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Predictive analytics pilot study: assessment of heterogeneity of treatment effects in two major clinical trials
Investigator (PI): Kent, David M
Performing Organization (PO): (Current): Tufts Medical Center, Department of Medicine, Division of Internal Medicine and Adult Primary Care / (617) 636-5400
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2017
Final Year: 2019
Record Source/Award ID: PCORI/RR-1705-0001
Funding: Total Award Amount: $546,780
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Although "evidence-based medicine" has become the dominant paradigm for shaping clinical recommendations and guidelines, recent work has emphasized that there is a fundamental incongruence between the evidence available from clinical trials (i.e., the overall or average effect of a treatment in the study population) versus the information that is needed for most medical decisions (i.e., what treatment is best for an individual patient). While the field of clinical therapeutics has long understood that treatments may work differently in different patients (a concept known as heterogeneity of treatment effect), developing reliable methods for the estimation of individual patient effects has proven difficult. Investigators have traditionally tried to discover heterogeneity by re-examining treatment effects for subgroups defined by single variables, but these analyses are generally inadequate and not truly representative of the complex combination of factors that represent individual patients. Multivariable risk stratification may more effectively identify patients in whom a therapy offers a greater potential net benefit (that is, benefit minus harm). This project re-analyzes two randomized clinical trials with the intent to identify patients at potentially higher risk of experiencing either benefits or adverse effects from the interventions studied (i.e., a positive or negative net benefit), in order to better personalize individual decision making. The first trial involves patients with insulin resistance and a prior stroke or TIA deciding whether to take pioglitazone for the prevention of stroke or myocardial infarction, given its adverse event profile of weight gain, peripheral edema, and bone fractures. The second trial examines the potential for cardiotoxicity among women with breast cancer receiving anthracycline chemotherapy.
MeSH Terms:
  • Anthracyclines /therapeutic use
  • Breast Neoplasms /*drug therapy
  • Clinical Trials as Topic
  • * Data Interpretation, Statistical
  • Decision Making
  • Edema /prevention & control
  • Evidence-Based Medicine
  • Fractures, Bone /prevention & control
  • Humans
  • Hypoglycemic Agents /therapeutic use
  • Insulin Resistance
  • Ischemic Attack, Transient /*drug therapy
  • Multivariate Analysis
  • Myocardial Infarction /drug therapy
  • /prevention & control
  • Patient Safety
  • Pilot Projects
  • Program Development
  • Program Evaluation
  • Randomized Controlled Trials as Topic
  • Risk
  • Stroke /drug therapy
  • /prevention & control
Country: United States
State: Massachusetts
Zip Code: 02111
UI: 20181700
Project Status: Completed