Pharmaspectra interview: What makes a difference when identifying and engaging experts in rare disease?

Q&A with Dr Frank Tennigkeit, Senior Director, Paediatric Development at UCB Biosciences

Dr Frank Tennigkeit is Senior Director, Paediatric Development at UCB Biosciences. He talked to us about his 15+ years of experience in Clinical Development and Medical Affairs and what makes a difference when identifying and engaging experts in rare disease.

Pharmaspectra: What is the focus of your current role?

FT: Currently, I lead a team of experts from different functions to develop drugs for children with rare diseases. We develop the strategies and pediatric plans for submission, and conduct pediatric trials. These data should lead ultimately to the approval of the treatments and to bringing the drug to patients. In this context, we work a lot with external experts. Drug development is a team sport we work with many internal experts and externally with many experts as well.

Pharmaspectra: What is different about expert identification and engagement in rare diseases?

FT: In rare diseases, there are very few patients, and also very few experts. And there is also very little published data in some indications. Some are very new, there’s almost nothing published, maybe one trial, or no trial has ever been done. So, we need to understand the patient needs. Therefore, it’s essential to talk to experts who treat these patients and work with them and find the right patients. In these rare indications, because there are so few patients, this is inspirational, and very interesting, this is totally new, white space. And you want to really understand the disease to identify the right parameters to measure the disease of the patient optimally, to really bring out the value and demonstrate the benefits of your treatment.

Pharmaspectra: What are some of the essential elements for optimal expert identification and engagement?

FT: In several diseases, there are many experts, and there’s a lot of information out there, so we want to take into account all of this information. But on the other hand, we want to be flexible, to identify the right experts for the right tasks. And they can be very diverse – from identifying biomarkers, understanding the disease pathways, understanding how many patients are there in this indication – these are very different tasks.

Having a complete data set is very important. Because we work globally, these experts live across the world, in Asia and in America and Europe. And, they work in many different fields within rare diseases – involving not only therapy, but also diagnostics, and biomarkers. So you have a whole host of information that you need to integrate for this program to work, with several different experts across the globe.

For expert identification, it is important to use multiple sources, ideally, as many as you can. So not only publications, but also abstracts, claim to trial information, presentations at conferences. Again, this is especially important in rare disease, because not everything is published. You need to identify the few expert statements that are made from conferences, for example – that gives you a real-time update. What someone published 10 years ago may not be what they’re working on today, but if you see an abstract, and see this is going to be presented in a few months, you know this is up-to-date information, and you should be engaging with this expert.

Pharmaspectra: How do you manage your expert engagement across the spectrum of diseases?

FT: We work with a platform for expert identification and we use it both for broad indications like Alzheimer’s and epilepsy, and also for rare disease indications. Of course, the focus in established indications is you have many more experts, you have already established drugs, so we use it to understand the additional niche and more about the competition – what is new for your drug, what’s the added benefit. Whereas in rare disease, you really need to find the right experts because there are so few patients and you don’t know who’s actually treating them, and who provides you the best information on these patients. We also use it to collect scientific data and bring out scientific data for the first time. These patients often suffer in that the disease is not recognized, and no one knows anything about that and how to treat it optimally. All of these things together in rare disease highlight the critical relevance of working with the right experts.

Pharmaspectra: If you’re in a company entering a new therapeutic area, how does a comprehensive data set support your work?

FT: Expert identification using comprehensive data is especially important in rare diseases where you have very few patients, and often medical research is evolving – particularly in new diseases that are newly-defined, for example, by biomarkers. It is critical to identify the right expert and bring all these experts together. Often, one expert has a few patients, another in another region has a few patients, but if you bring the data together, you really get solid information, enabling you to advance treatments for all of these patients faster.

Pharmaspectra: What are some examples of how data-driven expert identification has helped you and your teams?

FT: We work in a new indication that has only recently been identified, and there’s a question for the right biomarker. So we’ve worked with biomarker experts, discovering that there have been experts already out there identifying the right biomarkers, and you have different assay types. The question is, what’s the best assay type to identify these patients, and to provide these diagnostic biomarkers as fast as possible? Because that leads to the identification of the disease and the optimal treatment. On the other hand, again, this disease was not recognized. In fact, it didn’t have a clear disease definition and a code. So you cannot find these patients in big databases. We worked with several experts with experience in recognizing this disease and we’re setting up a big registry to identify all the patients and summarize the available information, the needs of the patients and the disease course. We help them set up this registry to again bring therapies faster for these patients.

For different disease areas, you have adult experts and pediatric experts. It’s very important to have the whole universe of experts, and then to identify the right pediatric experts, because they are located in different hospitals, pediatric patients have different needs, often different treatments. To identify these experts, it is essential to have the right platform with all of the available data. This is key to our work and to bringing drugs faster to children with severe diseases.

Pharmaspectra: How has the platform helped collaboration in your team?

FT: This data and platform supports collaboration – with the experts but also within our team to have the same level of evidence, so we all know that these are the right experts to talk to; we all agree we have a neutral system for expert identification so it’s not based on relationships for example. Or we really know who is the right biomarker expert who sees these children, who is contributing to treatment guidelines. Internally, it enables us to have a unified way of identifying this expert to work together.

Pharmaspectra: What inspires you the most about work with experts in rare disease?

FT: Overall, we and the experts have the same goal. We want to bring drugs and effective therapies to children with severe rare diseases. You need to identify the right experts and work with them. We have different information so only together can we bring new treatments forward. If we all bundle our forces, we can really make a difference. First, understanding the disease, designing the right patient-centric studies to have the optimal data and finally to bring the drug to the patients.