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Apples and oranges? Interpreting success in HIV prevention trials
Lori L Heise1, Charlotte Watts, Anna Foss
1Social and Mathematical Epidemiology Group, Department of Global Health and Development, London School of Hygiene and Tropical Medicine,WC1H 9SH London, UK. lori.heise@lshtm.ac.uk
Background:
In the last decade, several large-scale, clinical trials evaluating the efficacy of novel HIV prevention products have been completed, and eight are currently underway or about to be reported. Little attention has been given in the literature to the level of protection sufficient to warrant introduction, and there is concern that using the term "efficacy" to describe the effect of user-controlled methods such as microbicides may mislead policymakers.
Design:
We review how the fields of family planning, vaccine science and mathematical modelling understand and use the terms efficacy and effectiveness, and explore with simple mathematical models how trial results of user-controlled products relate to common understandings of these terms.
Results:
Each field brings different assumptions, a different evidence base and different expectations to interpretations of efficacy and effectiveness - a reality that could cloud informed assessment of emerging data.
Conclusion:
When making judgments on the utility of new health technologies, it is important to use standards that yield appropriate comparisons for the innovation and that take into account the local epidemic and available alternatives.
Insights
Understanding HIV prevention product efficacy is crucial for policymakers. Different scientific fields interpret "efficacy" and "effectiveness" uniquely, potentially impacting assessments of new HIV prevention methods.
Area of Science:
- Public Health
- Epidemiology
- Health Technology Assessment
Background:
- Numerous clinical trials for novel HIV prevention products have concluded or are nearing completion.
- There is a lack of consensus on the required level of protection for introducing new HIV prevention strategies.
- The term "efficacy" may be misapplied to user-controlled methods like microbicides, potentially misleading policymakers.
Purpose of the Study:
- To examine the distinct interpretations of "efficacy" and "effectiveness" across family planning, vaccine science, and mathematical modeling.
- To analyze how trial results for user-controlled HIV prevention products align with common understandings of these terms using mathematical models.
Main Methods:
- Comparative review of terminology and assumptions in family planning, vaccine science, and mathematical modeling.
- Application of simple mathematical models to explore the relationship between trial outcomes and conceptual definitions of efficacy and effectiveness.
Main Results:
- Each scientific field utilizes different assumptions, evidence bases, and expectations when interpreting efficacy and effectiveness.
- Divergent interpretations across fields can complicate the informed assessment of emerging data on HIV prevention products.
Conclusions:
- Judgments on the utility of new health technologies require appropriate comparative standards.
- Consideration of the local epidemic context and available alternative interventions is essential for evaluating new health technologies.
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