ICER’s influence & election effects: a BioCentury podcast

Usdin and Editor in Chief Simone Fishburn also discuss the likelihood of new leadership at NIH, and what kind of organization might support continued translation and collaboration between public and private sectors, as has occurred during the pandemic.

Meanwhile, as COVID-19 vaccine development continues to march on, Associate Editor Virginia Li and Usdin weigh the benefits and downsides of using the expanded access pathway versus emergency use authorizations to roll out the first vaccine candidates. 

A transcript of the episode follows.

[00:00:00] Virginia Li: Welcome to BioCentury this Week, I'm Virginia Li, BioCentury Associate Editor and I'm joined by

[00:00:10] Simone Fishburn: Simone Fishburn, Editor in Chief

[00:00:13] Steve Usdin: Steve Usdin, Washington Editor

[00:00:15] Lauren Martz: Lauren Martz, I head Translation and Clinical Development coverage

[00:00:19] Virginia Li: Today Steve will be giving us the latest from Washington as we head into tomorrow's election. But first Lauren is here to discuss the growing influence of ICER in discussions of drug pricing among payers and biopharmas, and the role it could play under the next administration regardless of who's in office. So Lauren, to start us off what does ICER do, and what role does it have in the industry today?

[00:00:40] Lauren Martz: ICER is a nonprofit organization that acts as a health technology assessment agency in the U. S . U.S. is one of the few countries that doesn't have a national agency to do that. And so we've been seeing that ICER has really stepped into a relatively influential role, after speaking with payers, in assessing the value of drugs, in looking at the cost-effectiveness, the clinical benefit, and the budget impact that new therapies have, especially high impact new therapies.

[00:01:13] Simone Fishburn: I have a question actually and Steve you may know the answer to this as well, historically has ICER been one of the main forces pushing for value-based drug pricing?

[00:01:25] Steve Usdin: There's a lot of people who are pushing for value based pricing. The problem of course is how do you define value. The way that a pharmaceutical CEO would define value, and an insurance company CEO would define value, and a patient would define value are quite likely to be different. Right, and then you've got the second question which is really the elephant in the room which is once you figured out how valuable something is, then how do you translate that value into a price. How much of the value that a drug company creates should that company, obtain and how much of that should go to society. Those are value judgements, and no amount of analytical information is going to answer that question.

[00:02:08] Simone Fishburn: And so Lauren, if I understand right, what you're saying is ICER is sort of moving to a position in the U. S. among the industry as being highly respected for the model it's come up with for assessing value, and putting a price, let's say, exactly on that?

[00:02:23] Lauren Martz: I think so, there are many different organizations. There are a lot of academic groups that are developing different frameworks to try to assess value, a lot of payers have their own metrics as well. But ICER sort of stands out as an independent third party and someone that's been able to do this quickly. Especially as they're getting more buy-in from the pharmaceutical and biotech players. Whether or not they are pushing for this value based pricing they're definitely creating a tool that will help assist that.

[00:02:51] Steve Usdin: When you say a buy-in from the biopharmaceutical industry, I'm wondering whether if that's true, because in the past when I spoke with biotech CEOs privately, a lot of them really have a lot of problems with ICER they're really critical of it. I'm wondering if what you're saying is that's changed, or is it just the difference between what they say in public and what they'll say in private?

[00:03:12] Lauren Martz: I don't know that's changed. I've still heard quite a bit of that, but if you look at the people who are funding ICER a lot of that's coming from the biotechs and pharmas, as well as the payers, and other charitable organizations. But what we're seeing is that payers are putting more weight on the ICER analyses. And while the threshold prices that they're creating don't actually have any weight. You know, if a pharma wants to price their drug a certain way it doesn't actually matter what ICER has suggested. Payers have historically used some tools to control how much a new drug is getting prescribed if it's priced way above that threshold. The fact that payers are accepting this may be changing the perception from some biotech players.

[00:03:56] Simone Fishburn: And Lauren, I think you gave a couple of examples, in your very nice story last week, of where companies had not really hewed to ICER's recommendations, and what ended up happening was it exactly that payers sort of restricted the amount that they ended up getting prescribed and put downward pressure on the price. Can you elaborate on those?

[00:04:18] Lauren Martz: Sure, a good example is the PCSK9 inhibitors we've just seen few examples but they're priced way above the threshold, and ended up not doing that well in the first year on market and the prices came down. And then drugs that were launched by the same companies later were then launched within the ICER threshold. I don't know how much the companies really do buy in, I guess that might've been the wrong phrase. But I think there is maybe a growing influence on the way that some drugs are priced.

