Latest / BioSpace / The race for access: How the FAIR Act could reclaim U.S. biotech leadership
Transcript
- speaker-0: My name is Jennifer Smith Parker, Director of Insights at Biospace, and you're listening to Deneatured. In this episode, I speak with Max Bronstein, CEO of Aviva Strategies, and Teresa Barnes, Chief Executive Warrior at PF Warriors. We explore why clinical trial access in the U.S. is becoming a competitiveness issue, and how the FAIR Act's push for harmonized, accelerated reviews could help the U.S. keep pace with Europe and Canada while getting patients into studies faster. speaker-1: Mm-hmm. speaker-0: Teresa and Max, thank you so much for joining this episode of Denatured. Really excited to talk about this topic. So if you could introduce yourselves, that would be great. Teresa, why don't we start with you? speaker-1: Hi, I'm Teresa Barnes. I'm, as you mentioned, Chief Executive Warrior of PF Warriors, which is a patient organization that works specifically in pulmonary fibrosis or idiopathic pulmonary fibrosis, but we actually have patients with a litany of different ⁓ origins of pulmonary fibrosis. And ⁓ I've worked in this space and worked on Capitol Hill relative to rare disease and relative to pulmonary fibrosis for it seems like most of my adult life. And I've known Max for a long time. I'm excited about ⁓ what might be possible for the future for a patient. speaker-0: Brilliant. Max, how about you? speaker-2: First of all, thank you so much for for having us, ⁓ Jennifer. Teresa, always a pleasure to be with you. I've been working in health policy, more importantly, health advocacy for two decades. And ⁓ I've been lucky enough to crisscross multiple sectors in the course of my career, including the nonprofit world. Worked at multiple biotech companies, both of which are or were rare disease companies, I should say. And I've I've also done multiple runs in government. So one of my last roles in government. I led health innovation during the Biden administration. I was very involved in rare disease and deeply involved in the launch of a brand new agency called ARPAH. Nowadays I run ⁓ this health advocacy firm called Aviva Strategies and we routinely partner with patient organizations and biotechnology companies. speaker-1: And he's a rock star, he didn't mention that part. speaker-2: ⁓ On my business card. It's on my business card. speaker-0: the reason why we're doing this podcast actually, just for that Ella. Brilliant. So let's go ahead and kick this off. So tell us about the Fair Act. I've already dropped this acronym at the very beginning and ⁓ what it aims to accomplish. speaker-1: So the FAIR Act is a bipartisan bill and it is making its way through Congress. The reality is a lot of patients don't have access to clinical trials that are outside the United States. ⁓ in fact, none in in America do. And this bill would make the access to clinical trials that are promising outside the US and drugs that are approved outside the US to make their way to the patients here. So it's a fast path to hope for our patients. speaker-2: ⁓ I want to talk a little bit about the problem that this bill is intended to solve, which when you take a step back and you look at the fact that there are thirty million Americans with the rare disease. So that's one in every ten people that that you know are directly affected by a rare disease. And that doesn't actually count for family members and the communities that are also impacted. So basically we have this overwhelming amount of ⁓ unmet medical need in this space given the number of patients with rare diseases. And if you think about the fact that the vast majority of those diseases, they have no FDA approved treatment. And the majority also don't even have a clinical trial going on for them. So clinical trials, of course, are how we get to an FDA approved treatment. We can't understate the importance of those trials and the importance of those trials. being available to patients in the US. So that's part of the problem. The other component is that when we look out across the world, we see China and other nations that are getting very deeply involved in the race to really lead the world in biotechnology and biomedical innovation. So just recently China actually eclipsed the United States in terms of the number of registered clinical trials. So That was a major wake up call to folks like us who worry about health policy and health advocacy and what this ultimately means for patients. So I wanted to give a sense of of what the the sort of scope and the scale of the problem is. And I I also want to say just a quick word about what this means if you're a patient. So at the outset, we talked about the number of patients who are affected by rare diseases. Imagine you've been waiting for five years or ten years for a clinical trial to open up. that clinical trial opens up, it's just halfway across the world and nowhere near where you live. That is exactly the scenario that we're trying to fix with the FAIR Act because we don't want you as patients to get left behind. That speaker-1: That's