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