Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

July 1, 2022

The U.S. Food and Drug Administration’s Accelerated Approval pathway allows for the use of surrogate endpoints to make therapies more quickly available for unmet medical needs. About 82 percent of the drugs approved under the designation have been for orphan indications. But controversy around its use to win approval for Biogen’s Alzheimer’s disease drug Aduhelm last year set lawmakers off on an effort to reform how the pathway is used and to place new requirements on drugmakers. The healthcare consulting firm Vital Transformation recently did an analysis on the effects potential changes to the Accelerated Approval pathway could have and found that as many as two-thirds of treatments approved this way would no longer reach patients. We spoke to Duane Schulthess, CEO of Vital Transformation, about proposed reforms to the Accelerated Approval pathway, the findings of his firms’ analysis, and why these changes could have dire consequences for rare disease drug development..



Daniel Levine: Duane, thanks for joining us.

Duane Schulthess: It’s my pleasure. Thank you.

Daniel Levine: We’re going to talk about accelerated approval, the controversy around the U.S. Food and Drug Administration designation, and a recent analysis from your firm, Vital Transformation, conducted on the effects potential changes to the designation would have on drug development. Let’s start with the designation itself. What is accelerated approval and why was the designation created?

Duane Schulthess: The accelerated approval came out of a lot of the requests and the challenges in developing HIV and AIDS treatments in the late eighties and early nineties. For those who don’t have gray hair like I do, the reality is, when the AIDS pandemic hit, it was ravaging the homosexual and gay community, particularly in San Francisco and New York, and then globally. The fact was we didn’t have any effective treatments and people were dying mysteriously. Then we found out, okay, this is a virus, ironically, it was a coronavirus at the time, [and] we had no practical treatments for them. And so the reality was we needed a pathway to start putting experimental drugs into people’s bodies quickly that showed promise. So there developed a pathway that would be able to use drugs that had promising indications, not necessarily endpoints. And for those who may not know what an endpoint is, when you say a hard endpoint and when this is used in clinical research, basically that’s are you alive or dead. Did you survive? Are you alive five years after having cancer? These are called hard endpoints. Did your melanoma return? Can you continue to walk? If you have Duchenne muscular dystrophy, that’s what we call a hard endpoint. And often these things evolve over time. Now, if you’re dealing with an emergency situation like you had with HIV and AIDS, time was not a commodity we had to spend overwhelmingly. We didn’t have much of it. So we needed to find ways to start testing these drugs that showed promise. And we would do so with something called a surrogate endpoint, which means not a hard endpoint, but a proxy, something that we could use that should be deterministic of outcomes. For example, in cancer, do you relapse or not, if you don’t relapse and we’re showing that this drug stops you from relapsing, that would be a good surrogate endpoint for overall survival. So, in 1992, we passed the accelerated approval that allowed us to use drugs that show promise with these secondary/surrogate endpoints; these proxies for hard outcomes, in lieu of having an outcome, where we had high unmet medical need. And that’s really what the accelerated approval was. In 1992 we rolled it out in the U.S., then the EMA came a few years later with what they called conditional approval. And generally it’s been considered a huge success. Obviously this led to AZT and many of the multi-combo therapies that are now very successfully treating HIV AIDS to the point where it’s a chronic condition. Now, this didn’t exist 20, 30 years ago. So that’s what the accelerated approval was.

Daniel Levine: How does the designation work? Who’s eligible and what rights or responsibilities does this bestow a drug developer?

Duane Schulthess: Sure. Basically it’s the FDA’s call and essentially what we’re looking for are areas of high unmet medical needs. So these are places where we don’t have a treatment. There’s not currently an available pathway to treat people. It’s often late stage, failed cancers for which we don’t have good treatments. For example, leukemia. Rituximab works extremely well in 60 to 65 percent of cases. People come in with a lymphoma of some type or a B cell lymphoma, and they walk out cured. But what do you do in those cases where they don’t come out cured? And what if the treatments we have don’t work? So essentially what we did is we were looking for drugs and treatments that would provide promise in areas where we don’t have current treatments. And that’s a call by FDA. It’s defined as an unmet medical need. And essentially what that does is use this surrogate endpoint to say, okay, we’re stopping tumor growth here. We think we’ll have outcomes. And then the FDA requires you to do what’s called a confirmatory trial, and then when you satisfy that agreement, you “convert,” and then you’ve fulfilled your obligation. So, you have to do a parallel—it’s not really a phase 4—sort of post-marketing evaluation and measuring that we do now for pharmacovigilance. It’s more of like a phase 3/4 simultaneously. So, you’re doing confirmatory evidence, but it’s a full approval. There’s enough evidence to say, yep, we need it. You get the approval. But then we expect a certain amount of evidence with a certain amount of people in the median. When we looked at the ones that have cleared over the last 20 years, the median’s about three years. So, it takes about three years, half of the drugs we saw convert in three years. So, they’ve fulfilled their obligations with the FDA.