true. And you know, in pulmonary fibrosis where I spend most of my time, thirty percent of the trials that are currently underway that are phase two, meaning they're very close to the end of the regulatory pathway. Most of the that thirty percent are outside of the country. Wow. And for a disease like pulmonary fibrosis, which is which is deadly and it's usually deadly in short order in three to five years. But in my family, my father died of the disease as did all four of his siblings. And my dad lived the longest, which was a year and a half post-diagnosis. And I can tell you, speed is everything. And having to wait isn't possible for these patients. And having a third of our trials basically being held elsewhere, they're out of reach for our patients. And and our patients talk to those patients, right? Because we at PF Warriors, we have virtual patient events all the time, every month at least. And a lot of these patients are outside the country. So when they say, ⁓ I'm in this trial and they share it with another patient and they say, Well, how can we get in that trial? And then they realize it's outside the states and so they can't get involved, it's it's a really that's a big blow for patients. speaker-0: And I think you make an interesting point, Theresa. One of the podcasts I did earlier this year was about idiopathic pulmonary fibrosis. And sure enough, both of the companies I spoke with were European. And they were running their trial starting in Europe. So there we go. We actually have in a real life scenario. So the FAIR Act has moved from concept to bipartisan house support. So how would you describe a trajectory so far? speaker-2: happy to to jump in here. ⁓ so first of all, I need to give a shout out to Congressman ⁓ Sessions from Texas and Peters from California. So that's our sort of bipartisan dynamic duo in the House. They have been amazing to work with and really incredible champions for For biomedical innovation. So ⁓ House is just the first step though. So we we've gotten the bill introduced in the House. We're trying to get it to a higher level of support by bringing more co-sponsors onto the bill. Our next step is to get a bill introduced in the Senate. Senate always tends to move a little more slowly, they're a little more deliberative. So it can take longer to get a a bill cooking in the Senate, but that's our goal. ⁓ and then we want to have it actually cross the finish line, which gets signed into law. So That's a little bit about the the state of play and and where we're hoping to go next with the legislation. speaker-1: Again, speed is everything. So the faster we get the co sponsors in the House, the faster it can make it into the Senate. And I think that's really kind of where the rubber meets the road for our patients right now is getting that done. speaker-0: And for the audience, if you want to find out more about the FAIR Act, ⁓ the website to go to is FaiRactnow dot com. So let me ask you when you talk about competitiveness, because that's obviously a very important topic tied into what we're saying here, what signals are you seeing that Congress views clinical trial access as a competitiveness issue, not just a patient access problem? speaker-2: I think we're seeing a lot of encouraging signs that Congress is open to new ideas in this space and it's not just Congress, it's the FDA itself. So that's really exciting because when you get Congress interested, when you get the agency, the FDA in this case, and you have voices in the administration all saying, Hey, we need to do something differently or our global biotechnology dominance is at risk, like that means that there's what we call a policy window to get something done. So there are specific ⁓ vehicles coming up in Congress that may be a really good fit for something like the FAIR Act. So we're hoping to have this legislation catch a ride on that, if not sooner. But we're seeing so much desire, interest and encouraging signs from Congress that I think we have a real shot at getting this done and ultimately passing a bill that's gonna be really meaningful for the rare disease patient community. speaker-1: Yeah, and the folks that are already on the bill, I mean, they're passionate about it. And I think that helps to to drive other bipartisan ⁓ leaders to get on board. And the patient community can really help with that. We've done it for years. I like to think we've made progress, but when you look at where our patients are, when you really dig down and say, Okay, how well are our patients after twenty years of profound research and more than thirty drug trials that have been held, although we only have three approved therapies. And unfortunately, our patients don't survive even with the therapies that are available to them. And the only way to survive the disease is lung transplant and less than 1% get that. So that's not a real option for the majority of our patients. And it's not sustainable either. Obviously there are limitations with that. But I think that this is an opportunity with ⁓ you know, Max has had so much experience in the rare