[00:04:49] Steve Usdin: What's going to be interesting is going to be if there are attempts, when there are attempts, to revive drug price controls in the next Congress, whether there's an explicit role for ICER there or not. Some of the legislation that's been proposed in the past has included roles for ICER, and some of it has attempted to carve them out a bit I think that's going to be very interesting to see.

[00:05:10] Lauren Martz: It will be. A lot of the payers that I spoke with think that there's going to be a role, of course that's something that we'll have to see. And the question is, if there is a role what that would be whether there's going to be an agency created that sort of replicates what ICER's doing, or if the government might contract ICER for specific work, or if they might just use that as a reference point, or as one of the many reference points, to help try to assign value to drugs independently.

[00:05:36] Simone Fishburn: And the sense you got from talking to folks was that ICER was going to want to remain independent?

[00:05:40] Lauren Martz: Yeah, I did speak with ICER as well and they're not giving too many clues about what they'd like to see happen going forward. But a lot of the payers said the thing that makes the most sense for ICER and for whichever administration we have is to have ICER remain an independent organization.

[00:05:56] Virginia Li: Switching gears a bit here Steve, what should we be thinking about heading into tomorrow's election?

[00:06:02] Simone Fishburn: There's a big one...

[00:06:02] Wait wait wait don't say what you actually think!

[00:06:13] Steve Usdin: Look leaving it leaving it just in the the realm of the you know the biopharma world, one of the things that I think is interesting and I think it's important to remember is that whatever happens tomorrow, or whenever we know what the results are Donald Trump is still going to be President at least through January 20th, right. That's a critical time for containing COVID. Cases and fatalities are way too high now, and they're likely to increase as a winter drives people in doors, and holidays bring families together. Just recently Trump has said that he's going to fire Fauci that's something that would have tremendous symbolic importance. I'm not sure that it would affect the actual response because the White House hasn't been listening to Fauci advise for months, and his deputies can run the Institute for Allergy and Infectious Diseases.

[00:07:03] Simone Fishburn: Steve, regardless of who's the next President do you think we're likely to see a new director of NIH, which is another position really important for our industry?

[00:07:11] Steve Usdin: I think yes definitely. I don't think that Francis Collins is going to stay there much longer. My understanding from people who have been in touch with him is that he's long planned to retire shortly after the election, regardless of the outcome of the election. I think that if Biden wins, he's certainly going to want to put his own person there, not due to any animosity towards Francis Collins but I think that he'll want to have his own team. I think that if Trump wins, that Collins would go. So either way I think we're going to see somebody else new there.

[00:07:43] Simone Fishburn: I think that's going to be really interesting to watch, you know even in a Biden administration, I wouldn't expect just to snap back to what NIH has been doing, well I dunno it's such a big organization and resistant to change...

[00:07:56] There's been for a long while now, what's the balance the translational versus basic research, what's the role of NIH. I think the pandemic has accentuated that it's really shown the importance of investment in biomedical science and innovation, much of which - almost all labs around the country have some degree of NIH funding. I think that will be something we'll continue to report on. It'd be interesting to see whether there's a sort of a wholesale change in thinking at NIH, or just an operational change in terms of being more effective.

[00:08:29] Steve Usdin: Or if there's a move to create new institutions for translational research, which is what I would advocate. There is pending legislation to create new institutions for translational science. Personally I think that's the best way to go. I think that NIH is not likely to have the kind of flexibility to do what's needed. What Operation Warp Speed (OWS) has demonstrated is what can happen when you have industry and government working together in a highly targeted way against specific problems. And I think that one of the lessons that could come out of COVID-19 will be the need to create new institutions, and new ways of collaborating between government and industry, and hopefully patients also.

[00:09:15] Simone Fishburn: Some of the things with that Steve, you know when I talk and this is even at the director level with NIH Institute Directors, two things are clear - there's this broad consensus on two things. One is that the Institute structure is a mess. You have brain science going on in lots of places, you have sort of a hodgepodge of missions, and of budgeting. It's really not the way you would design something from the ground up, it's something that sort of evolved over time. I think the most recent one that they added is (NCATS) I believe.

[00:09:45] I think that given all that we've seen this year, we may see more attention, maybe more funding go to the National Institute of Minority Health and Health Disparities (NIMHD); I spoke with the director of that institute for our back to school package. It's really important and I think there's just a growing understanding through COVID of the need for equity in health outcomes and therefore including minorities, and various underserved communities in clinical trials. And so that Institute is dedicated to promoting and enabling that. So I think that might be one part of NIH where we see a difference.