Daniel Levine: What’s been the impact of the designation on the development of new therapies for rare diseases?

Duane Schulthess: Well, it’s been hugely impactful, obviously. I think one of the key things that we uncovered, which was quite unexpected, was first, the high amount of orphan conditions that this treats. It’s over 80 percent. Four out of five of these approvals are for orphan conditions, which roughly affect less than five and 10,000 people. So we’re talking pretty small indications. These are pretty narrow areas. A lot of them are oncology, particularly over the last 10 years, we see a lot of cancer drugs, but a lot of that’s been driven by three drugs alone, Keytruda, which is a very successful late-stage oncology product, Optivo, which is now curing 50 percent of people with melanoma, which five years ago was virtually untreatable and was essentially a death sentence, and Gleevec, which is arguably one of the most effective cancer drugs ever invented. So, they hold over 50 percent of the secondary indications. These other pathway indications can be given once a drug is approved. So you see as a lot of action in oncology, but what it’s also done is given a leg up for a lot of these very, very small orphan conditions, for example, Goucher’s disease—Genzyme’s drug, one of the first “orphan conditions.” This is a frightfully small population, 50-70 people a year maybe. The entire clinical trial was only 40 people. So, it was a vehicle and a vector to allow that treatment for Goucher’s disease by Genzyme to come to market, and without it, frankly, it probably wouldn’t have happened because there’s no way you would get the hard point evidence that would be normally requested under a phase 3 trial without the ability to use that “surrogate” endpoint.

Daniel Levine: The designation became the center of controversy with the approval of Biogen’s Alzheimer drug in part because the agency went against the recommendations of its advisory panel that found there wasn’t adequate evidence of efficacy and concerns about the validity of the surrogate endpoint it was using. There’s long been concerns about drug companies not following through on required studies and ineffective drugs remaining on the market. What’s underlying the debate on accelerated approval?

Duane Schulthess: Well, John Dwyer, who was on our panel at BIO and obviously very involved with us against Alzheimer’s, is a well-known entrepreneur sold several companies to Unisys. He’s quite pointed in his criticism. He said, look, this is about budget period. Sue Peschin from the Alliance for Aging Research in Washington, DC is of the same opinion, as is George Redenberg, many known, well known, very respected philanthropists in the United States. The reality is, in our evidence and what we saw when we did our research, overwhelmingly you can determine the length of time it takes to fulfill the FDA obligations by the size of the clinical trial. We ran a statistical model. We did a multiple regression on this, and the evidence was really quite strong.

I mean, there’s less than a one in 2,500 probability that this is occurring with random chance. So the P value is four out of 10,000, which statistically means this is a pretty strong statistical relationship that says, Hey, something’s going on here. So, overwhelmingly what we saw in our data was the smaller, the clinical trial, the longer it’s going to take to fulfill that confirmatory evidence with the FDA. So rather than there being some grand cabal conspiracy about organizations not fulfilling their obligations, we really saw that you can actually model this and map this quite clearly and see that not only does it have an effect on the ability to close and confirm evidence—I mean, if you’re only dealing with a micro orphan of 100, 200 patients here, let’s not forget Duchenne muscular dystrophy is 400 patients a year, that’s the entire population—the ability to address that in a closed outcomes trial where you’re dealing with someone who’s being diagnosed at 12 years old and then basically they’ll live until their twenties, you’re talking a decade or more for some of these people. And the ability to run that on a short outcomes based trial in less than three years is a fallacy. It’s just not going to happen. I know there’s some controversy around this, but frankly, I think what happened with Alzheimer’s, and the Biogen drug Aduhelm, is that this was going to be a pretty big pile of sticker shock, unfortunately. We were talking tens and hundreds of millions of dollars a year. Interestingly, if we don’t have the drug, currently Alzheimer’s is about a trillion dollars a year, depending on which study you look at, in total cost and some cost for the U.S. economy. The question is, would we have gotten an end point out of that or not? I’ve been quoted in STAT. I wrote an article and I was very critical of the amyloid beta thesis, calling it a failed hypothesis. However, when you look at that Nature article from 2015, where they correlate the biomarker with cognitive decline, it does show evidentiary response there. Unfortunately it takes 10 years to develop that pathway. And unfortunately, I just don’t know. We’ve tried, so far no success, but to trash the entire pathway because of Biogen basically having a failed two-arm phase 3 trial and then coming back with the accelerated approval seems quite shortsighted. I think basically this was an opportunity for people who wanted to sit on the accelerated approval to score some political points. Unfortunately, the big losers are going to be the patients who are sitting on those orphan drugs that currently don’t have treatment because this is very, very, very risky. And I’m not the only person saying that. Scott Gottlieb and Mark McClellan in Endpoints said basically the same thing. There’s a lot of us who work and do the analysis and look at the data who are saying, “Hey, wait a minute. This may be a bridge too far.”

