Basics of Clinical Research PDF

Basics of Clinical Research Dr. Ahmed Atia  What is Clinical Research/ Human Subject Research? “A systematic investigation, including research development, testing and evaluation, designed to develop or contribute to generalizable knowledge.” “Any activity...

Basics of Clinical Research Dr. Ahmed Atia  What is Clinical Research/ Human Subject Research? “A systematic investigation, including research development, testing and evaluation, designed to develop or contribute to generalizable knowledge.” “Any activity designed to test a hypothesis, permit conclusions to be drawn and thereby to develop or contribute to generalizable knowledge.  Good Clinical Practices Known as GCP’s They are rules/regulations for the conduct of research Sometimes called “Good Research Practices” Standard for the design, conduct, performance, monitoring, analyses and reporting of research Even though established for drug studies, they dictate appropriate conduct for all research  Types of Study Designs 1. Observational Designs 2. Experimental Designs – interventional studies  Types of Observational Studies Cohort Studies A group of subjects followed over time Purpose: defining the incidence and investigating potential causes of a condition (incidence) Can be prospective – investigator chooses a sample group and measures characteristics in each subject over a period of time that might predict outcomes Can be retrospective – same as prospective, except all data collection and follow-up has happened in the past; only possible if adequate data is available  Types of Observational Studies Cross-Sectional Studies Similar to cohort studies except all the measurements are made at one time point with no follow-up Purpose: describing variables and their distribution patterns (prevalence) Strength – fast and inexpensive since there is no follow-up or waiting time for outcome  Case-Control Studies Two groups of people examined for the same outcome Group 1 – “cases” or a population of people with a certain disease Group 2 – “controls” or a population of people without that same disease Purpose: compare prevalence of risk factor(s) in subjects with the disease (cases) versus subjects without the disease (controls)  Types of Study Designs 1. Observational Designs 2. Experimental Designs – interventional studies  Experimental Studies These studies evaluate the effects of an intervention Types of interventions: Behavior modification (eg. a walking program to improve weight loss) Drug (eg. a new investigational drug or studying a drug for off- label use – subject to FDA regulations) Device (eg. a new investigational – subject to FDA regulations)  Phases of Experimental Studies Phase I: Unblinded studies of a small number of healthy volunteers to test safety of treatment (can sometimes use people with the disease) Phase II: Randomized studies of relatively small number of people with the disease to test dose ranges and/or efficacy of treatment Phase III: Randomized studies of large number of people with the disease to test efficacy of treatment Phase IV: Large experimental studies or observational studies conducted after treatment has been approved by the FDA to assess performance of treatment (called Post-Market Studies) Steps in Launching a Research Project Idea Detailed Literature Review Study Question Detailed Study Protocol Funding (if needed) Approval from Physicians, IRB, Hospital Implementation  Stating the Question/Hypothesis A research idea (for an observational or intervention study) should be structured into a well-built clinical research question or hypothesis with the following PICO components: Population of interest Intervention to be tested Comparison strategy Outcome(s)  Study Questions The type of question determines the study design that should be used to answer the question.  Pro to c o l Writing C o m p o ne nts of a C linic al Re se arc h Pro to c o l  Final Preparation Present proposal to division/department for practical advice and approval of procedures that impact patient management should be done earlier in the process (before finalizing protocol) if problems are anticipated  IRB/CPHS Approval Approval required before enrollment begins Required for all research (retrospective or prospective, observational or interventional) any activity involving human subjects that is designed to yield generalizable knowledge END SHOW