The importance of patient-reported outcomes in clinical trials and strategies for future optimization

Mercieca‐Bebber, Rebecca; King, Madeleine; Calvert, Melanie; Stockler, Martin R.; Friedlander, Michael

doi:10.2147/prom.s156279

Cited by 378 publications

(361 citation statements)

References 65 publications

Supporting

Mentioning

353

Contrasting

Unclassified

Order By: Relevance

“…Indeed, with regard to ERNs, two key drivers have been identified:, on the one hand, medical experts and scientists (because of the existing interdependence of research and care in this highly specialized field) and on the other hand patients and their families, including the patient organizations [379]. In the set-up of clinical trials, patient reported outcomes (PRO) are also increasingly gaining importance [380].…”

Section: How Can We Keep In Touch With Changes Of Marfan Syndrome?mentioning

confidence: 99%

Features of Marfan syndrome not listed in the Ghent nosology – the dark side of the disease

Kodolitsch

Demolder

Girdauskas

et al. 2019

Expert Review of Cardiovascular Therapy

View full text Add to dashboard Cite

Introduction: The revised Ghent nosology presents the classical features of Marfan syndrome. However, behind its familiar face, Marfan syndrome hides less well-known features. Areas covered: The German Marfan Organization listed unusual symptoms and clinical experts reviewed the literature on clinical features of Marfan syndrome not listed in the Ghent nosology. Thereby we identified the following features: (1) bicuspid aortic valve, mitral valve prolapse, pulmonary valve prolapse, tricuspid valve prolapse, (2) heart failure and cardiomyopathy, (3) supraventricular arrhythmia, ventricular arrhythmia, and abnormal repolarization, (4) spontaneous coronary artery dissection, anomalous coronary arteries, and atherosclerotic coronary artery disease, tortuosity-, aneurysm-, and dissection of large and medium-sized arteries, (5) restrictive lung disease, parenchymal lung disease, and airway disorders, (6) obstructive-and central sleep apnea, (7) liver and kidney cysts, biliary tract disease, diaphragmatic hernia, and adiposity, (8) premature labor, and urinary incontinence, (9) myopathy, reduced bone mineral density, and craniofacial manifestations, (10) atrophic scars, (11) caries, and craniomandibular dysfunction, (12) headache from migraine and spontaneous cerebrospinal fluid leakage, (13) cognitive dysfunction, schizophrenia, depression, fatigue, and pain, (14) and activated fibrinolysis, thrombin, platelets, acquired von Willebrand disease, and platelet dysfunction. Expert commentary: Future research, nosologies, and guidelines may consider less well-known features of Marfan syndrome. ARTICLE HISTORY

show abstract

Section: How Can We Keep In Touch With Changes Of Marfan Syndrome?mentioning

confidence: 99%

Features of Marfan syndrome not listed in the Ghent nosology – the dark side of the disease

Kodolitsch

Demolder

Girdauskas

et al. 2019

Expert Review of Cardiovascular Therapy

View full text Add to dashboard Cite

show abstract

“…However, it might be challenging to identify the correct PROMs to use in a study, to administer them at the right time, and to minimize and deal with missing data. 7,21 Many PROMs are tailored to measure the patient perspective in particular areas, for example, symptom-specific (eg, pain, fatigue, and anxiety) and disease-specific instruments that are most relevant to a patient population. In contrast, there are generic instruments that do not focus on a particular disease or symptom but aim to assess HRQOL in general, allowing comparisons between different conditions, and providing valuable data for economic evaluation…”

Section: Introductionmentioning

confidence: 99%

Patient‐reported outcome measures in studies of myelodysplastic syndromes and acute myeloid leukemia: Literature review and landscape analysis

