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Expanding Research Horizons – Beyond Clinical Trials

How do we categorize different kinds of research – and why?

In new drug development we categorize research by stage: Preclinical, Phase 1, Phase 2 and Phase 3 clinical trials.  The research question that we ask at every stage is different: does the drug work in a disease model, is it safe in humans, does it work in human disease, and does it have clinically meaningful benefit?

At the Infectious Diseases Institute (IDI), the purpose of research is to provide evidence that informs health care policy1 and optimizes treatment of disease.  I’ve developed a framework for categorizing types of research that’s relevant to the questions IDI asks in seeking to understand and treat disease and to implement its recommendations both in Uganda and via the World Health Organization.


When little is known about a disease, cross sectional surveys can help understand the epidemiology, or incidence and type of disease.  An observational cohort study where patients are closely monitored over time while receiving standard of care treatment can help researchers identify areas of unmet need.


Classical randomized controlled clinical trials fit in this category, where a new treatment is compared with standard of care treatment in a carefully selected patient population to see if the new treatment is superior.


It’s not enough for IDI to prove that a new treatment works; the next step is to demonstrate that it can be translated into real life, and show that the treatment works as well in a rural health center as it does at a center of excellence like IDI. Implementation or Operational research is used to demonstrate that a new health care policy can be scaled up nationally.

Cost Effectiveness3

In a resource-limited setting it’s vital that new treatments are shown to improve not only the benefit to patients but also to lower the cost, to maximize the overall health care bang for the buck.  Cost effectiveness or pharmacoeconomic research helps to justify spending money on newer treatments that provide superior benefit to patients.


  1. Is Evidence-Based Government Possible?” Philip Davies, 2004
  2. WHO Alliance for Health Policy and Systems Research: Implementation Research Platform
  3. Evidence from Cost-Effectiveness ResearchNoyes and Holloway, NeuroRx Vol 1 (3), July 2004


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