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Rare Disease Summit Recap


Greater Than One’s Chief Strategy Officer, Andrew Bast, shares his takeaways from 2024 Rare Disease Summit in Philadelphia, PA.

Andrew Bast, Cheif Startegy Officer, Greater Than One

Andrew Bast, Cheif Startegy Officer, Greater Than One

Greater Than One’s Chief Strategy Officer, Andrew Bast, came away from the 2024 Rare Disease Summit in Philadelphia, PA, held this year on March 18th-21st, feeling empowered and inspired. I recently connected with him for a Q&A on his experience and what takeaways he found most fascinating and profound.

Miranda Schmalfuhs: Did you just follow a dedicated track for content during the summit, or did you bounce around? What did you attend?

Andrew Bast: So generally, the areas I want to go are the roundtables and the discussions because that's where I find the most interesting debates, quite frankly. That's when you get different perspectives, different points of view, and you can see how different organizations are tackling the challenge of rare disease.

I attended about five of them. One that I found to be really interesting was done by Raregivers™, which was an entire debate and dialogue about those who care for people with rare diseases, and how it’s so important that they themselves take time to care for themselves, and to do so without guilt or shame. Because it is important for their own health, physical and mental, that they actually take time to focus on their own bodies, their own mental state, their own health. Rare Disease is such a complex area. It's not just about the latest scientific discoveries, it really is about the people with whom we're all invested.

MS: Recently on Pharmaceutical Executive, we’ve been talking a lot about personalization. I feel like there truly is nothing that is more personal than cell and gene therapy, and the therapies for rare diseases. They have to be so personal. Patients are quite literally shipping their cells out to be made into these therapies. I's such fascinating science.

AB: It is. I would say Miranda, for me, I had three big takeaways from this rare disease conference. One of them, of course, is about the emotional investment that one finds at these conferences. They truly are emotional, but inspirational at the same time. That is a consistent trend, and I'm glad to experience it. This year, I noticed a much bigger shift into the important role that AI will play and how AI is the future of medicine, in particular the future of rare disease medicine. The third one is the need—we must find these patients. We must find them, we must locate them, we must diagnose them; and we have even greater tools, more powerful tools at our disposal than ever before. It still remains an inspirational place to go and it reminds me of why I chose this industry as my vocation and why I'm staying in it.

MS: I'd like to dive in a little bit more to the shift to AI. Can you tell me a little bit more about what was presented on that topic?

AB: The role of AI in rare disease is fascinating, because what it does is it marries the understanding of the clinical pathway, the pharmacologic properties of the agent that you're discovering, and the genomic sequence that each of us have as individuals — very distinct. So, with AI, all of that can be modeled well in advance. And Paneer Sharma, the CEO of Lantern Pharma said it best, “AI is the future of medicine. Without AI, there is no future.” He was very strongly opinionated on that, because of the critical, important role it plays, and it leapfrogs so much effort, both in terms of dollars, resources, and time to get these treatments to market. Then when you do model that out, he made a bold prediction, one that I actually noted, because I've never heard of it before. “Because of AI, if it is applied appropriately, there should never be in rare disease, another phase III clinical trial failure.” That's a bold statement. And you just when you take a step back, and you go, “There really shouldn't be, right?” If we've mapped out the pathway, if we know everything there is about the pharmacologic properties of this molecule, and we know where the genomic sequence or where in any of the properties of the individual patients plays — there should be no clinical trial failures anymore in the future. I found that to be the most exciting declaration of the conference.

MS: Absolutely, that's very profound. You just think about how much it potentially shortens research cycle, and how much quicker therapies can get to market quicker, and in turn, to patients. I want to go back to the Raregivers™ because that’s similar to something I’ve seen recently called ‘Carefluencers.’ The people that are providing the care, we're seeing people trusting those people as a voice in the community, but as you mentioned before neglecting their own mental health and care to provide for their loved ones. Curious to your thoughts on that?

