Dan Gorenstein: We’ve arrived at the fourth and final installment of our series “The Aftermath of Aducanumab.” 

This week, we’ve heard some of the messy and complicated questions that this pricey, and controversial new Alzheimer’s drug, also known as Aduhelm, has raised for doctors, patients and payers.

Today, we hear what’s on the minds of pharmaceutical industry insiders.

From the studio at the Leonard Davis Institute at the University of Pennsylvania, I’m Dan Gorenstein, and this is Tradeoffs.

DG: Luke, if you would, just start by introducing yourself, please. Your name and title.

Luke Timmerman: Sure, I’m Luke Timmerman, the founder and editor of Timmerman report, a biotech industry newsletter.

DG: And what’s your background?

LT: I’ve been a biotech journalist for about 20 years.

DG: Based on your conversations with sources in the industry, Luke. What’s been the most common reaction to the aducanumab approval in one word?

LT: Outrage. People in the biotech industry want the FDA to be the guardian of safe and effective drugs. And in this case, they appear to have lowered their standards. When that happens, it potentially can reduce the confidence that physicians have in what an FDA approval means. It can shake the confidence of payers who count on the FDA to do a rigorous evidence-based analysis. It’s really the foundation of the industry. It’s kind of what separates prescription drugs from nutritional supplements. 

DG: Were you surprised with this ruling?

LT: I was, I was. The evidence in support of this application is very weak at best. There’s so many things here that are just really head-scratching like, why would the FDA surrender so much of their authority here? They said that they’re going to require the company to run a confirmatory trial, but this trial could take as long as nine years to complete. So in the meantime, Biogen will be able to market this drug. Physicians will be able to prescribe it. Medicare will be asked to pay a lot of money for it. And during all these years, we will not really know if it is bringing the clinical benefit that everybody wants to see. 

DG: So then Luke, what is the best explanation you’ve heard for why the FDA approved this drug, given all the doubts?

LT:  Well, I don’t think the FDA has clearly explained itself. I will say, though, that I have seen one previous situation with a prostate cancer drug that was a first-of-its-kind cellular therapy. And this was a controversial dataset much like aducanumab. I spoke to one of the advisory committee members who said he voted in favor of it because he thought that would stimulate better therapies to come. So I think it’s entirely possible that this is part of the motivation and it’s awfully hard to say no to, you know, very sympathetic victims when these people are being offered something that looks like hope.

DG: Earlier this week, after we interviewed Luke, the FDA did begin to explain its decision, releasing 83 pages of internal documents.

In one of the memos, an FDA official appeared to make the case that giving patients a chance to try a drug that might work was more important than waiting years for Biogen to prove the drug definitely works.

Acting Commissioner Janet Woodcock also turned heads this week with some public remarks defending the FDA’s approval by saying that, “empirical evaluation is not the only way of evaluating truth.”

DG: Do you think that Aduhelm’s approval is gonna drive more research into other Alzheimer’s treatments, Luke? 

LT: There has not been a lot of reason for hope and investors have looked at this field and been pretty discouraged. They’ve put their money in other areas that look more promising by and large. Other entrepreneurs could look at this, and be inspired to try the longshot idea that maybe wouldn’t have gotten funded before. So it could spark something of a rebirth of Alzheimer’s R & D. But it could also send the wrong message as well that we should double down on this amyloid hypothesis, which has been something of a blind alley in the scientific community for the last two decades.

DG: On cue…drugmaker Eli Lilly has now announced plans to seek accelerated FDA approval for a therapy similar to Aduhelm.

DG: How do you expect this to influence drugmakers’ moves outside of Alzheimer’s? Will we see denied drugs coming back from the dead new drugs with worse evidence? In essence, Luke, is this sort of open season for drugmakers?

LT: It could be perceived as an open invitation to take that failed study from a couple of years ago, do what’s called data dredging, like fish around in the dataset, try to find out if there might be some positive signals there in some subpopulation of patients, package it together, enlist the patient advocates on your side, who are often desperate, put pressure on your local Congressman and the FDA and the people in the media, and give it a whirl. And that opens up this kind of can of worms that most people in the pharmaceutical industry just don’t want to go into. 

