scholarly journals Outcome selection for tissue-agnostic drug trials for immune-mediated inflammatory diseases: a systematic review of core outcome sets and regulatory guidance

Trials ◽  
2022 ◽  
Vol 23 (1) ◽  
Author(s):  
Olalekan Lee Aiyegbusi ◽  
Lavinia Ferrante di Ruffano ◽  
Ameeta Retzer ◽  
Philip N. Newsome ◽  
Christopher D. Buckley ◽  
...  
Keyword(s):  
Systematic Review ◽  
Inflammatory Diseases ◽  
Core Outcome ◽  
Core Outcome Sets ◽  
Regulatory Guidance ◽  
Immune Mediated ◽  
Outcome Selection ◽  
Similarities And Differences ◽  
Selection For

Abstract Background Tissue-agnostic drug development provides a paradigm shift in precision medicine and requires innovative trial designs. However, outcome selection for such trials can prove challenging. The objectives of this review were to: Identify and map core outcome sets (COS), across 11 immune-mediated inflammatory diseases (IMIDs) in order to facilitate the selection of relevant outcomes across the conditions for innovative trials of tissue-agnostic drug therapies. Compare outcomes or endpoints recommended by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) to identify and highlight similarities and differences. Methods The Core Outcome Measures in Effectiveness Trials (COMET), International Consortium for Health Outcomes Measurement (ICHOM), FDA and EMA databases were searched from inception to 28th December 2019. Two reviewers independently screened titles and abstracts of retrieved entries and conducted the subsequent full text screening. Hand searching of the reference lists and citation searching of the selected publications was conducted. The methodological quality of the included peer-reviewed articles was independently assessed by the reviewers based on the items of the COS–Standards for Development recommendations (COS–STAD) checklist. Core outcomes from the included publications were extracted and mapped across studies and conditions. Regulatory guidance from FDA and EMA, where available for clinical trials for the IMIDs, were obtained from their databases and recommendations on outcomes to measure directly compared. Results Forty-four COS publications were included in the final analysis. Outcomes such as disease activity, pain, fatigue, quality of life, physical function, work limitation/productivity, steroid use and biomarkers were recommended across majority of the conditions. There were significant similarities and differences in FDA and EMA recommendations. The only instance where either regulatory body directly referenced a COS was for jSLE—both referenced the Paediatric Rheumatology International Trials Organization (PRINTO) COS. Conclusions The findings from this systematic review provide valuable information to inform outcome selection in tissue-agnostic trials for IMIDs. There is a need for increased collaboration between regulators and COS developers and inclusion of regulators as key stakeholders in COS development to enhance the quality of COS. Trial registration Not registered.

The evolution of instrument selection for inclusion in core outcome sets at OMERACT: Filter 2.2

2021 ◽  
Author(s):  
Lara J Maxwell ◽  
Dorcas E Beaton ◽  
Maarten Boers ◽  
Maria Antonietta D'Agostino ◽  
Philip G Conaghan ◽  
...  
Keyword(s):  
Core Outcome ◽  
Core Outcome Sets ◽  
Instrument Selection ◽  
Selection For

scholarly journals Core outcome sets in women's and newborn health: a systematic review

2017 ◽  
Vol 124 (10) ◽  
pp. 1481-1489 ◽  
Author(s):  
JMN Duffy ◽  
R Rolph ◽  
C Gale ◽  
M Hirsch ◽  
KS Khan ◽  
...  
Keyword(s):  
Systematic Review ◽  
Newborn Health ◽  
Core Outcome ◽  
Core Outcome Sets

scholarly journals Patients’ experiences and perceptions of Guillain-Barré syndrome: A systematic review and meta-synthesis of qualitative research

PLoS ONE ◽  
2021 ◽  
Vol 16 (2) ◽  
pp. e0245826
Author(s):  
Despina Laparidou ◽  
Ffion Curtis ◽  
Joseph Akanuwe ◽  
Jennifer Jackson ◽  
Timothy L. Hodgson ◽  
...  
Keyword(s):  
Systematic Review ◽  
Guillain Barre Syndrome ◽  
Link Type ◽  
Immune Mediated ◽  
Acceptable Quality ◽  
Guillain Barré Syndrome ◽  
Internet Searches ◽  
Guillain Barré ◽  
Full Text Screening

Background Guillain-Barré syndrome (GBS) is an immune-mediated polyradiculoneuropathy, with an incidence of 1-2/100,000 per year. Its severity is variable, ranging from very mild cases with brief weakness to severe paralysis, leading to inability to breathe independently, or even death. Currently there is limited evidence exploring the experiences of GBS patients. The aim of this study was to review patients’ experiences and perceptions of GBS and its variants at diagnosis, discharge and during recovery, by conducting a systematic review and thematic meta-synthesis of qualitative studies of patients’ experiences of GBS (and its variants). Methods We searched twelve electronic databases, supplemented with internet searches and forward and backward citation tracking from the included studies and review articles. Data were synthesised thematically following the Thomas and Harden approach. The CASP Qualitative Checklist was used to assess the quality of the included studies of this review. Results Our search strategy identified a total of 5,282 citations and after removing duplicates and excluding citations based on title and abstract, and full-text screening, five studies were included in the review and meta-synthesis; all included studies were considered of acceptable quality. Through constant discussions and an iterative approach, we developed six analytical themes following a patient’s journey from suspecting that they had a health problem, through to being hospitalised, experiencing ongoing difficulties, slowly recovering from GBS, adjusting to their new circumstances, and re-evaluating their lives. Conclusions Despite the variety of experiences, it was evident from all included studies that being diagnosed with and surviving GBS was a life-changing experience for all participants. Trial registration Protocol was registered (CRD42019122199) on the International Prospective Register of Systematic Reviews (http://www.crd.york.ac.uk/PROSPERO).

