06 / Standardizing outcomes measurement in cancer: A systematic review of core outcome sets

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.

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Development of ‘Core Outcome Sets’ for meningioma in clinical studies: The COSMIC project

Neuro-Oncology ◽

10.1093/neuonc/noab195.055 ◽

2021 ◽

Vol 23 (Supplement_4) ◽

pp. iv22-iv22

Author(s):

Christopher Millward ◽

Sumirat Keshwara ◽

Abdurrahman Islim ◽

Nisaharan Srikandarajah ◽

Tony Marson ◽

...

Keyword(s):

Systematic Review ◽

Clinical Studies ◽

Healthcare Professionals ◽

Clinical Effectiveness ◽

Core Outcome ◽

Core Outcome Sets ◽

Stakeholder Groups ◽

Patient Reported ◽

Effectiveness Trials ◽

Clinical Trial Activity

Abstract Aims To date, meningioma clinical trial activity has been limited, but a number of high-quality studies are underway, with more in development. There is heterogeneity in the outcomes reported in meningioma clinical trials. The COSMIC Project will develop two ‘Core Outcome Sets’ (COS) through comprehensive, transparent, consensus methodology; to ensure outcomes relevant to key stakeholders are reported within and across future meningioma clinical studies. The first will be for use in clinical effectiveness trials (COSMIC: Intervention), the second will be for use in clinical studies of incidental meningioma (COSMIC: Observation). Method Three systematic literature reviews will be performed to generate a long-list of outcomes potentially relevant to meningioma patients, healthcare professionals, researchers, and other stakeholder groups. The first systematic review will present the outcomes reported in published and ongoing meningioma clinical effectiveness trials. The second systematic review will present patient-reported outcomes (PRO) from the measurement tools utilised in meningioma PRO studies. The third systematic review will present the outcomes reported in published and ongoing clinical studies of untreated meningioma. Outcomes will be deduplicated, unique outcomes categorised according to the taxonomy presented by COMET, and the lists combined. The long-list of outcomes will be prioritised through two, 2-round, modified eDelphi surveys including meningioma patients, healthcare professionals, researchers, and other stakeholder groups. Undecided outcomes from both eDelphi surveys will be ratified at two, one-day consensus meeting, with representation from all key stakeholder groups. Results We have formed a study advisory group with international representation from key organisations. The project already has confirmed support from the International Consortium on Meningioma (ICOM), the European Association of Neuro-Oncology (EANO), the Response Assessment in Neuro-Oncology Patient-Reported Outcome group (RANO-PRO), the Society for Neuro-Oncology (SNO), British Neuro-Oncology Society (BNOS), Society of British Neurological Surgeons (SBNS), The Brain Tumour Charity (TBTC), and Brainstrust. Conclusion Standardising minimum outcome reporting in meningioma clinical effectiveness trials and meningioma clinical studies, through the development of these two COS will ensure outcomes reported are relevant to key stakeholder groups, including patients, whilst reducing research waste for a disease with increasing clinical trial activity. We seek to raise awareness of this project and invite participation from a wide range of stakeholders to ensure that the final COS reflects the opinion of the neuro-oncology community. Registration will take place via the study website www.thecosmicproject.org between June-August 2021.

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Choosing important health outcomes for comparative effectiveness research: 5th annual update to a systematic review of core outcome sets for research

PLoS ONE ◽

10.1371/journal.pone.0225980 ◽

2019 ◽

Vol 14 (12) ◽

pp. e0225980 ◽

Cited By ~ 13

Author(s):

Elizabeth Gargon ◽

Sarah L. Gorst ◽

Paula R. Williamson

Keyword(s):

Systematic Review ◽

Health Outcomes ◽

Comparative Effectiveness Research ◽

Comparative Effectiveness ◽

Effectiveness Research ◽

Core Outcome ◽

Core Outcome Sets ◽

Important Health

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Pain Measurement in Rheumatic and Musculoskeletal Diseases: Where To Go from Here? Report from a Special Interest Group at OMERACT 2018

The Journal of Rheumatology ◽

10.3899/jrheum.181099 ◽

2019 ◽

Vol 46 (10) ◽

pp. 1355-1359 ◽

Cited By ~ 1

Author(s):

Alessandro Chiarotto ◽

Ulrike Kaiser ◽

Ernest Choy ◽

Robin Christensen ◽

Philip G. Conaghan ◽

...