Stauder

Lambert

Desruol‐Allardin

et al. 2020

European J of Haematology

View full text Add to dashboard Cite

This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made. AbstractObjectives: This study aims to describe the use of patient-reported outcome measures (PROMs) in myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) studies and the PROMs landscape. Methods:A comprehensive literature review was performed in Medline/Embase (since 2000) and ClinicalTrials.gov (since 2013) to identify PROMs used in MDS and AML clinical studies. Additionally, PROMs included in approved drug labels since 2000 were reviewed. Results: Overall, 112 different PROMs were used in 168 published MDS studies and 152 PROMs were used in 172 AML studies. From ClinicalTrials.gov, 16 different PROMs were used in 22 ongoing registered studies in MDS, and 24 were reported in 41 AML studies. The most frequently used PROMs were cancer-specific (eg, EORTC QLQ-C30, FACT-An) or generic (SF-36, EQ-5D) instruments, whereas MDS-and AML-specific instruments (eg, QUALMS and QOL-E in MDS; FACT-Leu and EORTC QLQ-Leu in AML) were used in a minority of studies. Two EMA-approved drugs for MDS included PROMs in their label. EORTC QLQ-C30 is by far the most frequently used cancer-specific PROM in both MDS and AML studies. Conclusions: This research indicated an underuse of AML/MDS-specific PROMs for these two indications in clinical studies and labeling claims. However, AML/MDSspecific instruments in development might be considered in future studies. K E Y W O R D S acute myeloid leukemia, myelodysplastic syndromes, quality of life | 477 STAUDER ET Al.

show abstract

“…These evaluations, captured electronically or in person, in formats ranging from questionnaires to wearable devices, serve to provide a unique record of the patient's lived experience of a disease, its treatment and management, and the impact these may have on function and health‐related quality of life (HRQoL) . The inclusion of PROs as trial endpoints aims to ensure a comprehensive assessment of burden of disease and the impact of an intervention . In this way, PROs have established themselves as a central and indispensable component of the evidence evaluating medicines and can be used by clinicians, patients and policy makers to assess treatment choice, shape guidelines and enable regulatory and policy decisions based on the benefits and costs of treatment .…”

Section: Introductionmentioning

confidence: 99%

“…Reasons cited for the poor quality of PROs in some cancer clinical trials include methodological, cultural and practical issues . Choice and content of PRO measures are not always relevant to the aim of the study or may not reflect what is important to the patient . PROs are all too often dismissed as subjective in contrast to laboratory findings and as less important than survival endpoints .…”

Section: Introductionmentioning

confidence: 99%

“…In its guidance on PROs in regulatory submissions, the European Medicines Agency (EMA) refers to a range of issues including bias, timing of assessments and missing data . The practicalities of implementation may not be conducive to patients completing a PRO measure or to administrative staff supporting them in doing so . In some cases, compliance with completion of PRO measures is so poor that the data are not even analysed .…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Including the patient voice in the development and implementation of patient‐reported outcomes in cancer clinical trials

Addario

Geißler²,

Horn³

et al. 2019

Health Expectations

View full text Add to dashboard Cite

Portfolio Chair 4 | Deborah Maskens MA, MSM, Vice Chair 5 | Kathy Oliver BA, Chair and Co-Director 6 | Ananda Plate Chief Executive Officer 7 | Erin Schwartz MSW, VP of Global Engagement 8 | Nicole Willmarth PhD, Chief Mission Officer 9This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. Abstract Context: Patient-reported outcomes (PROs) are used in parallel with clinical evidence to inform decisions made by industry, clinicians, regulators, health technology assessment bodies and other health-care decision-makers. In addition, PRO data can also guide shared decision making and individual patient choice. Yet, the quality of many PROs in cancer clinical trials is suboptimal and requires improvement to add value to health care and policy decision making. Objective: To show how the integration of the patient and/or patient advocate at all stages of PRO development can help to realize the full potential of PROs. Methods: We examined the literature to show that the patient voice is often absent from the planning and implementation of PROs in cancer clinical trials. Good practice examples from the literature were combined with guideline recommendations, training or educational resources, and our own experience to create detailed practical steps for the inclusion of patients and/or patient advocates throughout PRO development.Results: Patient or patient advocates can play an active role in shaping PROs that are meaningful to the patient. They can contribute to content, choice of medium and implementation in a way that may support PRO completion and minimize missing data. Patients and their advocates can work to ensure PRO findings are disseminated appropriately in a way that is accessible to patients. Conclusion:This practical guidance aims to optimize PRO development and implementation in clinical trials, resulting in robust, relevant data that reflect the patient experience and that support decisions made by all stakeholders involved in research and health care.

show abstract

The importance of patient-reported outcomes in clinical trials and strategies for future optimization

Cited by 378 publications

References 65 publications

Features of Marfan syndrome not listed in the Ghent nosology – the dark side of the disease

Features of Marfan syndrome not listed in the Ghent nosology – the dark side of the disease

Patient‐reported outcome measures in studies of myelodysplastic syndromes and acute myeloid leukemia: Literature review and landscape analysis

Including the patient voice in the development and implementation of patient‐reported outcomes in cancer clinical trials

Contact Info

Product

Resources

About