Welcome to Pharma Talk Radio. This podcast is focused on how to better partner with patient organizations from the twenty twenty five Chief Medical Officer Summer three sixty. For more information on the CMO Summit, editorials, podcasts, or webcasts, please visit CMO three sixty dot org. Thank you and enjoy the podcast.
So I'm Joseph Palumbo, and yeah, I want to introduce these folks who are sitting next to me. They aren't quite remarkable individuals. In getting ready for this discussion, hearing their stories and their perspectives has really been quite fascinating, and I would like for them to share that with you, And assuming we have some time, we'll talk a little bit about my experience as well. But I think theirs
is really wonderful. So let me allow them please to introduce themselves in the order my left to right, and then after the introduction, we'll decide who goes first.
Sure to go offhabetical I hope. I'm Marie Abramson. I'm head of Clinical development and medical Affairs at Precision Biosciences.
I'm Laura Williams. I'm the Chief Medical Officer at our Daleks Small Biotech just outside Boston.
And I'm Peter Schmidt, I'm the president chief Scientific officer from BROW.
And Joseph PULLMBO at BIOV. So who's going first?
That's your call?
All right, I'm gonna go Laura first.
Great, So where where do you want me to start?
So talk to us? Why does this make sense? Why do we want to talk Well that's first of all, that's the wrong title. But why do we want to talk to patients and advocacy when we start a study, or even before we start a study? What makes that important and useful?
Yeah? I think at the forefront really of everything we do is the patient, right, and so you know when I think about the value of the patient's voice, that sort of reigned supreme in terms of all of the things that we do. And so for me, not only partnering with patients, but with organizations advocacy groups that connect
you to those patients is absolutely critical. It's interesting. In my last drug that we were able to get approved, it was for patients on maintenance dialysis to treat their hyperfocetemia, and it was quite a challenge, you know, we went through a couple of appeals processes. Crl ad come got it. Approved and I thought that was just great, right, good votes all of that, But what really was important was actually trying to make sure patients access to the drug.
And you don't really get a chance to do that if you haven't done the right partnering with patients as well as advocacy groups. And so for me, I think that was an excellent learning. It's not just getting a drug approved, but it's also making sure that the folks that you did all.
That work for actually have access to the drug. How did you engage them?
Yeah, So, I mean I think first and foremost, you have to identify the groups that you're most interested in. You have to ask the question, how can I help? In other words, you know, what are the things that are most important to you, your patients, the patient advocacy groups, and then what are the things that would be helpful to us because it's a shared relationship, right, And once you identify those things, you have to bring in and actually be real about. You know your intent about, you
know what it is you want want to gain. It's not just a one off where again you need this this from them, and so it's transactional. No, this is a real relationship. It you start building it early on, and you utilize it throughout every aspect of your drug development piece as well as commercialization.
So there's really that invitation to participate.
Absolutely.
Did you put together a patient advisory Absolutely?
I mean for both both our indications. One is an IBS C, the other one is, as I just said, in patients on dialysis. We have really strong patient advisory councils. And it was really obvious to me, and I think I shared this a couple of years ago when I was a year how important the patient voice was when we had the open public forum and one by one every patient you know, sort of came up in that
our session and basically spoke to the unmet need. And so just looking at the clinical efficacy, the safety, obviously all of those things are extremely important, but hearing why this matters to patients, I think stole the day and I.
Think that has to be very rewarding as a physician. Absolutely cool, We'll come back, Murray please. You have a wonderful story.
Yeah, you know, my perspective covers kind of multiple areas of my own experience, having been fortunate enough to do a fair bit of my clinical work, but subsequent to that in drug development, spending a substantial time at MSD, MERK and infectious diseases vaccines, among others, and then moving to biogen where there's always an un medical need and there's a lot of really needing patient populations that haven't
been tapped before. And I think that for me, there are multiple reasons to think about how to partner with a patient organization. The practical there are the ones that I perhaps don't have to convince a physician why it's important to be patient connected. I think from a practical perspective, I would say that it falls into multiple categories. One is the premise that all patients are not the same.
