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Medical Affairs in Rare Disease and Gene Therapy – Why a different approach?
In today’s episode of the MAPS “Elevate” podcast, we’ll be looking at considerations for Medical Affairs in Rare Disease and Gene Therapy and reviewing the following questions:
Fatima Ahmad 00:00
Hello and welcome everyone to the Medical Affairs Professional Society podcast and today we are on the Focus Area Group of Gene Therapy and Rare Disease and this is part of a two-part Podcast Series. My name is Fatima Ahmad, and I am the Vice President of Global Medical Affairs at Hansa Biopharma. And I’ll be the moderator for this podcast. Currently, I am the Co-Lead of the Gene Therapy Rare Disease Focus Area Working Group. And I’m very pleased to bring this content to you. But before we start, just a disclaimer, the views expressed in this recording are those of the individuals and do not necessarily reflect on the opinions of MAPS, or the companies with which they are affiliated. And this presentation is for informational uses only, and is not intended as legal or regulatory advice. And it’s my pleasure to introduce my distinguished guest today, Dr. Bartholomew Tortella. Bartholomew has been my boss and my mentor, and in a previous professional life, and I’m so happy to have him join us today. Bartholomew, welcome.
Bartholomew J Tortella 01:14
Thank you very much Dr. Ahmad and MAPS members. It’s my pleasure to be here today to talk a little bit about Medical Affairs and rare disease and gene therapy. I was in the pharmaceutical industry about 23 years and started in Medical Affairs but a little time in clinical development, but then returned to my home which I really felt was Medical Affairs and took on positions of increasing responsibility. And as Dr. Ahmad said, she and I were together as the Medical Affairs team at Spark therapeutics where we were very engaged with both rare disease and gene therapy. It’s my pleasure now to turn it back to her and look forward to our discussion.
Fatima Ahmad 02:00
Yeah, fantastic. And so the topic today that we’re talking about is the role of Medical Affairs in rare disease and gene therapy. And why does it necessitate a slightly different approach from other therapeutic areas? So we’ll dive into that a little bit and explore how we think about it differently, or how do we think about it, period? So, as a starting out this discussion, Bartholomew, how do you see the role of Medical Affairs evolving in the era of advanced therapeutics, like gene therapy, which often is investigated for rare disorders?
Bartholomew J Tortella 02:45
Yeah, Medical Affairs for rare diseases, and specifically, gene therapy is a whole different animal than the classic Medical Affairs that we associate in the past. And there’s several reasons for that, certainly, because Rare Disease is a highly technical area involves very complex diseases. And then piled on top of that is the complexity of gene therapy, a technology which is rapidly developing and important to understand not only from our side and Medical Affairs, but also the practicing medical community. I tend to think of the role of Medical Affairs as a three legged stool when it comes to rare disease and gene therapy. First of all, unlike many other areas of Medical Affairs, we are scientific educators, actually, for the practicing medical community. And I say that because many of the things in gene therapy that clinicians did not learn about in their graduate training, in their residency, their fellowship, and they’ve never had a patient so that the Medical Affairs professionals are often experts in their particular area of gene therapy. And as they go to the individual practicing medical community, they need to be very well versed, the basic science, the immunology, the very specific nuances of the clinical trials, and how those clinical trials hopefully will result in a an approved product. This is much more complicated than it would be if you were talking about small molecules or other well known therapies and the clinicians expect the Medical Affairs professionals to have a very high level of scientific expertise. Second of all, Medical Affairs is well known for insight gab. It’s no different here. But the Insight gathering is a lot more nuanced in that you won’t necessarily get questions about the gene therapy, you’ll get questions about the gene therapy and the rare disease interaction. You’ll get questions about the immunology, the control of the immunology, the different approaches to the clinical trials. And all those questions turn into valuable insights that you bring back, not only to your Medical Affairs colleagues, quite frankly, but also to your clinical development colleagues, and then to your research colleagues, especially at the immunology ones, in addition to our marketing colleagues, who were always interested in the pulse of the medical community, because those scientific insights would span the research area, the clinical development area and the Medical Affairs area, which is really quite unique to Medical Affairs from Rare Disease. And then finally, we really are our pharmaceutical biotech companies, doctors. And they look at us for consultations. This may be joined by a question, or perspective, or us giving spontaneously some advice on how to pursue not only a marketing approach, but very much more commonly a Clinical Affairs approach, or a scientific approach. And we need to be thinking on many levels, the basic science level, the clinical development level, the Medical Affairs level, and that unique piece of the equation that is unique to Medical Affairs. What would the practicing medical community say? How would they react to this particular research effort, or this particular portion of a clinical trial? So for those reasons, being specific scientific educators, sophisticated insight gathers, and in house medical professional consultants, you can see the rare disease, gene therapy, Medical Affairs, takes Medical Affairs to the highest level.
