given to sites and patients on PRO (FDA guidance) Training improves patient compliance (Ross et al., 2012) When? Before patients begin study participation Adapted to the study population F/F training + elearning, videos (demonstration)+ printed documentation is recommended with patients and staff (all trial personnel) (for the patients to take away with them), Q/A sessions. Simple! Refresher training throughout the duration of the study (especially for home based ePRO) (patient + staff) Include the patient ePRO training in the PTC as part of the site visit to ensure the site carries it out Have some means of reporting and tracking which sites have trained which patients Helpdesk 24/7 in local language
Logistic (II) •
Feasability testing (e.g. download connection is available; toll free numbers work, device testing)
•
Shipping ‐ Customs issues in some countries
Conclusion
• It’s our responsibility to ensure the Patient’s voice is heard ... accurately • ePRO is one vehicle to efficiently capture that voice • Ehealth and mhealth technology allows to go one step further and decrease some measurement bias (See M. Desseilles’ presentation)