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NE In Conjunction with The 28th Annual Clinical Conference on Diabetes
Florida Network Symposium
Desmond Schatz MD
University of Florida
Presented as part of Clinical Recruitment Task Force JDRF, 2012
Increase Participation in Clinical Trials
Accelerate the Path to Better Treatments and a Cure for Type 1 Diabetes A National Vision • A Pilot Program in Florida
Participate to Accelerate
Accelerate the pace of human clinical research
GOAL
Increase participation in registries and biobanks
Speed recruitment and enrollment into human clinical trials
l
l
STEPS
l Educate and promote within Florida programs l Collaborate with research centers, healthcare
providers, JDRF, Helmsley, ADA, etc. l Reach out to broader T1D community
Burden of Diabetes in USA (2012)
Diabetes Rising Morbidity/Mortality Economic Burden + =Prevalence/Incidence:
§ 22.3 million Americans
§ 6.3 million undiagnosed
§ 1.6 million cases/year
§ 10% type 1 (1/300)
High rate as evidenced by:
§ > 246,000 deaths or > 600 per day
§ Shortened life span
§ 2-4x risk MI, stroke
§ 75% hypertensive
§ 47,000 new cases RD/yr
§ 12,000 – 24,000 new cases blindness/yr
§ >82,000 amputations/yr
2012 Medical costs:
§ $245 billion
§ ~ 1/8 health care dollars
§ 27% of all medications ($77 billion of $286 billion)
0
10
20
30
40
50
60
70
1950 1960 1970 1980 1990 2000
Finland
Sweden
Colorado
Germany
Incidence/100,000/ yr in children 0-‐14 yr
T1D Incidence (# new cases/yr) is doubling every 20 yrs
Relative Percent Increase of Type 1 Diabetes Incidence in
Finland 1965-1996 (32 yrs)
Incidence of Type 1 Diabetes Growing Worldwide
Diabetes Research Strategic Plan (2012)
Ø Gene$c basis of type 1 diabetes, type 2 diabetes, and obesity
Ø Type 1 diabetes and autoimmunity Ø The beta cell Ø Type 2 diabetes: a mul$-‐dimensional disease
Ø Obesity Ø Development of an ar$ficial pancreas Ø Clinical research and clinical trials Ø Special needs for special popula$ons Ø Diabetes complica$ons Ø Clinical research to prac$ce Ø Resource and infrastructure needs
Available on NIDDK website
Time (yrs)
β -C
ell M
ass
100%
0%
Ris
k, T
rigge
rs
Prevent Onset of
Autoimmunity
Recent Onset Established Complications
Preserve Beta Cells
Pre-Diabetes
Aut
oant
ibod
y +
Stop
Autoimmune Attack
Prevent, Arrest,
Reverse Complications
“Dys
glyc
emia
”
Improve Glucose Control
Restore Beta Cell Function
Research Progress at All Stages of Type 1 Diabetes
NE
Ø Baptist Diabetes Associates Ø Diabetes and Hormonal Disease Center Ø JAEB Center Ø Metabolic Research Institute Ø Nemours Children’s Clinic Ø Orlando Diabetes & Endocrine Specialists Ø Sanford-‐Burnham Medical Research Institute Ø Tallahassee Endocrine Associates Ø The Center for Diabetes and Endocrine Care Ø Translational Research Institute for Metabolism and Diabetes Ø University of Florida Ø University of Miami Ø University of South Florida
Florida T1D Research Programs & Institutions (Incomplete)
Tampa Bay
Univ of Florida Gainesville
Florida State Univ Tallahassee
North Florida Nemours Jacksonville
Nemours Orlando
Sanford-‐Burnham Orlando
Central Florida
Florida Suncoast
Greater Palm Beach County
South Florida
Univ of Miami Miami
Nemours Pensacola
Florida Research Centers
U of South Florida Tampa
Jaeb Center Tampa
NE
Ø American Diabetes Association
Ø Helmsley Charitable Trust
Ø JDRF
Ø NIDDK
Ø Industry (various)
Ø Sanford Burnham Medical Research Institute
Ø Institutional
Funding Sources (Incomplete)
NE
Ø Observational
Ø Prevention
Ø Interventional
Ø Treatment
Ø Complications
Ø Behavioral
Types of Studies (Incomplete)
NE
Ø TrialNet Natural History Study Ø T1D Exchange Ø Type 1 Diabetes Genetics Consortium (T1DGC) Ø Prospective Assessment in Newborns for Diabetes Autoimmunity (PANDA) Ø Dietary Amino Acids and Insulin Sensitivity in Children with Type 1 Diabetes Mellitus Ø Cognitive and Neuroanatomical Consequences of Type 1 Diabetes in Young Children Ø LADA Ø Immunological Studies in T1D
Observational Studies (Incomplete)
NE
TrialNet Prevention Studies: TrialNet Ø Teplizumab for Prevention of Type 1 Diabetes in Relatives “At Risk” Ø Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus • Anti-‐CTLA-‐4 (Abatacept) prevention of dysglycemia and diabetes in
double Ab+ subjects
Interventional Studies Ø Reversing Type 1 Diabetes after it is Established Ø ATG-‐GCSF Ø Efficacy and Safety Study of DiaPep277 in Newly Diagnosed Type 1 Diabetes Adults (DIA-‐AID2)
Prevention and Interventional Studies (Incomplete)
NE
