Setting Priorities For Comparative Effectiveness Research In IBD:
Results Of An International Provider Survey, Expert RAND Panel, And Patient Focus Groups
A. Cheifetz1, G. Melmed2, B. Spiegel3, J. Talley4, S. Devlin5, L. Harrell6, P. Irving7,*, J. Jones8, G. Kaplan5, P. Kozuch9, M. Sparrow10, F. Velayos11, L. Baidoo12,B. Bressler13, C. Siegel14
1Beth
Israel Deaconess Medical Center, Boston, MA 2Cedars-Sinai Medical Center, 3VA Greater Los Angeles Healthcare System, 4CURE Digestive Disease Research Center , Los Angeles, CA, 5University of Calgary, Calgary, Canada, 6University of Chicago,
Chicago, IL, 7Guy's and St. Thomas' Hospitals, London, United Kingdom, 8University of Saskatchewan, Saskatoon, Canada, 9Jefferson University, Philadelphia, PA, 10The Alfred Hospital, Melbourne, Australia, 11University of California San Francisco, San
Francisco, CA 12University of Pittsburgh, Pittsburgh, PA, 13University of British Columbia, Vancouver, Canada, 14Dartmouth-Hitchcock Medical Center, Lebanon, NH
INTRODUCTION
Top 10 Comparative Effectiveness Research Priorities
Compare the effectiveness of…
 Comparative effectiveness research (CER) is an
emerging field that compares the relative
effectiveness of alternative strategies to prevent,
diagnose, or treat patients that are typical of day-today clinical practice.
In 2009 the Institute of Medicine (IOM) published a
priority list for CER for all of medicine.
To assist the field of IBD in CER it is necessary to
develop priorities specific to Crohn’s and colitis.
AIM
Withdrawal of biologic therapy vs. withdrawal of
immunomodulator therapy vs. continued combination
therapy in Crohn’s disease patients who achieved
remission on combination therapy
Efficacy and safety of infliximab vs. cyclosporine vs.
surgery in hospitalized steroid-refractory ulcerative colitis
Using biomarkers (genetics, immunology) vs. standard of
care in predicting response and toxicity to therapy of IBD
Different TNF inhibitors for the induction and maintenance
of remission and mucosal healing in Crohn's disease
6MP versus anti-TNF agents in preventing clinical and
endoscopic post-op recurrence of Crohn's disease after
resection
Anti-TNF alone versus anti-TNF+6MP in patients with
moderate to severe Crohn's disease failing 6MP
Scheduled post-operative colonoscopy vs. clinical followup guiding post-operative management
Tailoring therapy to mucosal healing vs. standard clinical
factors on long-term outcomes in IBD
Standard anti-TNF dosing vs. trough-level anti-TNF dosing
in IBD
The efficacy and safety of long-term immunomodulation
(6MP/AZA) versus biologic (anti-TNF) therapy in Crohn's
disease and UC
 To develop a priority list of CER studies for IBD.
METHODS
 Following the IOM's approach for developing CER
priorities, we surveyed practicing GIs to determine
their most important CER topics in IBD.
 The survey was electronically distributed to an
international sample of GIs from the membership of:
American College of Gastroenterology
Canadian Association of Gastroenterology
Australian IBD Association
British Society of Gastroenterology
 In addition, an international panel of 13 IBD
experts (BRIDGe group) independently proposed
CER studies.
 Two patient focus groups were convened to
determine their opinions of the most important IBD
research topics.
All candidate CER studies were compiled into 21
topic categories.
 CER studies were presented to the expert panel
using the validated RAND/UCLA method.
Rated using a standard 1-9 point scale
(1=lowest priority; 9=highest; >7=high priority).
Following the initial ratings the panel met in
person to re-rate priorities after group discussion.
Disagreement was assessed using a validated
index.
RESULTS
CONCLUSIONS
 234 unique CER questions.
 We systematically developed a list of high
priority CERs for IBD based on a survey of
GIs, expert review, and patient input.
 Based on the initial round of ranking and
clinical relevance, the RAND panel
selected 87 CER questions to re-rate in
person.
 40 were rated as high priority.
 Disagreement was observed in only 5
of 87 CER questions.
 The panel then distributed points to the
highest rated CER questions to develop
the top 10 CER priorities for IBD.
This list can guide IBD researchers and
funding agencies towards the most
important CERs.
www.BRIDGeIBD.com
The panel meeting was supported by an
unrestricted grant from Warner Chilcott, Abbott,
and Shire. Unrestricted funding for the BRIDGe
group is provided by Abbott, UCB, Salix and
Warner Chilcott.