Study design for a comprehensive assessment of biologic safety using multiple healthcare data systems

Pharmacoepidemiol Drug Saf. 2011 Nov;20(11):1199-209. doi: 10.1002/pds.2196. Epub 2011 Sep 15.

Abstract

Background: Although biologic treatments have excellent efficacy for many autoimmune diseases, safety concerns persist. Understanding the absolute and comparative risks of adverse events in patient and disease subpopulations is critical for optimal prescribing of biologics.

Purpose: The Safety Assessment of Biologic Therapy collaborative was federally funded to provide robust estimates of rates and relative risks of adverse events among biologics users using data from national Medicaid and Medicare plus Medicaid dual-eligible programs, Tennessee Medicaid, Kaiser Permanente, and state pharmaceutical assistance programs supplementing New Jersey and Pennsylvania Medicare programs. This report describes the organizational structure of the collaborative and the study population and methods.

Methods: This retrospective cohort study (1998-2007) examined risks of seven classes of adverse events in relation to biologic treatments prescribed for seven autoimmune diseases. Propensity scores were used to control for confounding and enabled pooling of individual-level data across data systems while concealing personal health information. Cox proportional hazard modeling was used to analyze study hypotheses.

Results: The cohort was composed of 159,000 subjects with rheumatic diseases, 33,000 with psoriasis, and 46,000 with inflammatory bowel disease. This report summarizes demographic characteristics and drug exposures. Separate reports will provide outcome definitions and estimated hazard ratios for adverse events.

Conclusion: This comprehensive research will improve understanding of the safety of these treatments. The methods described may be useful to others planning similar evaluations.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Aged
  • Autoimmune Diseases / drug therapy
  • Autoimmune Diseases / epidemiology*
  • Autoimmune Diseases / therapy*
  • Bias
  • Biological Products / adverse effects*
  • Biological Products / therapeutic use
  • Cohort Studies
  • Databases, Factual*
  • Delivery of Health Care
  • Humans
  • Medicaid / statistics & numerical data
  • Medicare / statistics & numerical data
  • Middle Aged
  • Models, Statistical*
  • New Jersey
  • Pennsylvania
  • Propensity Score
  • Proportional Hazards Models
  • Research Design
  • Retrospective Studies
  • Risk
  • Tennessee
  • Time Factors
  • United States
  • United States Food and Drug Administration / statistics & numerical data
  • Vulnerable Populations / statistics & numerical data
  • Young Adult

Substances

  • Biological Products