Clinical trials are used to scientifically evaluate new medical therapies. There are two main types of clinical trials: hypothesis-generating trials that explore potential treatments but cannot rigorously test hypotheses, and hypothesis-testing randomized controlled double-blinded trials, which are considered the gold standard. These trials help reduce bias by randomly assigning participants to treatment groups, blinding participants and researchers, and using intention-to-treat analysis for all participants. Tissue engineering presents challenges for clinical trials due to the need for long-term outcome data.
Clinical trials are used to scientifically evaluate new medical therapies. There are two main types of clinical trials: hypothesis-generating trials that explore potential treatments but cannot rigorously test hypotheses, and hypothesis-testing randomized controlled double-blinded trials, which are considered the gold standard. These trials help reduce bias by randomly assigning participants to treatment groups, blinding participants and researchers, and using intention-to-treat analysis for all participants. Tissue engineering presents challenges for clinical trials due to the need for long-term outcome data.
Clinical trials are used to scientifically evaluate new medical therapies. There are two main types of clinical trials: hypothesis-generating trials that explore potential treatments but cannot rigorously test hypotheses, and hypothesis-testing randomized controlled double-blinded trials, which are considered the gold standard. These trials help reduce bias by randomly assigning participants to treatment groups, blinding participants and researchers, and using intention-to-treat analysis for all participants. Tissue engineering presents challenges for clinical trials due to the need for long-term outcome data.
Clinical Trials are the scientific basis for therapeutics
What are the types of trials?
HYPOTHESIS-GENERATING are trials that cannot rigorously test a hypothesis, give you testable hypotheses for interventions and their outcomes, include: case studies - trying a novel treatment method on a few patients/one), case control study - trying a novel treatment method on a small group of patients with a control group that is age matched, gender matched, and ethnicity matched cohort study - follow two groups of people in the same demographic, one of whom has a certain condition, then look at outcomes (not as valid bc of confounding factors) confounding factors - separate factors that can systematically affect results prospective vs retrospective HYPOTHESIS-TESTING TRIALS/ GOLD STANDARD - randomized, controlled, double-blinded clinical trials randomized - participants randomly assigned control/active participation controlled - receives current standard of care, not necessarily just placebos (on a case-by-case basis) double-blinded - neither participants or caregivers/study evaluators know who is in which group (surgeries CANNOT be double-blinded) What are the sources of bias? Four types of bias: Selection bias - bias in the selection of patients for the intervention; combat through randomization and blinding Performance bias - bias in terms of the overall treatment a patient receives whether they be in the control group or not, combat through blinding and randomization Detection bias - bias in the detection of an effect, bias in the measurement of a clinical endpoint by the caregiver; combat through blinding and randomization Attrition bias - bias with regard to trial dropouts/non compliance; combat through intention-to-treat What is the gold standard? How might this present issues for tissue engineering?
HYPOTHESIS: If I perform an intervention then I will see difference in outcome/clinical
endpoint. If I implant cardiac stem cells in patients with heart failure, then I will see fewer heart attacks in this group versus the control group receiving the standard of care Clinical endpoints: blood pressure blood glucose levels enzyme levels cholesterol levels advantage: short term results are visible, BUT are not the most clinically meaningful number of major coronary events exercise tolerance visits to hospital overall survival (YOU WILL NEVER BE WRONG WITH THIS ANSWER) most clinically meaningful BUT results take longer Define intervention Define target patient population inclusion criteria exclusion criteria meant to preserve safety Define clinical endpoints