User-only design to assess drug effectiveness in clinical practice: application to bisphosphonates and secondary prevention of fractures

Pharmacoepidemiol Drug Saf. 2014 Aug;23(8):859-67. doi: 10.1002/pds.3650. Epub 2014 Jun 9.

Abstract

Purpose: Different strategies applicable to control for confounding by indication in observational studies were compared in a large population-based study regarding the effect of bisphosphonates (BPs) for secondary prevention of fractures.

Methods: The cohort was drawn from healthcare utilization databases of 13 Italian territorial units. Patients aged 55 years or more who were hospitalized for fracture during 2003-2005 entered into the cohort. A nested case-control design was used to compare BPs use in cohort members who did (cases) and who did not experience (controls) a new fracture until 2007 (outcome). Three designs were employed: conventional-matching (D1 ), propensity score-matching (D2 ), and user-only (D3 ) designs. They differed for (i) cohort composition, restricted to patients who received BPs straight after cohort entry (D3 ); (ii) using propensity score for case-control matching (D2 ); and (iii) compared groups of BPs users versus no users (D1 and D2 ) and long-term versus short-term users (D3 ).

Results: Bisphosphonate users had odds ratios (95% confidence interval) of 1.20 (1.01 to 1.44) and 0.95 (0.74 to 1.24) by applying D1 and D2 designs, respectively. Statistical evidence that long-term BPs use protects the outcome onset with respect to short-term use was observed for user-only design (D3 ) being the corresponding odds ratio (95% confidence interval) 0.64 (0.44 to 0.93).

Conclusions: User-only design yielded closer results to those seen in RCTs. This approach is one possible strategy to account for confounding by indication.

Keywords: bisphosphonates; confounding; fracture; pharmacoepidemiology; propensity score; user-only design.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Aged, 80 and over
  • Bone Density Conservation Agents / administration & dosage
  • Bone Density Conservation Agents / therapeutic use
  • Case-Control Studies
  • Confounding Factors, Epidemiologic
  • Databases, Factual / statistics & numerical data*
  • Diphosphonates / administration & dosage
  • Diphosphonates / therapeutic use*
  • Female
  • Fractures, Bone / prevention & control*
  • Hospitalization
  • Humans
  • Italy
  • Male
  • Middle Aged
  • Observational Studies as Topic / methods*
  • Propensity Score
  • Research Design
  • Retrospective Studies
  • Secondary Prevention / methods
  • Time Factors

Substances

  • Bone Density Conservation Agents
  • Diphosphonates