Document Detail

Role of disease risk scores in comparative effectiveness research with emerging therapies.
MedLine Citation:
PMID:  22552989     Owner:  NLM     Status:  MEDLINE    
BACKGROUND: Usefulness of propensity scores and regression models to balance potential confounders at treatment initiation may be limited for newly introduced therapies with evolving use patterns.
OBJECTIVES: To consider settings in which the disease risk score has theoretical advantages as a balancing score in comparative effectiveness research because of stability of disease risk and the availability of ample historical data on outcomes in people treated before introduction of the new therapy.
METHODS: We review the indications for and balancing properties of disease risk scores in the setting of evolving therapies and discuss alternative approaches for estimation. We illustrate development of a disease risk score in the context of the introduction of atorvastatin and the use of high-dose statin therapy beginning in 1997, based on data from 5668 older survivors of myocardial infarction who filled a statin prescription within 30 days after discharge from 1995 until 2004. Theoretical considerations suggested development of a disease risk score among nonusers of atorvastatin and high-dose statins during the period 1995-1997.
RESULTS: Observed risk of events increased from 11% to 35% across quintiles of the disease risk score, which had a C-statistic of 0.71. The score allowed control of many potential confounders even during early follow-up with few study endpoints.
CONCLUSIONS: Balancing on a disease risk score offers an attractive alternative to a propensity score in some settings such as newly marketed drugs and provides an important axis for evaluation of potential effect modification. Joint consideration of propensity and disease risk scores may be valuable.
Robert J Glynn; Joshua J Gagne; Sebastian Schneeweiss
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Publication Detail:
Type:  Comparative Study; Journal Article; Research Support, N.I.H., Extramural    
Journal Detail:
Title:  Pharmacoepidemiology and drug safety     Volume:  21 Suppl 2     ISSN:  1099-1557     ISO Abbreviation:  Pharmacoepidemiol Drug Saf     Publication Date:  2012 May 
Date Detail:
Created Date:  2012-05-03     Completed Date:  2012-09-04     Revised Date:  2013-06-25    
Medline Journal Info:
Nlm Unique ID:  9208369     Medline TA:  Pharmacoepidemiol Drug Saf     Country:  England    
Other Details:
Languages:  eng     Pagination:  138-47     Citation Subset:  IM    
Copyright Information:
Copyright © 2012 John Wiley & Sons, Ltd.
Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, 1620 Tremont Street, Boston, MA 02120, USA.
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MeSH Terms
Comparative Effectiveness Research*
Confounding Factors (Epidemiology)*
Dose-Response Relationship, Drug
Epidemiologic Research Design
Hydroxymethylglutaryl-CoA Reductase Inhibitors* / administration & dosage,  therapeutic use
Models, Statistical*
Myocardial Infarction / epidemiology*,  prevention & control
Outcome and Process Assessment (Health Care)
Propensity Score*
Randomized Controlled Trials as Topic* / statistics & numerical data
Risk Factors
Grant Support
Reg. No./Substance:
0/Hydroxymethylglutaryl-CoA Reductase Inhibitors

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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