AB: Partly because most of these people who are caring for rare disease patients, they were never formally trained or equipped for this role. We grow into a caregiving role when we're dealing with loved ones, that's just how we are socially and culturally shaped. But there's no formal training or playbook that comes along with that. And so, when people who are in that caregiver role, recognize that they may be struggling and need help, where do they turn? And so that's one of the fundamental reasons why when I heard what Raregivers™ was doing, I said, “This is just phenomenal.” Because what they're doing is they're making it okay to ask for help, and more importantly, they're making it a safe place for those caregivers to say, “I need to prioritize myself as well.” Rare disease is such a category full of emotion, inspiration, and let's be honest — frustration. But to hear that those who are the most essential in rare disease, isn't just the researchers, but are those that are actually providing care for these patients, whether they are parents, siblings, or children. They themselves tend to get lost in the entire conversation. The assumption is that those caregivers need to be there. That they will step up — but they themselves need help.

Usually when those caregivers finally are at that tipping point, a lot has to happen to them, and that suffering is too much. No one deserves to suffer, including the caregiver. So, to be able to help recognize and understand that there is support out there, that there's opportunities to connect and receive that support is actually very vital, because they don't want to abandon their core mission, which is taking care of their loved one. But they will not be able to do that core mission if they themselves are incapacitated.

Just when I heard the two of them speak, it became so personal because most of the people in rare disease have actually come to this area because of family stories, personal stories, friends’ stories. When I heard the CEO at Elpida Therapeutics, Terry Pirovolakis, a parent of a child, who actually recognized that there was very little the industry could do for him — he then leaned in, he became educated in the science, he became educated in the pathology of the disease, on different clinical approaches. That's where he became the creator and CEO of this pharma; that's how he channeled and manifested his vocation. And now he is actually serving as a mentor and guide to other families, other parents, who are in similar situations. So, while his scientific discovery wasn't able to help his son because his son had progressed too far — it will help others other children with that disease.

MS: I think it's interesting, how people are they grow into these positions of being a caregiver, that they don't necessarily have the certification. We don't necessarily talk about the responsibility or even guilt felt by the caregiver of “Am I doing this right?” In a perfect world, we'd able to afford a medical professional or a nurse to come and perform this care, but with the rising prices we're seeing 1/3 of Americans are skipping healthcare visits because they can't afford it as it is. It’s likely we’re probably seeing more people that are taking that route, because it's cheaper to have a family member do it.

AB: Part of the challenge with that comes back to how we as people approach the future, right? So, we always were hardwired to be optimistic about the future. We always view the future with rose colored glasses — it's a necessity for us as a species as a civilization to do that. If we thought that tomorrow is going to be worse than today, we would never get out of bed. So, we always have to think that tomorrow is going to be better today. That's what propels us forward. But it also means that we tend to discount what might happen in the future, which is why splitting of pills and skipping doses in areas like that occur. The old adage in the business is, when is that one cigarette that will give you lung cancer? You don't know. So, we gamble with our health, it's not a surprise. But when we do that on the preventive area, it actually can come back to really bite us.

It's one of those reasons why I was so excited by Wegovy. I know obesity is the opposite of rare disease, but what it does is because of what the Wegovys, Mounjaros, and Zepbounds are doing is actually getting ahead of more complicated diseases — diseases that will kill. So, I find it so short sighted on policymakers and payers to actually not cover some of these preventative treatments. You can connect the line between obesity to diabetes to cardio metabolic disease and chronic kidney disease, and you can see how the escalating threat to health rises. And if you can get it early, you actually preempt all of those others.

But again, that's human nature, right? We try to kick the can down the road is what we do. What I was excited about also was the stratification on the business side of rare disease discovery and commercialization. And the fact is, we talked about finances and how people react to it, organizations react on the basis of a financial perspective. The financial picture in those diseases where there's one to like single digit double digit patients. It's very rare to find anybody exploring scientifically, doing any R&D in there, because there will be no commercial opportunity. And that's when you get the father like Terry Pirovolakis coming out and creating Elpida is not going to be solving rare diseases that hit hundreds or thousands of patients. It is that rare. But then you have others like Lantern and that get into that that window between 100 to 2000 patients. That is the province of these small cap commercializing entities of some of these pharma. Once you get up to that 2000 patient area, that's when the Takeda's and the Novo Nordisk lean in, because they're scaled for those populations.

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