Diabetes Clinical and Translational Research:Rewards and Challenges

Ruth S. Weinstock MD PhDMedical Director, Joslin Diabetes Center and Clinical

Research Unit Professor of Medicine and Chief, Endocrinology, Diabetes

and MetabolismSUNY Upstate Medical University

Syracuse NY

October 24, 2011

Disclosures

Research funding: NIH/NIDDK, Helmsley Charitable Trust

Speaker bureaus/honoraria: none

Multicenter clinical trials:

Diamyd, Eli Lilly, Pfizer, Ultradian Diagnostics Inc., GlaxoSmithKline, Macrogenics, MiniMed

Prevention and Management of Diabetes:Types of Clinical Research

• Initiation:– Investigator vs. non investigator

• Focus:– Medications – Devices– Behavioral interventions – Epidemiology– Quality of life– Health services– Others

Sponsors

Federal: NIH, DOD, VA, CMS, otherFoundations/non-for-profits (ADA, JDRF, others)State health departments (usually limited funding and indirect

cost recovery)Industry:

Phase I: evaluate safety, determine dose, side effects (n=20-80)Phase II: evaluate effectiveness, further safety (n=100-300)Phase III: confirm effectiveness, monitor side effects, compare to standard Rx (n=1000-3000)Phase IV: collect more information re: risks, benefits, uses

Hybrids

Why Participate?

• Intellectual benefits: contribute to understanding of disease and development of new therapies

• Academic rewards

• Early experience with new treatments

• Free services/medications to the needy

Requirements

• Trained staff• For subjects: benefits > risks• Adherence to GCP (Good Clinical Practice) principles• Compliance with federal regulations (Protection of Human

Subjects)• IRB approval• Ability to meet recruitment and retention goals• Meticulous record keeping• Sufficient funding (careful budgeting!)• Dependent upon study: EMR, statistical/study design support,

specific facilities eg Clinical Research Unit• Must follow protocol exactly!

Considerations for Participate in Clinical Trials

• Do you have the required?– Patient population– Collaborators– Ancillary services– Equipment– Facilities (e.g. inpatient vs outpatient, pantry,

pharmacy, laboratory, specimen processing)– Trained, dedicated staff– Time (P.I. etc)– Other

• Is budget sufficient?• Duration of study?

Current Diabetes Grants at Upstate Medical University

NIH (n=6)

Industry-sponsored multicenter clinical trials (n=13)

Foundation (Type 1 Diabetes Exchange)

NYS Dept of Health Diabetes Center of Excellence (support community outreach, professional education and pilot projects e.g. school telemedicine)

Upstate Clinical Diabetes NIH Grants

Investigator-initiated: SHINE, DSPSupport salaries (% effort), other costsHighest indirect cost recoveryMost difficult to obtainGreatest intellectual/academic gratification

SBIR (Small Business Innovation Research)Partner with small business (new CGMS device)Joint federal and industry sponsorship

Multicenter trials: TODAY, TrialNetFunding: traditional (% effort) or accrual based

Diabetes Industry-Sponsored Multicenter Clinical Trials

• Usually little input from investigators re: design• Competitive enrollment• Funding: based on recruitment and retention• Indirect cost recovery: 30—50% of NIH• Need to meet enrollment and retention goals• May be discontinued at any time• Need significant number of subjects to break

even

So…Should We Participate?

Carefully consider all factors (budget, patient population, staffing, other support) before committing

Once committed, need to perform to expectations

ENJOY!

THANK YOU!