Fatima Ahmad 07:22
That’s fine. That’s a fantastic construct, Bartholomew, so the three legged stool scientific educators gathering insights, and in house medical professionals. And you know, one of the things that you and I discovered when we were working together is, you know, clinical experts. In a particular disease area, there are experts there, but they’re not gene therapy doctors. And so part of the Medical Affairs remit was to actually give them that expertise and give them that familiarity and ease with the topic at hand, so that they are then equipped in having those conversations in turn with the patient. I mean, they can talk to the patient about the specialty at length for sure. But when talking to them about trials, clinical trials and gene therapy, that was where the real gap was, and where Medical Affairs could step in and help educate and make them comfortable and having those those dialogues
Bartholomew J Tortella 08:30
do your job. You’re exactly right. And that also made us invaluable partners, to the clinical development, because we would often bring back insights to them, which would allow them to craft their protocols in a more expeditious fashion. It would allow us to open doors to them if we didn’t want to do their work their clinical trial. It’s we’re not, but just exactly as you said that familiarity with the practicing medical community really allowed our clinical development colleagues to go further faster, because of the support that we could offer.
Fatima Ahmad 09:13
Yes, yes, exactly. So in summary, you know, the specialized skill sets, Medical Affairs professionals need teaching skills, listening skills, and as you and I just talked about being steeped in clinical medicine, but also in gene therapy as a field of technology. As one very esteemed colleague, Dr. Cathy Hi once said that, you know, in concept gene therapy is very simple, but in practice, it is not the case. And so, as the science evolves, and as we learn more and our understanding develops, that is both went hand in hand in bringing that information over to the Kol and the HCP. He’s, but we were helping to educate. Very good. So maybe Switching gears, a little bit of gene therapy and rare disease, particularly go hand in hand, just coming to rare disease now for a bit. It from your perspective, are there, you know, any major differences in approach to Medical Affairs planning and rare disease, whether or not there is a gene therapy or novel therapeutic involved? What are your thoughts?
Bartholomew J Tortella 10:30
Yeah, I think rare disease really calls for your highest level of performance as Medical Affairs professionals. And the reason I say that is, the number of practitioners involved in any rare disease is very small. And so you must be laser focused on them, going to them with the highest level of expertise and preparedness. And they really expect that they don’t want somebody just in the first few years of Medical Affairs to go to them on a company know, obviously, you can cope with a colleague as you as they learn the craft of Medical Affairs. And they expect you to have read all the latest information on your area of rare disease, all the traditional treatments, all the upcoming treatments, and be very well aware of the mechanisms of action of all of those things, not on a theoretical but really on a molecular level. Because so much of what happens in rare disease happens at a molecular level. And the treatments being investigated in the current wave of rare disease and gene therapy are all at the molecular level. So I think the first thing I would say is, it’s a very high level of scientific expertise that transcends the normal approach that Medical Affairs colleagues can take. The second thing is it’s not just giving the same approach to everybody. And as you pointed out, just recently, in what you said, you are going to be interacting with rare disease colleagues who are experts in that rare disease. But you’re also going to be really interacting with individuals who treat that rare disease, but not in the same intensity and focus on I’ll give you an example. We both work in haemophilia, there are a number of patients with hemophilia who receive their treatment at internationally known hemophilia treatment centers. So obviously the approach and discussion when you’re talking with the faculty of an international hemophilia treatment center as a particular style and approach, but many patients also retreated further hemophilia by hematologists, at hospitals, which are not international centers, but they have a clear need for knowledge, understanding insight, and they have a whole different series of questions that those individuals at the International centers. So the Medical Affairs practitioner must be ready to talk to an international expert on one day, and the next day to walk into a situation where you have an individual who knows well, that rare disease, but it’s not at the same position in their knowledge or their practice, as an individual who does the hemophilia as their life’s work. And it’s that ability to flex and take a bespoke approach, which is really very, very important to effective and important communication in Medical Affairs, rare disease.