It's not a monolithic group, and I think for anyone this means that you have to get multiple opinions to understand whether the feasibility of your protocol makes sense and it's separationable.
That's one that's to be doable.
One two is because patients aren't a monolithic group, you have to make sure you're addressing a diverse population, bringing in depending on the phase of your study, the therapeutic area, that you're bringing in a wide variety of patients into your study. This may not be fashionable at this moment in time, but I have believed it for a long time and I still.
Do better days are ahead.
Yeah, So I think that there are multiple ways to do this. I think with the limitations of working in a drug company, you can. It's more difficult to do it individually, one on one, because you do run into the issues of one individual representing the entire patient. So I think patient organizations provide you an opportunity, and there's
a diversity of patient organizations that can do this. There's some that are more advocacy based, there's some that are more scientific based, there's some that are more political based. And I'll give an example to a companies which you I may And so from my own experience working in medical research operations, the goal of course is to be able to enroll your study and have success at the end with label and so clearly the feasibility component is
very important. Connecting with the right patient group is also very important. Building your credibility within the industry is also very.
And there's that wording and that credibility right invitation and the credibility.
And I have to say, based on my own professional and personal experience, based on health conditions within my family based on conditions that I've observed within my community. I think that it's particularly important to reach out to multiple patient organizations for this reason because what I've observed, For example,
I'll share personally. I think I shared on the line, Yes, you know, my brother passed with ALS a few years ago, and I happen to be working at a company that's pioneering within the ALS space, and it was at this time, and what I recognized was several things. One, the people who are being diagnosed with the disease aren't well connected necessarily. There's a lot of disparity and disconnection within the medical
community that prevents patients from getting access. That's correct, And so I think as a company, you have to be creative and thoughtful depending on your therapeutic area, depending on your disease, depending on the unmet medical need, depending on the moral urgency of the situation. In the case of ALS, reaching people earlier, reaching a diverse group, if you're looking for precision medicine and you're looking specifically for a particular genotype.
All the more reasons you did successfully.
And then when you talk about different patient groups, there are multiple different ways to do that, and I think different patients relate and connect to it in different ways. And again I think it requires a diversity of outreach to make that possible.
Yeah. Absolutely, and congratulations on having done that the right way.
I don't know if I did make it the right way, but by tried my best, I mean in the case of our trials, we were able to enroll them. I always feel like I've failed because ultimately we haven't solved
the problem yet. Yeah, it's an iterative process, but I think for when you interact with the patient, at least for me, I derive energy from that and you realize it orients you to do the right thing and to think about a particular problem in a more well, should I say, a higher level of thinking than just operationally from point A to point B.
Yeah. Absolutely, it goes both ways. It is energizing to be able to be a physician. So next to me is Peter Schmidt, who happens to be one of my good friends, and he came up here anyway. He has a very subtle sense of humor. And you guys can't see this, but he has mermaids on his tie and he told me he was going for a nautical theme, and so his braces have pirates, So thank you for that.
So you've been working in a number of fields in neuroscience for quite a number of years, both as an academic, a leader in advocacy of course, and as a developer. So your perspective can, in some ways, I don't know, sort of go over the top of everything else we've been talking about here. You've seen it in a lot of different directions. So comments, what would you what would you like to tell us?
So I spent about a decade working leading programs for Parkinson's Disease Advocacy Group, and one of the challenges that you all will face working with advocacy is that for the most of these organizations, their exposure to pharma is through the commercial division. Those are the people who often go out and first make contact, and they have a very different set of rules and a very different way
of engaging. And I always tell I'm currently the chairman of the Davis Finney Foundation for Parkinson's, and I tell them there is no better engagement than a development stage company. It's hope, it's opportunity, it's a way that people The most valuable donation a patient can make is their participation in a clinical trial. Absolutely that is the number one value. But many of these advacy groups don't understand that, and so it's important to go out early and make contact.
