Abstract
There are many consequences of heart failure (HF), including symptoms, impaired health-related quality of life (HRQoL), and physical and social limitations (functional status). These have a substantial impact on patients' lives, yet are not routinely captured in clinical trials. Patient-reported outcomes (PROs) can quantify patients' experiences of their disease and its treatment. Steps can be taken to improve the use of PROs in HF trials, in regulatory and payer decisions, and in patient care. Importantly, PRO measures (PROMs) must be developed with involvement of patients, family members, and caregivers from diverse demographic groups and communities. PRO data collection should become more routine not only in clinical trials but also in clinical practice. This may be facilitated by the use of digital tools and interdisciplinary patient advocacy efforts. There is a need for standardization, not only of the PROM instruments, but also in procedures for analysis, interpretation and reporting PRO data. More work needs to be done to determine the degree of change that is important to patients and that is associated with increased risks of clinical events. This ‘minimal clinically important difference’ requires further research to determine thresholds for different PROMs, to assess consistency across trial populations, and to define standards for improvement that warrant regulatory and reimbursement approvals. PROs are a vital part of patient care and drug development, and more work should be done to ensure that these measures are both reflective of the patient experience and that they are more widely employed.
Original language | English (US) |
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Pages (from-to) | 478-487 |
Number of pages | 10 |
Journal | European Journal of Heart Failure |
Volume | 25 |
Issue number | 4 |
DOIs | |
State | Published - Apr 2023 |
Funding
This article was generated from discussions at the 18th Global Cardiovascular Clinical Trialists (CVCT) Forum held online in December 2021 ( https://www.globalcvctforum.com/ ). The CVCT Forum is a strategic workshop for high level dialogue between clinical trialists, industry representatives, regulatory authorities, and patients. The authors would like to thank Pauline Lavigne and Steven Portelance (unaffiliated, supported by the CVCT Forum) for contributions to writing, and editing the manuscript.
Keywords
- Clinical trials
- Health-related quality of life
- Heart failure
- Minimal clinically important difference
- Patient partner
- Patient-reported outcomes
ASJC Scopus subject areas
- Cardiology and Cardiovascular Medicine