Presentazione standard di PowerPoint · R2S2 2nd Edition –Porto Potenza Picena (MC) ITALY - May...

R2S2 2nd Edition – Porto Potenza Picena (MC) ITALY - May 13-18, 2019 How to develop a research project Maria Gabriella Ceravolo

How to develop a research project

(selection of outcome measures,inclusion/exclusion criteria, ethical

aspects, randomization, blindassessment, sham therapy …)

Maria Gabriella Ceravolo

Member of the European Academy of Rehabilitation Medicine

Neurorehabilitation Clinic, United Hospitals of Ancona, Dept of Experimental and Clinical Medicine

Faculty of Medicine, Politecnica delle Marche University, [email protected]

May 15th 2019

mailto:[email protected]


DISCLOSURE

I HAVE NO CONFLICTS OF INTERESTS TO DISCLOSE


WHY DO RESEARCH IN PMR?

● Achieve a better understanding of the problems faced by persons with

disabilities and to define the underlying mechanisms of impairment,

reduced activity, and limited participation

● Understand the potential benefit of pharmacological and technological

rehabilitative interventions

● Inform our clinical decisions

● Find ways of increasing activity and participation of persons with

disabilities

● Influence health (and reimbursement) policy

● Contribute new knowledge that applies outside of the field of PM&R and

that is relevant to other medical specialties


ATTRIBUTES OF RESEARCH

▪ THEORETICAL: concerned with developing, exploring or testing

the theories or ideas that researchers have about how the world

operates.

▪ EMPIRICAL: based on observations and measurements of real

phenomena

▪ NOMOTHETIC: concerned with the nomothetic -- the general case --

rather than the individual. Although we study individuals, usually we

are interested in generalizing to more than just the individual.

▪ PROBABILISTIC: based on probabilities. All inferences have

probabilities associated with

▪ CAUSAL: most research is eventually interested in looking at cause-

effect relationships.


WHAT IS EB-PRACTICE

EVIDENCE BASED PRACTICE

Is the conscientious, explicit, and judicious use

of current best evidence

in making decisions about the care of individual

patients.

Clinical expertise is integrated with the best

available external clinical evidence from

systematic research

David Sackett

Archie Cochrane


THE VIRTUOUS CYCLE LEADING TO BEST PRACTICE

“Integration of best research evidence with clinical experience and patient values”

(Sackett et al., 2000)


THE ROAD TO RESEARCH: MILESTONES

THE QUESTION

DESIGN

SAMPLE

MEASURE

ANALYZE

CONCLUDE


Steps of Evidence Based Practice

Ask • PICO question

Acquire

• Study design

• Type of evidence

Appraise

• Validity

• Effect

Apply

• Feasibility

• Integration into practice


THE QUESTION: BACKGROUND or FOREGROUND QUESTIONS?

PROGNOSIS

DIAGNOSIS

ETIOLOGY / RISK :

FOREGROUND QUESTIONS:

designed to solve specific problems arising from clinical

practice

TREATMENT

IS THERE ANY CAUSAL RELATIONSHIP

BETWEEN X AND Y?

WHAT IS THE DIAGNOSTIC ACCURACY OF THE

“NN” CLINICAL/INSTRUMENTAL ASSESSMENT?

WHAT IS THE NATURAL HISTORY OF

RECOVERY AFTER STROKE? WHAT IS THE

PROGNOSTIC ROLE OF FACTOR “Z”?

WHAT IS THE EFFICACY OF TREATMENT “T”,

COMPARED TO “T1”?

CASE-CONTROL STUDIES

COHORT STUDIES

CROSS-SECTIONAL

STUDIES

COHORT STUDIES

COHORT STUDIES AND

RANDOMIZED

CONTROLLED STUDIES

EACH

QUESTION

HAS

ITS

OWN

DESIGN


- PATIENT: any feature characterising the patient sample

- INTERVENTION : the treatment, risk factor, diagnostic test under

study

- COMPARISON: the alternative treatment, risk condition,

diagnostic test, to be compared with the INTERVENTION

- OUTCOME (s): the measured event (s)

- (T) = time factor (optional)

FOREGROUND QUESTIONS:

answer a need for clinical information related to a

specific patient, an intervention or therapy.

THE PICO(T) APPROACH helps to focus your question:

LITERATURE

REVIEW


DIFFERENT STUDY DESIGN FOR

DIFFERENT QUESTIONS

CAUSAL

STUDIES

RELATIONAL STUDIES

DESCRIPTIVE STUDIESdesigned to describe

phenomena, discriminating

recurrent /sporadic events ,

mean/ modal/variable events..

designed to look at the relationships

between two or more variables.

designed to determine whether one or more

variables (e.g., a treatment variable) causes or

affects one or more outcome variables.


