RCTs are the gold standard for comparing therapies’ efficacy and safety but can have strict eligibility criteria and may not reflect real-life clinical practice. Electronic medical records such as those from the Predictive Health Intelligence Environment (PHIE) database may be used to assess clinical effectiveness in real-world populations missed by clinical trials. We describe here the proportion and characteristics of patients with T2DM who would have been excluded from RCTs using eligibility criteria adapted from the EDITION 1-3 and BRIGHT trials.
Records from people with T2DM, with a basal insulin prescription on the index date (1 March 2015) and ≥1 HbA1c value in the previous 12 months, were evaluated. Exclusion criteria included HbA1c <7.0 %/>10.0 %, systolic blood pressure >180 mmHg and/or diastolic blood pressure >95 mmHg, or key comorbidities (e.g., heart failure or stroke) in the previous 12 months.
Of 191,218 evaluated records, 157,873 (83%) met ≥1 exclusion criterion; only 33,345 (17%) could have been included in RCTs (Table). Excluded patients were older, with more previous hypoglycemia vs. included patients and were less likely to have HbA1c between 7 and 9 %.
This divergence in clinical characteristics and patient numbers highlights the need for real-world studies assessing clinical effectiveness in populations of interest typically excluded from RCTs.
D. Mauricio: Advisory Panel; Self; Merck Sharp & Dohme Corp., Sanofi. Speaker’s Bureau; Self; Almirall, S.A., Boehringer Ingelheim International GmbH, Lilly Diabetes, Merck Sharp & Dohme Corp., Novo Nordisk A/S, Sanofi, Urgo Medical. J. Westerbacka: Employee; Self; Sanofi. Stock/Shareholder; Self; Sanofi. C. Nicholls: Employee; Self; Sanofi. J. Wu: Employee; Self; Sanofi US. R. Gupta: None. A.A. Menon: None. B. Eliasson: Advisory Panel; Self; Amgen Inc., AstraZeneca, Merck Sharp & Dohme Corp. Speaker’s Bureau; Self; Boehringer Ingelheim International GmbH. Other Relationship; Self; Eli Lilly and Company, Novo Nordisk A/S, Sanofi.