Figures
- Figure 1
Studies have shown that classical efficacy RCTs exclude about 90% for a) asthma and 95% for b) COPD routine care populations due to strict inclusion and exclusion criteria. a) For asthma clinical-trial patients criteria included: visual analogue scale (VAS) <2; historical reversibility in FEV 12% within the last 12 months; absence of significant co-morbidity; nonsmoker or if previous smoker (XS) a smoke burden <10 pack-years; regular use of ICS; symptomatic asthma (defined as either the use of short acting β2-agonist daily or nocturnal awakening due to asthma at least once a week). b) For COPD clinical-trial patients criteria included: VAS >7.5, absence of significant co-morbidity; smoker (S) or XS; a smoke burden of >15 pack-years; no history of hay fever indicating presence of atopy. Reproduced from [5] with permission from the publisher.
- Figure 2
Integrated research framework, bounded by population and ecology of care axes running from highly selected to managed care populations (Y-axis) and from highly interventional to observational study design approaches (X-axis). Adapted from [10] with permission from the publisher.
- Figure 3
Long-term adherence to ICS. Short-term cRCTs will miss the interaction between real-life adherence and treatment outcomes. Reproduced from [20] with permission from the publisher.
- Figure 4
The COMPACT study demonstrated the difference in outcomes associated with management of only lower airways inflammation (budesonide) compared with systemic (upper and lower airways) inflammation management (montelukast) in asthmatic patients without (a) and with (b) rhinitis. Reproduced from [31, 32] with permission from the publishers.
- Figure 5
Mean (95% CI) difference between non-smokers and smokers with asthma, Suggesting alternatives to higher-dose ICS may be required. *: p≤0.01 for smokers versus non-smokers. Reproduced from [36] with permission from the publisher.
Tables
- Table 1
Common similarities and differences between the design of classical and pragmatic RCTs
Feature of trial design Classical RCT Pragmatic RCT Randomisation Yes Yes Control group Yes Yes Setting/ecology of care • Highly controlled
• Specialised centres (secondary or tertiary)• Pragmatically controlled
• Usual care (> primary care)Patient population • Highly selected
• Confirmed diagnosis
Narrow (“pure”) population• Pragmatically selected
• Clinical diagnosis
Broad (“real-life”) populationInclusion/exclusion criteria Many Few Adherence Very good (stimulated and monitored) Low (real-world adherence) Therapy • Blinded (single- or double-blind); or
• Open-labelUsually open-label to allow for effects of different technologies e.g. device or mode of administration Comparator Placebo; and/or active treatment Active treatment Outcome Efficacy Real-life effectiveness (comparative effectiveness) Safety Usually short-term Short-term and long-term
