Real-World Data and Randomised Controlled Trials: The Salford Lung Study.

Area of Science:

• Respiratory Medicine
• Clinical Trial Design
• Pharmacoeconomics

Background:

• Traditional double-blind randomized controlled trials (DBRCTs) assess treatment efficacy under controlled conditions with selected patients, limiting real-world applicability.
• Pragmatic effectiveness trials aim to measure treatment benefit in routine clinical practice, reflecting diverse patient populations and natural behaviors of patients and healthcare professionals (HCPs).

Purpose of the Study:

• To evaluate the real-world effectiveness of fluticasone furoate/vilanterol in patients with chronic obstructive pulmonary disease and asthma.
• To demonstrate the feasibility and value of a large-scale, prospective, pragmatic randomized controlled trial (RCT) in a real-world setting.

Main Methods:

• The Salford Lung Study (SLS) was a phase III, pragmatic RCT utilizing electronic health records to assess medication effectiveness.
• Engaged general practitioners and community pharmacists in clinical research to enhance real-world data collection and patient engagement.
• Focused on maximizing external validity by mirroring routine clinical practice while maintaining scientific rigor and robust safety data interpretation.

Main Results:

• The SLS successfully evaluated a pre-licensed medication in a real-world setting, providing effectiveness data representative of broader patient populations.
• Demonstrated the importance of integrating clinical research within routine care pathways.
• Highlighted the value of collaborative engagement with HCPs and patients in large-scale pragmatic trials.

Conclusions:

• Future pragmatic effectiveness RCTs should align trial infrastructure with routine care.
• Broad patient recruitment is crucial for generalizability and applicability of findings.
• Patient and HCP engagement, alongside careful study design with relevant outcomes and pre-planned analyses, ensures robust and meaningful trial results.