Transcript INTRODUCING THE RELIEF REGISTRY

INTRODUCING THE
RELIEF REGISTRY
Dr. Jaron Rabinovici
Sheba Medical Center, Israel
Personal Experience with Focused Ultrasound
 Performed the second uterine fibroid FUS treatment (First
treatment at Harvard Medical School)
• Participated in the first studies, pivotal study
• Since then more than 300 uterine fibroid treatment at our Center
 Treated the first patient who got pregnant after FUS
• Published the first “pregnancy paper” on 52 pregnancies after
 Introduced the possibility to treat adenomyosis
• First study to demonstrate clinical efficacy and safety
 Head of gynecological activity of Insightec Focused Ultrasound
Center at Sheba Medical Center, Israel
 Many invited lectures
© 2013 by Focused Ultrasound Foundation
© 2013 by Focused Ultrasound Foundation
Acceptance of Focused Ultrasound
Treatment for Uterine Fibroids
Over the last decade ~ 10,000 treatments in ~ 100
centers!
Centers that managed to obtain some form of
reimbursement managed to perform many
procedures!
© 2013 by Focused Ultrasound Foundation
Problems in the Implementation
of Focused Ultrasound for Fibroids
 New technology
• Physicians are conservative and “afraid” of new/unfamiliar approaches
• Focused Ultrasound is available only in a limited number of centers
• High “entry fee”
• Need for good and extensive scientific data
• Most data based on one center, limited observational studies
• Need for extensive post-marketing experience
• Lack of extensive long-term experience/data
 Multiple Alternatives
• Big competition, “difficult sale”
 Treatment price
• Limited adoption by insurance carriers
© 2013 by Focused Ultrasound Foundation
Problems in the Implementation
of Focused Ultrasound for Fibroids
 New technology
• Physicians are conservative and “afraid” of new/unfamiliar approaches
• Focused Ultrasound is available only in a limited number of centers
• High “entry fee”
• Need for good and extensive scientific data
• Most data based on one center, limited observational studies
• Need for extensive post-marketing experience
• Lack of extensive long-term experience/data
 Multiple Alternatives
• Big competition, “difficult sale”
 Treatment price
• Limited adoption by insurance carriers
© 2013 by Focused Ultrasound Foundation
“Needed” Studies for Focused Ultrasound
Randomized Controlled Studies?
RCT’s vs “gold standard” (which?)
• Difficult to impossible to perform.
• Random decision of invasive versus non-invasive
treatment?
• Difficult recruitment, very expensive, variable results
from various centers can affect outcome, examines a
predefined patient population.
© 2013 by Focused Ultrasound Foundation
“Needed” Studies for Focused Ultrasound
Registry Studies?
• Prospective entry of consecutive patients can provide
data on clinical procedures – “real life”
• Able to gather data on many patients within a limited
time frame and follow them up for a prolonged period
of time
• The resulting reports and publications should provide a
large body of evidence that will be difficult to disregard
© 2013 by Focused Ultrasound Foundation
Gathering Evidence, Expanding Access
Focused Ultrasound for Uterine Fibroids
Registry Goal: Provide large scale evidence on
safety, efficacy and long-term durability of Focused
Ultrasound, to facilitate reimbursement and drive
rapid patient and physician acceptance
© 2013 by Focused Ultrasound Foundation
 REgistry for Leiomyoma: International Efficacy of Focused
Ultrasound
 3-year, single-arm, prospective, global registry
 1,000 women treated with MR-guided FUS at 30+ treatment
sites
 Led by Steering Committee of international, leading experts
 Funded by philanthropy and industry
© 2013 by Focused Ultrasound Foundation
Steering Committee
 Chair: Jaron Rabinovici, MD, Sheba Medical Center, Israel
 Anne Roberts, MD, University of California, San Diego
 Elizabeth A. Stewart, MD, Mayo Clinic, Rochester
 Robert K. Zurawin, MD, Baylor College of Medicine, Houston
 Elizabeth David, MD, Sunnybrook Health Sciences Centre,
Toronto, Canada
 Wladyslaw M. Gedroyc, MD, St. Mary's Hospital, London, UK
 Young-Sun Kim, MD, Samsung Medical Center, Seoul, South
Korea
 Matthias Matzko, MD, Amper Kliniken, Dachau, Germany
 Harsh Rastogi, MD, Indraprastha Apollo Hospital, New Delhi,
India
© 2013 by Focused Ultrasound Foundation
RELIEF Registry
Hypothesis
Hypothesis: Focused Ultrasound will provide a
durable improvement for women with symptomatic
uterine fibroids.
