Ethical Considerations for the Inclusion of Patient-Reported Outcomes in Clinical Research

Abstract

IMPORTANCE Patient-reported outcomes (PROs) can inform health care decisions, regulatory decisions, and health care policy. They also can be used for audit/benchmarking and monitoring symptoms to provide timely care tailored to individual needs. However, several ethical issues have been raised in relation to PRO use. OBJECTIVE To develop international, consensus-based, PRO-specific ethical guidelines for clinical research. EVIDENCE REVIEWThe PRO ethics guidelines were developed following the Enhancing the Quality and Transparency of Health Research (EQUATOR) Network's guideline development framework. This included a systematic review of the ethical implications of PROs in clinical research. The databases MEDLINE (Ovid), Embase, AMED, and CINAHL were searched from inception until March 2020. The keywords patient reported outcome* and ethic* were used to search the databases. Two reviewers independently conducted title and abstract screening before full-text screening to determine eligibility. The review was supplemented by the SPIRIT-PRO Extension recommendations for trial protocol. Subsequently, a 2-round international Delphi process (n = 96 participants; May and August 2021) and a consensus meeting (n = 25 international participants; October 2021) were held. Prior to voting, consensus meeting participants were provided with a summary of the Delphi process results and information on whether the items aligned with existing ethical guidance.FINDINGS Twenty-three items were considered in the first round of the Delphi process: 6 relevant candidate items from the systematic review and 17 additional items drawn from the SPIRIT-PRO Extension. Ninety-six international participants voted on the relevant importance of each item for inclusion in ethical guidelines and 12 additional items were recommended for inclusion in round 2 of the Delphi (35 items in total). Fourteen items were recommended for inclusion at the consensus meeting (n = 25 participants). The final wording of the PRO ethical guidelines was agreed on by consensus meeting participants with input from 6 additional individuals. Included items focused on PRO-specific ethical issues relating to research rationale, objectives, eligibility requirements, PRO concepts and domains, PRO assessment schedules, sample size, PRO data monitoring, barriers to PRO completion, participant acceptability and burden, administration of PRO questionnaires for participants who are unable to self-report PRO data, input on PRO strategy by patient partners or members of the public, avoiding missing data, and dissemination plans. CONCLUSIONS AND RELEVANCEThe PRO ethics guidelines provide recommendations for ethical issues that should be addressed in PRO clinical research. Addressing ethical issues of PRO clinical research has the potential to ensure high-quality PRO data while minimizing participant risk, burden, and harm and protecting participant and researcher welfare.

Ethical and practical considerations related to data sharing when collecting patient-reported outcomes in care-based child health research

“…There has been some discussion about ethical collection and use of PROs in clinical care, focused on the integrating PRO data collected during research into patients' electronic health records, with options ranging from no disclosure to a detailed informed consent process including an opt-in requirement [22][23][24]. A set of ethical guidelines for research including PROs has recently been published [25], but they do not specify how research PROs can be integrated into care [25]. In pediatrics, limited attention has been paid to ethical questions surrounding PRO collection and data sharing in cohort or registry studies where consent and assent, child and family preferences, and clinical practice guidelines may change over time (i.e., in accordance with a child's age or capacity to provide consent).…”

Key considerations to reduce or address respondent burden in patient-reported outcome (PRO) data collection

“…Increasingly, research ethics committees and institutional review boards, in their assessment of PRO research, are considering the burden of PRO collection on study participants 96 . An objective assessment of the potential burden could assist such committees in their decision-making process.…”

Patient-Reported Outcome Measures in Clinical Research

“…8 A guideline for the ethical considerations in the use of patient-reported outcomes has been published. 9 How Was a Patient-Reported Outcome Measure Applied in This Case?…”

“…There are international guidelines for high-quality trial protocols involving patient-reported outcome measures and for reporting the results of patient-reported outcome measure–based end points in clinical trials . A guideline for the ethical considerations in the use of patient-reported outcomes has been published …”