Fatima Ahmad 13:58
Yeah, those are great points. I think one other area that I’m thinking of, as you were speaking, Bartholomew was that patient organizations in rare disease are very active. And typically the rare disease patient, depending on the disease tends to be informed, very well informed about treatment options that are in research are under investigation. And so as the Medical Affairs professional, and as we’re writing out our medical plans, that’s another area that we have to keep in mind integrating the patient voice, looking at our tactics and strategy, and liaising with patient advocacy groups within our within our area as well as with patient advocacy colleagues within our organization to bring that view into the Medical Affairs plan.
Bartholomew J Tortella 14:55
No problem. You hit it right on the head. All of us In Medical Affairs at one point or another treated patients, and somehow when you went into BioPharm, or farm, you found yourself distanced from the patients. But that is the real treat the real part that enlivens and energizes everybody in Medical Affairs, rare disease is the interaction with the patient societies. And indeed, with the patients themselves, they often have patients, society meetings, or Medical Affairs attends and discusses. And so you’re back in your element, you’re back in that area that gives you energy, when you find yourself interacting with patients, either on a leadership basis because of their patient advocacy group, or actual individual patients, who also want a little bit of information about the science and the rare disease aspects of their condition. And I found professionally, that was one of the aspects of Medical Affairs rare disease that was most energizing and most satisfying for me, because I got back to the patient, which of course, is our North Star in whatever we do in Medical Affairs.
Fatima Ahmad 16:17
Yes, absolutely. It really was just very, very rewarding. Understanding and, and, you know, just being closer to the patient in this area, that in some other areas, and actually being able to visualize how this therapy could could help a patient population that really has no other treatment options. Okay, well, this is such such a great conversation, and I’m really enjoying exploring these topics with you. So as you know, one of the one of the goals of our conversation here is to help our Medical Affairs colleagues, figure out if this is an area, they would like to work in both rare disease and gene therapy novel therapeutics, like gene therapy, which are very exciting in concept, but as we touched upon earlier in reality may differ. So, in your experience, and your perspective, what are some of the challenges that our Medical Affairs colleagues may want to think about as they consider working in this area?
