Global heart failure epidemiology versus enrolment in pivotal trials: A formidable mismatch.

ABSTRACT:

Aims

Randomized clinical trials (RCTs) that inform international clinical practice guidelines should adequately represent regions burdened with disease. We aimed to assess the geographic representativeness of pivotal heart failure (HF) RCTs using two methodological approaches.

Methods and results

We assessed the global geographic distribution of HF cases using the Global Burden of Disease 2021 dataset. We then assessed the geographic representativeness of pivotal phase 3 RCTs that have shaped international guidelines using two metrics: the representation index (RI), a ratio of regional trial sites to disease distribution, and the participant-to-prevalence ratio (PPR), a ratio of regional trial participants to disease distribution. In 2021, there were 55.4 million people with

SUBMITTER: Baudry G 

PROVIDER: S-EPMC12370579 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature