Medicine Research Slides

7/25/2019 Medicine Research Slides

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Writing aWriting a

Research ProtocolResearch Protocol

Henry Parkman, MDHenry Parkman, MDGI SectionGI Section

Department of MedicineDepartment of Medicine


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Goals of PresentationGoals of Presentation

Importance of writing a research protocolImportance of writing a research protocol

when conducting a research studywhen conducting a research study

Types of research protocols with differentTypes of research protocols with different

formatsformats

Suggestions for writing a research protocolSuggestions for writing a research protocol

Example of a research protocolExample of a research protocol


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What is a Research ProtocolWhat is a Research Protocol

A research protocol is a document that describesA research protocol is a document that describes

the background, rationale, objectives, design,the background, rationale, objectives, design,methodology, statistical evaluation of the data,methodology, statistical evaluation of the data,

and organization of a clinical research project.and organization of a clinical research project.


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Need for Written Research ProtocolNeed for Written Research Protocol

forces the investigators to clarify their thoughts and to thinkforces the investigators to clarify their thoughts and to think

about all aspects of the studyabout all aspects of the studystudystudy

conduct, analysis ofconduct, analysis of

the datathe data

guide for a team working on researchguide for a team working on research

help ensure study ishelp ensure study is

performed similarly by different people over timeperformed similarly by different people over time

essential if study involves research on human subjects or onessential if study involves research on human subjects or on

experimental animals, in order to get institutionexperimental animals, in order to get institutions ethicals ethical

approvalapproval

essential component of a research proposal submitted foressential component of a research proposal submitted for

fundingfunding

use to start writing a manuscript when study completeduse to start writing a manuscript when study completed


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Developing a Clinical Research ProtocolDeveloping a Clinical Research Protocol -- 11

Start with a good questionStart with a good question --

one for which theone for which the

answer matters either to other researchers in theanswer matters either to other researchers in the

field, practicing clinicians, or patientsfield, practicing clinicians, or patients

Convert the question to a hypothesis by assertingConvert the question to a hypothesis by asserting

a positiona position

Construct the protocolConstruct the protocol

Generate measures of exposure (treatments)Generate measures of exposure (treatments)and outcomeand outcome

Test the hypothesis by making a comparison inTest the hypothesis by making a comparison in

two or more groupstwo or more groups


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Developing a Clinical Research ProtocolDeveloping a Clinical Research Protocol -- 22

Develop a plan for data collection andDevelop a plan for data collection andmanagementmanagement

Determine the statistical methods forDetermine the statistical methods foranalysis, consult with statisticiananalysis, consult with statistician

Estimate the magnitude of expected differenceEstimate the magnitude of expected differencebetween the two groups, as a basis for determiningbetween the two groups, as a basis for determiningsample size (power calculation)sample size (power calculation)

Assess feasibility of the study:Assess feasibility of the study:

Can enough people be obtained for the study?Can enough people be obtained for the study?

Can the outcome events be observed andCan the outcome events be observed andsuitably analyzed?suitably analyzed?


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Research Protocol FormatResearch Protocol Format Project titleProject title Project summaryProject summary

Project descriptionProject description

RationaleRationale

ObjectivesObjectives

Subjects to study: Inclusion/Exclusion criteriaSubjects to study: Inclusion/Exclusion criteria

MethodologyMethodology

Data management and analysisData management and analysis

Sample size needed for the studySample size needed for the study

Ethical considerationsEthical considerations

Recruitment plans, CompensationRecruitment plans, Compensation

Risks, Benefits of the studyRisks, Benefits of the study

Informed Consent, Approvals (IRB)Informed Consent, Approvals (IRB) ReferencesReferences


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Project TitleProject Title

Should be descriptive of the study, butShould be descriptive of the study, but

conciseconcise

May need to be revised after completion ofMay need to be revised after completion ofthe writing of the protocol to reflect morethe writing of the protocol to reflect more

closely the sense of the studyclosely the sense of the study


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Project Summary (Abstract)Project Summary (Abstract)

The abstract should concisely summarizeThe abstract should concisely summarize

the elements of the protocolthe elements of the protocol

The summary should stand on its own, andThe summary should stand on its own, andnot refer the reader to points in the projectnot refer the reader to points in the project

descriptiondescription

Although present first in protocol, writtenAlthough present first in protocol, written

last, being taken from the subsequent partslast, being taken from the subsequent parts


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Project Rationale (Background and Significance)Project Rationale (Background and Significance)

Sets the stage for why the research project should beSets the stage for why the research project should be

done. Cite appropriate references.done. Cite appropriate references.

