Nikki Totton1, Steven Julious2, Dyfrig Hughes3, Jonathan Cook4, Katie Biggs2, Lizzie Coates2, Andrew Cook5, Catherine Hewitt6, Simon Day7. 1. School of Health and Related Research, University of Sheffield, Sheffield, UK. n.v.totton@sheffield.ac.uk. 2. School of Health and Related Research, University of Sheffield, Sheffield, UK. 3. Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, UK. 4. Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK. 5. Wessex Institute, University of Southampton, Southampton, UK. 6. York Trials Unit, Department of Health Sciences, University of York, York, UK. 7. Clinical Trials Consulting & Training Limited, Buckingham, UK.
Abstract
BACKGROUND: Depending on the treatment to be investigated, a clinical trial could be designed to assess objectives of superiority, equivalence or non-inferiority. The design of the study is affected by many different elements including the control treatment, the primary outcome and associated relationships. In some studies, there could be more than one outcome of interest. In these situations, benefit-risk methodologies could be used to assess the outcomes simultaneously and consider the trade-off between the benefits against the risks of a treatment. Benefit-risk is used within the regulatory industry but seldom included within publicly funded clinical trials within the UK. This project aims to gain an expert consensus on how to select the appropriate trial design (e.g. superiority) and when to consider including benefit-risk methods. METHODS: The project will consist of four work packages: 1. A web-based survey to elicit current experiences and opinions, 2. A rapid literature review to assess any current recommendations, 3. A two-day consensus workshop to gain agreement on the recommendations, and 4. Production of a guidance document. DISCUSSION: The aim of the project is to provide a guideline for clinical researchers, grant funding bodies and reviewers for grant bodies for how to select the most appropriate trial design and when it is appropriate to consider using benefit-risk methods. The focus of the guideline will be on publicly funded trials however, the vision is that the work will be applicable across research settings and we will connect with other organisations and committees as appropriate.
BACKGROUND: Depending on the treatment to be investigated, a clinical trial could be designed to assess objectives of superiority, equivalence or non-inferiority. The design of the study is affected by many different elements including the control treatment, the primary outcome and associated relationships. In some studies, there could be more than one outcome of interest. In these situations, benefit-risk methodologies could be used to assess the outcomes simultaneously and consider the trade-off between the benefits against the risks of a treatment. Benefit-risk is used within the regulatory industry but seldom included within publicly funded clinical trials within the UK. This project aims to gain an expert consensus on how to select the appropriate trial design (e.g. superiority) and when to consider including benefit-risk methods. METHODS: The project will consist of four work packages: 1. A web-based survey to elicit current experiences and opinions, 2. A rapid literature review to assess any current recommendations, 3. A two-day consensus workshop to gain agreement on the recommendations, and 4. Production of a guidance document. DISCUSSION: The aim of the project is to provide a guideline for clinical researchers, grant funding bodies and reviewers for grant bodies for how to select the most appropriate trial design and when it is appropriate to consider using benefit-risk methods. The focus of the guideline will be on publicly funded trials however, the vision is that the work will be applicable across research settings and we will connect with other organisations and committees as appropriate.
Entities:
Keywords:
Benefit-risk; Health technology assessment; Randomised controlled trials
Authors: M K Murphy; N A Black; D L Lamping; C M McKee; C F Sanderson; J Askham; T Marteau Journal: Health Technol Assess Date: 1998 Impact factor: 4.014
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