Trials with patient-reported outcomes registered on the Australian New Zealand Clinical Trials Registry (ANZCTR)

Mercieca-Bebber, Rebecca; Williams, Douglas; Tait, Margaret-Ann; Roydhouse, Jessica; Busija, Lucy; Sundaram, Chindhu Shunmuga; Wilson, Michelle; Langford, Ailsa; Rutherford, Claudia; Roberts, Natasha; King, Madeleine; Vodicka, Elisabeth; Devine, Beth

doi:10.1007/s11136-018-1921-5

Cited by 30 publications

(23 citation statements)

References 17 publications

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“…A more recent review of the Australian New Zealand Clinical Trials Registry determined that 45% of trials registered from 2005 to March 2017 included PROs and had a strong increase in the proportion of trials with PROs registered over time ( r =0.74, P =0.009). 7 Similarly, between 2000 and 2015, the Center for Devices and Radiological Health reported an increase of over 500% in pre-market submissions including PRO measures. 8…”

Section: Introductionmentioning

confidence: 99%

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

Mercieca‐Bebber

King

Calvert

et al. 2018

PROM

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Patient-reported outcomes (PROs) can be included in clinical trials as primary or secondary endpoints and are increasingly recognized by regulators, clinicians, and patients as valuable tools to collect patient-centered data. PROs provide unique information on the impact of a medical condition and its treatment from the patient’s perspective; therefore, PROs can be included in clinical trials to ensure the impact of a trial intervention is comprehensively assessed. This review first discusses examples of how PRO endpoints have added value to clinical trial interpretation. Second, it describes the problems with current practices in designing, implementing, and reporting PRO studies, and how these problems may be addressed by complying with guidance for protocol development, selecting appropriate PRO measures to match clinically motivated PRO hypotheses, minimizing the rates of avoidable missing PRO data, analyzing and interpreting PRO data, and transparently reporting PRO findings.

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Section: Introductionmentioning

confidence: 99%

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

Mercieca‐Bebber

King

Calvert

et al. 2018

PROM

Self Cite

374

320

View full text Add to dashboard Cite

show abstract

“…Patients reported outcomes (PRO) are de ned as "any report coming directly from patients, without interpretation by physicians or others, about how they function or feel in relation to a health condition and its therapy" [31]. Gradually, the use of PROs in clinical trials has increased signi cantly since 2005 [32][33][34]. PROs can be used to determine affected individual's experience particularly concerning improvement or aggravation of subjective symptoms, to stratify participants, to re ne clinical trial design and to illustrate the risk-bene t balance allowing to choose the personalized better treatment [34].…”

Section: Discussionmentioning

confidence: 99%

Families driven drug development and clinical trials: a pilot study in Dravet Syndrome to delineate what really matters

Teng

Barco

Marie³

et al. 2021

Preprint

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Background Dravet syndrome (DS) is a developmental and epileptic encephalopathy. Patients rapidly develop drug resistant seizures but patients with DS develop non-seizures disorders that are often age related. We aimed to identify the caregivers’ opinion on the outcome measures that matters in clinical trials in individuals with DS and their correlation with the age of the individual with DS. Methods We conducted a prospective international study with convenience sample based on a 11-closed questions survey developed with three European patients’ advocacy groups (PAG) for DS (France, Italy and Germany). The items were about seizures and daily life outcomes that a clinical trial should target according to family opinion. Items were scored from 1 (not important at all) to 5 (highly important). Statistical analyses were performed to evaluate country (ANOVA and khi2 tests) and age effect (Spearman's ρ). Results Hundred and fifty-three caregivers answered the survey (68%: France, 28%: Germany and 24%: Italy; affected individuals’ characteristics: 86 males, age: 11.4 [25th -75th percentile:7-20.4] years). Demographic characteristics were not significantly different between countries. Families ranked as important almost all the items proposed. However, most of the items related to daily life had the highest rank in all 3 countries compared to items about seizures (p = 0.02). Positive correlation between age and age at diagnosis (ρ = 0.26, p = 0.02) and negative correlations between age and targeting seizure duration and between age (ρ =-0.25, p = 0.005) and targeting the need of referral to hospital (ρ =-0.26, p = 0.005) were identified. Conclusions This study emphasized the DS families’ expectations from therapies beyond seizure efficacy. These data can help to adapt patients-centered outcome measures in future clinical and real-life trials in DS.

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“…Moreover, PRO data are often poorly reported 9–11 or not included in trial publications 12 . Despite this evidence, a substantial, and growing, number of randomized controlled trials (RCTs) across all cancer types now include PROs as primary and/or secondary endpoints 13,14 . Poor PRO protocol content and subsequent poor outcome reporting reduce the extent to which PRO results reach and inform clinical interactions and decision making, while simultaneously devaluing the contribution of trial participants providing this information.…”

Section: Introductionmentioning

confidence: 99%

International perspectives on suboptimal patient‐reported outcome trial design and reporting in cancer clinical trials: A qualitative study

et al. 2021

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Purpose Evidence suggests that the patient‐reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non‐reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions. Methods Semi‐structured interviews were conducted with four stakeholder groups: (1) trialists and chief investigators; (2) people with lived experience of cancer; (3) international experts in PRO cancer trial design; (4) journal editors, funding panelists, and regulatory agencies. Data were analyzed using directed thematic analysis with an iterative coding frame. Results Forty‐four interviews were undertaken. Several factors were identified that could influenced effective integration of PROs into trials and subsequent findings. Participants described (1) late inclusion of PROs in trial design; (2) PROs being considered a lower priority outcome compared to survival; (3) trialists’ reluctance to collect or report PROs due to participant burden, missing data, and perceived reticence of journals to publish; (4) lack of staff training. Strategies to address these included training research personnel and improved communication with site staff and patients regarding the value of PROs. Examples of good practice were identified. Conclusion Misconceptions relating to PRO methodology and its use may undermine their planning, collection, and reporting. There is a role for funding, regulatory, methodological, and journalistic institutions to address perceptions around the value of PROs, their position within the trial outcomes hierarchy, that PRO training and guidance is available, signposted, and readily accessible, with accompanying measures to ensure compliance with international best practice guidelines.

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Trials with patient-reported outcomes registered on the Australian New Zealand Clinical Trials Registry (ANZCTR)

Abstract: Our findings suggest growing use of PROs in the assessment of health and interventions in ANZ. Our review identifies trial categories with limited patient-reported information and provides a basis for future work on the impact of PRO findings in clinical care.

Cited by 30 publications

References 17 publications

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

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

Families driven drug development and clinical trials: a pilot study in Dravet Syndrome to delineate what really matters

International perspectives on suboptimal patient‐reported outcome trial design and reporting in cancer clinical trials: A qualitative study

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