Daniel Levine: There is legislation winding its way through Congress. How dramatic a change in accelerated approval might we expect to see?

Duane Schulthess: Well, it depends on where it lands. Unfortunately, a lot of these proposals that have been coming out of the U.S. Congress have been quite mercurial and hard to predict. I got my crystal ball out and unfortunately, it’s not really giving me good answers. I wish I could tell you the Pallone bill that was initially proposed three, four months ago that we started looking at, which is why we got involved with this debate post the work we did for Biogen, what was obvious to us is when you start putting a five year cap, [you’re] basically throwing out the accusation through legislation that anybody who’s taking longer than five years to close with the FDA is a bad actor. That’s just substantially not born out by the data. Basically, what you’re doing then is wiping out the 85 percent of untreated orphan conditions with the incidence rate of less than one in 1 million, which is where we are. The vast majority, the overwhelming majority, of orphan treatments that still need to be developed are micro orphans of less than one in 1 million incidence rate. And the idea that anybody would be able to do a hard outcome endpoint research study with FDA in less than five years for a population that minuscule is completely they’re kidding themselves. And they’re being intellectually radically dishonest. So, the Pallone bill would’ve been awful. Fortunately, cooler heads have prevailed, but it’s taken four months of work. And a lot of patients, patient groups jumping up and down and pulling their hair and threatening to eat worms in order to get that taken out in PDUFA and the written negotiations under PDUFA right now. But, the fact that they were willing to stand by that so long tells me that there’s a lot of regulators there who don’t care, or at least are ill-informed enough where they’re willing to play politics with this and, and monkey around with this pathway and potentially threaten something that could save lives. And that I find particularly obnoxious.

Daniel Levine: There’s also controversy over the accelerated approval pathway allowing for additional or secondary indications. You discuss the three cancer drugs. Why are these secondary indications controversial and how do a handful of drugs skew these numbers?

Duane Schulthess: The three drugs, I mentioned, Optivo, Gleevec, and Keytruda are responsible for 52 percent of all the accelerated approval secondary indications that we looked at in our research. So overwhelmingly these three drugs are basically driving those indications. Now, why is that? Well, Keytruda is an incredibly effective, late-stage oncology treatment that actually has some credibility and works on solid tumors. If you have metastatic late-stage phase 4r cancer, and you are probably looking at dying, the ability to basically move that out a few years hoping to find a cure would be good, I would think. And Keytruda has been shown to be very, very effective, as has Gleevec, as has Optivo. So, the idea that there is some grand cabal mystery with FDA and that there’s some grand corruption going on here is simply ridiculous. It’s absurd. These are three drugs that are on the WHO’s essential medicines list. I mean, these are highly respected, very, very good oncology [drugs] and the ability to be able to move those around and move them into indications where you show some ability to stall tumor growth, I honestly don’t see why that’s a bad thing now. They’re not cheap. No, they’re not cheap, and what we’re seeing here is effective treatments that are very, very good and maybe are the only ones that are working late-stage [and] are getting used a lot because oncologists are seeing that they work. I really have an issue with saying that that’s a bad thing. I mean, if I was in a situation and, Keytruda, $50,000 a dose under Medicare, according to the analysis we’ve seen from the MPAC report—okay, that’s not cheap, but it’s not hugely expensive for those drugs. And eventually Keytruda—I think it’s been on the market for six, seven years and probably got seven more years—then it’ll go generic. So this is the agreement we have. They’ve developed this drug, it works, it will become generic and it’ll be very effective then too. So, it’s not like there’s a big mystery here and what we also need to remember is a lot of the orphan drugs that have been developed that are non-oncology. Now again, overwhelmingly the vast majority, 60-70 percent over the history of the pathway are oncology. A lot of those were HIV drugs earlier. So, it’s been closer to 70-80 percent over the last decade. Fine. But the reality is that 20 to 30 percent are non-cancer orphan indications. A lot of them are genetic indications with a biomarker, and you’re not going to get a secondary indication. It’s just not going to happen. So it’s really biasing a lot of the decisions around three highly effective drugs that oncologists themselves and hematologists are saying, hey, we need this. This is the last option we’ve got in the drawer. I honestly don’t see—I guess the other option is we stopped prescribing it and let people die six months or a year earlier. That seems tremendously cynical to me.

