# A landscape review to identify what matters to patients with thrombotic cardiovascular diseases and patient-reported outcome instruments which can be used to capture the patient experience

## TL;DR

This study reviews what matters to patients with thrombotic cardiovascular diseases and identifies patient-reported outcome instruments that can capture their experiences.

## Contribution

The study provides a landscape review to identify patient priorities and suitable PRO instruments for thrombotic cardiovascular diseases.

## Key findings

- Qualitative literature identified key symptoms and HRQoL impacts reported by patients.
- Generic PROs are used, but disease-specific instruments meeting FDA guidance are lacking.
- HTA agencies discuss PROs but lack specific recommendations for thrombotic indications.

## Abstract

Thrombotic cardiovascular diseases profoundly impact patients’ health-related quality of life (HRQoL). However, patient-reported outcome (PRO) instruments that are disease-specific or antithrombotic-treatment focused, developed according to US Food and Drug Administration (FDA) guidance on PROs, and can be used in clinical trials, are lacking. The aim of this study was to understand concepts important to patients diagnosed with coronary artery disease (CAD) or acute coronary syndrome (ACS), atrial fibrillation (AF), or stroke who require antithrombotic treatment for reducing risk of future thrombotic events (indications being evaluated for an investigational new drug), identify PROs that measure relevant symptoms and impacts, and determine acceptability of PROs from a health technology assessment (HTA) perspective.

A landscape review, conducted between January 2009 and October 2020, included a search of qualitative literature (OVID), a review of PRO instruments using multiple sources (e.g., OVID and clinical trials databases), and a survey of HTA decisions for antithrombotic medications.

The qualitative literature review identified 27 publications used to develop a high-level conceptual summary of symptoms and HRQoL impacts reported by patients. The instrument landscape review indicated that generic PROs have been utilized for thrombotic indications, but disease-specific, fit-for-purpose instruments are lacking, and the HTA review revealed that although HTA agencies discussed PRO instruments, evidence of specific recommendations was not found.

To ensure patients’ experiences, perspectives, and priorities are incorporated into drug development and evaluation, a core set of PROs for thrombotic indications that meet health authority guidance and are acceptable to HTA agencies is needed.

The online version contains supplementary material available at 10.1007/s11136-024-03790-1.