disease space. He's made so many things happen. I feel like having his voice here about this particular bill makes all the difference because he communicates so well. He knows the rare disease space so well that honestly I think we're in a great position. We just need ⁓ people to encourage their members to sign on now and move it and move it through the channels. So I'm excited about it. speaker-2: Thanks for the pressure, Theresa. speaker-0: Right. If that's not a hit, hit nudge nudge, I don't know what is on this one. So you both talked about patients waiting months or it could be years for trials that already exist abroad. So how does a Fair Act turn that gap into a structural pathway? And why is that shift essential for keeping the US from falling behind Europe, Canada, and the UK? Nonetheless, China, as you mentioned, meth. speaker-2: Yeah. So the the way the bill actually works mechanistically is it does three things that are actually very straightforward. The first thing it does is it identifies what we refer to as trusted regulatory partners. So ⁓ in the bill that's the MHRA, which is the FDA equivalent in the United Kingdom. The EMA, which is FDA equivalent in Europe and Health Canada, which I I think we know where that is. So those are the the trusted regulatory authorities that we identify in the bill. And basically the bill says that if there is a trial or an approved product that has been greenlit or allowed in those regulatory regions, it would create an voluntary but accelerated pathway for sponsors to work with FDA to get those same. trials or products approved in the US. And then the last piece of the bill is that periodically FDA has to report on this pathway and whether it's being leveraged or not leveraged. And the report has to go to Congress so that there is transparency and an understanding about how the bill is is impacting biomedical research and biomedical innovation. So fundamentally those are the pieces of the bill if we can get this done What we want to see is we wanna close this gap, this innovation gap that's growing with regards to access to clinical trials and with regards to products that have been approved in these other countries but aren't yet obtainable by patients in the US. So we really wanna see the that gap close and and not grow. And we think the FAIR Act can be a huge part of of the solution here. speaker-1: And I I can think of real world examples in just in my life over the last several years. And one, I remember a a drug being approved in the EU. And ⁓ our patients didn't have access to it. It was actually turned down here. ⁓ and ⁓ and so guess what? The ones that were physically and financially able left the country to get the medications that they needed. ⁓ and unfortunately most people could not do that. And so I've got another one young patient who desperately needed wanted to get into a trial. And I remember making calls and begging the CEOs of companies to to allow some of our patients that didn't fit the inclusion criteria perfectly, would they consider would they get special allowance or something for someone like that? And what I discovered is it wasn't the company's decision. not to bring their trials here. It was another decision that was made. I can think of another patient who actually had family in another country and was able to get citizenship there. It took months and was able to get a therapy that was already available there that he could not get in the US because the clinical trials were not available. So I think the reality for our patient, it this is a real, this is a real opportunity to change lives. And I think that's why we're in it. speaker-2: I I think you you said it so well, Teresa, and and this is exactly the situation that no US patient should ever have to face. I mean, imagine having a rare progressive life-threatening disease. The trial opens up and your only shot is to move abroad or upend your entire life, move away from your friends and family at the hope of getting into a clinical trial. That is something that no patient, no family should should have to bear. on top of having to deal with all of the challenges that arise from having ⁓ a rare progressive condition. speaker-0: Yeah, these are very stark examples of where we're falling short in the US. So thank you for pointing that out. So one thing I find very interesting about this bill is there's a thirty day harmonized review, and that's a departure from the status quo. So what convinced you that this accelerated timeline is both ethically necessary for patients and strategically necessary for US biotech leadership? speaker-2: When you look at the fact that the diseases in this bill are life-threatening and progressive, the one thing that these patients do not have is the luxury of time. So when we looked at how long it was taking for some of these review decisions to be made at FDA, we said this is unacceptable. ⁓ we need to find a way to speed up the process because imagine waiting months and months and months for the FDA to make a decision with some of these diseases. Six months means the difference between