Daniel Levine: Talk about the Vital Transformation analysis. As a matter of disclosure, this was funded by industry, but what were you seeking to determine?

Duane Schulthess: If I can also add it? It wasn’t just industry. It was also the CEOI of the World Economic Forum, which is an association with the patient group Us Against Alzheimer’s. So, there were a couple of nonprofits involved in this too, and the BIO organization, as well, which is a nonprofit organization.

Daniel Levine: What were you seeking to determine?

Duane Schulthess: Well, we just wanted to dig through the data, but what we do at our firm, we don’t make any predetermined opinions. We let the data guide us. We like to open up the hood and start pulling the engine apart and seeing if there’s an engine knock, we want to know what’s going on in there. We want to know if it’s the pistons, the rings, or if you got a main bearing going out. We like to look at the data and see what’s really there. We don’t come in with predetermined opinions. We like to follow the actual numbers and that’s what I found so interesting with this study. We wanted to just see what was really going on with the accelerated approval. Was there an enormous amount of controversy around the ability? Was there a grand cabal stopping hard endpoints and doing a traditional FDA-approved phase 3 trial, were surrogate endpoints really just a scam and they weren’t valuable? Would patients be better served without this pathway? Those were sort of the three questions—that’s the rhetoric. If you read in the media what people are saying, we wanted to determine if that was true or not. So we wanted to test those straw men that are being thrown out at the pathway. We wanted to see how much of this was legit and how much was just air in the tire.

Daniel Levine: And how did you go about conducting this study? What was the methodology you used?

Duane Schulthess: Well, what we did is we extracted the entire cohort of the last 20 years of accelerated approvals, removing any drugs that were repurposed. You’ll see like half a dozen or a dozen of these things—there’s a blue dye that’s used to determine oxygenation from 1956, that gets repurposed for an orphan condition. And again, they’re not the majority, they’re a slim minority, so we removed that stuff. And then we just want to look, okay, we have primary first new indications, and we have all the secondary indications—what’s really going on here. What’s the time to market, how much has been invested, what’s the real return on investment for these things? If people are investing, there’s a high failure rate in biotechnology and biopharma overall, the failure rate, people like to say is 90 percent, which is true, but that belies the fact that neurological disorders fail 99 percent of the time, oncology fails 92 to 93 percent of the time. So as an investor, you’re taking on a heck of a lot of risk and you want to know practically what’s going to happen from the standpoint of your investment if you have a change to this pathway. So, we looked at the amount of money using two studies—one by the academic named Jayasundara, and one by DiMasi. A lot of activists don’t like the DiMasi study because they say it’s too high. A lot of the industry doesn’t like the Jayasundara because they say it’s too low. So, we decided to use them both—we’d say that would be our range. And we’d say, look, we’ll use them both as a range and that’ll be somewhere in between. There probably lies the truth. So, we used those two cost bases. We scaled it based on the size of the clinical trials and all that’s declared in all 206 therapies and approvals that we found in the data set. And then we modeled it out using the actual trial cost, using these two studies for cost basis and then looked at the actual revenue that’s been created by these drugs once they’ve been approved. And then we determined if they would have a fair rate of return. And if they wouldn’t, they would probably be withdrawn from the market. And that’s what we wanted to determine. How many would be with withdrawn if this wasn’t there and how many would still come to market?

Daniel Levine: Before we talk about the actual findings, I think it’d be useful to explain one concept for listeners. That’s a key financial metric known as net present value. Can you explain what’s meant by net present value when we talk about that with regards to drugs and its relevance?