DG: When we come back, we ask Luke the 56,000 dollar question…is this drug worth the price.

MIDROLL

DG: We’re back with biotech journalist Luke Timmerman, who’s closely watched the FDA and the saga of this Alzheimer’s drug aducanumab for years.

Luke, let’s turn to the price of this treatment cause it’s made some jaws drop. 

What’s been the reaction inside the biotech industry to Biogen’s $56,000 decision?

LT: This greatly concerns everyone I talked to in the biopharmaceutical industry, because it could provoke a payer backlash and a bigger populist backlash. Like, look, there was a lot of heroic work that was done during COVID-19. And now here comes along a company with an Alzheimer’s drug that’s really unproven and they’re setting this egregiously high price. This is the kind of thing that gave pharmaceuticals a bad name in this country and a lot of people are worried and upset that this could boomerang back and actually chill all kinds of other drug development.

DG: That boomerang is already coming back at drugmakers.

Yesterday, June 24th, Democratic Senator Elizabeth Warren and Republican Bill Cassidy called for a hearing on Aduhelm, asking whether Congress should give Medicare more power to tie the price of a drug to its clinical effectiveness. 

Biogen is feeling the heat. Just the day before, the company said, “We stand ready to work with public and private payers to address pricing,” an eyebrow raising statement…and a nod to widespread doubts that $56k a year is a fair price for a drug of questionable efficacy. 

DG: Is there a quote that you’ve gotten from an industry executive that captures the concern around Biogen’s putting a really big price tag on a drug that has shown so little evidence that it actually does much?

LT: I spoke to one executive who shared that he had spoken to someone at Biogen and said, “What the hell are you guys doing?” And he told me, he thought, you know, maybe they’d price it at $12,000 and that even made him a little queasy. It isn’t like something that he would recommend to his mom and dad because he’s looked at the evidence. But yeah, that price just strikes many people, even within the pharmaceutical industry, as egregious. 

DG: Anything from investors? Are you hearing that investors are sort of eager to pour money into more risky stuff now?

LT: I think biotech investors have had a pretty healthy appetite for risky things the past few years anyway. We really are in a golden age of biomedical research and development. It’s been raising a lot of money. It’s been rapidly expanding across different therapeutic areas, and I think a lot of investors would like to keep it that way. And when they see a cloud of doubt come from the FDA, it makes a lot of them nervous.

DG: I mean how big of a deal is this decision, Luke, when it comes to the FDA’s reputation, or how people perceive the agency and its standards?

LT: Can I tell you a little personal anecdote? So I’ve been covering this sector for a long time, and I speak with my friends and family about things like the vaccines. And one of the things that I’ve told them is about that FDA process, that part of what makes us the world leader in biomedicine is that we have this really rigorous, transparent process. They don’t just read a paper and take somebody’s word for it in industry or at Harvard or Stanford or anywhere else. They do their own diligence. So when I see something like what happened with aducanumab it’s demoralizing. I kind of feel like I have egg on my face and it gives me some doubt, like a little bit of doubt, about what it is they’re doing. And I think there are a lot of people in industry that are asking themselves that same question.

DG: Luke, thank you very much for taking the time to talk to us on Tradeoffs.

LT: Thanks Dan, for having me.

DG: When you step back and look at all the Aduhelm news that’s unfolded, it’s pretty wild.

Just this week, bipartisan senators called for a hearing…FDA leaders published a rare op-ed defending their decision…drugmaker Biogen signaled it might actually give up some ground on price…and Health and Human Services Secretary Xavier Becerra said his agency is looking closely at the drug’s cost.

…and Aduhelm has only been approved for 19 days.

Thanks to all of you, our listeners, for tuning in all week to this special series our team worked so hard to put together.

This is a fast moving story we plan to follow closely and we look forward to sharing with you what we learn.

And if you have any show ideas, pitch us at info@tradeoffs.org

I’m Dan Gorenstein and this is Tradeoffs.