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?”

My current plan for the content is to talk about

  • Overview of BR
    • What is a framework?
    • What is quantitative vs qualitative?
    • What are the different initiatives in this field?
  • Stakeholders like NICE, ISPOR, ….
  • Patient initiatives and other ongoing topics (IMI projects)
  • What is the role of the statistician?
  • What is the EFSPI/PSI special interest group on benefit-risk doing?
  • What are the recommendations regarding simple steps that everybody could do?

If you are new to this topic, would that be something, that you would like to hear about or is there something else? If you have already some expertise or experience in the benefit-risk area, would you add something different? Regarding my last point – what recommendations would you have?

Please leave a comment and I promise, that I will reply!

Please note: I reserve the right to delete comments that are offensive or off-topic.

Leave a reply and share your perspective for others to learn

6 thoughts on “What would you like to know about BR as a beginner?

  1. Hi Alexander,

    Great, I`m looking forward to your talk!

    I do have a suggestion which you may want to park for another opportunity as it is falls in the post-approval phase but at least let me suggest it so that you know what it is …

    What about the benefit-risk (b-r) assessment from the payer perspective? How do we incorporate the b-r framework into the budget impact analysis (BIA)?

    From a b-r perspective, what types of modeling could we perform to provide into the BIA to guide the impact by incoporating the b-r assessment?

    How might we think about modifying the b-r plots to incorporate BIA or the Cost-Effectiveness Analysis to graphically convey to this stakeholder?

    Thanks,
    Pritibha

    • Hi Pritibha,

      Interesting comment… we have also been working in a small group to blend the BR assessments for regulatory activities and those for health technology assessment. We recognise that there are some overlaps in terms of requirements and also methodologies. We hope that the two assessments could be more streamlined in the near future to increase efficiency and consistency.

      Would you mind sharing how you handle the BR assessment from the payer perspective within your organisation?

  2. Hello Alexander!

    I am excited to see that the methodological issues surrounding benefit-risk assessment continue to be of interest to many, and many more are interested in the area but are not quite sure how to get involved.

    For this talk, I would be interested to hear about your personal account, as an industry statistician, on how the various BR initiatives and stakeholders have helped you promote and shape the benefit-risk assessments within your organisation, and more widely within the EFSPI community. One of the advantages that I have seen is the camaraderie that develops, and the openness to discuss and to share expertise and resources towards a common goal. This in turn created an SME platform of mutual benefit and respect that extends beyond that of any organisation is capable of acting alone.

    Being new to the pharmaceutical industry, I am very interested to learn how to get involved in areas such as benefit-risk assessment especially if it is not exactly your “day job”. I may have certain advantages having been working in the area itself for several years, but other interested individuals may find it difficult to even start (or getting back into) not having dedicated time to pursue it. I find myself asking, how, repeatedly…

  3. Alexander,
    In addition of what you have already plan for the content of your talk, we (like Shahrul) would also be interested to hear how you have shaped the Structured Benefit-Risk Assessment within your organization.
    To embed new processes is challenging and we are keen to hear how companies are managing it.

    • Thanks for the suggestion. I’ll talk about this but I also want to give some thoughts here.

      1. I think it is helpful to get an understanding on who is already working on this. Is there a dedicated group like at Lilly, that is focusing on benefit-risk. Who are the stake holders there? What is their interest? Are they working on all aspects or maybe they focus on certain parts of the business like regulatory submission? Is this all centralized in the head-quarter or are the activities going on in the different affiliates? HTA related topics might be more local as the HTA submission teams might be local. Are there health economists involved in these HTA submission and do they run patient preference studies on a local level?

      2. Then, I think, it is good to build a network or maybe there is already an existing one. This could be an informal network, that meets more or less regularly to share what’s going on. If you can contribute to this network regularly, this will grow your profile and help you to shape things in the future.

      3. Think about how you can improve your deliverables by including benefit-risk features into it? E.g. if you are writing a paper about the main outcomes of a study – can you include an effects table or plot into this? Share this experience with your colleages and your network.

      4. The case studies then can create new expectations on things and these then might lead to different processes if necessary.

      5. Finally, I’m sure it helps if you have gained some credibility by contributing to the overall stats community on the topic. If your name is known in the field, this will help you to gain credibility within your organization as well. This will open up new opportunities within the organization, which in turn helps you with projects outside your company.

      Was that helpful?