You don't have to go out with your checkbook, but just go out and have someone from your team or or someone from your office go out and make contact and talk to them about make sure they understand that you can that during your clinical development you cannot be promotional about your molecule. That you can't. They're afraid that they're going to be railroaded into something, but that's not lawful,
and they don't understand that. So you need to help them to understand how clinical research represents the most, you know, the best possible way that an patient advoacy group can work with industry. And I truly believe that, and that I tell that to the groups that I advise. The other thing that you need to watch out for is that, like in any space, and the politics were discussed this morning,
so I'm not discussing politics. There is a Dunning Krueger effect that you will see in patient advocacy, just as you see in so many domains. The people who come to you with the patients who come to you with the greatest confidence are the ones who are going to have preset ideas about which scientific avenues are going to be productive and which one they don't want to get engaged in the staff of Patient advocacy groups can help you get past that and find the people who hesitate.
I used to be.
On a panel for the Department of Defense funding Parkinson's research, and I got a friend of mine with a patient with Parkinson's. He got him appointed to be the patient representative, and every year he would say, I'm not smart enough. I don't think I should go back, and I would say, well, that's why you need to keep staying on this panel, and they kept him on that panel longer than any other participant because his insight and input was so valuable.
He has finally said he can't do it anymore. It's not his diseases progress and he can't travel. But he, for I think ten years, was a productive represented the patient voice, and his value came from the fact that he knew that he was not the embodiment of Parkinson's. He represented a community and so working. It's great to engage with these groups and work with the staff to identify the people who are going to have an open mind,
who are going to listen. One of the things that we've seen is that, you know, working in neurology, there are so many new approaches than there used to be. When I started working in Parkinson's disease, people thought there were only really two promising avenues, one of which was going to cure the disease stem cells and gene therapy dope minergic gene therapy, and those are not really major folks.
I Now in clinical development, there are a ton of different molecular pathways that we're focusing on, and the Parkinson's community does not understand them, and so it's crucial that well doing outreach, that we bring scientific training. We teach people what are what's going to be productive, what's going to why we think that this avenue might be effective.
And you can always get an independent SCIENTI as somebody who's not on your team, but at a university near where the headquarters is there always you can always find somebody who has the understanding and the independence to be able to talk about it honestly with that group. And that's what you'll need is just an honest discussion, Yes, this is something that's interesting, Yes it needs human trials for us to truly understand if this is the breakthrough that we hope it will be.
And again that's honesty credibility one more time. So before I say anything, you've all had an opportunity to talk to each other. He would you ask each other any questions based on what you've just heard?
Have lots of questions, you know, I guess from my
vantage point. You know, patient organizations are as different as the patients themselves, and so I guess from a Parkinson's perspective, are there and you don't have to name specific ones, but what I've observed in the ALF space and I know that Parkinson's space a bit coming from biogen, are there organizations that are more focused on advocacy or patient engagement that probably men amend themselves more to pharmaceutical development, education, patient awareness feasibility than others.
So I think in every domain, so that we used to say.
What are they?
Actually that was the real question is you know, could you share with me some of those thoughts?
So in every it used to be that in every disease you had one organization that was focused on care and one organization that's focused on search. But they will probably both be good targets. It depends on them, how on the personalities the people you talk to, It depends on the on You can't know in advance whether or not an organization has funded something, and sometimes if you look at their websites, you can see they're really pushing one concept and that's not what I'm doing, and that's
probably not the right group to reach out to. Sometimes they they are aligned, and they will be. Sometimes their research initiative aligns with your therapeutic approach, and then that's a great probably a great organization, unless they decide that there's the subtlety of the difference in what they're investing in is different. There are a lot of organizations who have taken a queue from the Cystic Fibrosis Foundation and think they're going to make billions by investing in a drug.
