FDA PRO Guidance


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The PRO Final Guidance announced December 2009 helps Sponsors and CROs understand how to include the patient viewpoint in the clinical trials that support market authorization for their medical products. The Guidance shows that FDA understands the pivotal role of PRO measures in establishing clinical benefit. This Insights edition is dedicated to ePRO mentioned within the Final Guidance, and intended to provide readers with an executive summary of the new document with respect to ePRO.

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FDA PRO Guidance

  1. 1. Final FDA Guidance Brings PRO and ePRO Mainstream Tools, Glossary & Appendix Provided for Dossier SubmissionThe PRO Final Guidance announced last eDecember helps Sponsors and CROsunderstand how to include the patientviewpoint in the clinical trials that supportmarket authorization for their medicalproducts. The Guidance shows that FDAunderstands the pivotal role of PRO measuresin establishing clinical benefit. This Insights edition is dedicated to ePRO mentioned within the Final Guidance, and intended to provide readers with an executive summary of the new document with respect to ePRO. Contents Key Takeaways from FDA Final Guidance 2 on PRO Diagram of the Conceptual Framework of 2 a PRO Instrument Recommended Key Strategies for 3 Sponsors Appendix may be used as a Dossier Outline 3 Other Links on Current Industry PRO 4 Developments
  2. 2. 2Final FDA Guidance Brings PRO and ePRO MainstreamKey Takeaways from FDA Final Guidance on PRO1 Figure 4. Diagram of the Conceptual Framework of a PRO Instrument Item 1 Item 2 Domain 1 Item 3 General Item 4 Concept2 Item 5 Item 6 Domain 2 Figure 3. Development of a PRO Instrument: An Iterative Process i. Hypothesize Conceptual Framework tl hypothesized potent or lite re/exper review te in on ork te in n/ ri in endpoint l yp or st tion) nt in deve ent v. Modify Instrument ii Adjust Conceptual , Framework and Draft Instrument PRO s ↕ Claim iv. Collect, Analyze, and Interpret Data iii. Confirm Conceptual Framework and ) Assess Other Measurement Properties s3 Section lll of the Final Guidance4As a result of this collaborative instructional document, ePRO use within clinical trials will significantly grow,as more therapies that include the patient perspective are developed by industry.
  3. 3. 3Final FDA Guidance Brings PRO and ePRO MainstreamSponsors should avoid the following:1. Direct PRO data transmission from the ePRO data collection device to the sponsor, clinical investigator, or other third party without an electronic audit trail.2. Exclusive e-source document control by the sponsor.3. Inability of the clinical investigator to maintain and confirm electronic PRO data accuracy.4. The existence of only one database without backup.5. Ability of any entity other than the investigator (and/or site staff designated by the investigator) to modify the source data.6. Loss of adverse event data.7. Premature or unplanned access to unblinded data.8. Inability of an FDA investigator to inspect, verify, and copy the data at the clinical site during an inspection.9. An insecure system where records are easily altered.10. Direct PRO data transmission of important safety information to sponsors, clinical research organizations, and/or third parties, without ensuring the timely transmission of the data to the clinical investigator responsible for the patients.1Please note that numbers 3 and 10 were not present in the Draft, and they both insist on the role of theClinical Investigator preparing and maintaining the patient data to meet the intention of the Final Guidance.1 This concept was not mentioned in the FDA Draft GuidanceRecommended key strategies for Sponsors,based on the Final Guidance:1. Plan an endpoint model that shows the relationship between your planned claims and the measures, including the PRO measures.2. Choose instruments for PRO that are capable of measuring what you want to know for your claim.3. Establish content validity first, then other measurement properties.4. Take advantage that PRO can be migrated to e-versions. Verify measurement properties based on the amount of change.5. If questionnaire completion is unsupervised, explain how you know the diaries were completed at specified times.6. Begin the selection and development of your PRO measures early, since this must be done by time of confirmatory trials.7. Include final versions of the questionnaires (on paper or electronic if appropriate) in protocols.8. Prepare PRO dossier using the Guideline’s Appendix.
  4. 4. 4Need More Detailed PRO Guidance Information?The FDA Final Guidance: Key Considerations forSponsors Collecting PRO and ePRO Data PHT Electronic Patient Reported Outcome (ePRO) System ComponentsPHT Corporation Comments on New FDA Guidancefor Industry. LogPad® SitePad® StudyWorks® eSense™ Sensors ePRO Designer Study ArchiveThe Pros of ePRO, PROVision™ Scientific Services –The mobile touch-screen Trial Success Program (TSP) ™ tablet for ePRO collected at sites PHT System Support Center Scientific Review and Validation Site Telecom Assessments Technology Transfer PHT LogPad – The mobile d Hand Held for home eDiariesPHT Corporation PHT Corporation Sàrl Insights Q1 2010500 Rutherford Avenue 2, chemin Louis-Hubert www.phtcorp.comBoston, MA 02129 USA 1213 Petit-Lancy, Geneva, Switzerland Copyright © 2010 PHT CorporationToll-Free: 877.360.2901 Phone: 41.22.879.91.00 Rev 3.10