What’s a publishable question?Focus on Clinical Research

September 16th, 2002

Elizabeth S. Higgs, MD, DTM&H, MIA

Overview: What’s a Publishable Question? Focus on Clinical Research

! Why conduct clinical research?– Three fundamental goals in clinical research.

! The primary research question– FINER

! Thinking in advance of where you might publish your research.

! Summary

Why Conduct Clinical Research?

! Standards for clinical care and health policy are data driven.

! Rigorous standards must be applied to the acquisition and reporting of knowledge in the clinical research setting such that “truth” can guide health care and health care policy.

! Absence of standards or rigor leads to erroneous or uncertain conclusions…e.g. dietary supplements in the U.S.

Conduct Clinical Research to generate truthful data about relevant clinical diseases.

! We want accurate, reliable data….we want scientific truth!

TRUTHFUL DATA

Clinical Care and Health Policy Standards

Health & well being of people!

informs

Fundamental Goal #1

A Fundamental Goal of Clinical Research to add to existing body of knowledge

! Want to add to existing scientific knowledge….

Natural History

Susceptibility

Pathogen etiology

Pathogenesis

Therapeutic Strategy

Fundamental Goal #2

Does your paper add to the existing body of knowledge? If so where & how?

! If you want to add to existing scientific knowledge….you must know the literature.

Natural History

Susceptibility

Pathogen etiology

Pathogenesis

Therapeutic Strategy

Fundamental Goal #2

Continuum of Clinical Research*

Description Exploratory Experimental

Describe populationAnd problem

Find Relationships Cause and Effect

•Case studies•Natural history

•Cohort studies•Case Control studies

•Randomized Controlled Trial

•Meta analysis*From Foundations of Clinical Research , Portney and Gross

Epidemiology

Fundamental Goal of CR is to generate results which have universal truth….or at least relevance outside study population.

! Want study results to be generalizeable…..– Study of therapy for neurocysticercosis want to say

that the study results are applicable to others outside the study with neurocysticercosis

TRUTH IN THE

UNIVERSE

FINDINGS IN THE STUDYInfer

Fundamental Goal #3

How is your research generalizable? Does it have implications outside Mali?

! Want study results to be generalizable…..if not state limitations around findings…– E.g. what are the malaria drug sensitivities in Mali?

Vs. what are the relevant issues to the development/evolution of drug resistance?

TRUTH IN THE

UNIVERSE

FINDINGS IN THE STUDYInfer

Fundamental Goal #3

Designing & Implementing

Drawing Conclusions

Research Question

Truth in Universe

Study Plan

Truth in The Study

Actual Study

Findings inThe study

design implement

infer infer

Process of Designing and Implementation a Research Project sets the stage for drawing conclusions*and the papers you will eventually write.

*Diagram from Designing Clinical Research S. Hulley, S Cummings

Primary Research Question… should eventually become the focus of a paper.

! Developing the primary research question, or primary research objective is the first step in designing research.

! Question that investigators are most interested in answering and one that meets goals of clinical research (truthful data, add to knowledge, and generalizable) .

! Should be carefully selected, clearly defined and stated in advance.

! Tied to well defined variables, or endpoints.! Study should statistically able to answer primary

objective.

Characteristics of a Good Question Research Question--FINER*

! Feasible! Interesting! Novel! Ethical! Relevant

*Designing Clinical Research S. Hulley, S Cummings

Characteristics of a Good Question Research Question--FINER*

! Feasible: – Adequate # subjects and Expertise– Time and money available– Manageable– Data to answer

! Interesting– To the investigator

! Novel– Confirms or refutes previous findings– Extends previous findings– Provides new findings

Designing the study

*Designing Clinical Research S. Hulley, S Cummings

Characteristics of a Good Question Research Question--FINER*

! Ethical– Multiple levels including investigator conviction

! Relevant– To scientific knowledge– To clinical and health policy– To future research directions

Designing the study

*Designing Clinical Research S. Hulley, S Cummings

One question? “What about all the other things I want to learn?”

! Secondary Objectives: can be related to primary objective or relate to other hypothesis.– Study may not be statistically able to answer these

questions! Common Error—avoid overloading (sinking)

the study with too many objectives and too much data collection.

Designing the study

Once develop research question, think about where you will publish.

! Types of manuscripts that would have a higher likelihood of being published by one of the first-tier general medical journals (The Lancet, NEJM, BMJ, JAMA, or Annals of Internal Medicine) Ken Schulz

– acceptance rates run between 5-10% " Reports of original research that would be likely to

change clinical practice or concepts about a disease.

! Reports should also be of general interest – they might venture into more than one specialty or be of such great novelty and importance that the journal’s readers should be informed of the findings.

First Tier Journals…

" Carefully conducted randomized controlled trials of treatments or other interventions have a much greater chance of being accepted for publishing.

" Generally, the methodology and the clarity of the report need to be impeccable.

! Carefully conducted analytical, observational studies (for example, comparative cohort or case-control) would have a reasonable chance of success, but lower than randomized trials.

Where to publish

First Tier Journals…

" Carefully conducted descriptive studies have the lowest likelihood of success. Simply describing or exploring a problem, particularly if in a small, defined population, would have a low probability of being published.

Where to publish

First Tier Specialists Journals…JID, ASTMH etc.

" acceptance rates run between 10- 40%1. Reports of original research that would be likely to

change clinical practice or concepts about a disease in the relevant specialty would also have priority here.

2. However, the reports need not have the general interest across specialties nor the great novelty and importance that one of the big five general medical journals require.

3. Generally, the methodology and clarity of the report need to be excellent, sometimes approaching the level of the first-tier general medical journal but frequently at a standard below them.

Where to publish

First Tier Specialists Journals…JID, ASTMH etc.

4. As with the general journals, randomized trials frequently receive priority attention. However, trials are more likely to compete on equal footing with analytical observational studies just because of what is submitted to these journals and because of editorial opinion.

5. Descriptive studies have a greater chance of success here than with the general medical journals, but the chances still are not good.

Where to publish

Second Tier Specialists Journals

Acceptance rates exceed 30% to 70%.1. Reports of original research need only pertain to the

specialty, whether the relevance is broad or only quite narrow.

2. The quality of the methodology and the clarity of the reports vary greatly, excellent to poor conduct. Also, the clarity of the reports range from clear to unintelligible.

Where to publish

Second Tier Specialists Journals

Acceptance rates exceed 30% to 70%.3. Randomized trials and observational studies probably

compete on equal footing here because well-conducted randomized trials would likely be published in one of the above two tiers and are unlikely to be submitted to a second-tier journal.

4. Descriptive studies are more likely to eventually end up being published, if anywhere, in a second-tier specialist journal.

Where to publish

International Clinical Research..added complexities

! Must have collaborative true partnership based on mutual respect and trust.

! Equally strong commitment to the research! Complexities of integration of research ethics from two

countries, including more ethical and regulatory requirements.

! Challenges with data management & transfer! Cultural differences which may impact on feasibility

and conduct of research.

Study Analysis and Reporting

! New standards for analysis and reporting, CONSORT Guidelines

! Evolving standards on publication of data bases led by genomics.

Summary..publishable questions….

! Three fundamental goals for clinical research– to generate truthful data– to add to existing body of knowledge– generate results which have universal truth, or generalizable in some

way.! The primary research question should fulfill general

goals for clinical research and FINER acronym (Feasible, Interesting, Novel, Ethical and Relevant) .

! Think about where you might publish prior to initiating studies.

! Keep up with the literature monthly!