And I don't know how firewall that is from programs at different places, but you may find that their challenge is there if they're invested in something.
Yeah, yeah, I observed it. I observe that too. There's some that are profit motivated, some are, you know, and it also depends on the disease, how organized they are,
at what stage they are. I've interacted with some spaces, let's say in mitochondrial disease, which is still very nascent because there's not adequate therapy in those spaces, And they're very different perspective of organization and structure than maybe in Alzheimer's or dementia program, which is vastly different in size, but also in focus and professional experience.
Groups that engage parents of children with childhood diseases can are often really great to work with. They want any benefit, anything is, they're interested in anything. They don't just want Holy Grail cure. They want anything that can make their child better, and.
Those good points to work. It's a really good point.
Any Yeah, I mean I think it depends on the disease. I mean when I look at again and patients in the renal space, and we're talking about chronic kidney disease more specifically kidney failure. These are folks with chronic diseases. And when you think about some of the advocacy groups that come to my you know, National Kidney Foundation, American Kidney Fund, their focus is multi factorial, right, It's it's it's disease awareness. I mean, specifically with something like chronic
kidney disease. We're up to ninety percent of folks don't even know they have it, right, and so disease awareness education, you know, championing you know, more transplants as opposed to maintenance dialysis, actually providing financial so to some of those patients who actually don't despite you know, you know, sort of our antiquated payment system for those patients they don't have enough to cover just you know, simple things. And so I think it depends on the disease. It depends
on the organization. But they've been great to work with, at least in the renal space. When I look at the IBS space, not as many advocacy groups, but there needs to be more.
So absolutely thank you for that. I want to leave about five minutes for question and answering, so I'll take a couple of minutes and sort of talk about my experience. I've been mostly working in the rare disease space, orphan disease since I came to Farmer from academia in the nineteen nineties. It's wonderful what the belong to a community. It's engaging in this way reminds us or reminds me
of the fiduciary relationship we have as physicians. It's very easy in farm it's very easy in academia to put on your scientific hat, and sometimes informer to put on your commercial hat and think of, well, I'm doing this because X. But in speaking to families and speaking with advocacy and talking to patients and people who don't know their patients yet about what they want and what's meaningful, it educates you. It makes you a better physician, it
makes you a better drug developer. It helps you to really hear the voice of the patient. There have been times where I brought things forward to global health authorities and not been sick successful and having the community there knowing that I had engaged with them and we were working together really helped to soothe a lot of that hurt when you know we didn't get to hear what we wanted to hear. I've taken that into my career and it's been one of the most fulfilling things to
be able to reflect what the community wants. And I
would say it's not just working with advocacy. I had the privilege of working in translational research in Japan for a number of years and one of the ways that we were able to advance molecules was to go into the laboratory and talk to the individual scientists about the cares they have in their families, the diseases, the conditions that are still unmet, because for many preclinical scientists, they read the literature, but they kind I do forget or
unaware that they're there to produce medicines and to return it to them and say, Okay, here's an opportunity to do something for someone you care about. Are you interested in making that molel you are interested in developing that program. It's been really a privilege to work with these communities.
So we have about five minutes left, and I'm hopeful that those of you who've worked in this field or or have thoughts will share those thoughts and experiences, because it's not just about bringing the advocacy group to the advisory panel. At the end of the day, it's about creating medicines. So any thoughts, Yeah, I had a question for you.
So many of us here in small biotech companies, so we don't have a big organization. We don't have anybody in patient advocacy necessarily at our companies. And if you're going into a rare disease where maybe there isn't a well defined patient organization or advocacy group. Do you have any suggestions of how we get to the patients, how we get the voice of the patients.
So let me first start by saying, we have about ten people in my company. Small company. It can be done with. Let me these folks provide you with some advice please.
