FDA’s accelerated approval pathway has a few potholes
The Health and Human Services Office of Inspector General has found that, in a few cases, FDA officials veered from the accelerated approval pathway.
The Food and Drug Administration sometimes uses a program called the accelerated approval pathway to get new drugs to the market quickly. Such drugs may not kill you, but they may not help much either. The Health and Human Services Office of Inspector General found that, in a few cases, FDA officials veered off the path. Social science research analyst Ivan Troy joined the Federal Drive with Tom Temin to provide the details.
Interview transcript:
Tom Temin: And just give us the background on this program. How far back does it go and how do they tend to use it and for what? The accelerated approval pathway.
Ivan Troy: So accelerated approval pathway started in 1992 and FDA, the Food and Drug Administration, can use it to approve drugs before those drugs can show that they have an actual clinical benefit to people. And it’s only used for drugs that treat serious conditions and where there is an unmet medical need.
Tom Temin: So this is important to note. It goes back to 1992. It wasn’t something that happened in the flurry of debate about FDA of recent years.
Ivan Troy: Yeah, I believe it really started because of HIV and the lack of drugs to treat that.
Tom Temin: All right. And do we have a sense of volume? How many products have come through that program since 1992?
Ivan Troy: At this point, it’s a little over 300 drugs, at least through FDA Center for Drug Evaluation and Research, which is what we looked at.
Tom Temin: And it’s drugs, but not devices?
Ivan Troy: Correct. Could be biologics, but not devices.
Tom Temin: All right. So what sparked this look-see and tell us your methodology?
Ivan Troy Sure. So in 2021, FDA approved the drug Aduhelm, which was for treating Alzheimer’s disease. And that approval was very controversial right from the start. A lot of people raised concerns in Congress, other medical researchers, some people within FDA. And what we didn’t know at the time was, well, is this the norm for accelerated approval or was this more of an outlier? And so that was the question that we set out to answer.
Tom Temin: What was controversial about that? That it only worked in a small percentage of cases or could it have actually harmed someone? I mean, my understanding of the program is that these items so approved may not help much, but they’re probably not dangerous either.
Ivan Troy: Right. They have to meet the same safety standards than any other approved drug. But in this case, for Aduhelm, there was concerns that using accelerated approval wasn’t really the most appropriate pathway forward. FDA approved the drug despite the concerns of some of its own reviewers. And there were some concerns that it didn’t document meetings with the drug company.
Tom Temin: All right. These are all program rules, but also not just.
Ivan Troy: Well, less rules than best practices, which we’ll get into, I think, in a moment.
Tom Temin: So, yeah, let’s go into that right now. What are the best practices? Why would, I guess, it has to do with what it is that gets through that approval process in the first place?
Ivan Troy: Well, I mean, just take a step back for a minute. What we found was that those concerns weren’t the norm. There were some outliers, including Aduhelm. And so we identified three drugs, and putting Aduhelm where they had some of these similar concerns. So there was Aduhelm. Talked about eteplirsen to the drug for treating Duchenne muscular dystrophy and hydroxyprogesterone caproate, or HPC, which is a drug to reduce the risk of preterm birth in certain women. And so these three drugs shared some of the same concerns as Aduhelm.
Tom Temin: And what sort of sample did you look at of drugs thus approved?
Ivan Troy: So we looked at 24 drugs that had gone through the accelerated approval pathway. Ten of them were recommended to us specifically by knowledgeable stakeholders and the rest were a random sample of drugs we took.
Tom Temin: So there was a little bit of a whistleblower, almost?
Ivan Troy: Less of a whistleblower, and more just, here are some drugs we think could use some scrutiny.
Tom Temin: And how does the FDA and the people involved, how do they decide this is something that is worthy of the accelerated pathway and this should go the regular route?
Ivan Troy: Well, again, it comes back to that sort of initial definition for serious conditions and where there is an unmet medical need. So, for example, it might be the only option. There might not be other existing treatments or it might be an improvement over existing treatments.
Tom Temin: We’re speaking with Ivan Troy. He’s a senior social science analyst in the Office of Inspector General at Health and Human Services. So you found 3 of 24 in the sample. And tell us what it is in more detail that didn’t happen that should have.
Ivan Troy: Sure. And again, let me reiterate that even though these are some concerning factors that we identified, they’re all allowed. So FDA has a lot of flexibility in how it administers this pathway. And so the three drugs that we identify, push that flexibility further than we saw in any other drugs. But everything is within that flexibility, within FDA’s authority. And so there are these three factors that we saw. And the first was that FDA approved all three drugs despite concerns from its own reviewers, some of whom didn’t support approval at all. Additionally, for Aduhelm and for eteplirsen, FDA Use and External Advisory Committee. And those committees didn’t support approval either. Second, for Aduhelm and for eteplirsen, FDA veered from its own best practices by evaluating analyses that the drug companies didn’t initially have. And then finally, for Aduhelm, some meetings between FDA and the drug company appear to be missing from the files that we reviewed and meetings for other drugs weren’t fully documented.
Tom Temin: So it’s OK to meet with drug industry officials? You just have to make sure that it’s known and disclosed.
Ivan Troy: Yeah, it’s an issue of transparency.
Tom Temin: Right. So then I guess someone could arrive at the conclusion, rightly or wrongly, that, ‘Well, you ignored the advisory board and you met with industry and didn’t tell anybody you were influenced by industry to get this on the market.’ Someone could see it that way.
Ivan Troy: Sure.
Tom Temin: All right. Well then, do you have recommendations then or simply follow these best practices?
Ivan Troy: We made two recommendations to FDA that we think will strengthen the pathway by putting in some better guardrails and increasing transparency. And both of these recommendations build on some steps that Congress has taken to do the same. And so the first recommendation builds on this accelerated approval council that Congress created a couple of years ago. And this council is comprised of senior leaders across FDA. And its charge is to ensure consistent use of accelerated approval across FDA. And so what we’re recommending is that FDA define specific factors that would require this council to weigh in on drug approvals, basically identifying outliers such as we did get some extra sets of eyes on them. And we also recommend that FDA better document meetings with drug companies in its administrative files. And we think this will increase transparency in FDA’s decision-making and bolster public confidence in those decisions. And Congress, also in its own investigation with Aduhelm, made a similar recommendation.
Tom Temin: Got it. And FDA generally accepted your recommendations?
Ivan Troy: Well, they agreed with the recommendation regarding meeting documentation, but not with the recommendation about the council.
Tom Temin: So that’s kind of an open disagreement item?
Ivan Troy: Yeah. I mean, we have an internal process. We can go back and forth at the agency, so we’ll see what happens. You can also track the progress of our recommendations on our website.
Tom Temin And by the way, I just had a question about your title, senior social science analyst. What does that mean in this context?
Ivan Troy: Basically, within the OIG, we’re doing evaluation work. So as opposed to any kind of clinical science or anything like that, we’re using more social science skills. So quantitative and qualitative data analysis, things like that. Interviews for large datasets.
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Tom Temin
Tom Temin is host of the Federal Drive and has been providing insight on federal technology and management issues for more than 30 years.
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