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Especialização

Curso de Princípios e Prática em Pesquisa Clínica

Module 1: BASICS OF CLINICAL RESEARCH

Will provide students with a solid understanding of the basic issues in the design of clinical trials. Outline the fundamental principles of clinical trials including study population, selection of questions, basic study design, randomization process and blindness.

Lecture 1 – Steve Freedman

Introduction to Clinical Trials:
•Why perform a clinical?
•What is a clinical trial?
•Phase I studies; Phase II studies; Phase III/IV studies
•Introduction to ethics of Clinical Trials

Lecture 2 – Felipe Fregni

Selection of the Questions:
•Primary question
•Secondary question
•Adverse effects
•Ancillary questions
•Natural history
•Frequent errors

Lecture 3 - Michele Hacker

Study Population:
•Definition of study population
•Issues on generalization
•Trade-off: Internal validity vs. external generalizability
•Examples from landmark studies

Lecture 4 – David Wypij

Basic Study Design:
•Observational studies
•Randomized control studies
•Nonrandomized concurrent control studies
•Historical controls/databases
•Cross-over designs
•Factorial design
•Studies of equivalence
•Large clinical trials

Online only - Participant Adherence:
•Considerations before participant enrollment
•Maintaining good participant adherence
•Adherence monitoring
•Special population of patients: the “skeptic”, the “pleaser”, “the information seeker”, “the hopeless”, “the money seeker”, “the professional research participant”, “the high maintenance participant”, “the noncompliant participant”

Lecture 5 – Joseph Massaro

Study Blinding:
•Type of Trials – unblinded trials
•Single Blind Trials
•Double-blind Trials
•Triple blind trials
•Special problems in double blind studies – matching of drugs, coding of drugs and assessment of blindness

Lecture 6 – David Wypij

The Randomization Process:
• Fixed allocation randomization
• Simple randomization
• Blocked randomization
• Stratified randomization
• Adaptive Randomization Procedures (baseline adaptive randomization procedures)
• Mechanisms of randomization

Lecture 7 – Paul Conlin

Recruitment of Study Participants:
• Strategies and Sources
• Monitoring
• Problems
• Reasons for participation
• Reducing dropout rates

Module 2: STATISTICS

Will introduce the basic statistical methods used in clinical trials. How to select and apply appropriate statistical methods to analyze data from clinical trials and presenting, interpreting, and discussing the analyses clearly and concisely.
Online discussion: Special topics

Lecture 8 – Roger Davis

Statistics – Basics:
• Data classification
• Data distribution
• Descriptive methods for categorical data
• Descriptive methods for continuous data

Clinician perspective- Munir Boodhwani
Case study 1: Importance of data distribution for clinical studies
Case study 2: Data classification in clinical research

Lecture 9 – Jessica Paulus

Sample Size:
• Dichotomous response variables (two independent samples, paired dichotomous response)
• Sample size for continuous response variables (two independent samples)
• Sample size for equivalency of interventional studies
• Estimating sample size parameters
Practical example: How to calculate sample size for a grant application

Clinician perspective- Munir Boodhwani
Using statistics for the study design and hypothesis testing - a clinician perspective

Lecture 10 – Felipe Fregni

Statistical Tests:
(note: only basics of statistical tests (for normal distribution) will be covered)
• Estimation of parameters
• Comparison of population proportions (Chi-square test and Fisher’s exact test)
• Comparison of population means (Student t-test, ANOVA)
• Correlation (Person correlation coefficient)
• Linear Regression

Lecture 11 – Roger Davis

Survival Analysis:
• Estimation of the survival curve (Kaplan Meier estimate)
• Comparison of two survival curves
• Covariate adjusted analysis
• Use of survival analysis in clinical research

Lecture 12 – Felipe Fregni

Other Issues in Statistics I:
• Missing data
• Intention-to-treat analysis
• Covariate adjustment

Lecture 13 – Felipe Fregni

Other Issues in Statistics II:
• Subgroup analyses
• Comparison of multiple variables
• Meta-analysis of multiple studies

