به نام ايزد يکتا Clinical Trial Design Dr. Khalili 1  The common types  Advantages and limitations  Usual applications.

Slides:

Advertisements

Similar presentations

Agency for Healthcare Research and Quality (AHRQ)

Advertisements

Randomized Controlled Trial

Experimental Design True Experimental Designs n Random assignment n Two comparison groups n Controls threats to internal validity n Strongest evidence.

Research Study Designs

Incomplete Block Designs. Randomized Block Design We want to compare t treatments Group the N = bt experimental units into b homogeneous blocks of size.

Designing Clinical Research Studies An overview S.F. O’Brien.

Study Designs in Epidemiologic

Introduction to Epidemiology

Bayesian Adaptive Methods

Analysis & Expressing Resultd in Clinical Trials Dr. Khalili.

Regression Discontinuity Design William Shadish University of California, Merced.

Design and Analysis of Clinical Study 12. Randomized Clinical Trials Dr. Tuan V. Nguyen Garvan Institute of Medical Research Sydney, Australia.

Clinical Trials Medical Interventions

Bios 101 Lecture 3: Experimental Designs

SMALL-N DESIGNS u Experiments conducted on only one or a few subjects u External validity is a problem u Internal validity is often very good - no individual.

Journal Club Alcohol and Health: Current Evidence July-August 2006.

25/5/2009 Dr. Salwa Tayel 1. 25/5/2009 Dr. Salwa Tayel2 Associate Professor Family and Community Medicine Department King Saud University By.

Chapter 12: Single-Subject Designs An alternative to experimental designs Purpose: To draw conclusions about the effects of treatment based on the responses.

Clinical trial The Way We Make Progress Against Disease Prof. Ashry Gad Mohamed Prof. of Epidemiology College of Medicine & KKUH.

1 Section IV Study Designs for Investigating Adaptive Treatment Strategies Murphy.

Sample Size Determination

Regression Approach To ANOVA

RANDOMIZED CLINICAL TRIALS. What is a randomized clinical trial?  Scientific investigations: examine and evaluate the safety and efficacy of new drugs.

Professor of Epidemiology and Biostatistics

Common Designs for Controlled Clinical Trials A.Parallel Group Trials 1.Simplest example - 2 groups, no stratification 2.Stratified design 3.Matched pairs.

Future Research Directions Improving the Chain of Recovery for Acute Stroke in Your Community.

CME Disclosure Statement The North Shore LIJ Health System adheres to the ACCME's new Standards for Commercial Support. Any individuals in a position.

Intervention Studies Principles of Epidemiology Lecture 10 Dona Schneider, PhD, MPH, FACE.

Chapter 11 Research Methods in Behavior Modification.

Reading Scientific Papers Shimae Soheilipour

Testing People Scientifically.  Clinical trials are research studies in which people help doctors and researchers find ways to improve health care. Each.

CLINICAL TRIALS.

Lecture 16 (Oct 28, 2004)1 Lecture 16: Introduction to the randomized trial Introduction to intervention studies The research question: Efficacy vs effectiveness.

CLINICAL TRIAL. Clinical Trials Strengths: – Best measure of causal relationship – Best design for controlling bias – Can measure multiple outcomes.

Introduction to Clinical Trials

Study Design. Study Designs Descriptive Studies Record events, observations or activities,documentaries No comparison group or intervention Describe.

1 Experimental Study Designs Dr. Birgit Greiner Dep. of Epidemiology and Public Health.

Gastroenterology Volume 142, Issue 4, April 2012, Pages 790–795 Tom W. Chu.

Department of Pharmacology L. M. College of Pharmacy, Ahmedabad-09.

Study design P.Olliaro Nov04. Study designs: observational vs. experimental studies What happened?  Case-control study What’s happening?  Cross-sectional.

Designs for Clinical Trials. Chapter 5 Reading Instructions 5.1: Introduction 5.2: Parallel Group Designs (read) 5.3: Cluster Randomized Designs (less.

Applied Epidemiology Sharla Smith. Discussion Assignments How to complete a discussion assignment –Read the chapters –Evaluate the question –Be very specific.

Responsibilities of state, community and private sector on research ethic Policy : looking back on recent CCR5 antagonists Hugues Fischer, TRT-5 (French.

Study Designs in Epidemiologic

Some terms Parametric data assumptions(more rigorous, so can make a better judgment) – Randomly drawn samples from normally distributed population – Homogenous.

Placebo-Controls in Short-Term Clinical Trials of Hypertension Sana Al-Khatib, MD, MHS Assistant Professor of Medicine Division of Cardiology Duke University.

