Mary B Codd. MD, MPH, PhD, FFPHMI UCD School of Public Health, Physiotherapy & Pop. Sciences



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HRB / CSTAR Grant Applications Training Day Convention Centre Dublin, 9 th September 2010 Key Elements of a Research Protocol Mary B Codd. MD, MPH, PhD, FFPHMI UCD School of Public Health, Physiotherapy & Pop. Sciences 1

HRB aims to fund Researchers and Research Teams new knowledge evidence of benefit to health, through patient-oriented research population health sciences research health services research Focus is similar for Health Research Awards and Training Fellowships 2

Research Protocol - I What is a Research Protocol? A formal document specifying what the study hopes to achieve and how it is to be conducted When should you write a Research Protocol? For every research project, no matter how small or seemingly insignificant Before doing anything else! 3

Research Protocol - II Why should you write a Research Protocol? to organise your thinking to clarify the question(s) being asked to facilitate discussion with colleagues to reduce the risk of digressions during project to compete for funding for submission to Research Ethics Committee to submit your MSc / PhD proposal & get it accepted! to ensure continuity of the project if necessary 4

Research Protocol - III How should you write a Research Protocol? Know the Key Elements of a Research Protocol Engage with a Multidisciplinary Team Give yourself adequate time! Deadlines within deadlines, e.g. Institutional requirements Be prepared to draft and redraft! 5

The Research Team. Breadth & depth of expertise in scientific / clinical / methodological areas Expertise in statistics, health economics, HSR, behavioural sciences, qualitative research, sociology, trialists (for RCTs) Add as Co-applicants / Collaborators Partner Organisations, e.g. academic, health service providers, government, charities Adds to Complexity, but synergistic for Quality 6

Key Elements of a Research Protocol 7

The Research Question Research usually begins with a general question of interest to you, which may arise from an observation within your area of work or interest a prompt or issue in your field of study prior research of relevance However, usually too general! Needs to be formulated into a specific question that can be investigated Once question defined, a systematic approach to the investigation must follow (research) 8

Current Knowledge / Background Size / nature of issue State the issue! Identify & summarise previous knowledge / work through comprehensive literature review, searches for relevant national data, pilot work, etc Seek collaboration / multidisciplinary input Relevance / Importance Identify the potential contribution / significance of your work to existing body of knowledge 9

Literature Review: Mechanics Literature Searches: Keywords / Terms: Type of Publication: Time Period: Citation Alerts: Referencing System: Which Databases? MeSH Databases / PubMed Original Research Papers Reviews Meta analyses Editorials / Letters Recent Do not ignore KEY old papers Sign Up EndNote / Reference Manager 10

Literature Review: Content Review published works to date on this question Identify gaps in current knowledge / What can your research contribute? e.g. Is there a literature specific to Ireland on this topic? Be critical of published literature and of your proposal Avoid the need to find a literature to support your preconceived ideas / what you have already done! Include relevant literature only! Beware of plagiarism! Review policies on plagiarism, e.g. http://www.ucd.ie/library/students/information_skills/plagiari.html 11

Specific Aims / Objectives Statement of the Question(s) to be answered Statement of the Hypothesis(es) to be tested Structure of the Statement of Objectives: To describe To examine To determine To analyse Structure of Statements of Hypotheses: Null hypothesis (Ho): There is no difference Alternative hypothesis (Ha): There is a difference 12

Study Design / Methods OBSERVATIONAL Descriptive Retrospective Cross-sectional Prospective Analytical Case-control Association Cohort EXPERIMENTAL Analytical Clinical trials Intervention trials 13

Sources of Participants / Data Population-based Clinic attendees Hospital inpatients Selected subgroups Specific disease subgroups (DM, HIV ) Specific exposures Patients with known familial or genetic characteristics Survey / Questionnaire Interview data Focus Groups Medical record review Employment records Registry data Vital statistics 14

Eligibility Criteria (Incl / Excl) DEMOGRAPHIC Age, Gender Occupational Ethnic Group Geographic Area BIOLOGICAL Specific parameters Specified ranges DIAGNOSTIC Decide on criteria prior to recruitment / inclusion Use standard criteria where possible Define cut off points Establish hierarchy of sources of diagnostic data 15

Populations / Sampling Objective of Sampling To reduce cost and workload while Maintaining representativeness and relevance for extrapolation of results to the larger population of interest Know your Sampling Frame (size, location, characteristics, availability, accessibility) 16

Sampling Methods Epidemiologic Sampling Simple random sampling Systematic sampling Stratified sampling Cluster sampling Stratified Cluster Sampling Convenience Sampling Caveats!! 17

Sample Size Estimation For almost every type of study Different inputs depending on study design Well laid out in statistical texts Trade off between power and practicality Be prepared to abandon study if necessary Get help! 18

Objective of Sample Size Estimation Descriptive: Analytical: RCT: Community Intervention: Adequate sample size to ensure desired precision of a single estimate / mean or proportion (prevalence) Adequate sample size to ensure statistical significance of difference in risk Adequate sample size to ensure statistical significance of estimate of change / benefit Sample size to estimate NNT / benefit to community at large 19