Module 3: PRACTICAL ASPECTS OF CLINICAL RESEARCH

The main features of clinical trials, including methodological and organizational considerations, and the principles of trial conduct and reporting will be discussed. Preparing the protocol for a trial, including data collection forms, logistical and budgetary issues are critical issues when designing a clinical trial.
Online discussion: Ethical and regulatory issues
- Distinguishing Clinical Research from Clinical Practice
- Equipoise and ethics of clinical research
- Ethical considerations in Randomized Clinical Trials
- Ethics of phase I research
- A moral theory of informed consent
- Children in Research
- Confidentiality
- Scandals and tragedies of research with human participants: Nuremberg, the Jewish chronic hospital and Tuskegee
- Special topic: ethical dilemmas in clinical research
- Special topic: Reasons patients refuse to participate in Clinical Trials
- Special topic: effective patient communication – challenges and recommendations
- Special topic: Psychiatric symptom-provoking studies: an ethical appraisal

Lecture 14 - Gretchen Brodnicki

Dean for Faculty Research and Integrity, Harvard Medical School Integrity in Research
• Disputes about authorship - when authorship fails
• The right or otherwise to publish data, patents and grant funding
• Scientific integrity and misconduct
• Publication practices
• Conflict of Interest

Lecture 15 - Frank Newby

The future of Clinical Data in Clinical Research
• Current Clinical Research Technology Applications
• State of Clinical Healthcare Information Technology
• Linking Healthcare and Clinical Research
– Standards - Value and Status
– Process Opportunities

Lecture 16 – Alan Zaslavsky

Design and Analysis of Surveys:
• Instrument design
• Design of survey administration
• Sample design
• Data collection
• Analysis
Online only – manuscript and grant writing
• Writing with clarity
• Writing in a non-native language
• Tables
• Figures
• Figures legends
• Rewriting the paper: reviewing style, spelling and second opinions

Lecture 17 – John Ferguson

Assessing risk and adverse effects in clinical research:
• Reporting of adverse effects
• Determinants of adverse effects (length of follow-up, frequency of events and individual susceptibility)
• Signal detection in pharmacovigilance – challenges and recommendations
• Safety risk management and clinical development
• Risk management principles for pharmacovigilance

Lecture 18 – Dennis LaCroix

The business of Clinical Research – negotiating contracts
• Budgeting for ancillary services
• Budgeting for personnel
• Budgeting for patient fees
• Budgeting for quality assurance
• Budgeting for overhead
• Negotiating the study budget (intellectual property, confidential information, publication and termination, and payment terms)

Lecture 19 – Caren Solomon

Deputy editor – New England Journal of Medicine
Manuscript submission:
• What makes a great paper?
• Tips to get your research published in high-impact journals
• What high impact journals are interested to publish?
• Is there bias in the review process?

Module 4: STUDY DESIGNS

Will introduce the main study designs used in clinical trials. Advantages and drawbacks of each design will be presented; including also the analytical methods and issues of different study designs.
Online discussion: International/global research
- HIV trials in developing countries
- Ethical Imperialism? Ethics in International Collaborative Clinical Research

Lecture 20 – James Ware

Non-inferiority designs:
• Superiority trials
• Non-inferiority designs
• Goals of NI designs
• Choosing the non-inferiority margin

Lecture 21 – Clarissa Valim

Observational Studies:
• Basic designs of observational studies
• Retrospective studies or cohort studies
• Sample size
• Bias and confounding
• Control of bias
• Control of the phenomenon of confounding

Lecture 22 – E. Francis Cook

Confounders in observational studies: using the method of propensity score:
• The issue of confounders in observational studies
• Methods to control for confounders
• The method of propensity score

Lecture 23 – Richard Kuntz

Other designs:
• Interim analyses
• Adaptive (flexible) design
• Studies with Medical Devices

Lecture 24 – Alexa B. Kimball

Vice Chair, Department of Dermatology, Massachusetts General Hospital
Associate Professor, Harvard Medical School
Experimental Design – Randomized Clinical Trials:
• Parallel-group design
• Stratified parallel group design
• Parallel group randomized block design
• Complete cross-over design
• Simultaneous treatments design
• Factorial design