Desmond Schatz MD University of Florida NE Florida Network Symposium In Conjunction with The 28th Annual Clinical Conference on Diabetes
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 Presented as part of Clinical Recruitment Task Force JDRF, 2012
Participate to Accelerate GOAL Accelerate the pace of human clinical research l Speed recruitment and enrollment into human clinical trials l Increase participation in registries and biobanks STEPS l l l Educate and promote within Florida programs Collaborate with research centers, healthcare providers, JDRF, Helmsley, ADA, etc. 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 $286 billion)
Incidence of Type 1 Diabetes Growing Worldwide 70 T1D Incidence (# new cases/yr) is doubling every 20 yrs 60 50 40 30 20 10 Incidence/100,000/ yr in children 0-14 yr Relative Percent Increase of Type 1 Diabetes Incidence in Finland 1965-1996 (32 yrs) Finland Sweden Colorado Germany 0 1950 1960 1970 1980 1990 2000
Diabetes Research Strategic Plan (2012) Gene$c basis of type 1 diabetes, type 2 diabetes, and obesity Type 1 diabetes and autoimmunity The beta cell Type 2 diabetes: a mul$- dimensional disease Obesity Development of an ar$ficial pancreas Clinical research and clinical trials Special needs for special popula$ons Diabetes complica$ons Clinical research to prac$ce Resource and infrastructure needs Available on NIDDK website
Research Progress at All Stages of Type 1 Diabetes Prevent Onset of Autoimmunity Stop Autoimmune Attack Preserve Beta Cells 100% β -Cell Mass Risk, Triggers Autoantibody + Dysglycemia Improve Glucose Control Restore Beta Cell Function Prevent, Arrest, Reverse Complications 0% Pre-Diabetes Recent Onset Established Complications Time (yrs)
Florida T1D Research Programs & Institutions (Incomplete) Baptist Diabetes Associates Diabetes and Hormonal Disease Center JAEB Center Metabolic Research Institute Nemours Children s Clinic Orlando Diabetes & Endocrine Specialists Sanford- Burnham Medical Research Institute Tallahassee Endocrine Associates The Center for Diabetes and Endocrine Care Translational Research Institute for Metabolism and Diabetes University of Florida University of Miami University of South Florida NE
Florida Research Centers Florida State Univ Tallahassee Univ of Florida Gainesville North Florida Nemours Jacksonville Nemours Pensacola Nemours Orlando U of South Florida Tampa Jaeb Center Tampa Tampa Bay Central Florida Florida Suncoast Sanford- Burnham Orlando Greater Palm Beach County South Florida Univ of Miami Miami
Funding Sources (Incomplete) American Diabetes Association Helmsley Charitable Trust JDRF NIDDK Industry (various) Sanford Burnham Medical Research Institute Institutional NE
Types of Studies (Incomplete) Observational Prevention Interventional Treatment Complications Behavioral NE
Observational Studies (Incomplete) TrialNet Natural History Study T1D Exchange Type 1 Diabetes Genetics Consortium (T1DGC) Prospective Assessment in Newborns for Diabetes Autoimmunity (PANDA) Dietary Amino Acids and Insulin Sensitivity in Children with Type 1 Diabetes Mellitus Cognitive and Neuroanatomical Consequences of Type 1 Diabetes in Young Children LADA Immunological Studies in T1D NE
Prevention and Interventional Studies (Incomplete) TrialNet Prevention Studies: TrialNet Teplizumab for Prevention of Type 1 Diabetes in Relatives At Risk 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 Reversing Type 1 Diabetes after it is Established ATG- GCSF Efficacy and Safety Study of DiaPep277 in Newly Diagnosed Type 1 Diabetes Adults (DIA- AID2) NE
Treatment Studies (Incomplete) Statins in Children with Type 1 Continuous Glucose Monitoring in Adolescents with Poorly Controlled Type 1 Diabetes Glutamine and Insulin Sensitivity in Type 1 Diabetes Immunosafety Study of Recombinant Human Insulins in Type 1 Diabetes Efficacy and Safety of Real- Time Continuous Glucose Monitoring in the Management of Type 1 Diabetes in Young Children (4 to <10 years old) Treatment to Target of Type 2 Diabetes Mellitus NE
Complication Studies (Incomplete) Islet Transplant Clinical Trials A Cellular Approach to the Treatment of Diabetic Maculopathy Type 1 Diabetes Recurrence in Pancreas Transplants Efficacy of Islet after Kidney Transplantation Ranibizumab for Edema of the Macula in Diabetes Dyslipidemia and Diabetic Retinopathy Insulin Resistance Intervention after Stroke Trial (IRIS) Early Indices of Auditory Pathology is Young Adults with Diabetes Cardiovascular Safety Study Comparing Type 2 Diabetes Patients NE
Participate to Accelerate GOAL Accelerate the pace of human clinical research l Speed recruitment and enrollment into human clinical trials l Increase participation in registries and biobanks STEPS l l l Educate and promote within Florida programs Collaborate with research centers, healthcare providers, JDRF, Helmsley, ADA, etc. Reach out to broader T1D community
Defining the Problem Problem Research progress is slowed by substantial challenges of patient recruitment Higher costs due to longer trials Consequences Evidence 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!
Benefits of Collaboration 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 C 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
Steps to Achieving Goals Develop a communication plan within Florida diabetes community Determine channels to promote research participation Websites Newsletters Social media Events
Risks to Avoid Promoting only individual center trials Promoting only certain favored trials Promising more outreach and services than we are prepared to provide Violating HIPPA laws
Example: TrialNet in Florida Population 2011: 19,000,000 Estimated number of people with T1D (assuming 1:250-300): Estimated number of eligible TrialNet participants: 63,000 76,000 200,000 + TrialNet new screens in 2011: approx 1,000 Participation rate over lifetime of study (estimated) < 4% University of Florida and University of Miami are among the top performers of TrialNet Clinical Centers for enrollment, suggesting a larger problem nationwide
Wide Scope of Type 1 Diabetes Studies in Florida Immunological Studies in Type1 Diabetes TrialNet Natural History Study TrialNet Oral Insulin Study Anti- CD3 Neulasta T1D Exchange Type 1 Diabetes Genetics Consortium Continuous Glucose Sensors in Youth: Biobehavioral Study npod Diagnosis ATG/GCSF Clinical Trial Artificial Pancreas Islet Cell Transplantation Diabetes and Hearing Loss Study Complications
Strengthen Partnerships Improved communication between research centers Statewide list of clinical trials and contact persons ONE FLORIDA Greater breadth of clinical trials across Florida (e.g. basic science, devices) Diminish barriers to enrollment (e.g. language, locations) Who really wants to participate?
Expected Oucomes Stakeholders T1D investigators and diabetes research centers Adult and pediatric endocrinologists Research Studies Statewide clinical trials Screening locations Spanish language resources Barriers Barriers to participation among T1D community Barriers to referrals by healthcare professionals