scholarly journals Core outcome set measurement for future clinical trials in acute myeloid leukemia: the HARMONY study protocol using a multi-stakeholder consensus-based Delphi process and a final consensus meeting

2019 ◽  
Author(s):  
Katharina M Lang ◽  
Kathryn L. Harrison ◽  
Paula R. Williamson ◽  
Brian J.P. Huntly ◽  
Gert Ossenkoppele ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Acute Myeloid Leukemia ◽  
Myeloid Leukemia ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Face To Face ◽  
Outcome Set ◽  
Multi Stakeholder ◽  
Stakeholder Consensus ◽  
Acute Myeloid

Abstract Background Acute myeloid leukemia is the most common acute leukemia in adults with an unacceptably low cure rate. In recent years a number of new treatment strategies and compounds were developed for the treatment of acute myeloid leukemia. There were several randomized, controlled clinical trials with the objective to improve patients’ management and patients’ outcome in acute myeloid leukemia. Unfortunately, these trials are not always directly comparable, as they do not measure the same outcomes and currently there are no core outcome sets that can be utilized to guide outcome selection and harmonization in this disease area. The HARMONY Alliance is a public-private European Network established in 2017, which currently includes 53 partners and 32 associated members from 22 countries. Amongst many other goals of the HARMONY Alliance, Work Package 2 focuses on defining outcomes that are relevant to each hematological malignancy. In accordance, a pilot study will be performed to define core outcome set in acute myeloid leukemia. Methods The pilot study will use a three-round Delphi survey and a final consensus meeting to define a core outcome set. Participants will be recruited from different stakeholder groups, including patients, clinicians, regulators and members of the European Federation of Pharmaceutical Industries and Associations (EFPIA). At the pre-Delphi stage a literature research was conducted followed by several semi-structured interviews of clinical public and private key opinion leaders. Subsequently the preliminary outcome list was discussed in several multi-stakeholder face-to-face meetings. The Delphi survey will reduce the preliminary outcome list to essential core outcomes. After completing the last Delphi round a final face-to-face meeting is planned to achieve consensus about core outcome set in acute myeloid leukemia. Discussion The pilot Delphi as part of HARMONY Alliance aims to define a core outcome set in acute myeloid leukemia based on a multi-stakeholder consensus. Such a core outcome set will help to allow consistent comparison of future clinical trials and real world evidence research and ensures that appropriate outcomes valued by a range of stakeholders are measured within future trials.

scholarly journals Core outcome set measurement for future clinical trials in acute myeloid leukemia: the HARMONY study protocol using a multi-stakeholder consensus-based Delphi process and a final consensus meeting

2020 ◽  
Author(s):  
Katharina M Lang ◽  
Kathryn L. Harrison ◽  
Paula R. Williamson ◽  
Brian J.P. Huntly ◽  
Gert Ossenkoppele ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Acute Myeloid Leukemia ◽  
Myeloid Leukemia ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Face To Face ◽  
Outcome Set ◽  
Multi Stakeholder ◽  
Stakeholder Consensus ◽  
Acute Myeloid

Abstract Background Acute myeloid leukemia is the most common acute leukemia in adults with an unacceptably low cure rate. In recent years a number of new treatment strategies and compounds were developed for the treatment of acute myeloid leukemia. There were several randomized, controlled clinical trials with the objective to improve patients’ management and patients’ outcome in acute myeloid leukemia. Unfortunately, these trials are not always directly comparable, as they do not measure the same outcomes and currently there are no core outcome sets that can be utilized to guide outcome selection and harmonization in this disease area. The HARMONY Alliance is a public-private European Network established in 2017, which currently includes 53 partners and 32 associated members from 22 countries. Amongst many other goals of the HARMONY Alliance, Work Package 2 focuses on defining outcomes that are relevant to each hematological malignancy. In accordance, a pilot study will be performed to define core outcome set in acute myeloid leukemia. Methods The pilot study will use a three-round Delphi survey and a final consensus meeting to define a core outcome set. Participants will be recruited from different stakeholder groups, including patients, clinicians, regulators and members of the European Federation of Pharmaceutical Industries and Associations (EFPIA). At the pre-Delphi stage a literature research was conducted followed by several semi-structured interviews of clinical public and private key opinion leaders. Subsequently the preliminary outcome list was discussed in several multi-stakeholder face-to-face meetings. The Delphi survey will reduce the preliminary outcome list to essential core outcomes. After completing the last Delphi round a final face-to-face meeting is planned to achieve consensus about core outcome set in acute myeloid leukemia. Discussion The pilot Delphi as part of HARMONY Alliance aims to define a core outcome set in acute myeloid leukemia based on a multi-stakeholder consensus. Such a core outcome set will help to allow consistent comparison of future clinical trials and real world evidence research and ensures that appropriate outcomes valued by a range of stakeholders are measured within future trials.

