Recommendations to address respondent burden associated with patient-reported outcome assessment

Aiyegbusi, Olalekan Lee; Cruz Rivera, Samantha; Roydhouse, Jessica; Kamudoni, Paul; Alder, Yvonne; Anderson, Nicola; Baldwin, Robert Mitchell; Bhatnagar, Vishal; Black, Jennifer; Bottomley, Andrew; Brundage, Michael; Cella, David; Collis, Philip; Davies, Elin-Haf; Denniston, Alastair K.; Efficace, Fabio; Gardner, Adrian; Gnanasakthy, Ari; Golub, Robert M.; Hughes, Sarah E.; Jeyes, Flic; Kern, Scottie; King-Kallimanis, Bellinda L.; Martin, Antony; McMullan, Christel; Mercieca-Bebber, Rebecca; Monteiro, Joao; Peipert, John Devin; Quijano-Campos, Juan Carlos; Quinten, Chantal; Rantell, Khadija Rerhou; Regnault, Antoine; Sasseville, Maxime; Schougaard, Liv Marit Valen; Sherafat-Kazemzadeh, Roya; Snyder, Claire; Stover, Angela M.; Verdi, Rav; Wilson, Roger; Calvert, Melanie J.

doi:10.1038/s41591-024-02827-9

Cited by 12 publications

(3 citation statements)

References 49 publications

(83 reference statements)

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“…It should also be noted that while it is important to understand a new treatment regimen from a patient’s perspective [ 3 ], the collection of such information can also place an additional burden on patients. In this study, patients were asked to complete 8 questionnaires, and questionnaire burden may have contributed to the missing data [ 36 , 37 ]. Similar problems were found in other studies, such as the Expanding Communications on Hemophilia A Outcomes registry, which had to be closed after 2 years due to objections of investigators and patients to the burden of multiple PROs, among other challenges [ 38 ].…”

Section: Discussionmentioning

confidence: 99%

Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study

Tran,

von Mackensen,

Abraham

et al. 2024

Research and Practice in Thrombosis and Haemostasis

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Section: Discussionmentioning

confidence: 99%

Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study

Tran,

von Mackensen,

Abraham

et al. 2024

Research and Practice in Thrombosis and Haemostasis

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“…This latter aspect needs to be considered as it may negatively impact rates of missing PRO data 68 and, in this respect, we note that recommendations to address respondent burden have been recently published. 69 This issue is relevant in the context of MDS trials, which often involve older patients and/or patients with fatigue who may have trouble with electronic interfaces and frequent requests to fill out questionnaires.…”

Section: Key Selected Aspects To Consider During Protocol Development...mentioning

confidence: 99%

Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Efficace,

Buckstein,

Abel

et al. 2024

HemaSphere

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Notable treatment advances have been made in recent years for patients with myelodysplastic syndromes/neoplasms (MDS), and several new drugs are under development. For example, the emerging availability of oral MDS therapies holds the promise of improving patients' health‐related quality of life (HRQoL). Within this rapidly evolving landscape, the inclusion of HRQoL and other patient‐reported outcomes (PROs) is critical to inform the benefit/risk assessment of new therapies or to assess whether patients live longer and better, for what will likely remain a largely incurable disease. We provide practical considerations to support investigators in generating high‐quality PRO data in future MDS trials. We first describe several challenges that are to be thoughtfully considered when designing an MDS‐focused clinical trial with a PRO endpoint. We then discuss aspects related to the design of the study, including PRO assessment strategies. We also discuss statistical approaches illustrating the potential value of time‐to‐event analyses and their implications within the estimand framework. Finally, based on a literature review of MDS randomized controlled trials with a PRO endpoint, we note the PRO items that deserve special attention when reporting future MDS trial results. We hope these practical considerations will facilitate the generation of rigorous PRO data that can robustly inform MDS patient care and support treatment decision‐making for this patient population.

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“…An excessive respondent burden may result in unwillingness to complete the questionnaires and, ultimately, will result in missing data. Therefore, extensive, and time-consuming surveys are generally unpractical ( 88 , 89 ).…”

Section: Challenges and Opportunitiesmentioning

confidence: 99%

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

Almeida,

Umuhire,

Gonzalez-Quevedo

et al. 2024

Front. Med.

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Patient experience data (PED), provided by patients/their carers without interpretation by clinicians, directly capture what matters more to patients on their medical condition, treatment and impact of healthcare. PED can be collected through different methodologies and these need to be robust and validated for its intended use. Medicine regulators are increasingly encouraging stakeholders to generate, collect and submit PED to support both scientific advice in development programs and regulatory decisions on the approval and use of these medicines. This article reviews the existing definitions and types of PED and demonstrate the potential for use in different settings of medicines’ life cycle, focusing on Patient-Reported Outcomes (PRO) and Patient Preferences (PP). Furthermore, it addresses some challenges and opportunities, alluding to important regulatory guidance that has been published, methodological aspects and digitalization, highlighting the lack of guidance as a key hurdle to achieve more systematic inclusion of PED in regulatory submissions. In addition, the article discusses opportunities at European and global level that could be implemented to leverage PED use. New digital tools that allow patients to collect PED in real time could also contribute to these advances, but it is equally important not to overlook the challenges they entail. The numerous and relevant initiatives being developed by various stakeholders in this field, including regulators, show their confidence in PED’s value and create an ideal moment to address challenges and consolidate PED use across medicines’ life cycle.

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Recommendations to address respondent burden associated with patient-reported outcome assessment

Abstract: Revision of recommendations and elaborations Suggestion to merge 2 recommendations Final agreed consensus statement with 19 recommendationsRecommendations proposed by the Delphi participants from Round 1 = 7 recommendations

Cited by 12 publications

References 49 publications

Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study

Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study

Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

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