Ø Statins in Children with Type 1 Ø Continuous Glucose Monitoring in Adolescents
with Poorly Controlled Type 1 Diabetes Ø Glutamine and Insulin Sensitivity in Type 1 Diabetes Ø Immunosafety Study of Recombinant Human Insulins in Type 1 Diabetes Ø Efficacy and Safety of Real-‐Time Continuous Glucose Monitoring in the Management of Type 1 Diabetes in Young Children (4 to <10 years old) Ø Treatment to Target of Type 2 Diabetes Mellitus
Treatment Studies (Incomplete)
NE
Ø Islet Transplant Clinical Trials Ø A Cellular Approach to the Treatment of Diabetic Maculopathy Ø Type 1 Diabetes Recurrence in Pancreas Transplants Ø Efficacy of Islet after Kidney Transplantation Ø Ranibizumab for Edema of the Macula in Diabetes Ø Dyslipidemia and Diabetic Retinopathy Ø Insulin Resistance Intervention after Stroke Trial (IRIS) Ø Early Indices of Auditory Pathology is Young Adults with Diabetes Ø Cardiovascular Safety Study Comparing Type 2 Diabetes Patients
Complication Studies (Incomplete)
Participate to Accelerate
Accelerate the pace of human clinical research
GOAL
Increase participation in registries and biobanks
Speed recruitment and enrollment into human clinical trials
l
l
STEPS
l Educate and promote within Florida programs l Collaborate with research centers, healthcare
providers, JDRF, Helmsley, ADA, etc. l Reach out to broader T1D community
Problem
Consequences
Evidence
Research progress is slowed by substantial challenges of patient recruitment
Defining the Problem
Higher costs due to longer trials
Fewer trials performed
Disincentive to sponsors
Slower progress
Participation rate in eg., TrialNet estimated to be < 4%
20,000 screenings yielded ~50 participants in Oral Insulin study
The Solution
A concerted and broadly collaborative effort to educate and inspire participation
in T1D clinical research
… a change of culture!
C
Enhanced Reputation
Expanded Outreach Success
Accelerated Pace of Cure
Mission-‐driven imperative
Heightened visibility and credibility
Collaborate efforts viewed positively among
constituents
New avenues to engage and provide value • Families and adult T1Ds • Research centers • Healthcare professionals • Professional societies
New avenues to engage volunteers and donors New opportunities for partnerships
Accelerated recruitment means decreased time and cost of clinical research, a “de-‐risk” factor for industry Accelerated recruitment translates into greater number of clinical trials Increased screening makes more prevention clinical trials possible High T1D Exchange participation speeds research
Large returns for small investment of time and funds
Benefits of Collaboration
Steps to Achieving Goals
Develop a communication plan within Florida diabetes community
Determine channels to promote research participation
Ø Websites Ø Newsletters Ø Social media Ø Events
Promoting only certain favored trials
Promising more outreach and services than we are prepared to provide
Violating HIPPA laws
Promoting only individual center trials
Risks to Avoid
TrialNet new screens in 2011: approx 1,000
Participation rate over lifetime of study (estimated)
< 4%
Estimated number of eligible TrialNet participants:
200,000 +
Estimated number of people with T1D (assuming 1:250-‐300):
63,000 – 76,000
Population 2011: 19,000,000
University of Florida and University of Miami are among the top performers of TrialNet Clinical Centers for enrollment,
suggesting a larger problem nationwide
Example: TrialNet in Florida
Diagnosis
TrialNet Natural History Study
TrialNet Oral Insulin Study
Anti-‐CD3 Continuous Glucose Sensors in Youth:
Biobehavioral Study
Artificial Pancreas
Diabetes and Hearing Loss Study
T1D Exchange
ATG/GCSF Clinical Trial Islet Cell
Transplantation
Type 1 Diabetes Genetics Consortium
nPOD
Neulasta
Immunological Studies in Type1 Diabetes
Complications
Wide Scope of Type 1 Diabetes Studies in Florida
Improved communication between
research centers
Statewide list of clinical trials and contact
persons
Greater breadth of clinical trials across Florida (e.g. basic science, devices)
Diminish barriers to enrollment (e.g.
language, locations)
ONE FLORIDA
Strengthen Partnerships
Who really wants to participate?
• T1D investigators and diabetes research centers
• Adult and pediatric endocrinologists Stakeholders
• Statewide clinical trials
• Screening locations • Spanish language resources
Research Studies
• Barriers to participation among T1D community
• Barriers to referrals by healthcare professionals
Barriers
Expected Oucomes