A multi-stakeholder Delphi consensus core outcome set for clinical trials in moderate-to-severe asthma (coreASTHMA)

2021 ◽  
Author(s):  
Vickram Tejwani ◽  
Hsing-Yuan Chang ◽  
Annie P. Tran ◽  
Jennifer Al Naber ◽  
Florian S. Gutzwiller ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Severe Asthma ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Delphi Consensus ◽  
Outcome Set ◽  
Multi Stakeholder

scholarly journals Using a web-based method to achieve stakeholder consensus about core outcomes for clinical trials is a viable option in the face of pandemic-related movement restrictions: Qualitative methods study (Preprint)

2021 ◽  
Author(s):  
Roulla Katiri ◽  
Deborah A. Hall ◽  
Derek J. Hoare ◽  
Kathryn Fackrell ◽  
Adele Horobin ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Core Outcome Set ◽  
Consensus Meeting ◽  
Core Outcome ◽  
Web Based ◽  
Face To Face ◽  
Outcome Set ◽  
Study Team ◽  
The Face ◽  
Single Sided Deafness

BACKGROUND Clinical trials that assess the benefits and harms of an intervention do so by measuring and reporting outcomes. Inconsistent selection and diversity in the choice of outcomes makes it challenging to directly compare interventions. To achieve an agreed core set of outcomes, a consensus methodology is recommended comprising a web-based Delphi survey and a face-to-face consensus meeting. However, UK-government regulations to control the pandemic prohibited plans for a face-to-face consensus meeting as part of the Core Rehabilitation Outcome Set for Single-Sided Deafness (CROSSSD) study. OBJECTIVE This article describes and evaluates the modifications taken by the CROSSSD study team to achieve consensus using web-based methods, but with minimal deviation from the original study protocol. METHODS The study team worked with healthcare users and professionals to translate the planned face-to-face consensus meeting in a web-based format, preserving key elements of the nominal group technique. A follow-up survey gathered evaluation feedback on the experience of the 22 participating members. Feedback covered (i) pre-meeting preparation, (ii) process of facilitated discussions and voting, (iii) ability to contribute, and (iv) perceived fairness of the outcome. RESULTS On balance, the web-based meeting achieved its original goals of open discussion, debate, and voting to agree a core outcome set for single-sided deafness. Hearing-impaired participants were fully engaged, but there were some methodological challenges. For the participants, challenges included building rapport, understanding, and delivering the tasks in hand. For the study team, challenges included the need for thorough preparation and managing the unpredictability of tasks on the day. CONCLUSIONS Sharing our experiences and lessons learned can benefit future core outcome set developers. Overcoming the challenges of delivering a web-based consensus exercise in the face of the pandemic can be applied more generally to maximise inclusiveness, enhance geographical access, as well as to reduce research costs. CLINICALTRIAL N/A

OP163 Health Technology Assessment Participation And Prioritization In Core Outcome Set Development

2019 ◽  
Vol 35 (S1) ◽  
pp. 35-35
Author(s):  
Elizabeth Clearfield ◽  
Jennifer Al Naber ◽  
Sean Tunis ◽  
Donna Messner
Keyword(s):  
Decision Making ◽  
Clinical Trials ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Core Outcome Set ◽  
Well Being ◽  
Health Technology ◽  
Core Outcome ◽  
Outcome Set ◽  
Multi Stakeholder

IntroductionA core outcome set (COS) is a minimum standardized set of agreed-upon outcomes for clinical trials of a specific condition. COS development can improve research by aligning stakeholder priorities for the outcomes most important in decision-making across the life-cycle of a product. It is important to include health technology assessment (HTA) representatives in COS development to ensure that outcomes useful to HTA are consistently included in clinical trials. Here we describe the role of HTA representatives in two COS projects: coreHEM, for gene therapy for hemophilia, a genetic blood clotting disease; and coreNASH, for nonalcoholic steatohepatitis (NASH), a progressive form of fatty liver disease that can lead to cirrhosis. We will describe the voting patterns of HTA representatives and consider aspects of their role in shaping the final COS.MethodsFor each multi-stakeholder COS, a modified Delphi process was utilized (three online surveys plus an in-person consensus meeting). Candidate outcome lists were compiled via a literature review complemented by participant interviews. Voters condensed and prioritized the lists by rating each outcome on a scale of 1-9 (not important-essential). Votes on each outcome were stratified by stakeholder group; HTA votes were compared with those of other stakeholders.ResultsHTA representatives made up 12.2 percent and 13.5 percent of the voters in coreHEM and coreNASH, respectively. They tended to give the highest votes to mortality outcomes, outcomes measuring the severity of disease, and outcomes related to a patient's quality of life, general well-being and general health perspective. HTA votes helped certain outcomes meet the inclusion criteria in the final voting rounds; without HTA voters, the “mental health status” outcome in coreHEM and the “hepatic-related mortality” and “liver transplantation” outcomes in coreNASH would have been eliminated.ConclusionsHTA participation in COS projects provides HTA representatives an opportunity to help shape COS in clinical research for better decision-making.