being able to walk, the difference between being able to talk on your own, to eat on your own. And in some cases it can be a life and death delay, which is totally unacceptable. So we felt we wanted to light a fire under the agency to really move these reviews along. So on the clinical trial side, it's a thirty day review, which is actually very similar to the current timeline. On the approved product side, it's quite accelerated. And that was intentional. ⁓ because if there are patients who are already getting an approved product in one of those regions we discussed earlier, we feel that US patients should have those same benefits and be able to get those products too, especially in instances where someone has a life threatening disease. Why should they have to wait longer in the US than patients abroad? speaker-1: A lot of our patients in the examples I gave and I remember them saying, I'm not gonna live that long. By the time it comes here, I won't be able to get the drug. And then you hear that there's a trial or a drug available elsewhere that will take years to get here and they realize that it's not gonna happen for them. What I love about this this bill, this opportunity is it is fair. It gives our patients a fair shake in what's already been vetted. Outside of our country by people we trust. And why would we not want our patients to benefit that? And you know what? It it also comes down to economics. Our patients are spending money, the ones that could, when we had approved therapies elsewhere, were leaving. They were spending money there, some were staying. And I'm not talking about for a year, I'm talking years that the US kind of missed out on that, on having our patients treated here. And also. Our patients trust their doctors. And the ones that left the country, I'm sure they ended up trusting those doctors. But there's nothing like someone that knows you, who's seen your case, who's looked at your files, who's monitored your progression, who can make a suggestion on what you may or may not choose, whether a trial might be good for you, whether a drug might be good for you. And instead, patients were sort of forced to make those decisions on their own because their doctors weren't doing the trials. So I think it really It really speaks to why patients need this now. And it's because they need their doctors to be able to help them here in the US. speaker-0: So if the Fair Act passes crossing fingers, toes, and everything else, what does success look like five years out in your view? Is it more US trial sites, faster access to ex US therapies, stronger biocompetitiveness, or a measurable shift in where companies choose to launch their most innovative studies? Or all of the above? speaker-2: Well, hopefully all of the above. I really want to see the US maintain its global leadership as the superpower for biomedical innovation. Right now, that is completely at risk. So this bill is a big part of of the solution. We want to see more trials happening in the US, including early stage trials, the vast majority of which are are going on abroad nowadays. So I think that would be one easy metric is how many phase ones, phase twos are actually happening in the US. And we want to see more options, particularly for the rare disease community. This is a community that is faced with overwhelming unmet medical need in terms of approved products and access to clinical trials. So we want to see more of those happening in the US and within reach of patients. I think those would be two ⁓ tremendous outcomes for sure. speaker-1: Yeah, and patients, I mean, they get to make a decision. If it's available here, it gets to be their choice. When it's not available in the US, it really isn't up to them. And unfortunately, powers everything when you're a patient. Your ability to advocate for yourself, your ability to sort of speak up and say, Here's what I need, here's what I want. And when it comes right down to it, what FDA is most concerned about is what are the patients willing to accept? What is their risk benefit? what does that equation look like? And what do they really want? And I can tell you, at least in our patient population, they want access. They want a chance. And and some of these therapies, they're promising therapies out there, but we're not going to see them in the US for years to come if things don't improve. And I think this bill is so easy. It's such an easy decision to make. Who wouldn't want patients to get therapies that are promising faster here in the in the States? speaker-0: Well, thank you so much, Teresa, and thank you, Max, for joining this episode of Denature. It's been a fascinating conversation and I'm very optimistic going forward, crossing everything that this bill moves on to the Senate, et cetera, ⁓ and then it's tacked on or standalone legislation. And again, for the listeners, it's Fairacnow.com if you want to find out more. If you'd like to listen to more episodes, you can turn to biospace dot com. And if you'd like to send me a pitch, my email is Jennifer.smithparker at biospace dot com. Thank you so much.