What do you consider to be the benefit-risk key drivers?

@DIA Pharmacovigilance Conference, London in May 2017

We keep asking ourselves how do we tackle benefit-risk assessment using advanced structured approaches in real life. It is difficult, especially when we know that benefit-risk balance is not constant over time. How should we approach it to begin with, and how do we update it when new evidence comes into the equations? Dr Kaatje Bollaerts (P-95) and I are chairing the next DIA Pharmacovigilance Workshop to discuss this topic.

Share your experience in this area so we can discuss this topic further, and hopefully take the harmonisation of structured benefit-risk assessment to the next level!

What would you like to learn about?

As we enter into 2017 it is a great time to plan for the year. The EFSPI/PSI special interest group on benefit-risks wants to make this as relevant as possible for you, but we need your help for this. Please add comments to this post to tell us, what you would like to hear about? What topics are you interested in? Are there any people, you would like to hear from? Or are you interested yourself to share your thoughts?

Please let us know!

What would you like to know about BR as a beginner?

I’m pretty excited as I was asked to present about benefit-risk at the first EFPSI workshop on regulatory statistics. The organizers have built a very good agenda with lots of good content and presenters.

I’ll talk after David Wright from the MHRA, who will give a regulatory perspective on the evolution of BR and the regulatory decision process. Georg Quartey (Roche) will speak after me about a variety of case studies. Thus, I’m in the middle. We agreed, that each presenter will have 20 minutes to speak and that the session will end with a 30 minutes podium discussion.

The goal of my presentation is to provide the perspective off an industry statistician on benefit-risk. I’m thinking especially about those statisticians, that rarely touched this topic in the past. My draft title is “What’s happening in benefit-risk and what is our role as a statistician?”

FDA on further Benefit-Risk framework – an outlook

PDUFA VI Draft Goals Letter just posted online

The Draft Goals Letter was just posted online here. There are lots of interesting things in this. Here is a quote from the Regulatory Decision Tools Subgroup that is especially interesting for Benefit-Risk:

“FDA discussed its proposal to explore the appropriateness of incorporating additional decision-analytic approaches that could inform FDA’s regulatory decision-making through the B-R Framework. As part of this proposal, the agency proposed convening at least one workshop to review potential approaches, such as the use of trial endpoints that capture ranked benefit and harm outcomes at the patient-level, and their applicability within FDA’s drug regulatory context. Industry indicated that the value in this proposed area of exploration would need to be weighed against other biostatistics-related proposals under discussion.”

Why should (nearly) every statistician know about benefit-risk assessment?


Benefit-risk assessment is at the heart of what our industry is all about.

A physician will only prescribe a treatment, if he is convinced that the benefits outweigh the safety concerns. A patient will only take a medication, where he trusts, that the treatment works and the side effects are less important than the anticipated help. And finally, of course a treatment will only be approved if the regulatory body determines that there is more to gain for the patients than to loose.

These things are really clear to everybody, but