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Children’s Health Research Summit

Children’s Health Research Summit. Session 4: Clinical Research & Clinical Trials. Sharon Murphy & Dan Hale, Co-Chairs. January 2008. Outline. - Definitions of Clinical/Translational Research. - Importance / Examples / Issues.

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Children’s Health Research Summit

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  1. Children’s Health Research Summit Session 4: Clinical Research & Clinical Trials Sharon Murphy & Dan Hale, Co-Chairs January 2008

  2. Outline - Definitions of Clinical/Translational Research - Importance / Examples / Issues - Survey of Active Pediatric Clinical Research Hematology/Oncology as an Example - Opportunities

  3. Definitions Clinical Research – Involves human subjects, typically in a face-to-face encounter Clinical Trials – Advance knowledge and promote discovery by following a specified protocol to determine safety, risks/benefits, and efficiency Today’s Trials are Tomorrow’s Cures

  4. Objectives of Clinical Trials Phase IThe earliest types of studies that are carried out in humans. They are typically done using small numbers of healthy subjects and are to investigate pharmacodynamics, pharmacokinetics and toxicity IICarried out in patients, usually to find the best dose of drug and to investigate safety IIIGenerally major trials aimed at conclusively demonstrating efficacy. They are sometimes called confirmatory trials and, in the context of pharmaceuticals, typically are the studies on which registration of a new product will be based IV Studies carried out after registration of a product. They are often for marketing purposes as well as to gain broader experience with using the new product

  5. Definition of Translational Research “ Translational research transforms scientific discoveries arising from laboratory, clinical, or population studies into clinical applications and practice.” Lab New Tools & New Applications Population Clinic

  6. The Translational Continuum Basic Science Discovery Early Translation Late Translation Adoption Dissemination • Promising molecule or gene target • Candidate protein biomarker • Basic epidemiologic finding • Partnerships and collaboration (academia, government, industry) • Intervention development • Phase I/II trials • Phase III trials • Regulatory approval • Partnerships • Production & commercialization • Phase IV trials – approval for additional uses • Health services research to support dissemination and adoption • (new drug, assay, device, behavioral, intervention, educational materials, training) • To community health providers • To patients and public • Adoption of advance by providers, patients, public • Payment mechanism(s) in place to enable adoption The #1 enemy of translational research is IP

  7. Clinical Trials Sponsors Locations NIH Academic Health Centers (AHCs)Pharmaceutical Companies DOD/VAFoundations 501(c)3 corporations Hospitals AHCs Community Clinics Doctors’ Offices Models Single or multi-institutional Consortia and cooperative groups Conduct of Clinical Trails is (largely) within AHCs which have the personnel, infrastructure, compliance

  8. Challenges to Clinical Research in AHCs Based on respondents to a survey of US Med Schools Pressure to See Patients Insufficient Clinical Revenues Recruiting Trained Researchers Availability of External Support Competition from CROs IRB Process Finding Research Subjects 93% 89% 75% 72% 48% 38% 37% Campbell, E.G., et al JAMA 286: 800-806, 2001

  9. Difficulties in Recruiting Children to Clinical Trials • Primary treating doctor’s reluctance to enroll patients • Lack of awareness of trials for accrual • Parent’s fears of perceived risks and mistrust of researchers • Sociodemographic factors • The child’s health status: • The more severe the illness, the greater the willingness to participate • The informed consent process • Inconvenient/unpleasant aspects of trial design Caldwell, P.H.Y., Murphy SB, et al Lancet 364:803-11, 2004

  10. Childhood ALL: Progressive ImprovementIn event-free Survival 1950-2000 100 2000 80 1985 60 1965 40 20 1950 0 0 1 2 3 4 5 Years From Diagnosis

  11. * p < 0.0001 by stratified log-rank test ** PHR, proportional hazards ratio, by Cox proportional hazards regression, adjusted for age, WBC, ploidy, gender, era of treatment, or size of treating institution. Conclusion: AA and Hispanic children with ALL have mortality rates 42% and 33% higher respectively than Whites; differences not explained by presenting prognostic factors.

  12. Pediatric AIDS/Lymphoma Network Cases Control Comparisons HIV + NHL+ HIV + NHL- Demographic Clinical Molecular Viral Phase I+II Trails - Response - Toxicity HIV - NHL+

  13. Survey of Active Clinical Research Protocols in the Division of Hematology/Oncology, Department of Pediatrics, UTHSCSA (2007) • Total Trials = 113, including • - 70 open Children’s Oncology Group (POG) protocols • - 10 transplant trials (PBMC, TTI) • - 7 industry sponsored, plus various registries, tissue/sera collection, etc. • Enrollments: 79 total, 57 oncology, 22 hematology • larger numbers on study, active, in follow-up

  14. Opportunities Further Develop/Expand the Childhood Cancer Survivors Program Relate research in long-term survivors of childhood cancer to long-term outcomes of other childhood illnesses: CF, genetic disorders, heart disease, transplants, diabetes, etc.

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