TY - JOUR
T1 - Patient-reported outcome measures and patient engagement in heart failure clinical trials
T2 - multi-stakeholder perspectives
AU - Zannad, Faiez
AU - Alikhaani, Jacqueline
AU - Alikhaani, Sadegh
AU - Butler, Javed
AU - Gordon, Jason
AU - Jensen, Klaus
AU - Khatib, Rani
AU - Mantovani, Lorenzo
AU - Martinez, Robin
AU - Moore, Wanda F.
AU - Murakami, Masahiro
AU - Roessig, Lothar
AU - Stockbridge, Norman
AU - Van Spall, Harriette G.C.
AU - Yancy, Clyde
AU - Spertus, John A.
N1 - Funding Information:
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.
Publisher Copyright:
© 2023 European Society of Cardiology.
PY - 2023/4
Y1 - 2023/4
N2 - 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.
AB - 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.
KW - Clinical trials
KW - Health-related quality of life
KW - Heart failure
KW - Minimal clinically important difference
KW - Patient partner
KW - Patient-reported outcomes
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U2 - 10.1002/ejhf.2828
DO - 10.1002/ejhf.2828
M3 - Review article
C2 - 36924142
AN - SCOPUS:85150898301
SN - 1388-9842
VL - 25
SP - 478
EP - 487
JO - European Journal of Heart Failure
JF - European Journal of Heart Failure
IS - 4
ER -