Validation of Electronic Systems to Collect Patient-Reported Outcome (PRO) Data - Recommendations for Clinical Trial Teams: Report of the ISPOR ePRO Systems Validation Good Research Practices Task Force

Validation of Electronic Systems to Collect Patient-Reported Outcome (PRO) Data - Recommendations for Clinical Trial Teams: Report of the ISPOR ePRO Systems Validation Good Research Practices Task Force Abstract. Outcomes research literature has many examples of high-quality, reliable patient-reported outcome (PRO) data entered directly by electronic means, ePRO, compared to data entered from original results on paper. Clinical trial managers are increasingly using ePRO data collection for PRO-based endpoints. Regulatory review dictates the rules to follow with ePRO data collection for medical label claims. A critical component for regulatory compliance is evidence of the validation of these electronic data collection systems. Validation of electronic systems is a process versus a focused activity that finishes at a single point in time. Eight steps need to be described and undertaken to qualify the validation of the data collection software in its target environment: requirements definition, design, coding, testing, tracing, user acceptance testing, installation and configuration, and decommissioning. These elements are consistent with recent regulatory guidance for systems validation. This report was written to explain how the validation process works for sponsors, trial teams, and other users of electronic data collection devices responsible for verifying the quality of the data entered into relational databases from such devices. It is a guide on the requirements and documentation needed from a data collection systems provider to demonstrate systems validation. It is a practical source of information for study teams to ensure that ePRO providers are using system validation and implementation processes that will ensure the systems and services: operate reliably when in practical use; produce accurate and complete data and data files; support management control and comply with any existing regulations. Furthermore, this short report will increase user understanding of the requirements for a technology review leading to more informed and balanced recommendations or decisions on electronic data collection methods.

Good Practices for Outcomes Research, Надлежащая практика изучения исходов, patient-reported outcome, сбор электронных данных, ePRO, PRO, валидация системы, electronic data collection, ePRO, PRO, systems validation

1. Coons S.J., Gwaltney C.J., Hays R.D., et al. Recommendations on evidence needed to support measurement equivalence between electronic and paper-based patient-reported outcome (PRO) measures: ISPOR ePRO Good Research Practices Task Force Report. // Value Health 2009;12:419-29. Available from: http:// www.ispor.org/workpaper/patient_reported_outcomes/ Coons.pdf. [Accessed March 26, 2013].

2. US Food and Drug Administration. Guidance for industry: patient reported outcome measures: use in medical product development to support labeling claims. December 2009. Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatory Information/ Guidances/ UCM193282.pdf. [Accessed February 19, 2011].

3. European Medicines Agency. Reflection paper on the regulatory guidance for the use of health related quality of life (HRQL) measures in the evaluation of medicinal products. 2005. Available from: http://www.emea.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003637.pdf. [Accessed February 22, 2011].

4. Paty J., Stokes T. Electronic diaries, part 1: what is a subject diary, and how do regulations apply. // Appl Clin Trials 2002. Available from: http://www. appliedclinicaltrialsonline.com/ appliedclinicaltrials/article/articleDetail.jsp?id=83521. [Accessed March 23, 2011].

5. Hufford M.R., Stokes T.E., Paty J.A. Collecting reliable and valid real-time patient experience data. // Drug Inf J 2001;35:755-65.

6. Paty J., Stokes T. Electronic diaries, part 2: the role of the clinical protocol in developing and implementing electronic diaries. Appl Clin Trials 2003. Available from: http://www.appliedclinicaltrialsonline.com/appliedclinicaltrials/article/articleDetail.jsp?id=90715. [Accessed March 23, 2011].

7. Chamberlain R. Computer Systems Validation for the Pharmaceutical and Medical Device Industries. (2nd ed.). Libertyville, IL: Alaren Press, 1994.

8. Gogates G.D. Software validation in accredited laboratories: a practical guide. June 2010. Available from: ftp://ftp.fasor.com/pub/iso25/validtion/adequate_ for_use.pdf. [Accessed July 5, 2012].

9. US Food and Drug Administration. Guideline on general principles of process validation. 1987. Available from: http://www.fda.gov/ MedicalDevices/DeviceRegulationandGuidance/ PostmarketRequirements/QualitySystemsRegulations/MedicalDeviceQualitySystemsManual/ucm122439.htm. [Accessed September 3, 2011].

10. US Food and Drug Administration. Guidance for industry process validation: general principles and practices. 2011. Available from: http://www.fda.gov/ downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM070336.pdf. [Accessed September 3, 2011].

11. European Commission Health and Consumers Directorate-General. The rules governing medicinal products in the European Union, volume 4: good manufacturing practice: medicinal products for human and veterinary use, annex 11: computerised systems. June 2011. Available from: http://ec.europa.eu/ health/files/eudralex/vol-4/annex11_01-2011_en.pdf. [Accessed September 1, 2011].

12. U.S. Food and Drug Administration. General principles of software validation: final guidance for industry and FDA staff. January 2002. Available from: http:// www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm126955.pdf. [Accessed August 12, 2011].

13. Stokes T. The Survive and Thrive Guide to Computer Validation. Buffalo Grove, IL: Interpharm Press, Inc., 1998.

14. Stokes T., Branning R.C., Chapman K.G., et al. Good Computer Validation Practices: Common Sense Implementation. Buffalo Grove, IL: Interpharm Press, Inc., 1998.

15. US Food & Drug Administration. Guidance for industry part 11, electronic records; electronic signatures - scope and application. August 2003. Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072322.pdf. [Accessed August 15, 2011].

16. Cardie M.L., Tucker N.G., Weiss L.M. Computer system validation. GMP Rev 2005;4:20-2.Fig. 1 - Sample of traceability matrix.488 // Value Health 16 (2013) 480 - 489.

17. Stokes T., Paty J. Electronic diaries, part 3: developing and validating electronic diaries: roles for the technical and clinical teams. // Appl Clin Trials 2003;6:68-78.

18. PIC/S. Good practices for computerised systems in regulated “GXP” environments, section 13 “testing” report PI 011-3, Pharmaceutical Inspection Convention, Geneva. September 2007. Available from: http://www.picscheme.org/publication.php?id=8. [Accessed August 12, 2012].

19. European Medicines Agency (EMA) ICH Topic E 6 (R1) Guideline for good clinical practice. July 2002. Available from: http://ichgcp.net/pdf/ich-gcp-en.pdf. [Accessed September 21, 2012].

20. Atkins T. User acceptance testing: finally some validation? Silverpath Technologies. 2009.Available from:http://silverpath.com/resources/Silverpath-UserAcceptan ceTestingWhitepaper-090203.pdf. [Accessed March 26, 2013].