Abstract

Background: Hyperlipidaemia is common and most patients can be managed with established oral therapies and lifestyle modifications. However, patients with complex high-risk lipid disorders require specialist input and access to novel therapies to achieve treatment targets. While new agents are progressing through late phase trials, the eligibility criteria of these studies may limit their applicability to real-world settings. Aim: Assessing the generalisability of novel lipid-lowering therapy (LLT) trials by mapping their eligibility criteria to a cohort of patients attending a specialist lipid clinic. Methods: An ethically approved study was conducted at a specialist clinic in St James’s Hospital, Dublin over 6 months, enrolling 370 patients. Demographics, clinical characteristics, lipid profiles, genetic testing results and LLT use were collected. A structured search of clinicaltrials.gov and euclinicaltrials.eu identified phase 3 pharmacological trials registered since 2018. Patient data were then mapped against trial eligibility criteria to estimate the proportion of clinic patients that would have been eligible for each study. Results: In total, 38 trials were identified, including 15 different novel LLTs. It was found that the percentage of the cohort (n = 370) that would have been eligible for these LLT trials ranged from 0% to 62.4%, with an average eligibility of 19% across the 38 trials. Further analysis of specific patient subgroups and corresponding trial categories was performed. Conclusions: The included novel LLTs trials are reasonably applicable to a specialist lipid clinic cohort, suggesting generalisability to a wider group of real-world patients seen in clinics. This study can help guide future trial design by aligning eligibility criteria more closely with real-world patient populations, ensuring safe and effective clinical practice.

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