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.

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 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.

scholarly journals Development of a Core Outcome Set for Clinical Trials of Traditional Chinese Medicine in Lung Cancer: a Study Protocol

2021 ◽  
Author(s):  
Mingyan Zhang ◽  
Hui Zi Chua ◽  
Bohan Niu ◽  
Kai Li ◽  
Junhua Zhang
Keyword(s):  
Lung Cancer ◽  
Decision Making ◽  
Clinical Trials ◽  
Methodological Quality ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Stakeholder Groups ◽  
The Core ◽  
Outcome Set

Abstract Background: Lung cancer (LC) is currently the leading cause of cancer death globally. LC accounts for a high mortality and incidence rate of cancer in both men and women. Radiotherapy and chemotherapy, though effective for some patients, have strong side effects and most of them only have palliative effect. Though many studies reported the effectiveness of traditional Chinese medicine (TCM) treatment on LC, the lack of methodological quality in clinical trials resulted in heterogeneous reporting of outcomes, making it difficult to compare and combine in different trials, limiting the validity of meta-analysis and reducing the value of clinical studies. A core outcome set (COS) could reduce outcome reporting bias and heterogeneity across studies of LC using TCM-related interventions. The aim of this study is to develop a standardized COS for LC in TCM clinical trials (COS-TCM-LC) to improve the methodological quality of TCM clinical studies to serve as a guidance in healthcare decision making for LC.Methods: The study has been developed according to the Core Outcome Set-STAandards for Development standards for the design of a COS study. The study process consisted 4 stages as follows: (1) Identifying a list of potential outcomes through systematic reviews of TCM RCTs and 2 clinical registry databases, qualitative surveys on patients and healthcare professionals to form an outcome pool and finally establishing a preliminary checklist of outcomes. (2) Selection of stakeholder groups. (3) Representatives of stakeholder groups will be invited to participate in a two-round Delphi survey. (4) A face-to-face consensus meeting will be held to determine the final COS-TCM-LC.Discussion: In this study protocol, we have followed the guidelines of COS-STAndardized Protocol (STAP) statement and checked the items in COS-STAndards for Developement (STAD). Developing a COS-TCM-LC will improve the quality of future RCTs on LC with the TCM interventions and promote better evidence-based clinical decision-making.Trial registration: This study is registered with the Core Outcome Measures in Effectiveness Trials database as study 1483 (http://www.comet-initiative.org/studies/details/1483).

scholarly journals Health technology assessment in sub-Saharan Africa: a descriptive analysis and narrative synthesis

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Samantha Hollingworth ◽  
Ama Pokuaa Fenny ◽  
Su-Yeon Yu ◽  
Francis Ruiz ◽  
Kalipso Chalkidou
Keyword(s):  
Decision Making ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Technical Assistance ◽  
Health Technology ◽  
Sub Saharan Africa ◽  
Main Theme ◽  
Narrative Synthesis ◽  
Policy Makers ◽  
Sub Saharan

Abstract Background Countries in Sub-Saharan Africa (SSA) are moving towards universal health coverage. The process of Health Technology Assessment (HTA) can support decisions relating to benefit package design and service coverage. HTA involves institutional cooperation with agreed methods and procedural standards. We systematically reviewed the literature on policies and capacity building to support HTA institutionalisation in SSA. Methods We systematically reviewed the literature by searching major databases (PubMed, Embase, etc.) until June 2019 using terms considering three aspects: HTA; health policy, decision making; and SSA. We quantitatively extracted and descriptively analysed content and conducted a narrative synthesis eliciting themes from the selected literature, which varied in study type and apporach. Results Half of the 49 papers identified were primary research studies and mostly qualitative. Five countries were represented in six of ten studies; South Africa, Ghana, Uganda, Cameroon, and Ethiopia. Half of first authors were from SSA. Most informants were policy makers. Five themes emerged: (1) use of HTA; (2) decision-making in HTA; (3) values and criteria for setting priority areas in HTA; (4) involving stakeholders in HTA; and (5) specific examples of progress in HTA in SSA. The first one was the main theme where there was little use of evidence and research in making policy. The awareness of HTA and economic evaluation was low, with inadequate expertise and a lack of local data and tools. Conclusions Despite growing interest in HTA in SSA countries, awareness remains low and HTA-related activities are uncoordinated and often disconnected from policy. Further training and skills development are needed, firmly linked to a strategy focusing on strengthening within-country partnerships, particularly among researchers and policy makers. The international community has an important role here by supporting policy- relevant technical assistance, highlighting that sustainable financing demands evidence-based processes for effective resource allocation, and catalysing knowledge-sharing opportunities among countries facing similar challenges.

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
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