Successful Concepts Study Rationale Literature Review Study Design Rationale for Intervention Eligibility Criteria Endpoint Measurement Tools.

1 Statistics in Drug Development Mark Rothmann, Ph. D.* Division of Biometrics I Food and Drug Administration * The views expressed here are those of the.

Welcome to Workshop #5: Accelerated Approval (AA) in Rare Diseases: Review of a White Paper Proposal Emil D. Kakkis, M.D., Ph.D. President and Founder.

Chapter 4 – Research Methods Different methods to answer different questions A.Does one factor cause another? The Scientific Method - obtain reliable information.

EXPERIMENTAL EPIDEMIOLOGY

Causal relationships, bias, and research designs Professor Anthony DiGirolamo.

August 20, 2003FDA Antiviral Drugs Advisory Committee Meeting 1 Statistical Considerations for Topical Microbicide Phase 2 and 3 Trial Designs: A Regulatory.

Some Design Issues in Microbicide Trials August 20, 2003 Thomas R. Fleming, Ph.D. Professor and Chair of Biostatistics University of Washington FDA Antiviral.

1 Study Design Issues and Considerations in HUS Trials Yan Wang, Ph.D. Statistical Reviewer Division of Biometrics IV OB/OTS/CDER/FDA April 12, 2007.

Reversal Designs. Overview One of the most important designs you can use Can be used in a variety of settings Can be very powerful in detecting changes.

Is the conscientious explicit and judicious use of current best evidence in making decision about the care of the individual patient (Dr. David Sackett)

Medical Research and Clinical Trials Rels 300 / Nurs October 2015.

Regulatory Considerations for Endpoints Ann T. Farrell, M.D. FDA/CDER/DODP.

Clinical Trials - PHASE II. Introduction  Important part of drug discovery process  Why important??  Therapeutic exploratory trial  First time in.

CHS502 Unit IV-1 Randomized Blinded Trial Ahmed A. Mirza, PhD.

CLINICAL PROTOCOL DEVELOPMENT

Clinical Research: Part 1

Why experimental design are important

Observational Studies and Experiments

Research Methods 3. Experimental Research.

11 Single-Case Research Designs.

انواع مطالعات توصيفي تحليلي مشاهده اي مداخله اي گزارش مورد گزارش موارد

Data Monitoring committees and adaptive decision-making

Presentation transcript:

به نام ايزد يکتا Clinical Trial Design Dr. Khalili 1  The common types  Advantages and limitations  Usual applications

Dr. Khalili2  Clinical Trial (on patients) vs.  Field Trial (on healthy people)  Community Trial (on communities)

Dr. Khalili3

4 After Before Intervention In phase II clinical trials OR in serious diseases such as cancers

Dr. Khalili5

6

7

8

9

10

Dr. Khalili11

Dr. Khalili12 Notice: the standard methods for sample size calculation and data analysis considering subject as analysis unit are not appropriate here.

Dr. Khalili13 Be relax for few minutes

Dr. Khalili14

Dr. Khalili15 Advantages:  Allows within-patients comparisons of treatments  Removes interpatient variability  Provides the best unbiased estimates for the differences between treatments  Decreases number of patients needed

Dr. Khalili16 Limitations: It is applicable where:  Objective measures for efficacy and safety are obtained  Chronic and relatively stable disease  Prophylactic drugs with relatively short half life  Relatively short treatment periods  Baseline and washout periods are feasible It increases the duration of the study Its analysis is not straightforward:  The paired design  The period and carry-over effects The effect of loss to follow-up

Dr. Khalili17 Sequence (Period) Effect: The order in which the therapies are given may elicit psychological responses. Patients may react differently to the first therapy given in a study as a result of the enthusiasm may diminish over time. Carryover Effect: If a subject is changed from therapy A to therapy B and observed under each therapy, the observations under therapy B will be valid only if there is no residual carryover from therapy A. There must be enough of a washout period to be sure none of therapy A or its effect remains.

Dr. Khalili18

Dr. Khalili19 To resolve the problom:

Two applications: 1.Quantifying the interaction between the two treatments 2.Opportunistic situations

Dr. Khalili21 A Full (a+1) * (b+1) Factorial Design for Combination Therapy of Two Components at a and b Dose Levels

Dr. Khalili22 Designs with ethical considerations Adaptive Randomization Zelen design Variations of placebo-controlled trials: Add-on design Replacement design Randomized Withdrawal design Sequential analysis

Dr. Khalili23 And more complex DESIGNS …