Clinical trials: conventional or pragmatic?

Temporal trends in evidence supporting therapeutic interventions in heart failure and other European Society of Cardiology guidelines

“…The impact of treatment is usually assessed on a range of outcomes over a prolonged follow‐up period, linking data from high‐quality registries to electronic health records (EHRs) 42 . The availability of an operative EHR system is key to performing RRCT, and this still represents a primary limitation in several countries 43 . There have been several RRCTs in areas other than HF, in which the first RRCT entitled ‘Spironolactone Initiation Registry Randomized Interventional Trial in Heart Failure with Preserved Ejection Fraction (SPIRRIT‐HFpEF)’ should soon be completed in Sweden.…”

“…In this ongoing PCT, patients are enrolled and randomized at any time during an HF hospitalization; dosing and frequency changes to the randomized therapy after hospital discharge are at the discretion of the patient's usual outpatient clinicians; no study‐specific, in‐person follow‐up visits are planned but periodical phone interviews from a centralized call centre for data collection. Many experts believe that this ‘rough’ methodology intentionally chosen to ‘represent the real world’ may lead to findings that could simply represent the play of chance rather than true associations, and an intense debate over ‘minimum standards’ of future clinical trials generating real‐world evidence is ongoing 43,46 …”

“… 42 The availability of an operative EHR system is key to performing RRCT, and this still represents a primary limitation in several countries. 43 There have been several RRCTs in areas other than HF, in which the first RRCT entitled ‘Spironolactone Initiation Registry Randomized Interventional Trial in Heart Failure with Preserved Ejection Fraction (SPIRRIT‐HFpEF)’ should soon be completed in Sweden. This study will evaluate the impact of spironolactone, a drug generically approved for HF, in real‐world HFpEF patients, with the goal of validating the conflicting results of the original RCT TOPCAT.…”

“…Many experts believe that this ‘rough’ methodology intentionally chosen to ‘represent the real world’ may lead to findings that could simply represent the play of chance rather than true associations, and an intense debate over ‘minimum standards’ of future clinical trials generating real‐world evidence is ongoing. 43 , 46 …”