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Published on: February 11, 2019
Clinical trial efficacy: what does it really tell you?
1National Jewish Medical and Research Center, and University of Colorado Health Sciences Center, Denver, Colorado 80206, USA.
The Journal of Allergy and Clinical Immunology
|October 31, 2003
Summary
Understanding clinical trial outcomes is key to assessing drug effectiveness. Objective measures like lung function (FEV1) are common, but health outcomes also provide crucial real-world insights for asthma treatments.
Area of Science:
- Clinical pharmacology
- Respiratory medicine
- Biostatistics
Background:
- Clinical trials aim to evaluate drug efficacy, necessitating an understanding of how study outcomes reflect real-world effectiveness.
- Assessing treatment impact on morbidity and mortality is a high priority in clinical research.
Purpose of the Study:
- To review the types of outcomes used in clinical trials for evaluating drug efficacy, particularly for asthma medications.
- To highlight the importance of objective measures and health outcomes in determining a drug's real-life effectiveness.
Main Methods:
- Review of common outcome measures in clinical trials, categorized as subjective, objective, and health-related.
- Discussion of specific objective measures, including lung function (e.g., FEV1), bronchial hyperresponsiveness, and inflammatory markers.
- Identification of key health outcomes such as reduced emergency care, hospitalizations, and mortality.
Main Results:
- Clinical trials commonly employ subjective (symptom scores, quality of life) and objective measures (lung function, inflammation markers).
- Forced expiratory volume at one second (FEV1) is the gold standard for efficacy, though adjustments may be needed for pediatric populations.
- Health outcomes, including reduced need for systemic glucocorticoids, emergency care, hospitalizations, and mortality, are increasingly important secondary or primary measures.
Conclusions:
- A comprehensive evaluation of drug efficacy requires considering a range of outcomes, from objective physiological measures to real-world health impacts.
- Further research is needed to refine outcome measures, especially in pediatrics, and to predict treatment response.
- Studies focusing on morbidity and mortality outcomes are critical for understanding the full impact of asthma therapies.
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