Duane Schulthess: Yeah. I mean obviously the big killer with biotech investing in developing a product is time. It takes a long time. This has always been the problem with Alzheimer’s disease. As I mentioned, the biomarker looks legit when you look at that 2015 Nature study, but it takes 10 years. And the fact is by the time you do your clinical research, you may only have 10 or 11 years of sales before your product goes generic and you lose your revenue streams. So, the question is, what is the investment you’re going to make today worth 10 years from now, five years from now, 12 years from now? So essentially if you think about this with interest like we have today, inflation, if you put $100 down today, with 10 percent inflation, something that you would buy today for $100, in seven years is $200, roughly give or take. Well, that works the opposite way because there’s another way to look at that. It’s not just if things cost more, it also means the value of your money is worth less now. So, what you have for a $100 now in 10 years is only worth $50. So, that $100 you’re looking at today is worth half that much. If you look at the other side of that equation of inflation, instead of requiring $200 to buy something, you could say: well, this $100 I have now is only going to be worth $50 10 years from now. So that’s sort of how a net present value works. It looks at your investment today and then looking at costs and inflation and risk. It then values what that would be in the future. So, it gives you a net idea based on how much money you’re going to be potentially earning five, 10 years from now. It allows you to balance in apples to apples, is this a good investment or not given the rate of return I will need to have. And obviously with 10 percent inflation, an 11 to 15 percent cost of capital is what you would need to charge against your money to reduce the value each year [and] is probably legit. We only used 11 percent, which is generally the industry standard, but with inflation running 8, 9, 10 percent, we could probably could have done 15 or 20, but we didn’t do that. We wanted to play this pretty straight bat as we would say in cricket. So, we didn’t do that. We kept it pretty close. But the reality is what that shows is even if you’re making what looks like good revenue, 10 years from now, you still have to account for that investment today, because otherwise, you can invest in apps, you can invest in clean energy, you can invest in electric vehicles, you name it. You don’t have to invest in biotech. And unfortunately, this is a very important business. It’s a life-saving business, but it’s still a business and investors have to know they can be made whole at the end of the day.

Daniel Levine: And what did your study find?

Duane Schulthess: Well, what did we find? I think the first thing that was interesting and this came as quite a shock given all the secondary endpoints and this rhetoric that there’s a bunch of bad actors there who aren’t closing their clinical trials in time with the FDA to convert and provide evidence. What we found is the proven accelerated approvals by the FDA, the median time of approval and conversion is three years. Half of the companies who are approved for the accelerated approval clear all their FDA requirements within three years. Again, that came as quite a shock, as matter of fact, a quarter in two years or less. So, quite surprising how the rhetoric doesn’t necessarily meet the data by 75 percent of the companies are clearing in four years. And then what you find is yeah, Genzyme took 18 years and why? Because they only had 40 people in their clinical trial and that’s basically the entire population. So, what you find is these drugs going out 18 years—that final 20 percent—that the Pallone bill would’ve basically wiped out had we put that in place. And the overwhelming majority of those are very, very small-need orphan indications that require long outcomes trials. So, contrary to the rhetoric, there’s real time that’s required here. And what we then found is if you start putting delays in requiring companies to like the Biogen example where we’re going to put coverage with evidence and you’re going to need to continue that phase 3 trial that you’ve just done for another three or four years, if we apply that to the whole population in the accelerated approval, a three year delay for example, if we use the Jayasundara numbers, which everyone knows or thinks is too low from a cost standpoint, you’re thinking about 35 percent would no longer have a positive net present value. So again, investors would probably walk away from a third. If we use the DiMasi, it’s two thirds. So somewhere between 33 and 66 percent, so probably in the midpoint there, about 50 percent would disappear immediately. That again was quite shocking to us. It just shows that very small changes to this thing that may sound good on the surface—you know the old expression, the road to hell is paved with good intentions. Well, there’s a lot of good intentions being paved out right now around the accelerated approval and the impacts could be quite devastating and more than I anticipated frankly.

Daniel Levine: And draw those impacts out specifically for rare disease therapies. What would they be?

