Evaluating the effectiveness of asthma treatment in real-life practice.
OBJECTIVE
The randomized controlled trial (RCT) is considered the gold standard methodology for determining the efficacy and tolerability of new treatments. However, RCTs cannot provide information on the effectiveness of interventions as they are used in real life. This study was conducted to investigate the effectiveness of montelukast, a leukotriene receptor antagonist, in the real-world management of asthma, through a large-scale, retrospective, observational study: the National Montelukast Survey.


METHODS
In order to ensure a robust methodology for the National Montelukast Survey we performed three pilot studies involving a total of almost 400 patients. During the pilots, the design of the study was extensively modified from a simple prescriber questionnaire used in the first pilot to the 'triangulated' methodology encompassing the perspectives of patient, prescriber and independent observer used in the National Montelukast Survey. Good levels of interobserver agreement confirmed the robustness of the final methodology.


CONCLUSIONS
Achieving a robust methodology was dependent on the extensive piloting. It is possible to collect reliable observational data relating to treatment outcomes. We believe our methods are likely to have more widespread applicability and offer a potential improvement over postmarketing surveillance.
Pearson
Mike
M
University Hospital Aintree, Liverpool, and The Royal College of Physicians of London, UK. Michaeal.pearson@rcplondon.ac.uk
Barnes
Neil
N
Thomas
Mike
M
Tate
Helen
H
Simnett
Sarah
S
D000085:Acetates / Q000627:therapeutic use*; D018927:Anti-Asthmatic Agents / Q000627:therapeutic use*; D001249:Asthma / Q000188:drug therapy*; D003521:Cyclopropanes; D019317:Evidence-Based Medicine; D006801:Humans; D010865:Pilot Projects; D011804:Quinolines / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic; D013440:Sulfides; D006113:United Kingdom
D000085:Acetates; D018927:Anti-Asthmatic Agents; D003521:Cyclopropanes; D011804:Quinolines; D013440:Sulfides; C093875:montelukast