How should I select the right endpoints when creating an Effects Table?

Results based on a survey among pharma and academia

The Effects Table (ET) has become one of the most common tools in the area of Benefit-Risk Assessment. It displays a concise summary of the key benefits and risks of a new product compared to either placebo or an existing product. See here on more insights into what an effects table is and why it is helpful.

On initial inspection, it may look simple to create. However, those who have already tried to do the exercise know that it is not at all easy.

IMI Project PREFER – patient preferences in benefit-risk assessments – replay of the webinar now available

By Conny Berlin and Rachael L. DiSantostefano

In our 1-hour webinar on the 12 of July 2017 (4:30pm CET, 3:30pm GMT, 10:30am EST) we had a presentation by Conny Berlin who is the industry project leader of the public-private IMI PREFER project and by Rachael L. DiSantostefano member of IMI PREFER. They talked about:

Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle: PREFER – an IMI Project

by Conny Berlin and Rachael L. DiSantostefano

The replay of the webinar including the Q&A is now available here:

Abstract:

The main objective of the IMI PREFER project is to strengthen patient-centric decision making throughout the life cycle of medicinal products by developing evidence-based recommendations to guide industry, Regulatory Authorities, HTA bodies, reimbursement agencies, academia, and health care professionals on how and when patient-preference studies should be performed and the results used to support and inform decision making.

What is this effects table and why should I create one?

8 reasons for creating an effects table

Originating from the EMA Benefit-Risk Methodology Project, the effects table has become one of the most commonly discussed tools in the area of benefit-risk assessment (PrOACT-URL (Oct16)). In fact, its prominence has grown significantly since February 2015 when it became a CHMP requirement for EMA clinical assessors to include an effects table in European Public Assessment Reports (EPARs).

So, what is it?

Benefit-Risk in HTA and best practices based on case studies

have a look at the webinar

In our webinar in Feburary 2017, we had 2 presentations from widely known speakers:

The emerging and merging fields of benefit-risk and health technology assessments

by Jason (Jixian Wang), Shahrul Mt-Isa and Susan Talbot, on behalf of the EFSPI BRA/HTA joint working group

Benefit-Risk Assessment via Case Studies: Key Considerations and Best Practices

by George Quartey

The webinar occured twice and the recording is now available here.

Here is some further background information about the content and the presenters (here is the pdf-file for further distribution):

The emerging and merging fields of benefit-risk and health technology assessments

jasonAbstract: Benefit-risk assessments (BRA) focus on clinical aspects of health care products and are often seen as purely regulatory activities, while health technology assessments (HTA)

Examples of benefit-risk assessments comparing antipsychotic treatments

2 publications on using BRAT and DCE in the same setting

Levitan et al and Katz et al have recently published research on the comparison of treatments against schizophrenia. Both papers are available under open access. Levitan et al. report on a clinical study results from two similar placebo controlled studies. The indirect comparison was not done via a classical Bucher method but using IPD data as described in Markowitz et al (2013) which also contains the basis for this post-hoc analyses. Levitan et al performed a post-hoc benefit-risk assessment using the Benefit-Risk Action Team framework. The key benefits and risks are reported using a value tree. The benefit-risk assessment differentiates between short term (8 weeks) and long term (40 weeks) outcomes. For both time points similar effects tables and associated plots are provided, that display the events per 1000 patients and the response or risk differences with 95% confidence intervals. Continuous outcomes like PANSS and CGI-S as well as weight increases were dichotomized using common criteria.

In a related paper Katz et al describe how patients and physicians in these clinical trials quantified preferences related to benefits and risks of antipsychotic treatments. Via the used discrete choice experiment, the authors assess the impact of prior patient adherence on the relative importance of treatment efficacy and formulation. This publication shows a nice incorporation of such a preference study within clinical trials and might be the first time, that such a DCE as successfully implemented in such a trial.

Did you find this reference helpful? Would you like to learn more about it? Please provide comments below.

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!