[00:10:23] The second thing I hear is the politics behind the cleanup inside reorganizing institutes is just a mess and will basically never happen, or not for a long time. So I just wonder if the idea of creating another institute would just add to that sort of feeling of...

[00:10:40] Steve Usdin: No, what I'm talking about and what there's pending legislation is not to create another Institute at NIH, it's to create institutions outside of NIH that would be responsible for translational science. It's very cumbersome to change it, it's not nimble enough to have the kind of large scale change that is needed on the timeframe that would be needed to do something, and that the better policy would be to create a new institution completely separate from NIH, and in some ways a rival to NIH. I think that the idea of having a little bit of competition for who has the best model for funding biomedical research would probably be a good thing.

[00:11:21] But getting back to the elections and what's going to happen this week, and in the coming months. I do think that it's going to be really important to look at what happens with the leadership of HHS, and the HHS Agencies to try to bolster their independence, and public confidence in those institutions during a really trying time that it's going to be coming up in the next couple of months.

[00:11:44] Virginia Li: So with the leading COVID-19 vaccines approaching their interim readouts, what sort of discussions are happening at the FDA right now regarding the initial rollout of those vaccines, and what sort of decisions do they have ahead of them?

[00:11:59] Steve Usdin: I think there's going to be interim readouts from Pfizer's, and Moderna's vaccine very soon sometime in the next few weeks. If it's a strong readout saying that there's a strong signal of efficacy, then obviously that sponsor's going to apply for Emergency Use Authorization (EUA). There's going to be an advisory committee meeting and they'll be a lot of debate about what should be done. Part of that debate, I wrote a story about it last week, is whether the interim readout should lead to Emergency Use Authorization (EUA), or whether it should lead to expanded use, or whether FDA should defer until there's more data. And there are people who are advocating all three of those paths.

[00:12:42] Virginia Li: So what would the pros and cons of an expanded use pathway be?

[00:12:48] Steve Usdin: So expanded access, which other people call "compassionate use" is a pathway that FDA has for allowing pre-approval access to medicines, it's different from Emergency Use Authorization (EUA). The main differences are that it would be slower, it would be much more under FDA's control, and it would probably be a more limited number of people who would be able to get access that way.

[00:13:14] There are some people who advocate expanded access precisely because it would be slower and more controlled.

[00:13:21] There are other people who oppose it precisely because it would be slower and more controlled.

[00:13:25] So where you stand on that really depends on whether you think that the risks of having an experimental vaccine widely used, outweigh the benefits of having an experimental vaccine out there and widely used. And you have to remember, and it's hard to forget that we're looking at this against the background of of a pandemic that's out of control.

[00:13:50] Simone Fishburn: Yeah, I think the way that we've previously talked about this discussion is sort of I don't know if I want to go as far as say irresponsible, but it would certainly be a weighty decision to give Emergency Use Authorization (EUA) that could end up scuppering the development of vaccines that could be better. There's no reason that the first vaccine to reach the goal line is necessarily the best one. On the other hand, you've got a hundred thousand new cases a day in the U.S. And not getting something out as fast and broadly as possible, by that view, is irresponsible. So it's not an easy one to solve.

[00:14:28] Steve Usdin: I think it is also not obvious that Emergency Use Authorization will hinder the development of other vaccines. If you look at technically in the way that FDA views it, is an Emergency Use Authorization (EUA) is not an approval, even though the president and others have portrayed it that way. A vaccine that's distributed under Experimental Use Authorization (EUA) is still experimental. And for that reason FDA believes that placebo controlled trials should continue and that the blinding, the trials that are ongoing shouldn't be unblinded.

[00:15:04] Simone Fishburn: Right. It really boils down to whether the companies with EUA will unblind their trails.

[00:15:10] Steve Usdin: And also the timing that they might do it. Moderna and Pfizer both said that they feel an ethical obligation to un-blind their trials after Emergency Use Authorization (EUA) has been granted. But they might wait for several months to do that, they might wait until a vaccine is widely available to do that. And if they did that, it would give them time to finish their trials and it would give other companies that are coming slightly behind them, time to get their phase three trials well underway.

[00:15:39] Virginia Li: That's all we have time for today. Registration is open for our 7th BioCentury-BayHelix China Healthcare Summit. This digital event runs from November 9th to the13th and includes strategic panels, one-on-one virtual meetings, company presentations, and two conference reports from our partner McKinsey. Register today to get immediate access to our pre-event program including BioCentury business intelligence on China biotech.

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