© 2013 by Focused Ultrasound Foundation
RELIEF Registry
Objectives
Primary Objectives: Provide large scale evidence
on the safety and long term efficacy of Focused
Ultrasound for the indication of symptomatic uterine
fibroids
Secondary Objectives: Assess the impact of the
treatment on medical resource utilization and
pregnancy outcomes
© 2013 by Focused Ultrasound Foundation
RELIEF Registry
Outcomes of Interest
 Primary endpoints:
• Treatment effectiveness using UFS-SSS
• Need/reason for alternative treatments
• Treatment safety using adverse events
 Secondary endpoints:
• Treatment satisfaction
• Recovery using return to work and/or normal activities
 Exploratory endpoints:
• Medical resources utilization
• Pregnancy outcomes
• General health-related quality life
© 2013 by Focused Ultrasound Foundation
RELIEF Registry
Registry Design
Target –1,000 women in two years, three years
follow-up.
Approximately 30 sites in:
• North America - U.S., Canada
• Europe - Germany, United Kingdom
• Asia - Korea, India, Israel
Each site will enroll approximately 50 consecutive
patients who receive Focused Ultrasound
© 2013 by Focused Ultrasound Foundation
PROTOCOL
1,000
planning
to be treated
using focused ultrasound
Complete
baseline
Survey
~30 Sites
Treatment Information:
Duration,
Number of fibroids
Treated fibroids
Fibroid volume, NPV
AEs
Self-report
© 2013 by Focused Ultrasound Foundation
Baseline:
Demographics
Gynecologic history
Uterine, fibroid anatomy
Future fertility
Symptoms and QoL
Treatment
Treatment form
completed
1 month,
6 month,
1-2-3 year surveys
Complete
study
Follow-up Measures:
Additional treatments
Treatment Satisfaction
Resource utilization
Pregnancy outcomes
AEs
Symptoms and QoL
RELIEF Registry
Patient Population
 Inclusion
• Patient is
—Intended to be treated for symptomatic uterine fibroids by focused
ultrasound (consistent with local labeling)
—Able to communicate over the phone/internet and can complete study
questionnaires
—Willing to be followed for three years
 Exclusion
• Patient is
—Pregnant at time of treatment
—Post-menopausal
© 2013 by Focused Ultrasound Foundation
RELIEF Registry
Publications
Treatment guidelines
Structure
Baseline data
Short-term results
Mid-term results
Long-term results
Other analyses TBD
© 2013 by Focused Ultrasound Foundation
RELIEF Registry
Status
Enrollment to begin second half this year
Sites to come onboard soon – potential US
locations to include:
Bethesda, MD
Boca Raton, FL
Boston, MA
Charlottesville, VA
Chicago, IL
Columbus, OH
© 2013 by Focused Ultrasound Foundation
Houston, TX
Nashville, TN
Rochester, MN
San Diego, CA
San Francisco, CA
Stanford, CA
REFERRING PATIENTS TO RELIEF
Every patient treated in the U.S. will be
eligible for the registry
RELIEF will systematically collect data and
provide evidence-based recommendations for
patient selection and outcomes
www.fibroidrelief.org/registry
© 2013 by Focused Ultrasound Foundation