Recruitment Issues Know your pool (Incidence / prevalence data) Feasibility / Organisation of recruitment Duration / How long do you have? Participant information / Consent issues Response / participant rate adjustments? Non participants / refusals characteristics? Compliance / drop outs / losses to follow up Impact on endpoints / data analysis? 20

Outcome of Interest Primary Endpoint What is it? Can it be defined precisely? Can it be measured? How do you plan to measure / document it? Secondary Endpoints Are there any? If yes, how many? (too many?) If no, should there be? 21

Methods of Evaluation - I Baseline / Follow-up Measure the same parameters in the same way at baseline and follow up Standardize to reduce measurement error Develop SOP s appropriate to the study Decide frequency of follow up measurements Advise caution re analysis of repeated measures in same individuals 22

Methods of Evaluation - II Subjective / Scaling methods Use pre-existing, pre-tested instruments Estimate validity and reliability Objective / Validity and Reliability Standardisation / calibration Measures of validity / accuracy Measure of reliability / reproducibility 23

Basic Data Management: Some truths Most (first-time) Researchers: Know they should write a Research Protocol before starting a project Can guess the key elements of a Research Protocol even without a Template Really don t think too much about the data required, i.e. what data to collect where to get / collect the required data how to collect the required data what to do with them once collected Shudder at the thought of Data Analysis / Statistical Tests! 24

Basic Database Management: Some truths Most (first-time) Researchers: At best, give some thought to how to handle project data but find it too uncomfortable / difficult to deal with Decide to cross that bridge when they come to it Muddle along somehow in the hope that out of a soup of figures will emerge some results Plan to find a friendly Statistician at a late stage in the process At worst, do not think about it until it s too late or not at all! 25

Basic Data Management: Elements 1. What Data? 2. Where to find them? 3. How to collect them? 4. How to ensure consistency in data collection? 5. How to collate data electronically? 6. How to ensure integrity (validity) of data in database? 7. How to ensure integrity (security) of data in database? 8. How to analyse, interpret and report findings? In so far as possible all aspects should be addressed and planned for at the before the project begins 26

Basic Data Management: Elements 1. What Data? Decide what Data Items (Variables) Group Items / Variables into Categories Administrative Demographic Attitudinal / Behavioural Clinical Economic Occupational 2. Where to Find? Sources of Data (Primary / Secondary) Public domain (e.g. Vital Stats, Registry Data) Requests from existing databases (e.g. ESRI / HIPE) Records available (e.g. Company, Medical) New data (e.g. questionnaires, clinical data) 27

Basic Data Management: Elements 3. How to Collect Data? 4. How to ensure consistency in Data Collection? (QA in data collection) Standardised Format Questionnaire Case Report Form Spreadsheet Standardised Terminology Data Dictionary to include the following on each variable Full Name & Abbrev to be used Definition Source(s) Type (Quantitative/Qualitative) Type (Continuous/Discrete/Categorical) Level of Measurement Coding Options (very important) 28

A Data Dictionary is a Database about a Database Centralised repository of information about data It catalogues what data are stored The characteristics of each data item / variable It defines the structure of a database itself, not that of the data held in the database There is no universal standard as to level of detail in a Data Dictionary 29

A Data Dictionary Is essential for training study personnel Underpins standardisation of data capture Facilitates assessment of data quality (QA) Ensures adherence to study protocol Invaluable at data analysis phase 30

What s in Data Dictionary? For each Variable: Column in which entered Abbreviation to be used (appropriate & intelligible) Full name of the Variable with Unit of Measurement Definition of the Variable (complete & agreed) Source(s) where and how to find information Type (Quantitative/Qualitative) Type (Continuous/Discrete/Categorical) Level of Measurement Coding Options 31

Basic Data Management: Elements 5. How to collate data electronically? Create a Database Excel / Access / Other Relational / Bespoke Enter data to proscribed format 6. How to ensure integrity (validity) of data in your database? (QA of database data) Standardised Routines for: Periodic review of data entered Identification of errors e.g. missing data, incorrect or illogical entries (the Data Dictionary is critical to this) Correction of errors (data cleaning / editing strategies, possible including review of source documents) Documentation / log of edits made 32

Basic Data Management: Elements 7. How to ensure integrity (security) of data in your database? 8. How to Analyse, Interpret & Report findings from your study? Data Security Measures Care / security of source documents Questionnaires / CRF s Personal Identification Care / security of hardware PC / Laptop / USB Software security Basic / sophisticated Statistical Analysis Standard statistical software Standard statistical techniques 33

Literature Cited Up to 30 allowed Use a software application which enables users to manage bibliographic references (e.g.endnote) References can be entered manually or downloaded from an online database Endnote can format them according to the requirements set out for your thesis http: //www.ucd.ie/library/students/information_skills/endnote/index.html 34

Relevant Appendices As relevant: Participant Information Leaflet Consent Form Research Ethics Committee Approval Questionnaire Focus Group Interview schedule Case Report Form for data collection Data Dictionary 35