Our research
team includes scholars at the University
of North Carolina School of Medicine, Vanderbilt
University Medical
Center, Wake Forest
University School of Medicine, and the University of Washington School of
Medicine.
The “Social
Construction
of
Benefit
in Gene Transfer Research” study, 1R01HG02087, is supported by
the
Ethical,
Legal and Social Implications (ELSI) Research Program of the
National
Human Genome Research Institute, and is currently funded as a
competitive renewal (2R01HG02087).
Patients
with serious disease who are potential
research
subjects are routinely described as “vulnerable” and as
disproportionately
anticipating direct benefit from participation in early-phase trials.
In our
initial grant
(1999-2003), we
explored how benefit in gene transfer research (GTR)
is
discussed
and understood, and whether and how the “therapeutic misconception”
exists in
GTR. Our data identified individual and
study-level factors associated with vulnerability and the therapeutic
misconception in GTR, but we also found that the relationship between
expectation of direct benefit for subjects and these factors is complex
and may
call into question some generally held assumptions (see Project Publications).
The current project uses our
rich qualitative and quantitative data in a deeper conceptual and
analytical
investigation of benefit in GTR, with the following aims:
1) Development of a
multi-factorial model of influence in GTR, based on research
relationships, to replace the static and problematic notion of vulnerability.
We will be analyzing research participation as a form of social
exchange, and
delineating a dynamic continuum of influences, ranging from “due” to “undue,”
affecting all parties to research relationships.
2) Development of a benefit threshold for assessing what may
be offered
as a “reasonable prospect of direct benefit” for subjects in
early-phase GTR.
This will include examination of how study endpoints relate to direct
benefit,
of specificity in discussion of direct benefit in early-phase research,
of the
effects of trial design features on direct benefit, and an assessment
of
collateral (“inclusion”) and societal benefit.
3) Application of our influence model and benefit threshold to a
close examination
of the “vulnerable” population of children in GTR studies, where
special
regulatory guidance, the dual problems of inclusion and access, and
growth and
change in the field of GTR affect how benefit in pediatric GTR is
viewed by
IRBs, investigators, and families.
Products of this research
will include
empirically and
conceptually based analyses, policy
recommendations, and guidance documents
disseminated
to research oversight bodies and policymakers, scientific and
professional
organizations for GTR and other research, and patient advocacy groups.