General Format:General Format:

The condition to be studiedThe condition to be studied

Treatments currently available, note gaps presentTreatments currently available, note gaps present

Specific treatment to be studiedSpecific treatment to be studied

Preliminary data for treatment in the condition to bePreliminary data for treatment in the condition to bestudiedstudied

Purpose of the studyPurpose of the study


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Research Objectives (Aims)Research Objectives (Aims)

Briefly describe the broad objectives and specific aims of thisBriefly describe the broad objectives and specific aims of thisstudy, including the hypotheses. Objectives should be simple,study, including the hypotheses. Objectives should be simple,

specific, and stated in advance of performing the research.specific, and stated in advance of performing the research.

E.g., To determineE.g., To determine ..

After statement of the primary objective, several secondaryAfter statement of the primary objective, several secondary

objectives may be mentionedobjectives may be mentioned

List research hypothesesList research hypotheses --

written as statements of what youwritten as statements of what you

expectexpect

Types of Specific AimsTypes of Specific Aims

Hypothesis aim, based on scientific hypothesis to be testedHypothesis aim, based on scientific hypothesis to be tested

Exploratory aim seeks to obtain useful data about a specificExploratory aim seeks to obtain useful data about a specific

questionquestion


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Study Title:Study Title:

Use ofUse of SancusoSancuso

in patients within patients withnausea and/or vomiting fromnausea and/or vomiting from gastroparesisgastroparesis

The hypotheses to be tested include:The hypotheses to be tested include:

1)1) SancusoSancuso

improves symptoms ofimproves symptoms of gastroparesisgastroparesis

2) Symptoms of nausea and vomiting improve to a2) Symptoms of nausea and vomiting improve to agreater degree than abdominal paingreater degree than abdominal pain

3) The beneficial response of3) The beneficial response of SancusoSancuso

is seen inis seen in

both diabetic and idiopathicboth diabetic and idiopathic gastroparesisgastroparesis

4) The symptom reduction occurs on the second4) The symptom reduction occurs on the second

day and continues throughout the treatmentday and continues throughout the treatment

coursecourse

HypothesisHypothesis --

ExampleExample


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The aim of this study is determine the efficacy ofThe aim of this study is determine the efficacy of SancusoSancuso

patchpatchin improving symptoms of nausea and vomiting in patients within improving symptoms of nausea and vomiting in patients with

gastroparesisgastroparesis..

The specific objectives of this study are:The specific objectives of this study are:

1) To determine the treatment response of1) To determine the treatment response of SancusoSancuso

inin

gastropareticgastroparetic

patients with nausea and/or vomitingpatients with nausea and/or vomiting --

TheThe

percent of patients treated withpercent of patients treated with SancusoSancuso

that have improvedthat have improved

symptoms of nausea, vomiting.symptoms of nausea, vomiting.2) To determine which specific symptoms of2) To determine which specific symptoms of gastroparesisgastroparesis

improveimprove

nausea, vomiting, early satiety, abdominalnausea, vomiting, early satiety, abdominal

distension, abdominal paindistension, abdominal pain3) To determine symptomatic responses in both diabetic and3) To determine symptomatic responses in both diabetic and

idiopathicidiopathic gastroparesisgastroparesis

4) To determine the time course of symptom improvement with4) To determine the time course of symptom improvement with

SancusoSancuso

for symptoms offor symptoms of gastroparesisgastroparesis

Study AimsStudy Aims --

ExampleExample


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Research Design and MethodologyResearch Design and Methodology

Research DesignResearch Design

The choice of the design should be explainedThe choice of the design should be explained --

how it will address studyhow it will address studyobjectivesobjectives

The research design, methods and procedures should help answer yThe research design, methods and procedures should help answer yourour

researchresearch question(squestion(s) as written in your study objectives and aims) as written in your study objectives and aims

Research Subjects or ParticipantsResearch Subjects or Participants

Inclusion/exclusion criteriaInclusion/exclusion criteria

In intervention studies, how will subjects be allocated to the tIn intervention studies, how will subjects be allocated to the treatment andreatment and

comparison groups?comparison groups?