Postinflammatory hyperpigmentation: protocol for development of a core outcome set for clinical trials

2021 ◽  
Author(s):  
Bianca Y. Kang ◽  
Sarah A. Ibrahim ◽  
Divya Shokeen ◽  
Daniel I. Schlessinger ◽  
Jamie J. Kirkham ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Outcome Set ◽  
Postinflammatory Hyperpigmentation

Identifying Outcomes in Clinical Trials of Fistulizing Crohn’s Disease for the Development of a Core Outcome Set

2019 ◽  
Vol 17 (9) ◽  
pp. 1904-1908 ◽  
Author(s):  
Christopher Ma ◽  
Claire E. Parker ◽  
Tran M. Nguyen ◽  
Reena Khanna ◽  
Brian G. Feagan ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Crohn’S Disease ◽  
Crohn's Disease ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Outcome Set

scholarly journals Acute severe paediatric asthma: study protocol for the development of a core outcome set, a Pediatric Emergency Networks (PERN) study

2019 ◽  
Author(s):  
Simon Craig ◽  
Franz E Babl ◽  
Stuart R Dalziel ◽  
Charmaine Gray ◽  
Colin Powell ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Severe Asthma ◽  
Working Group ◽  
Core Outcome Set ◽  
Asthma Severity ◽  
Core Outcome ◽  
Acute Severe Asthma ◽  
Paediatric Asthma ◽  
Future Studies ◽  
Outcome Set

Abstract Background. Acute severe childhood asthma is an infrequent, but potentially life-threatening emergency presentation. There is a wide range of different approaches to this condition, with very little supporting evidence, leading to significant variation in practice. To improve knowledge in this area, there must first be consensus on how to conduct clinical trials, so that valid comparisons can be made between future studies. We have formed an international working group comprising paediatricians and emergency physicians from North America, Europe, Asia, the Middle East, Africa, South America, Central America, Australasia and the United Kingdom. Methods / design. A five-stage approach will be used: (1) A comprehensive list of outcomes relevant to stakeholders will be compiled through systematic reviews and qualitative interviews with patients, families, and clinicians; (2) A Delphi methodology will be applied to reduce the comprehensive list to a core outcome set; (3) We will review current clinical practice guidelines, existing clinical trials, and literature regarding bedside assessment of asthma severity. We will then identify practice differences in clinical assessment of asthma severity, and determine whether further prospective work is needed to achieve agreement on inclusion criteria for clinical trials on acute paediatric asthma in the emergency department setting; (4) A retrospective chart review in Australia and New Zealand will identify the incidence of serious clinical complications in children hospitalized with acute severe asthma, such as intubation, ICU admission, and death. Understanding the incidence of such outcomes will allow us to understand how common (and therefore how feasible) particular outcomes are in an asthma ED population; (5) Finally, a meeting of the PERN asthma working group will be held, with invitation of other clinicians interested in acute asthma research, and patients / families. The group will be asked to achieve consensus on a core set of outcomes and to make recommendations for the conduct of clinical trials on acute severe asthma. If this is not possible, the group will agree on a series of prioritized steps to achieve this aim. Discussion The development of an international consensus on core outcomes is an important first step towards the development of consensus guidelines and standardised protocols for RCTs in this population. This will enable us to better interpret and compare future studies, reduce risks of study heterogeneity and outcome reporting bias, and improve the evidence base for the management of this important condition.

scholarly journals Co-producing a multi-stakeholder Core Outcome Set for distal Tibia and Ankle fractures (COSTA): a study protocol

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Nathan A. Pearson ◽  
Elizabeth Tutton ◽  
Alexander Joeris ◽  
Stephen Gwilym ◽  
Richard Grant ◽  
...  
Keyword(s):  
Core Outcome Set ◽  
Distal Tibia ◽  
Ankle Fractures ◽  
Core Outcome ◽  
Core Domain ◽  
Outcome Reporting ◽  
The Core ◽  
Outcome Set ◽  
Multi Stakeholder ◽  
Core Domain Set

Abstract Background Ankle fracture is a common injury with a strong evidence base focused on effectiveness of treatments. However, there are no reporting guidelines on distal tibia and ankle fractures. This has led to heterogeneity in outcome reporting and consequently, restricted the contribution of evidence syntheses. Over the past decade, core outcome sets have been developed to address this issue and are available for several common fractures, including those of the hip, distal radius, and open tibial fractures. This protocol describes the process to co-produce—with patient partners and other key stakeholders—a multi-stakeholder derived Core Outcome Set for distal Tibia and Ankle fractures (COSTA). The scope of COSTA will be for clinical trials. Methods The study will have five-stages which will include the following: (i) systematic reviews of existing qualitative studies and outcome reporting in randomised controlled trial studies to inform a developing list of potential outcome domains; (ii) qualitative interviews (including secondary data) and focus groups with patients and healthcare professionals to explore the impact of ankle fracture and the outcomes that really matter; (iii) generation of meaningful outcome statements with the study team, international advisory group and patient partners; (iv) a multi-round, international e-Delphi study to achieve consensus on the core domain set; and (v) an evidence-based consensus on a core measurement set will be achieved through a structured group consensus meeting, recommending best assessment approaches for each of the domains in the core domain set. Discussion Development of COSTA will provide internationally endorsed outcome assessment guidance for clinical trials for distal tibia and ankle fractures. This will enhance comparative reviews of interventions, potentially reducing reporting bias and research waste.

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