Bartholomew J Tortella 17:28
I think the first thing is to realize the amount of time education and focus that it takes to really gain command of the basic science and the clinical science of the rare disease, first of all, and of your particular modality, that would be for example, gene therapy, and to not start with a cursory example, but really dig into all the basic parts of the vector, all the basic parts of the molecular biology of the disease itself, and then put in the time necessary to really have a commanding knowledge of those. But don’t stop there. It’s a lifelong learning situation. And so Medical Affairs individuals are always the ones going to clinical develop and saying, What is new in the clinical trials, they’re going to the basic science group, and saying, what new things are you’re doing or new approaches you taking, so that they can maintain their level of knowledge, not just on what’s been published in the literature, although that is important, but also the cutting edge that is being put forward by their company itself. And that’s a never ending opportunity to stay current on all those things. And also, as things change in their rare disease, or their genes or therapy, to be aware of that. And that’s why I say that you have to read literature, I myself read seven to 10 journals a month. Not every journal cover to cover, but tables of contents, so that I would be able to know what’s happening in that area of the rare disease, and in that area of gene therapy. So get steeped in that art of clinical medicine, rare disease, molecular biology, what’s going on state of the art in gene therapy, and break it all down to a molecular level. Now, that’s the easy part, if you will, because all of us are good learners. We study hard. We’ve done well, etc. Now comes the hard part, you have to be able to break down that knowledge you’ve obtained into easily and quickly understandable units, because no one’s gonna sit down and talk three hours with you is busy clinicians, busy researchers, practitioners of any stripe don’t simply have that much time, the hard thing is to take your extensive knowledge base, and put that into understandable discussions, time limit of 10 or 15 minutes, if you will. And that’s a tremendous task, because you have to know what to leave in what to leave out how to answer questions, and then have a particular sense of whether you’re talking to that international expert, and how you’re going to explain things to them. Or that person who is a specialist in their area, but not necessarily an international expert. And that’s where I see a lot of people fall down. They know the knowledge, they control the area. But when you talk to them, it’s like they’re talking to or reciting some of the material they’ve read. And what I’ve always said is, tell me the story of what you’re trying to communicate, break it down, use examples, keep the line with straight forward. And that I think, is one of the things that separates the beginning Medical Affairs expert who is very erudite book read from the superior, well seasoned Medical Affairs practitioner, who can take that knowledge and somehow explain it effortlessly, in a way that can be understood by the audience to whom they’re speaking.
Fatima Ahmad 22:02
You know, as you speak, I’m reminded of one of the unique challenges that I encountered a while working in this space. And so as we looked at how to develop the HCP landscape in advance of a gene therapy, I realized that there were three distinct groups of KOLs. So you have your clinical experts who are experts in the disease area. And then you have your scientific experts, who are actually very well versed in gene therapy have written extensively around it around the technology, the vector, for example. And then there is a very small group of individuals who know both. And that’s typically a handful of people for a given disease area. So as a Medical Affairs person, as you think about developing education, or thinking, think about maybe developing educational reviews, you have to bring these three groups together. Because peer to peer education, as we know, is is highly effective. But what peers are you talking about? So the clinician peers, you know, the disease area, peers are going to be looking for peers in their group, the scientists are going to be looking for peers in their group. But then to make have a paper be truly considered effective or widely read or recognized as a clinical expert point of view. You need both groups, all three groups, in fact, and how do you identify and bring them together as a cohesive author group? That’s also one of the unique challenges that I encountered working in this space?
Bartholomew J Tortella 23:56
Oh, yes, very much so. And then I have to resist the patient to dumb things down. You never want to dumb things down. So one of the things why I went to business school and one of the things they taught us in business school is exactly what you have articulated. See the picture in your audience’s head, take a sense of the audience and those three archetypes that you articulated, will stand every Medical Affairs practitioner in rare disease and gene therapy in very good stead as they walk into it and prepare mentally for a discussion. ask themselves, what is the picture in this individual’s head? How do I best Connect? How do I best interact? And how can I best help that person in their educational pursuit? You’re spot on?
Fatima Ahmad 24:54
Yeah, it really is building a community of experts, isn’t it? Well, great So I think as we are wrapping up this first podcast on the topic, my last question to you, Bartholomew is looking at how fast the field is developing and evolving. Do you think that there might be specialized roles within Medical Affairs that may be coming up that are different from current roles, either in the rare disease or gene therapy, or a combined space? What are your thoughts?
