Benefit – Risk

Benefit – Risk is a large methodological area which is commonly used within the regulatory industry to trade-off the benefits of a treatment with the risks, however this is not commonly transferred to NIHR trials.

This section aimed to bring to focus the use of benefit-risk methods which could be implemented in NIHR projects by bringing together useful resources and researchers in this area to share experiences and best practice.

The group collated a number of online resources about benefit-risk (see links below).

Links from Working Group Members

• Owen, R. Webinar on individualised benefit-risk Career Young Statistician Session, PSI conference in June 2020 (Access to PSI Members only).
• Totton, N. & Julious, S. Benefit-Risk Assessment Breakout Session NIHR Statistics Conference 2019.
• Totton, N., Julious, S., Hughes, D. & Cook, J. MRC-NIHR Methodology Guideline Development on Utilising Benefit-Risk Assessments within Clinical Trials ICTMC Conference 2019 (PS1C-01).
• Hughes, D. Waddingham, E. et al. Recommendations for benefit-risk assessment methodologies and visual representation. Pharmacoepidemiology and Drug Safety 2016.
• Nixon, R., Dierig, C., Mt-Isa, S., Stockert, I., Tong, T., Kuhls, S., Hodgson, G., Pears, J., Waddingham, E. et al. A case study using the PrOACT‐URL and BRAT frameworks for structured benefit risk assessment. Biometrical Journal 2015.
• Waddingham, E. et al. A Bayesian approach to probabilistic sensitivity analysis in structured benefit‐risk assessment. Biometrical Journal 2015.
• Hallgreen, C., Waddingham, E. et al. Benefit–risk assessment in a post‐market setting: a case study integrating real‐life experience into benefit–risk methodology. Pharmacoepidemiology and Drug Safety 2014.

Other Useful Links

• PROTECT Group This website is a thorough overview of many different elements of benefit-risk assessment and many useful resources from their work. This is a good place to start to get a better understanding on benefit-risk in practice.
• PSI Benefit-Risk Special Interest Group This group have put together a blog which contains links to many useful articles and information as well as blog posts explaining specific aspects of benefit-risk assessment.
• Food and Drug Administration (FDA) Guidance
• European Medicines Agency (EMA) Guidance
• Systematic Review completed by the PROTECT group Mt-Isa, S. et al. Balancing benefit and risk of medicines: a systematic review and classification of available methodologies. Pharmacoepidemiology 2014.