MAIN COMPONENTS OF STUDY DESIGNS

MANIPULATIONOF

INDEPENDENT VARIABLES

MEASUREMENT OF

DEPENDENT VARIABLES

(OUTCOME)

STATISTICAL ANALYSIS

OF THE CAUSAL

RELATIONSHIP CONCLUSIONS


• CONTROL GROUP ----> chance • RANDOMISATION ----> selection bias• BLINDNESS ---- > assessment bias• SAMPLE SIZE ESTIMATION • STATISTICAL SIGNIFICANCE • Intention to treat analysis• CLINICAL SIGNIFICANCE ??

(Jakobsen et al BMC Medical research Methodology 2014)


Blinding

•Participants•Healthcare providers•Outcomes assessors•Data analysts•Data safety and monitoring committee•Writers•OPEN TRIAL (trial in aperto)

CONSORT Statement 2010 Cartabellotta AMD 2011

Data collectors


MAIN SOURCES OF FALLACIES

ECOLOGICAL FALLACY.: conclusions about individuals are

based only on analyses of group data

EXCEPTION FALLACY.: conclusions about groups are

based on analyses of exceptional

cases.


MEASUREMENT

DESIGN

EXTERNAL VALIDITY

ANALYSIS

CONSTRUCT VALIDITY

INTERNAL VALIDITY

CONCLUSION VALIDITY

THE YIN AND YANG OF

RESEARCH METHODOLOGY

THEORY

INTRODUCTION

TO RESEARCHRESEARCH

PROBLEM

FORMULATION

SAMPLING

PRACTICE

TYPES OF VALIDITY PRACTICE AREAS


RESEARCH VALIDITY the best available approximation to the truth of a

given proposition, inference, or conclusion

Trochim, William M. The Research Methods Knowledge Base, 2nd Edition. http://www.socialresearchmethods.net/kb/


RESEARCH VALIDITY the best available approximation to the truth of a

given proposition, inference, or conclusion

Trochim, William M. The Research Methods Knowledge Base, 2nd Edition. http://www.socialresearchmethods.net/kb/

IS THERE A RELATIONSHIP

BETWEENTHE CAUSE AND EFFECT?

IS THE RELATIONSHIP CAUSAL?

CAN WE GENERALIZE

TO OTHER PERSONS,

PLACES, TIMES?

CAN WE GENERALIZE

TO THE CONSTRUCT?


ASSESSING RESEARCH VALIDITY

EXTERNAL VALIDITY

CONCLUSION VALIDITY

AND

INTERNAL VALIDITY


% TRIALS MEETING EACH SINGLE REQUIREMENT


BEST PRACTICE: BEYOND EBP

BEST PRACTICE is PRACTICE THAT

on rigorous evaluation,

demonstrates success

has had an impact

can be replicated

METHODOLOGY CRITERION

EFFICACY CRITERION

EFFECTIVENESS CRITERION

EXTERNAL VALIDITY CRITERION

KNOWLEDGE TRANSLATION (≠ DISSEMINATION)

EVIDENCE-

BASED

PRACTICE

WHAT IS KNOWN

WHAT IS DONE

(Johns Hopkins Bloomberg School of Public Health)

orientation

insight

acceptance

change

maintenance

it takes 17 years

to translate 14%

of research findings

into clinical practice

R2S2 2nd Edition – Porto Potenza Picena (MC) ITALY - May 13-18, 2019 How to develop a research project Maria Gabriella CeravoloProf. Maria Gabriella Ceravolo – Dept. Experimental and Clinical Medicine – Neurorehab. Clinic - ANCONA

WHAT’S IN A NAME? THE CHALLENGE OF DESCRIBING

INTERVENTIONS IN SYSTEMATIC REVIEWS

Hoffmann TC, et al. BMJ Open 2015;5:e009051. doi:10.1136/bmjopen-2015-009051

Mean percentage of systematic reviews, per Template for

Intervention Description and Replication (TIDieR) checklist

item, in which interventions were described ‘adequately for all’ or

‘adequately for some’ of the interventions in the review, for

Cochrane and non-Cochrane reviews.


CHALLENGES IN THE APPLICATION OF EBP AND

KNOWLEDGE TRANSLATION

IN REHABILITATION MEDICINE

SCARCITY OF RCTs

Most evidence addressing the effectiveness of clinical or policy

interventions rests on nonrandomized studies

RCTS ACCLAIMED AS THE HIGHEST LEVEL OF EVIDENCE

Perception that rehabilitation research suffers from a

lack of methodologic rigor.

PAUCITY OF SYSTEMATIC REVIEWS

Perception that rehabilitation is evidence-poor


…HOWEVER “HIGH-LEVEL” RESEARCH DESIGNS

may not be the best solutions to given research questions,

may not be applicable to all populations or situations,

often have limited external generalizability,

can be equally or more flawed than other study designs.

Finally, even if they are METHODOLOGICALLY SOUND

THE VALUE OF THE RESEARCH QUESTION can be POOR

CHALLENGES IN THE APPLICATION OF EBP AND

KNOWLEDGE TRANSLATION

IN REHABILITATION MEDICINE


TO BE CONTINUED…….

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