Bartholomew J Tortella 25:26
I think it’s already here. I think those Medical Affairs individuals who are in rare disease, and in gene therapy, or one of the other novel therapeutic regimens represent the elite of Medical Affairs. And that puts on them a particular responsibility, in that they have to teach and mentor other members of the Medical Affairs community, on what that area of expertise looks like. So it’s not your regular Medical Affairs, it’s a very specialized and we have a responsibility to teach, and mentor Medical Affairs professionals as they get there. The second thing I also believe is, it’s not just sufficient to go and see your clinical professionals or work in your own region, it’s very important to get involved in leadership of professional societies. And MAPS is the perfect example. To give you an example. When you get started Medical Affairs, you’re looking around, you’re trying to learn the craft, you’re looking at those people more expert, and as I said, many of them are already in the rare disease, gene therapy. But the one thing that marks the rare disease gene therapy group is they’re also leaders in their profession. So if you were to take a look at MAPS, I would recommend first of all, some member I mean, that’s there’s no question about that. Second of all, just don’t stop there. Go to the meetings, even whether there’s local meetings, or regional or national, and then second, raise your hand. Yeah, I’ll be on that committee, for all organize that meeting. And that’s the way you take your practitioner thing of Medical Affairs to that next level, because it gives you the opportunity to see people from other areas of the country, other disciplines, and to sharpen your leadership skills, which are extremely important when it comes to Medical Affairs, gene therapy, rare disease, because that’s the leader, leading edge of Medical Affairs right now, in, in my judgment. So be aware that you are at the elite level, if you’re in rare disease, gene therapy, be aware that that comes with responsibilities of teaching, and mentoring. And be aware that you need now to get involved in those professional societies like MAPS, not only just as a member, but also an active member who works in committees, conferences, meetings, to really help all those individuals in Medical Affairs, bring the needed information to the practicing medical community and the patient care groups.
Fatima Ahmad 28:28
Yeah, very, very well said. And, you know, I would I would just add that as, as professionals, we tend to get obviously very, very busy in our day to day, our day jobs. And sometimes it seems, okay, how am I going to find the time to engage in these types of endeavors. But honestly, what I’ve learned in my time working at MAPS, it’s actually a sanity check. That actually has helped me feel less isolated when I talk within a group of, you know, like minded Medical Affairs professional colleagues, you know, they may be working in Big Pharma, medium sized small biotech, and you come to realize that we, you know, we face very similar situations. And so that is actually a great break for your mind is tired from the day to day work, to come together in this peer to peer type of conversation, and simply learn and relax because you’re not really fulfilling any, you know, specific objective at the time. It’s really holistic learning. So that’s great, great advice.
Bartholomew J Tortella 29:44
One of the things, one of the things I’d like to say is you said, Where am I going to find time for this? So I’d like to pick that up and amplify it. Yep. Is your supervisors responsibility to make Time for you to do this. I was blessed throughout all of my time in pharma, with bosses, who always told me what are you doing to develop your career? And what can I do to help you. And I was given performance ratings, based in part on how members of our team, develop their careers, went on to get promoted, went on to take local, regional, national and international positions of responsibility. So please don’t feel that you have to do it alone. And I know you and I know this very well, because we’ve had many conversations, but bring your boss, your manager, supervisor, whatever you call them into this equation, because it is part of their job as their as a leader in the organization, wherever they are, to take specific efforts to develop the career and capabilities of colleagues within their organization. So please bring them in, because you don’t have to do it alone. And I find one of the most fulfilling parts of my career has been those individuals who’ve gone on like yourself to take executive leadership positions in in many areas of BioPharm and Medical Affairs.
Fatima Ahmad 31:33
Fantastic reminder, and, and very well said, Bartholomew, and I truly appreciate the support that I received from you. And likewise, encourage listeners to explore these opportunities within your organizations as well. So sadly, our time together has come to an end. This has been a very enjoyable conversation, and I hope our listeners find it useful as well. As we wrap up, I would just like to reiterate that, you know, we encourage you to engage in conversations about gene therapy and rare disease with other MAPS members via the MAPS LinkedIn page. And please do visit Medical Affairs.org/events to participate in our upcoming webinars and in-person conferences. And as I said in the beginning of the podcast, this is a multi-series conversation so we will be back with more exploring this topic. So Bartholomew, thank you very much for your time. Thank you. Take care. Okay, well, bye now.
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