Patterns of reporting health-related quality of life outcomes in randomized clinical trials: implications for clinicians and quality of life researchers

Brundage, Michael; Bass, B.; Davidson, Judith; Queenan, John; Bezjak, Andrea; Ringash, Jolie; Wilkinson, Anna N.; Feldman‐Stewart, Deb

doi:10.1007/s11136-010-9793-3

Cited by 131 publications

(104 citation statements)

References 26 publications

(33 reference statements)

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“…Prior reviews indicated suboptimal PRO reporting [10,11], which limited the potential for PRO evidence to impact practice, thus representing a waste of research effort. CONSORT-PRO aims to facilitate translation of high-quality PRO evidence to clinical practice and policy [12].…”

Section: Preliminary Evidence On the Uptake Use And Benefits Of The mentioning

confidence: 99%

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

et al. 2017

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Section: Preliminary Evidence On the Uptake Use And Benefits Of The mentioning

confidence: 99%

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

et al. 2017

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“…In a review published in 2011, Brundage et al reported that in an identified sample of 794 RCTs, PROs were primary endpoints in just 26% of the total trials; in the 56% of the total trials where the rationale for the selected PROs was documented, 28% of the RTCs gave information about missing data and 64% of the RTCs analyzed the PRO results in the context of other endpoints [15]. The sub-optimal protocols for reporting PROs in clinical trials are also confirmed by two recent articles focused, respectively, on ovarian cancer and advanced breast cancer, in particular regarding timing of administration of PROs instruments, monitoring of PROs compliance, handling of missing data, the analysis plan for PROs and results discussion [16,17].…”

Section: How To Include Pros In Clinical Trials: the Consort Pro Extementioning

confidence: 99%

Patient-reported outcomes in clinical trials

Joly

Mittica

2016

CBN

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Patient-reported outcomes (PROs) are progressively being included in clinical trials to provide information about treatment benefits identified by the patients themselves that extend the data on traditional clinical trial endpoints, such as disease free survival, overall survival, progression-free survival, and response rate. PROs may have a greater impact for patients than other endpoints. For example, patients may be prepared to forgo some increase in progression-free survival in return for reduced treatment-related toxicity. PROs may also be an indicator of disease response and have value as prognostic factors. This article discusses the way PROs can be defined and incorporated into clinical trials to enhance the value of clinical trials data and improve the understanding of the clinical benefits of a specific treatment, not only for health care professionals, but for patients and caregivers. The importance and relevance of a patient-centered perspective and shared decision making in defining value and determining treatment benefit is increasingly recognized. However, despite the acknowledged value of PROs, their inclusion in clinical trials remains far from ideal. New guidelines from the research community and technological improvements in data collection and analytics will increase the quality and the importance of PROs as standard methods for the evaluation of medical studies and in the drugs approval process.

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“…Therefore, orphan drug manufacturers may lack the incentive to collect additional data, such as PROs, in the drug development process. Indeed, PROs are rarely reported in clinical trials of drugs for rare diseases [4,8]. Here, we sought to identify and characterise PRO claims in a comprehensive set of FDA orphan drug approvals from a 5-year period.…”

Section: Introductionmentioning

confidence: 99%

Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration

Jarosławski

Auquier

Borissov³

et al. 2018

Journal of Market Access & Health Policy

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Background: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks.Objective: We sought to assess the usage of patient-reported outcome (PRO) claims in a comprehensive set of US FDA orphan drug approvals dated between 1/1/2012 and 31/12/2016, and characterize them.Study design: Orphan drug approval documentation was obtained from the US FDA website. Drug Package Inserts (PI) were analyzed to extract information on PRO-related language.Results: Among 178 drugs that met inclusion criteria, 16 (9%) products approved for 16 orphan indications contained PRO language in the Clinical Studies section of the PI. All PRO instruments concerned disease symptoms, and two also referred to patient functioning. The most common PRO instrument was a bleed-specific rating scale for four products approved for the treatment or prevention of bleeding episodes in patients with genetic bleeding disorders.Conclusions: There is a need to implement public incentives for academic development of PRO instruments for rare conditions and for regulatory policies that mandate the collection of PRO endpoints in pivotal trials of orphan drugs.

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Patterns of reporting health-related quality of life outcomes in randomized clinical trials: implications for clinicians and quality of life researchers

Cited by 131 publications

References 26 publications

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

Patient-reported outcomes in clinical trials

Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration

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