Duane Schulthess: Well again, 82 percent of the accelerated approval pathway are orphan conditions, period. And the overwhelming ones that are small niche, genetic based, the Gaucher disease, they’re the ones that are taking more than five years. So, you’re really attacking the smallest, narrowest indications when you start throwing around this idea that there’s a lot of bad actors out there. Now look, I’m not saying that there aren’t bad actors. That’s fine. This is the beauty of statistics. We’re not looking for specifics. We’re not looking for an adhoc example of one actor. What we’re looking at is the population as a whole. And what we can say is overwhelmingly the actors do behave logically given the fact that these smaller indications take longer, and that makes sense. That makes sense because it just takes longer to develop the evidence.

Daniel Levine: One of the concerns that your study raised was the threat of having arbitrary timelines for the completion of confirmatory trials being set to five years or less. How real a possibility do you see that being and what would be the consequence of that?

Duane Schulthess: Well, Nick Shipley and John Murphy from BIO and I had a long podcast on their Vital Health podcast. And we asked this specific question and Nick Shipley, who obviously deals with the Hill all the time. His comment was, and I’m paraphrasing here slightly, but it’s like, “well look, they went down this road a long time for about six months and it was only under PDUFA and a lot of pulling and a lot of patient groups getting very, very upset that this changed.” So yeah, I do think this is a risk, absolutely, because they were willing to keep this in for a long time, a long time. My concerns around a lot of these decisions are the fact that these are becoming political decisions around a process that is deeply data driven and economic. The investors and the scientists who are developing these drugs know the evidence that they require. They know the amount of time and cost it’s going to take to do it. And you’re already dealing with neurological disorders, particularly with Alzheimer’s of a failure rate of 99.5 percent. Ninety-nine point five percent of all clinical developments in Alzheimer’s fail and have failed over the last 30 years. So, if you start ratcheting up these requirements and ratcheting down the ability to even get a fair rate of return, and then you’re increasing the cost, investors and biotech companies are just going to walk away from these indications. And this is the implication that these politicians, unfortunately, politically don’t understand. There are real fundamental, logical, economic consequences to these decisions that we can measure and count. And we talk to the investors regularly. We talk to the clinical developers regularly. They understand this better than just about anyone. This is not rhetoric. This is reality. You will not get a fair rate of return. You will not be able to recoup your investment. Particularly with the equity markets, my God NASDAQ fell 50 percent last week at one point, but it’s come back a bit. But in 2020 Q1 before COVID hit, 80 percent of all the listings on the NASDAQ were biotech listings. So the equity markets are vital to this. Liquidity is key. Cash is king. And if we start messing with this and killing this ability to get a fair rate of return by putting up these arbitrary timelines, this is going to go away, because nobody’s going to be able to invest in these. The numbers will not add up.

Daniel Levine: The rare disease community doesn’t like to think about the power economics plays in guiding drug development decisions and the consequences policy changes can play in that. Given the large unmet need that rare conditions represent, how concerned should people be about changes to accelerated approval and, should people who care about rare diseases, about evolving legislation?

Duane Schulthess: Well, absolutely. I know economics is a dirty word and I know that we want to be altruistic. I completely understand that nobody is going into biotech because they are like Daffy Duck rolling around in their gold balloon in their cartoon basement with Huey, Dewey, and Louie rolling around throwing money at each other. That’s not how this works. The reality is there are other industries that have a far better rate of return. If you look at the New York University analysis of sector by sector, last year, the pharmaceutical sector had a net profitability of 14 percent, that’s slightly less than the soft drink sector, contrary to popular rhetoric. This is not an overwhelmingly profitable sector. It’s been harder and harder to get a fair rate of return on these assets. The fact is there’s high risk here and you’re talking billions and billions of dollars of investment are required. You know the old joke I like to say is, “A boat is a hole in the water. You throw money. Well, a biotech company is a furnace. You throw boats.” This is a very, very expensive business. It’s a very uncertain business. And frankly, the accelerated approval has been a vital tool in promoting the ability of American, particularly American small biotech, under 500 million a year give or take, has been dominating the development of these assets. over half of all assets approved in the last 10 years, 363 assets approved by the FDA, over half of them came from small U.S.-based biotech, predominantly from California, Massachusetts, and North Carolina—that’s globally. We we’re dominating the sector. The U.S. is leading innovation in this space and the accelerated approval’s been a hugely important part of that. To negate that or ignore that is really, really doing a huge disservice and to the taxpayer, as well as industry and people who are developing this.

Daniel Levine: Duane Schulthess, CEO of the healthcare consulting firm, Vital Transformation. Duane, thanks so much for your time today.

Duane Schulthess: It’s my pleasure. Happy anytime at all.

This transcript has been edited for clarity and readability.

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