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.

1. Final FDA Guidance Brings PRO and ePRO Mainstream
Tools, Glossary & Appendix Provided for Dossier Submission
The PRO Final Guidance announced last
e
December 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.
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
Final FDA Guidance Brings PRO and ePRO Mainstream
Key Takeaways from FDA Final Guidance on PRO
1 Figure 4. Diagram of the Conceptual Framework of a PRO Instrument
Item 1
Item 2 Domain
1
Item 3
General
Item 4 Concept
2 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
s
3 Section lll of the Final Guidance
4
As 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
Final FDA Guidance Brings PRO and ePRO Mainstream
Sponsors 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.1
Please note that numbers 3 and 10 were not present in the Draft, and they both insist on the role of the
Clinical 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 Guidance
Recommended 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
Need More Detailed PRO Guidance Information?
The FDA Final Guidance: Key Considerations for
Sponsors Collecting PRO and ePRO Data
PHT Electronic Patient Reported Outcome
(ePRO) System Components
PHT Corporation Comments on New FDA Guidance
for Industry. LogPad®
SitePad®
StudyWorks®
eSense™ Sensors
ePRO Designer
Study Archive
The 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 eDiaries
PHT Corporation PHT Corporation Sàrl Insights Q1 2010
500 Rutherford Avenue 2, chemin Louis-Hubert www.phtcorp.com
Boston, MA 02129 USA 1213 Petit-Lancy, Geneva, Switzerland Copyright © 2010 PHT Corporation
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