Translating New Medical Therapies Into Societal Benefit
The Role of Population-Based Outcome Studies
- Christopher M. Booth, MD, FRCPC;
- William J. Mackillop, MB, ChB, FRCPC
- Author Affiliations: Division of Cancer Care and Epidemiology, Queen's University, Kingston, Ontario, Canada.
Since this article does not have an abstract, we have provided the first 150 words of the full text.
- KEYWORDS:
- DRUG THERAPY
- EMERGENCY TREATMENT
- HEART FAILURE
- ISCHEMIC ATTACK, TRANSIENT
- OUTCOME ASSESSMENT (HEALTH CARE)
- POPULATION
- RADIOTHERAPY
- RANDOMIZED CONTROLLED TRIALS AS TOPIC
- RESEARCH
- RESEARCH DESIGN
- SPIRONOLACTONE
- STROKE
- UTERINE CERVICAL NEOPLASMS
Phase 4 studies have traditionally monitored safety of new drugs and occasionally served as marketing tools for pharmaceutical companies. In the 1990s these “seeding studies” came under criticism for the substantial influence industry had on study design and interpretation.1,2 Recently, innovative study designs and access to large electronic databases have allowed investigators to evaluate outcomes in the general population and provide information regarding uptake, safety, and outcomes in the real world.3 These population-based outcome studies may have important potential roles in clinical research. In this Commentary, we describe several examples that demonstrate the emerging roles of these studies and propose that they could be considered as adjuncts to and occasionally as substitutes for randomized controlled trials.
Contemporary Population-Based Outcomes Studies
Spironolactone in Patients With Heart Failure
In 1999, Pitt et al4 evaluated the addition of spironolactone to standard medical therapy for congestive heart failure. The authors reported that spironolactone was associated with an 11% absolute …
