scholarly journals coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Ellen Tambor ◽  
Matoya Robinson ◽  
Lewis Hsu ◽  
Hsing-Yuan Chang ◽  
Jennifer Al Naber ◽  
...  
Keyword(s):  
Decision Making ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Delphi Panel ◽  
Core Outcome ◽  
Cell Disease ◽  
Consensus Process ◽  
Core Outcome Sets ◽  
Multi Stakeholder ◽  
Stakeholder Consensus

Abstract Background With the dramatic increase in the pipeline for new sickle cell disease (SCD) therapies in recent years, the time is ripe to ensure a robust body of evidence is available for decision making by regulators, payers, clinicians, and patients. Harmonization of the outcomes selected across interventional trials enables optimal post-trial appraisal and decision making through valid pooled analyses and indirect comparisons. We employed a structured, multi-stakeholder consensus process to develop core outcome sets (COS) for use in clinical trials of SCD interventions. Methods CoreSCD utilized a modified Delphi method adapted from the standards recommended by the Core Outcome Measures in Effectiveness Trials (COMET) Initiative. An initial list of candidate outcomes was developed through a targeted literature review and input from an 11-member advisory committee. A 44-member multi-stakeholder Delphi Panel was established and included patients and family members, advocates, clinicians, researchers, payers, health technology assessors, representatives from government agencies, and industry representatives. Patients/advocates comprised 25% of the Delphi Panel and orientation and training was provided prior to the consensus process to ensure all were prepared to participate meaningfully. Panelists completed three rounds of an online survey to rate the importance of candidate outcomes for inclusion in the COS. Summary data was provided between each voting round and an in-person consensus meeting was held between the second and third round of voting. Consensus rules were applied following each round of voting to eliminate outcomes that did not meet predetermined criteria for retention. Results Consensus was reached for two core outcome sets. The final COS for trials of disease-modifying therapies includes ten outcomes and the COS for trials of acute interventions includes six outcomes. Both core sets include clinical outcomes as well as outcomes related to functioning/quality of life, resource utilization, and survival/mortality. Conclusions Use of the COS in clinical development programs for SCD will help to ensure that relevant, consistent outcomes are available for decision making across the product lifecycle.

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.

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.

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.

scholarly journals coreNASH: Multi‐stakeholder Consensus on Core Outcomes for Decision Making About Nonalcoholic Steatohepatitis Treatment

2021 ◽  
Author(s):  
Elizabeth Clearfield ◽  
Veronica Miller ◽  
Joseph Nadglowski ◽  
Katherine Barradas ◽  
Jennifer Al Naber ◽  
...  
Keyword(s):  
Decision Making ◽  
Nonalcoholic Steatohepatitis ◽  
Core Outcomes ◽  
Multi Stakeholder ◽  
Stakeholder Consensus

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 A protocol for the Development of Core Outcome Sets for Endodontic Treatment modalities (COSET): an international consensus process

2021 ◽  
Author(s):  
Ikhlas El karim ◽  
Henry F Duncan ◽  
Siobhan Cushley ◽  
Venkatesh Nagendrababu ◽  
Lise-Lotte Kirkevang ◽  
...  
Keyword(s):  
Clinical Studies ◽  
Endodontic Treatment ◽  
Treatment Modalities ◽  
Core Outcome ◽  
Consensus Process ◽  
International Consensus ◽  
Core Outcome Sets ◽  
Key Stakeholders ◽  
Dental Practitioners ◽  
Patient Reported

Abstract BackgroundThe outcome of endodontic treatment is generally assessed using a range of patient and clinician-centred, non-standardised clinical and radiographic outcome measures. This makes it difficult to synthesise evidence for systematic analysis of the literature and the development of clinical guidelines. Core outcome sets (COS) represent a standardised list of outcomes that should be measured and reported in all clinical studies in a particular field. Recently, clinical researchers and guideline developers have focussed on the need for integration of a patient-reported COS with clinician-centred measures. This study aims to develop a COS that includes both patient reported outcomes and clinician centred measures for various endodontic treatment modalities to be used in clinical research and practice.MethodsTo identify reported outcomes (including when and how they are measured), systematic reviews and their included clinical studies, which focus on the outcome of endodontic treatment and were published between 1990 and 2020 will be screened. The COSs will be defined by a consensus process involving key stakeholders using semi-structured interviews and an online Delphi methodology followed by an interactive virtual consensus meeting. A heterogeneous group of key ‘stakeholders’ including patients, general dental practitioners, endodontists, endodontic teachers, clinical researchers, students and policy-makers will be invited to participate. Patients will establish, via interactive interviews, which outcomes they value and feel should be included in a COS. In the Delphi process, other stakeholders will be asked to prioritise outcomes identified from the literature and patients interviews, and will have the opportunity at the end of the first round to add outcomes that are not included, but which they consider relevant. Feedback will be provided in the second round, when participants will be asked to prioritise the list again. If consensus is reached, the remaining outcomes will be discussed at an online meeting and agreement established via defined consensus rules of outcome inclusion. If consensus is not reached after the second round, a third round will be conducted with feedback, followed by the online meeting. Following identification of a COS, we will proceed to identify how and when these outcomes are measured.DiscussionUsing a rigorous methodology, the proposed consensus process aims to develop a COS for endodontic treatment that will be relevant to stakeholders. The results of the study will be shared with participants and COS users. To increase COS uptake, it will also be actively shared with clinical guideline developers, research funders and the editors of general dental and endodontology journals.Study registrationThe study is registered in COMET (https://comet-initiative.org/Studies/Details/1879)

Decision-making in pediatric hematopoeitic cell transplantation

2018 ◽  
Author(s):  
◽  
Ginny L. Schulz
Keyword(s):  
Decision Making ◽  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Family Members ◽  
Real Life ◽  
Decisional Conflict ◽  
Cell Disease ◽  
Decision Choice

[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Hematopoietic cell transplantation (HCT) is a treatment option utilized to cure many childhood disorders, with cancer, sickle cell disease (SCD), and aplastic anemia being a few. The decision of whether or not to pursue HCT involves uncertainty and risk. Pediatric patients with SCD and their families undergo a decision-making process distinct from patients with childhood cancer. Patients with sickle cell disease (SCD) realize an awareness of unpredictable disease progression and conflict when considering HCT, while patients with cancer perceive an urgency for survival. We applied Connor's Framework of Decisional Conflict and a multiple-case study approach to describe the level and source of decisional conflict in patients with SCD and their families in a real-life decision choice of HCT. The contribution of the pediatric patient in the decision was also described in our four families. Observation, survey, and interview methods allowed us to gain an in-depth understanding of their conflict. Through pattern-matching and cross-case synthesis, we found that the level and sources of conflict among 11 participants varied within and across family members. Some experienced levels that resulted in a delayed decision or feeling unsure. The patients and their family members ultimately navigated their uncertainties together to come to consensus on whether to proceed with HCT. Future studies should implement individualized interventions from a family research perspective to decrease the decisional conflict this population faces.

scholarly journals How I treat sickle cell disease with hematopoietic cell transplantation

Blood ◽  
2019 ◽  
Vol 134 (25) ◽  
pp. 2249-2260 ◽  
Author(s):  
Elizabeth O. Stenger ◽  
Shalini Shenoy ◽  
Lakshmanan Krishnamurti
Keyword(s):  
Decision Making ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Cell Disease ◽  
Hematopoietic Stem

Hematopoietic stem cell transplantation can be curative for sickle cell disease, but decision-making is often complex. This How I Treat provides a perspective on how to negotiate this process for an individual patient.

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