TY - JOUR
T1 - Eligibility of Asian and European registry patients for phase III trials in heart failure with reduced ejection fraction
AU - Lim, Yvonne Mei Fong
AU - Asselbergs, Folkert W.
AU - Bagheri, Ayoub
AU - Denaxas, Spiros
AU - Tay, Wan Ting
AU - Voors, Adriaan
AU - Lam, Carolyn Su Ping
AU - Koudstaal, Stefan
AU - Grobbee, Diederick E.
AU - Vaartjes, Ilonca
N1 - Publisher Copyright:
© 2024 The Authors. ESC Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology.
PY - 2024/12
Y1 - 2024/12
N2 - Aims: Traditional approaches to designing clinical trials for heart failure (HF) have historically relied on expertise and past practices. However, the evolving landscape of healthcare, marked by the advent of novel data science applications and increased data availability, offers a compelling opportunity to transition towards a data-driven paradigm in trial design. This research aims to evaluate the scope and determinants of disparities between clinical trials and registries by leveraging natural language processing for the analysis of trial eligibility criteria. The findings contribute to the establishment of a robust design framework for guiding future HF trials. Methods and results: Interventional phase III trials registered for HF on ClinicalTrials.gov as of the end of 2021 were identified. Natural language processing was used to extract and structure the eligibility criteria for quantitative analysis. The most common criteria for HF with reduced ejection fraction (HFrEF) were applied to estimate patient eligibility as a proportion of registry patients in the ASIAN-HF (N = 4868) and BIOSTAT-CHF registries (N = 2545). Of the 375 phase III trials for HF, 163 HFrEF trials were identified. In these trials, the most frequently encountered inclusion criteria were New York Heart Association (NYHA) functional class (69%), worsening HF (23%), and natriuretic peptides (18%), whereas the most frequent comorbidity-based exclusion criteria were acute coronary syndrome (64%), renal disease (55%), and valvular heart disease (47%). On average, 20% of registry patients were eligible for HFrEF trials. Eligibility distributions did not differ (P = 0.18) between Asian [median eligibility 0.20, interquartile range (IQR) 0.08–0.43] and European registry populations (median 0.17, IQR 0.06–0.39). With time, HFrEF trials became more restrictive, where patient eligibility declined from 0.40 in 1985–2005 to 0.19 in 2016–2022 (P = 0.03). When frequency among trials is taken into consideration, the eligibility criteria that were most restrictive were prior myocardial infarction, NYHA class, age, and prior HF hospitalization. Conclusions: Based on 14 trial criteria, only one-fifth of registry patients were eligible for phase III HFrEF trials. Overall eligibility rates did not differ between the Asian and European patient cohorts.
AB - Aims: Traditional approaches to designing clinical trials for heart failure (HF) have historically relied on expertise and past practices. However, the evolving landscape of healthcare, marked by the advent of novel data science applications and increased data availability, offers a compelling opportunity to transition towards a data-driven paradigm in trial design. This research aims to evaluate the scope and determinants of disparities between clinical trials and registries by leveraging natural language processing for the analysis of trial eligibility criteria. The findings contribute to the establishment of a robust design framework for guiding future HF trials. Methods and results: Interventional phase III trials registered for HF on ClinicalTrials.gov as of the end of 2021 were identified. Natural language processing was used to extract and structure the eligibility criteria for quantitative analysis. The most common criteria for HF with reduced ejection fraction (HFrEF) were applied to estimate patient eligibility as a proportion of registry patients in the ASIAN-HF (N = 4868) and BIOSTAT-CHF registries (N = 2545). Of the 375 phase III trials for HF, 163 HFrEF trials were identified. In these trials, the most frequently encountered inclusion criteria were New York Heart Association (NYHA) functional class (69%), worsening HF (23%), and natriuretic peptides (18%), whereas the most frequent comorbidity-based exclusion criteria were acute coronary syndrome (64%), renal disease (55%), and valvular heart disease (47%). On average, 20% of registry patients were eligible for HFrEF trials. Eligibility distributions did not differ (P = 0.18) between Asian [median eligibility 0.20, interquartile range (IQR) 0.08–0.43] and European registry populations (median 0.17, IQR 0.06–0.39). With time, HFrEF trials became more restrictive, where patient eligibility declined from 0.40 in 1985–2005 to 0.19 in 2016–2022 (P = 0.03). When frequency among trials is taken into consideration, the eligibility criteria that were most restrictive were prior myocardial infarction, NYHA class, age, and prior HF hospitalization. Conclusions: Based on 14 trial criteria, only one-fifth of registry patients were eligible for phase III HFrEF trials. Overall eligibility rates did not differ between the Asian and European patient cohorts.
KW - Clinical trials
KW - Disease registry
KW - Generalizability
KW - Heart failure
KW - Patient eligibility
UR - http://www.scopus.com/inward/record.url?scp=85197803423&partnerID=8YFLogxK
U2 - 10.1002/ehf2.14751
DO - 10.1002/ehf2.14751
M3 - Article
AN - SCOPUS:85197803423
SN - 2055-5822
VL - 11
SP - 3559
EP - 3571
JO - ESC heart failure
JF - ESC heart failure
IS - 6
M1 - doi.org/10.1002/ehf2.14751
ER -