I was whispering, but I guess not in my microphone. I was just saying, at my current company, it's a very small company, right, it's a gene editing company, and they're working in various areas. One of the areas happens to be mitochondrial disease. And so that is an example
that you're talking about. It's not well organized, but if you compare to big pharma and all of that, that actually I get the opportunity to dip into it and get to meet and interact, and it's a very rewarding thing because they actually don't come to you with preconceived ideas and they're very welcoming of all interactions that they have.
The community itself is still relatively small, it's still very scientifically based, and they have these events where both patients, their families, the scientific community, the medical community all come together. And what I've observed is on the early side of a diseases, that's kind of where it starts. Then it gets really big where you wouldn't have that in a cardiovascular some cardiovascular conferences or some let's say, I don't know,
let's make it expand it out to even Parkinson's. I would say it would be big enough in size that you might not have that same. So I think part of it is just reaching out and making sure you're interacting. If it's a global effort to do it globally, there's usually a couple major organizations in the US within this space, within Europe, within this space, within Asia, within this space,
and just sit down and build a human relationship. And that seems to be a common theme that we've heard this afternoon, is building that connection.
And the other place you might look is academic medical centers often have the day day events for their diseases.
No, I mean, I don't have anything different there. I think again, it depends on the space. You were speaking in terms of rare diseases. I think in in in the case of you know, well known more populated chronic ny disease. There there are there are a lot of patients out there, but I think it's still important to be able to show them right who you are and that you're there for them, not for any other you know,
sort of altruistic way. And so wherever it is, whether it's rare, whether it's uh, you know, more chronic disease states, I think building those relationships is just really key. I mean, we I love I mean of all the meetings that I have when we meet with our patients, when we have our patient council meetings, that's the best part of the day.
It's very meaningful. I would suggest again, as you just heard, reach out to the academics in the field. I've worked a lot in childhood developmental disorders. Reach out to those physicians, and frankly, it's a wonderful way to get a sense of whether you're going to be welcome, right. If you can explain yourself well and they say cool, that will be part of the additional invitation. I'm trying to see a face yes please.
Albert kim ra a Ventures. One of the things we've noticed about patient engagement and organizations, which by the way, is absolutely fundamentally important and critical new biotech sponsors is that in certain situations, the patient advacy a advocacy groups and organizations don't necessarily reflect some of the patient opinions. Could the panel comment on that topic when the advocacy groups aren't necessarily aligned with some of the patients they represent.
And second, how do you all feel about compensating patients for their time in clinical trials, which is a polarizing issue. Let's say in oncology versus more common diseases.
Who wants to shot at that?
I can talk a little bit about the latter, and maybe you know, compensations are very complicated. You don't want to be in a position of inducement. The fact of the matter is clinical trials are biased ways of doing studies. People are more educated, more affluent, centrally located in certain geographic centers. Its gender, and its race. And I think one of the ways to be cognizant to bring in a more diverse population is to understand the hardships that
patients have. And sometimes people have to leave work, people have to stay family members have to give up things, and it's economically not possible. And so for that reason, I think it depends on the therapeutic area and what you're talking about compensation, But it really is something you can focus on again of giving people the right amount of money to make up for the time lost to participate in a particularly important venture. But it's a balance of ethics and regulatory ability.
Who would like the last one? When advocacy doesn't necessarily represent everyone.
Harari and his book Sapiens says that groups form because they have a shared belief in something that's not true. And that's true of patient at groups as well as all other groups. So if you find a group whose identity is based on something that is not that is contradictory to what the hypothesis for your intervention, you probably need to find it another.
Group or work them over on the long run. I at this point would say thank you to all, and also historically thank you to the AIDS community for really bringing this to the four or forty years ago, because before that experience, which I lived through, we didn't listen as much, and I think we now ask ourselves are we listening enough? And I hope we continue to listen. Thank you, thank you.
We hope you enjoyed the podcast. For more information, about the CMO Summit three sixty editorials, podcasts, or webcasts, please visit CMO three sixty dot org. Thanks for listening.