What are the criteria for discontinuation?What are the criteria for discontinuation?

InterventionsInterventions

If an intervention is performed, a description is given of the dIf an intervention is performed, a description is given of the drugs orrugs or

devices to be used, and whether they are already commercially avdevices to be used, and whether they are already commercially available,ailable,or in phases of developmentor in phases of development

ObservationsObservations

What observations will be made, how they will be made, and howWhat observations will be made, how they will be made, and how

frequently will they be madefrequently will they be made

Sample size neededSample size needed


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Data Management and AnalysisData Management and Analysis

Describe the statistical methods to be used for each studyDescribe the statistical methods to be used for each studyobjectiveobjective

The protocol should provide information on what types of dataThe protocol should provide information on what types of datawill be obtained and how the data will be managedwill be obtained and how the data will be managed

Describe plans for data and statistical analyses, including theDescribe plans for data and statistical analyses, including thetiming of interim and subgroup analyses, as appropriatetiming of interim and subgroup analyses, as appropriate

Describe which subjects will be included in the data analysisDescribe which subjects will be included in the data analysis

for each of your study objectives (e.g., all participants, allfor each of your study objectives (e.g., all participants, all

participants dosed, all evaluable participants, etc.)participants dosed, all evaluable participants, etc.)

What criteria will be used to stop the study, if necessary?What criteria will be used to stop the study, if necessary?


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Sample SizeSample Size

The protocol should provide information and justification on theThe protocol should provide information and justification on the

sample sizesample size

the number of patients needed to be studiedthe number of patients needed to be studied

A larger sample size than needed to test the research hypothesisA larger sample size than needed to test the research hypothesis

increasesincreasesthe cost and duration of the study and will be unethical if it ethe cost and duration of the study and will be unethical if it exposes humanxposes human

subjects to any potential unnecessary risk without additional besubjects to any potential unnecessary risk without additional benefitnefit

A smaller sample size than needed can also be unethical if it exA smaller sample size than needed can also be unethical if it exposes humanposes humansubjects to risk with no benefit to scientific knowledgesubjects to risk with no benefit to scientific knowledge

Calculation of sample size has been made easy by computerCalculation of sample size has been made easy by computer

software programs. The principles underlying the estimation of tsoftware programs. The principles underlying the estimation of thehesample size should be well understoodsample size should be well understood


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Ethical considerationsEthical considerations

Risks, Benefits of the studyRisks, Benefits of the study

Recruitment plansRecruitment plans

Compensation for subjects in studyCompensation for subjects in study

Informed Consent, IBR ApprovalInformed Consent, IBR Approval


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Getting started on a Research ProtocolGetting started on a Research Protocol

Read and understand the instructionsRead and understand the instructions

IRB, pharmaceutical, society, NIH grantIRB, pharmaceutical, society, NIH grant

submissionsubmission

Know the time linesKnow the time lines

Deadline for submissionDeadline for submission

Know the formatKnow the format

Electronic/PaperElectronic/Paper

Notify collaborators in advanceNotify collaborators in advance

letters, CVletters, CV

Frequent reviews and critiques by othersFrequent reviews and critiques by others


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GI Research Project Proposal PresentationsGI Research Project Proposal Presentations

During the September and October months, our GI ResearchDuring the September and October months, our GI Research

Conferences are devoted to hearing the projects of the second anConferences are devoted to hearing the projects of the second and thirdd third

year GI fellows and the GI motility fellowyear GI fellows and the GI motility fellow

The presentation is 10 minutes long with 5 minutes for questionsThe presentation is 10 minutes long with 5 minutes for questions

Fellows go over their presentation with their research mentor prFellows go over their presentation with their research mentor prior to theior to the

presentationpresentation

General format for the power point slides:General format for the power point slides:

Title of Project, your name, and name of Research Mentor: 1 slidTitle of Project, your name, and name of Research Mentor: 1 slidee

Background for project: 1Background for project: 1--2 slides2 slides

Aims and Hypotheses: 1 slideAims and Hypotheses: 1 slide

Methods for the project: 1Methods for the project: 1--4 slides4 slides

What is hoped to be found in the data, importance of project: 1What is hoped to be found in the data, importance of project: 1--22

slidesslides

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Medicine Research Slides