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Pain Measurement ◽

Research Agenda ◽

Online Survey ◽

Musculoskeletal Diseases ◽

Core Outcome ◽

Core Outcome Sets ◽

Pain Experience ◽

Key Questions

Objective.Establishing a research agenda on standardizing pain measurement in clinical trials in rheumatic and musculoskeletal diseases (RMD).Methods.Discussion during a meeting at the Outcome Measures in Rheumatology (OMERACT) 2018, prepared by a systematic review of existing core outcome sets and a patient online survey.Results.Several key questions were debated: Is pain a symptom or a disease? Are pain core (sub)domains consistent across RMD? How to account for pain mechanistic descriptors (e.g., central sensitization) in pain measurement?Conclusion.Characterizing and assessing the spectrum of pain experience across RMD in a standardized fashion is the objective of the OMERACT Pain Working Group.

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Mortality Outcomes in Dermatology: An Exploration of Core Outcome Sets and Cochrane Skin Systematic Review (Preprint)

JMIR Dermatology ◽

10.2196/34140 ◽

2021 ◽

Author(s):

Torunn E. Sivesind ◽

Mindy D. Szeto ◽

Shahzeb Hassan ◽

Peter Tugwell ◽

Robert P. Delllavalle

Keyword(s):

Systematic Review ◽

Core Outcome ◽

Core Outcome Sets

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Developing core economic outcome sets for asthma studies: a protocol for a systematic review

BMJ Open ◽

10.1136/bmjopen-2017-017054 ◽

2017 ◽

Vol 7 (8) ◽

pp. e017054 ◽

Cited By ~ 4

Author(s):

Natalia Hounsome ◽

Deborah Fitzsimmons ◽

Ceri Phillips ◽

Anita Patel

Keyword(s):

Systematic Review ◽

Core Outcome Set ◽

Ethics Committees ◽

Economic Outcomes ◽

Economic Evaluations ◽

Core Outcome ◽

Core Outcome Sets ◽

Outcome Set ◽

Life Years ◽

Starting Point

IntroductionCore outcome sets are standardised lists of outcomes, which should be measured and reported in all clinical studies of a specific condition. This study aims to develop core outcome sets for economic evaluations in asthma studies. Economic outcomes include items such as costs, resource use or quality-adjusted life years. The starting point in developing core outcome sets will be conducting a systematic literature review to establish a preliminary list of reporting items to be considered for inclusion in the core outcome set.Methods and analysisWe will conduct literature searches of peer-reviewed studies published from January 1990 to January 2017. These will include any comparative or observational studies (including economic models) and systematic reviews reporting economic outcomes. All identified economic outcomes will be tabulated together with the major study characteristics, such as population, study design, the nature and intensity of the intervention, mode of data collection and instrument(s) used to derive an outcome. We will undertake a ‘realist synthesis review’ to analyse the identified economic outcomes. The outcomes will be summarised in the context of evaluation perspectives, types of economic evaluation and methodological approaches. Parallel to undertaking a systematic review, we will conduct semistructured interviews with stakeholders (including people with personal experience of asthma, health professionals, researchers and decision makers) in order to explore additional outcomes which have not been considered, or used, in published studies. The list of outcomes generated from the systematic review and interviews with stakeholders will form the basis of a Delphi survey to refine the identified outcomes into a core outcome set.Ethics and disseminationThe review will not involve access to individual-level data. Findings from our systematic review will be communicated to a broad range of stakeholders including clinical guideline developers, research funders, trial registries, ethics committees and other regulators.

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