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Chair:
Donald L. Patrick PhD, MSPH, Professor, Department of Health Services, University of Washington, Seattle, WA, USA
Leadership Team:
Laurie B. Burke RPh, MPH,
Director, Study Endpoints and Label Development, Office of New
Drugs, CDER, FDA, Washington, DC, USA
Chad Gwaltney PhD,
Assistant Professor (Research), Department of Community Health,
Brown University, Providence, RI, USA and Senior Scientist,
PRO Consulting, Pittsburgh, PA, USA
Nancy Kline Leidy PhD,
Senior Vice President of Scientific Affairs, United BioSource,
Bethesda, MD, USA
Mona L. Martin RN, MPA,
Executive Director, Health Research Associates, Inc, Seattle,
WA, USA
Lena Ring PhD, Principal Scientist in PRO Strategy, HEOR, R&D, AstraZeneca, Södertälje, Sweden and Associate Professor, Pharmaceutical Outcomes Research, Department of Pharmacy, Uppsala University, Sweden

Goal:
The task
force will develop the following topics and provide recommendations for the
following research methods:
- Methods for generating
items for a new PRO instrument including item wording, evaluating the
completeness of item coverage, and performing initial assessments of clarity
and readability. These are often called concept\item elicitation studies.
- Methods for documenting
item development including the qualitative interview guide used in item
elicitation, sample composition (inclusion and exclusion criteria) and size,
training of interviewers, and methods for demonstrating and documenting that
saturation has been achieved or no new content is being elicited.
Recommendations will be developed for each of these subtopics.
- Methods for coding of
qualitative data from item generation, development of the qualitative
coding frame, and application of this frame including evaluation of
inter-coder reliability.
- Methods of cognitive
interviewing including patient understanding of items and evaluating
appropriateness of recall period and response options
- Methods
for tracking item development through the various stages of research and
preparing this tracking for submission to regulatory agencies.
Background / Overview:
Content
validity is the extent to which an assessment instrument measures the important
aspects of the concept assessed. A patient reported outcome (PRO)
instrument measures a concept relevant and important to the patient’s condition
and its treatment. For PRO instruments, items and domains as reflected in
the scores of an instrument should be important to the target population of
patients and comprehensive with respect to patient concerns concerning the
concept being assessed. Documentation of target patient population input
in item generation, as well as evaluation of patient understanding through
cognitive interviewing, can provide the evidence for content validity.
The
importance of PRO instrument content validity is stressed both by the US Food
and Drug Administration Draft Guidance (US Food and Drug Administration, 2006)
and European Medicines Agency Reflection Paper on the Regulatory Guidance for
the Use of Health Related Quality of Life (HRQL) Measures (EMEA, 2006). These
guidance documents describe how PRO or HRQL measures are used as effectiveness
end points and how study results are used to support approval or in the US
context, label claims and advertising. Although these documents include
discussions on instrument evaluation, study design, and data analysis, they do
not provide developers or industry specific recommendations on how to gather and
prepare documentation of content validity. The FDA guidance provides a
summary of the kinds of evidence needed to support a conclusion that content
validity is adequate for the purpose proposed.
A
previous ISPOR Good Practices Task Force has taken up the issues involved in the
evaluation of content validity of existing PRO instruments, Evaluating
and Documenting the Content Validity of Patient-Reported Outcome Instruments to
Support Medical Product Labeling and Promotional Claims in the United States:
Report of the ISPOR PRO Existing Instruments & Their Modifications Task Force.(Rothman,
2009).
http://www.ispor.org/TaskForces/documents/Rothman.pdf
The report outlines the broad need for documentation and discusses the issues
involved when existing PRO instruments are modified or evaluated for content
validity.
However,
it does not take up the specific methodological practices involved in (1)
designing studies to gather evidence of content validity and (2) methods for
evaluating and documenting content validity. This proposed TF report
will build upon this work with a more specific focus on two methodological
issues within the scope of a manuscript submitted for review by ISPOR membership
and submitted for publication to Value in Health.
Timeline effective 3/31/10:
Activity: |
Deadline: |
Task Force approved by ISPOR Board of Directors |
March 23, 2009 |
Small group meeting at ISPOR 14th Annual International Meeting |
May 2009 |
Decision made to produce 2 manuscripts - manuscript content consensus reached |
August 2009 |
Face to face working meeting at ISOQOL |
October 2009 |
Drafting and discussion |
November 2009 – April 2010 |
PRO Short Course on Content Validity & PRO Forum at ISPOR 15th Annual International Meeting |
May 2010 |
TC discussion of ISPOR meeting and comments with task force members |
Week of 5/24 |
Manuscripts revised based on attendees’ and task force members’ comments. |
June – July 2010 |
Donald sends finalized manuscript to EM |
July 20, 2010 |
Manuscripts circulated to the ISPOR PRO Reviewer Group for comments |
July 22 – August 6, 2010 |
Manuscripts circulated to ISPOR membership for comments |
August 16 – September 7, 2010 |
TC discussions of ALL reviewer comments |
August 24 & August 30, 2010 |
| Revisions |
September 2010 |
| Discussion of revised manuscripts |
October 2010 |
| Finalize and format manuscripts |
October 2010 – November 2010 |
| Submit to Value in Health |
Mid November 2010 |
| Press Release Due |
On ViH early online publication |
Task Forces Index |
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