whether a medical strategy, treatment, or device is safe and effective for humans. • May show which medical approaches work best for certain illnesses or groups of people. • Produce the best data available for health care decision-making. National Institutes of Health, 2014
to consider: • Requirements for treatment administration/adherence assessment • Rate of occurrence of outcomes of interest • Participant health care needs • Cost of participant visit • Participant convenience/fatigue
possible (national guidelines, validated procedures from other studies) • Technology can improve quality: • Teleforms, eForms, vioFFQ • Digital Monitoring Devices • Systems for data management
• Data management often a consideration in scientific review for federally submitted grants • Data entry screens designed, when possible, to mirror paper data collection forms to minimize errors in data entry • Allows for use of validation checks (including determination of eligibility for trial) and data entry rules
visits, participant compliance • Can be customized or off-the-shelf • Enables centralized data management, especially valuable in large multi-center trials • Also allows for easy export of complete data sets to existing data warehouses or repositories and sharing of data between groups
for new collaborators to propose ancillary studies using samples, data, etc. already collected • Data, samples usually controlled by the study group not a data repository • ACCORD (diabetes) • Women’s Health Initiative (HRT) • ARIC (atherosclerosis) • NIH funding mechanisms may be available for ancillary studies
Consumption • 34% of adults over 20 years of age are overweight; additional 34% considered obese1 • Over 20 million people with diabetes, 50 million with pre-diabetes in the US2 • Estimated healthcare burden of diabetes: more than $197 billion in US in 20103 1NHANES 2007-2008 2CDC. National diabetes fact sheet:2007. 3Zhang P et al. Global healthcare expenditure on diabetes for 2010 and 2030. Diabetes Res Clin Pract 2010.
by NIH • 3,234 participants at 27 sites across the US • Participants randomly assigned to one of four interventions: • Intensive lifestyle intervention • Metformin • Placebo • Troglitazone (stopped early due to potential for liver toxicity)
on behavior modification, caloric restriction, and increased physical activity • Goal of 7% weight loss • Individual intervention delivered by professional case managers (RDs, RNs, and exercise physiologists) in a clinical setting • 4 mg/dL reduction in fasting glucose and 4.9% weight loss in lifestyle intervention group over 2.8 mean years of follow-up; 58% reduction in incidence of diabetes
• the process of applying discoveries generated during research in the laboratory, and in preclinical studies, to the development of trials and studies in humans • the study and facilitation of the application of research findings to the community, aimed at enhancing the adoption of best practices Bench research Clinical Research Community application
to be community-located, group-based, and peer-led • Comparison group • Number of participants • Measurement of weight AND glucose as outcomes • Length of follow-up
activity, and weight loss) administered through a community-based diabetes prevention program model will have a beneficial and clinically meaningful impact on glucose and insulin metabolism, and markers of the metabolic syndrome.
based translation of DPP • Testing a group-based behavioral lifestyle change strategy versus usual care • Fasting glucose is the primary outcome; assessment visits every six months • Intervention delivered through a local Diabetes Care Center with RDs and Community Health Workers (CHWs) Katula J. et al. Contemp Clin Trials. 2009 Sep 13.
BMI, waist circumference, and fasting blood glucose achieved during the first year • These results largely maintained in the second year as compared to the enhanced usual care condition • All achieved with lay community health workers and community based systems with high potential for dissemination
conducted in populations that were similar enough that we can even compare the results? • Results demonstrate that HELP PD was effective, but was it as effective as DPP? • Could HELP PD be a less expensive/more sustainable model than DPP for large-scale dissemination efforts?
from NIDDK Central Repository • Goals: • compare the baseline characteristics of HELP PD and DPP participants • contrast the magnitude of relative effects observed in the two trials • compare the cost effectiveness of the two interventions
granted for one-year time periods (can be renewed) • Three divisions: • Biosample Repository (Rockville, MD) • Genetics Repository (Piscataway, NJ) • Data Repository (Calverton, MD)
of the HELP PD Lifestyle Intervention for 2 years: $850 • Per capita direct medical cost of the DPP Lifestyle Intervention for 2 years: $2,631 • No other translations of DPP to date have done a comparable economic analysis Lawlor M. et al. Am J Prev Med. 2013 April. 44 (Suppl 4).
of cardiovascular disease (CVD) events at rates 2 to 4 times higher than people who do not have diabetes • Risk factors for CVD events: • Glycemia (blood sugar control) • Lipids (HDL and TG) • Systolic blood pressure
To test three complementary medical treatment strategies for type 2 diabetes to enhance options for reducing the very high rate of major CVD morbidity and mortality
2 diabetes who are at high risk for having a CVD event because of existing clinical or subclinical CVD or CVD risk factors: 1. Does a therapeutic strategy that targets a HbA1c of < 6.0% reduce the rate of CVD events more than a strategy that targets a HbA1c of 7.0% to 7.9% (with the expectation of achieving a median level of 7.5%) ? 2. In the context of good glycemic control, does a therapeutic strategy that uses a fibrate to raise HDL- C/lower triglyceride levels and uses a statin for treatment of LDL-C reduce the rate of CVD events compared to a strategy that only uses a statin for treatment of LDL-C? 3. In the context of good glycemic control, does a therapeutic strategy that targets a systolic blood pressure (SBP) of < 120 mm Hg reduce the rate of CVD events compared to a strategy that targets a SBP of < 140 mm Hg?
and Canada • 10,251 participants • Double 2 X 2 factorial design: participants randomized to either intensive or standard glycemia and: – Intensive or standard blood pressure – Blinded lipid trial
of a major cardiovascular disease event (nonfatal MI or stroke or cardiovascular death) • MIs, strokes, and deaths adjudicated by a committee masked to treatment assignment • Other outcomes include total mortality, microvascular outcomes, HRQL, and cost- effectiveness • Sub-studies on diabetic retinopathy (Eye) and cognitive decline (MIND)
to higher mortality rates in the intensive compared with the standard glycemia treatment strategies • Treatment for all participants was stopped on June 30, 2009 and transitioned to their own physicians • Participants still being followed in a non- treatment observational study (ACCORDION)
deaths in the intensive group – this was unexpected. There was a lower rate of non-fatal heart attacks in the intensive group. Overall fewer people died in both glycemia groups than expected. • There was more hypoglycemia, more weight gain & more adverse events in the intensive group – but these do not explain the higher risk of death. • Bottom line – the standard treatment was safer than the intensive treatment in ACCORD.
• Pooled analyses that have included data from the ACCORD datasets have provided important information on the safety of available diabetes and cardiovascular drugs • Ancillary studies have also provided significant information on the relationships between the management of diabetes and eye disease, cognitive decline, and bone health
A1c (blood sugar) in adults with diabetes • Meal replacements can be effective for weight loss in diabetics • Soy protein has demonstrated positive influences on body composition, metabolic risk factors, and lipids
meal replacement on glycemic control and metabolic effects in patients with type 2 diabetes over a period of 12 months in different populations • Primary outcome variable is HbA1c • Other outcomes include fasting blood glucose, fasting insulin level and insulin resistance (HOMA), risk factors for cardiovascular disease, and diabetes-related metabolic factors and complications.
to receive either group-based lifestyle intervention or meal replacement regimen • 2:1 randomization scheme • Participants followed for one year; seen every 2 months
that favor use of Rx interventions to reduce blood sugar levels and manage disease- paradigm shift to look at non-pharmacological treatments • Comparison with other trials that have targeted weight loss (LookAhead)
trials- can impact policy and future funding • Validate results across populations • Determine trends or patterns that may be undetectable in smaller samples- safety of drugs or interventions
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