Non-Scientific Factors on Research and Education: Publication Bias
Seth S. Leopold, MD
Associate Professor
Department of Orthopaedics and Sports Medicine
University of Washington

Industry and Orthopaedics
Physician — Public
Collaborative outreach / public education
Physician — Patient
Research to determine best practice
Physician — Physician
CME

Headlines: Public Concerns
“Company tried to bar report that HIV vaccine failed”
“MDs and the pharmaceutical industry: A growing embarrassment and liability”
“Premature discontinuation of clinical trials for reasons not related to efficacy, safety, or feasibility”
“MDs and the pharmaceutical industry: Is a gift ever just a gift?”

Data: Funding and Research
Authors of practice guidelines
87% funded through industry
59% rec’d products from supporting companies
Denied relationship affected choice
Meta-Analysis: 1140 studies
Industry sponsorship related to positive outcome
Also related to limits on publication and data sharing
Orthopaedic Results
Industry funding related to positive outcome
Most severe in arthroplasty (83% vs 45%, p<0.004)

Data: Funding and CME
>40% CME funding is from industry
Up from 17% in 1994
$569 million in 2001; 22% increase since 2000
Evidence that industry uses CME as a “marketing tool”
Physicians attending company’s courses disproportionately prescribe their products

Orthopaedic Relationships
Public: Confidence?
Bias, or mere appearance of bias?
In this context, does that matter?
Headlines: MD’s “on the take” from industry
Patients: Best Practice?
Quality research or publication bias?
Physicians: CME?
Education or marketing?

Issue: The Relationship
“A good relationship is based on trust…

Issue: The Relationship
“Good name in man and woman, dear my lord, is the immediate jewel of their souls…”

Academic Orthopaedic Goals
How do we
Earn / keep public’s confidence?
Discover best practice?
Disseminate results of our research?
What effect do non-scientific factors have on these goals?

“CAGE” for Industry Relations

Example: MIS THA
How many of you do hip replacements?
Of those, how many have had a patient ask you about “Minimally Invasive”?

Example: Less-Invasive THA
Peer-reviewed papers on Mini THA: 0
Peer-reviewed papers on MIS THA: 0
MD’s marketing MIS to public: Countless
Courses teaching MIS to surgeons: Countless

Patients in the Middle
Public: Confidence?
Patients: Best Practice?
Publication Bias?
Our Research…Past and Future
Physicians: CME?

External Factors and Outcome
Evidence from non-surgical specialties
Non-scientific factors associated with positive outcomes
Pharmaceutical industry funding
Country of origin
Relationship with tobacco/EtOH industry

External Factors and Outcome
Orthopaedics: No data, despite
Strong industry presence
Apparent positive-outcome bias (AAOS 2000)

Positive Outcome Bias
Increased conditional probability that a research will be

•Funded
•Accepted for presentation
•Published

If conclusions are positive

Positive Outcome Bias
Insidious: Hard to detect
Need to look at large number of studies
Harmful: Overestimation of treatment effects
Review articles
Meta-analysis
Economic analysis
Evidence-based medicine

Hypotheses
Receipt of commercial funding
Country of origin
Statistician as co-investigator

Are associated with positive outcome

Methods
All papers over a one-year period
3 Journals
JBJS-A
J Arthroplasty
Am J Sports Med

Methods
Two experienced reviewers
“Rotating” review process
3rd reviewer adjudicated prn

Methods
Blinded review
Authors’ names
Departmental affiliations
Countries of origin
Sources of funding
Presence of a statistician

Data Collected
Classified by study design
Outcome (positive or negative)
315 articles reviewed
95.6% were classifiable

Definition of “Positive”
Emphasizes safety or efficacy
Emphasizes cost-effectiveness
Statistically significant difference found
If specific criteria not met, study not considered “positive”

Description of Literature
11 of 315 (3.5%) randomized
32 of 315 (10.2%) stated hypothesis
66 of 315 (21%) prospective
159 of 315 (50.5%) controlled

Associated w/Positive Outcome
Receipt of commercial funding
78.9% of industry-funded studies were positive
63.3% of non-industry-funded studies were positive
p=0.025

Industry Funding and Outcome
Largest contribution:
J Arthroplasty
83% vs. 45% positive if funded in JA

Not Associated with Outcome
Presence of statistician (p=0.935)
Country of origin (p=0.248)

Reasons for Industry Effect
Benign
In-house research before collaboration
More $ ® More specimens ® Less b-error

Reasons for Industry Effect
Troubling
Restrictive covenants
Early termination of studies by funding source
Psychological or economic influences on investigator

Conclusions
Industry funding associated with positive outcome
Potential source of positive-outcome bias

What Did We Really Show?
Industry-funded papers more likely to conclude positively
Positive papers more likely to have received industry funding

Smoke = Fire?
Possibly
F/U work in other specialties: Bias, not just appearance of bias, was confirmed
ML Callaham (JAMA v280), JM Stern (BMJ v315)
Documented misbehaviors already discussed
But not proven
Consider a different denominator
Published papers (Our study): Appearance of bias
Abstracts submitted for presentation
Manuscripts submitted for publication

Bias vs. Appearance of Bias
Consider universe of all experiments submitted
Address alternative explanations for “apparent bias”
Statistical power
Study quality
Actual publication bias

Power
Is there a difference between positive and negative trials in terms of power?
Control for any systematic differences in power between positive and negative studies

Study Quality
Are positive studies “better” studies as a group?
Industry says “yes,” cites different
Designs
Funding levels
Support networks
Need to control for potential systematic differences in quality

Publication Bias: If So, Where?
Investigator
Avoid studies to show equivalence ($, time)
Unexpectedly negative result? Self-censor
Reviewer
Framing effects
Journal
Funding sources?

Follow-Up Study
H1: Non-scientific factors do not affect likelihood that submitted studies will conclude positively
Control for:
Sample size
Study quality
H2: Positive outcome does not affect the likelihood that study will be published
Denominator: All submissions to a journal
Comment on more than just “apparent bias”

Conclusions
Industry collaboration is here to stay
Study to determine its impact and effects
Realize unique partnership opportunities
Research on publication bias critical
If non-scientific factors impact outcomes, these MUST be identified / controlled as any other confounding variable
Peer-review process may need change
Disclose funding sources earlier in review

THANK YOU
ACKNOWLEDGEMENTS:
Winston J. Warme, MD
E. Fritz Braunlich, MD
Susan Shott, Ph D
Fredric M. Wolf, Ph D
UW Friends of Orthopaedic Research and Education