1 / 22

Accelerating the Dissemination and Translation of Clinical Research into Practice

Accelerating the Dissemination and Translation of Clinical Research into Practice. The View from NHLBI Susan B. Shurin, MD Deputy Director, NHLBI May 9, 2008. Disclosures. Accelerating the Dissemination and Translation of Clinical Research into Practice.

rosine
Download Presentation

Accelerating the Dissemination and Translation of Clinical Research into Practice

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Accelerating the Dissemination and Translation of Clinical Research into Practice The View from NHLBI Susan B. Shurin, MD Deputy Director, NHLBI May 9, 2008

  2. Disclosures Accelerating the Dissemination and Translation of Clinical Research into Practice The Following Faculty have No Relevant Financial Relationships with Commercial Interests Dr. Susan Shurin Podium Session

  3. Three Translational Roadblocks on the Way Toward Improved Public Health Funding Source National Institutes of Health Others Implementation Whose Responsibility? Funding Source Whose Responsibility? Translation Into Clinical Practice Translation Into Humans Health Services Research Effectiveness Research Outcomes Research Comparative Research Translational Research Improved Public Health Basic Science Research Clinical Trials 2nd block is integrally tied into Funding of health care delivery Modified from Crowley, W. F. et al. JAMA 2004;291:1120-1126.

  4. Contributions to Change in Life Expectancy, U.S., 1970-2000 Cardiovascular Disease Increase Due to CVD = 3.9 Years Perinatal Disease CHD Stroke Other CVD Injuries Cancer Net Increase = 6.0 Years COPD HIV/AIDS Other causes -1 0 1 2 3 4 5 Change in Life Expectancy (Years)

  5. Framingham Heart Study Downtown Framingham, MA (circa 1960) • Risk Factors for Heart Attack and Stroke • High blood pressure • High cholesterol • Cigarette smoking • Diabetes mellitus • Parental or sibling history • Obesity

  6. Age-Adjusted Death Rates for Coronary Heart Disease, U.S., 1950-2004 Deaths/100,000 Population 600 Risk Factors defined 1961 Risk Factors Primary and Secondary Prevention 500 Physical Intervention CASS 1983 TIMI 1985 400 NHBPEP 1972 300 HDFP 1979 200 BHAT 1981 NCEP 1985 100 ALLHAT 2002 CPPT 1984 0 1960 1980 1950 1970 1990 2010 2000 Year

  7. Rates of pharmacologic therapy at admission and discharge. p=0.15 p<0.001 p<0.001 p<0.001

  8. Rates of procedures in patients who fulfilled AHCPR Guidelines for cath and CABG p=0.035 p<0.001 p<0.001 p=0.082 p=0.040 p=0.002

  9. The Dilemma of Sickle Cell Disease • The first (>60 years), and possibly the best understood, molecular disease • Several billion $US invested in research • Vastly improved survival rates • Now a chronic disease, debilitating to many patients • Poor access to care, implementation of research discoveries to alleviate burdens

  10. Increases in Life Expectancy of Patients with Sickle Cell Anemia MSH 50 PROPS I 40 National Sickle Cell Act 30 Life Expectancy (Years) 20 STOP 10 0 1900 1910 1920 1930 1940 1950 1960 1970 1980 1990 2000 Year

  11. Cumulative Mortality in Patients With Sickle Cell Anemia in the MSH and in Follow-up Steinberg, M. H. et al. JAMA 2003;289:1645-1651.

  12. NIH Consensus Conference Feb 25-26, 2008

  13. Efficacy of hydroxyurea • Strong evidence is found in support of the efficacy of hydroxyurea use in adults. • Variable evidence is available in the preadolescent population. • No well-designed clinical trial evidence in infants is available. • Although the evidence for efficacy of hydroxyurea treatment for children is not as strong, the emerging data are supportive.

  14. Effectiveness of hydroxyurea • One problem in determining effectiveness is the lack of a precise estimate of the number of people who have sickle cell disease in the United States and the number of people actually receiving hydroxyurea. • Most published studies have strict entry criteria, meaning some patients with co-morbidities who might benefit have not been assessed. • Overall, data regarding effectiveness are very limited.

  15. Harms of hydroxyurea • Short-term, dose-related, usually temporary, and reversible effects • decrease in white blood cell count, decrease in platelet count, • decreased sperm counts or increased sperm abnormalities in men • dryness and darkening of the skin and nails. • No high-quality evidence supports increased incidence of cancer or birth defects • Moderate evidence shows that hydroxyurea does not affect growth rate in patients who have sickle cell disease. • The data currently available are reassuring re: risks of both short- and long-term harms of hydroxyurea treatment. • The risks of hydroxyurea in adults are acceptable compared to the risks of untreated sickle cell disease.

  16. Barriers to hydroxyurea treatment • Only three studies specifically address barriers, and none addresses hydroxyurea interventions. • Patient and parent/family/caregiver barriers include: • Fears about cancer, birth defects, infertility, and uncertainty of other potential long-term risks; • Lack of knowledge of hydroxyurea as a therapeutic option for sickle cell disease

  17. Provider barriers • Limited number of physicians who have expertise in the use of hydroxyurea for sickle cell disease; • Provider bias and negative attitudes toward patients who have sickle cell disease and their treatment; • Lack of clarity in hydroxyurea treatment regimens and • Undertreatment.

  18. System barriers • Financing: lack of insurance and coverage; • Geographic isolation, • Limited access to comprehensive care models; • Problems in transitioning from pediatric to adult care.

  19. Future research needs • A comprehensive registry of all patients who have sickle cell disease that will be followed prospectively. • Studies to better define the mechanism of action of hydroxyurea, as well as optimal dosing, titration, and monitoring. • Studies that identify factors that predict clinical hydroxyurea response or nonresponse. • Effectiveness studies considering when to begin use of hydroxyurea to prevent or treat complications of sickle cell disease and how long to continue the therapy. • Evaluation of the utility and cost-effectiveness of the comprehensive care and medical home models for the delivery of hydroxyurea treatment

  20. Major barriers for improving care: SCD • No one makes money • Commitment to ensuring quality of care is individual, not societal • Access to care is often difficult, and quality variable • To primary care providers, this is a rare genetic disease • Survival is vastly improved • Burden of disease is vastly increased

  21. Added Barriers for Sickle Cell Patients • The disease affects primarily persons many of whom are struggling with other challenges, including • Lower socioeconomic status • Historically disadvantaged racial and ethnic group • Educational disadvantage • The chronic disease affects physical energy, and may cause neurocognitive damage

  22. Needed • Both care provision and research need to extend into the community • Lessons from other settings: patient advocacy groups must be part of the process • Results of research need to be clear to patients, families and primary care providers • Patients should be able to access both primary and tertiary care providers, with provision of a medical home

More Related