Jun 11, 2021 - Health

Biogen says FDA didn't push for trial before Alzheimer's approval

The blue Biogen logo on its white headquarters building and a tree in the foreground.

Photo: Adam Glanzman/Bloomberg via Getty Images

Two Biogen executives said in an interview the company's newly approved Alzheimer's drug is priced fairly, and that it didn't conduct another clinical trial before approval because the FDA didn't push them to do so.

Why it matters: The scientific consensus is the drug, Aduhelm, has not been proven to work. But Biogen and the FDA are content with "hopefully" validating the $56,000 drug's efficacy at some unknown point in the future.

This interview was with Maha Radhakrishnan, Biogen’s chief medical officer, and Chirfi Guindo, Biogen's head of global product strategy and pricing, and has been edited for brevity and clarity.

Axios: The FDA approved Aduhelm under the condition that Biogen run a confirmatory trial. When will the company start that?

Maha Radhakrishnan: We're in the process of discussions of what the study should look like. As things become more clear to us, we'll be sure to share that publicly.

A: The FDA said they were going to give up to nine years to submit trial data. Do you plan on taking that long?

MR: Our plan is to be able to finalize a study design, the patient population, the site, the countries, et cetera. What we do not have in our control ... we do have milestones on recruitment and enrollment. Whether it will pan out in the real world as we expect is the question, which is why we usually give ourselves time, but with the hope that we can can expedite and accelerate the study readout.

Q: Why didn't Biogen do this trial in 2019, when you were parsing through the data again?

MR: That's a good question. But I do want to go back a minute to the amount of information, the substantial volume of evidence that we submitted to the FDA as part of our filing package. We submitted data on eight clinical programs ... so based on the negotiations we've had with the regulators, we are about to embark on a path where we will actually look to validate and verify the data that we have seen in our clinical program.

Q: But going back to my question, why didn't Biogen conduct another trial before submitting an application to the FDA?

Chirfi Guindo: I think we were informed by our engagement with the regulator, and we were prepared to do what was necessary in order to get Aduhelm to the finish line. We're pleased to be in a situation now where we can provide access to these patients and their families, while we continue to generate data going forward.

A: But why not start the trial when you were looking at the data in 2019? One of the phase 3 trials failed outright, and then the other showed some signs of amyloid reduction. Didn't you feel you at the least needed a tiebreaker?

MR: When we decided to go back into discussions with the FDA, after really looking at the data, we were essentially respecting the guidance from the FDA in terms of what was needed to submit the [drug application]. All companies follow the pathway given by the regulators.

A: So, in 2019, did the FDA say you didn't have to run another trial just submit an application?

MR: Anytime a company gets into a regulatory dialogue, we usually listen to the advice and feedback that we get.

A: Why does Biogen believe Aduhelm's $56,000 list price is fair, especially considering this hasn't been proven to work? This could potentially balloon Medicare spending.

CG: We've consulted with experts, health economists, clinicians, as well as payers and policy leaders, because we take this responsibility very seriously. We were guided by the value that Aduhelm has and will have going forward. The second principle is the commitment that we have to continue to research and develop innovative medicines in, not only Alzheimer's but also other areas of neuroscience. These are all areas that are very risky. And then lastly, we are committed to working with the payers, working with public policymakers, to ensure that Aduhelm is affordable for patients. That is really what is most important to patients: affordability, and then the sustainability of the whole system.

A: But doing just a little math on how Medicare pays for these drugs results in tens of billions of dollars in added spending. So it's going to raise out-of-pocket costs significantly, but also Medicare premiums.

CG: We anticipate that there will be probably around 10% of Medicare patients who would be really exposed to a high copay. We have been talking with [the Centers for Medicare & Medicaid Services] to see if there's something that can be done for those patients. On the total budget impact, we do not anticipate a very rapid uptake. This is not a treatment that is one pill, once a day that your average family physician can prescribe.

A: Even if you get only 200,000-300,000 Alzheimer's patients, we're talking upwards of $17 billion. That'd make it the drug Medicare spends most on by a long shot, no?

CG: I don't believe we will get into those tens of billions of dollars, at least not in the initial few years. But if you look at the bigger picture, the system collectively spends $600 billion a year on Alzheimer's care. Just imagine you have a treatment that over time can mitigate some of that cost.

A: But those analyses assume the drug works. How can you make those types of assumptions of saved costs if Aduhelm hasn't been proven yet?

MR: You keep saying it has not worked. The data from our studies actually talk to the evidence seen in the study, which is what we're looking to verify by the next study. And again, in the last 20-plus years ... there has been really no treatment, no hope for patients. What Aduhelm offers, hopefully, is a hope to really be able to integrate these patients back into society, give them back a quality of life.

Go deeper