scholarly journals Outcomes and outcomes measurements used in intervention studies of pelvic girdle pain and lumbopelvic pain: a systematic review

2019 ◽  
Vol 27 (1) ◽  
Author(s):  
Francesca Wuytack ◽  
Maggie O’Donovan
Keyword(s):  
Systematic Review ◽  
Intervention Studies ◽  
Core Outcome Set ◽  
Pelvic Girdle ◽  
Pelvic Girdle Pain ◽  
Cochrane Library ◽  
Core Outcome ◽  
Outcome Set ◽  
Pregnancy And Postpartum

Abstract Background Pelvic girdle pain is a common problem during pregnancy and postpartum with significant personal and societal impact and costs. Studies examining the effectiveness of interventions for pelvic girdle pain measure different outcomes, making it difficult to pool data in meta-analysis in a meaningful and interpretable way to increase the certainty of effect measures. A consensus-based core outcome set for pelvic girdle pain can address this issue. As a first step in developing a core outcome set, it is essential to systematically examine the outcomes measured in existing studies. Objective The objective of this systematic review was to identify, examine and compare what outcomes are measured and reported, and how outcomes are measured, in intervention studies and systematic reviews of interventions for pelvic girdle pain and for lumbopelvic pain (which includes pelvic girdle pain). Methods We searched PubMed, Cochrane Library, PEDro and Embase from inception to the 11th May 2018. Two reviewers independently selected studies by title/abstract and by full text screening. Disagreement was resolved through discussion. Outcomes reported and their outcome measurement instruments were extracted and recorded by two reviewers independently. We assessed the quality of reporting with two independent reviewers. The outcomes were grouped into core domains using the OMERACT filter 2.0 framework. Results A total of 107 studies were included, including 33 studies on pelvic girdle pain and 74 studies on lumbopelvic pain. Forty-six outcomes were reported across all studies, with the highest amount (26/46) in the ‘life impact’ domain. ‘Pain’ was the most commonly reported outcome in both pelvic girdle pain and lumbopelvic pain studies. Studies used different instruments to measure the same outcomes, particularly for the outcomes pain, function, disability and quality of life. Conclusions A wide variety of outcomes and outcome measurements are used in studies on pelvic girdle pain and lumbopelvic pain. The findings of this review will be included in a Delphi survey to reach consensus on a pelvic girdle pain - core outcome set. This core outcome set will allow for more effective comparison between future studies on pelvic girdle pain, allowing for more effective translation of findings to clinical practice.

scholarly journals Patient involvement to inform the design of a clinical trial in postbariatric hypoglycaemia

2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Matthias Hepprich ◽  
Marc Y. Donath ◽  
Lars G. Hemkens
Keyword(s):  
Quality Of Life ◽  
Bariatric Surgery ◽  
Clinical Trial ◽  
Primary Outcome ◽  
Evidence Based ◽  
Core Outcome ◽  
Online Questionnaire ◽  
Core Outcome Sets ◽  
German Speaking

Abstract Background Bariatric surgery may lead to symptomatic postprandial hypoglycaemia as a major side effect without established therapy so far. We aimed to develop an evidence-based study design of a clinical trial that tests treatment options and can provide useful patient-relevant evidence. Methods We searched systematically for guidance of core outcome sets to determine the most relevant types of outcomes and duration of such a trial. Our search comprised literature databases, a database of core outcome sets and self-help organizations. We then developed a simple online questionnaire based on interviews with German-speaking patients with postprandial hypoglycaemia after bariatric surgery. We recruited participants by reaching out to all German speaking endocrinologists in Switzerland and large Swiss bariatric centres. We asked for preferences regarding outcome types and acceptable duration of being included in a corresponding clinical trial. Results The literature search did not identify evidence-based guidance for informing our study design. Experience of clinical and research routine as well as patient interviews helped in identifying potential outcomes and the design of an online questionnaire. Therein, a total of 29 persons started the questionnaire and 22 answered questions related to the primary outcome. Of these, 17 (77.3%) deemed quality of life more relevant as primary outcome than the rate of hypoglycaemic episodes. A trial length of four weeks or longer was regarded as acceptable for 19 of 21 respondents to this question (91.4%) and of six months or longer for 12 respondents (56%). Conclusions In situations with no other guidance, a simple questionnaire may help to inform trial design decisions. This study identifies a patient preference for “quality of life” as a primary outcome and supports the evidence-based conception of a patient-centred clinical trial in postbariatric hypoglycaemia.

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