Until recently, assessment of the benefit risk balance for a medicine has been entirely informal, but there is now growing interest among drug regulators and pharmaceutical companies in the possibilities of more formal approaches to benefit-risk decision-making, including those which explicitly take patient perspectives into account. Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium (PROTECT) was a project funded under the Innovative Medicines Initiative as a collaboration between academic, pharmaceutical, regulatory and patient organizations. Based on work from PROTECT we review current methodological approaches, and illustrate them with the case-studies where benefit-risk is finely balanced. We will introduce the PROTECT Benefit-Risk Roadmap, designed to help those wishing to find their way through this evolving arena (http://protectbenefitrisk.eu/), and highlight recent statistical developments and current challenges.
Deborah Ashby holds the Chair in Medical Statistics and Clinical Trials at Imperial College London, where she is also Co-Director of Imperial Clinical Trials Unit and Deputy Head of Imperial School of Public Health. Her research interests are in clinical trials, risk-benefit decision making for medicines, and the utility of Bayesian approaches in these areas. She led the benefit-risk workstream of the IMI-funded PROTECT project, bringing together academic, pharmaceutical, regulatory and patient expertise to determine best practice, and innovate on both underpinning methods and communication of the benefits and risks of medicines.
Professor Ashby has sat on the Commission on Human Medicines and often advises the European Medicines Agency. She chairs the HTA Commissioning Board, and is Deputy Chair of the HTA Programme for the UK National Institute for Health Research, and is Chair of the Population Research Committee for Cancer Research UK. Deborah was awarded the OBE for services to medicine in 2009, appointed an NIHR Senior Investigator in 2010, elected a Fellow of the Academy of Medical Sciences in 2012 and is President-Elect of the Royal Statistical Society.