Background

A little over a year ago, my employer submitted trial data to the FDA relating to the efficacy of one of the company's diagnostic products that was, at that time, already being used under an EUA (Emergency Use Authorization). The new data involved a direct performance comparison between the company's product and a class of competing products already on the market. We planned to publish the data, but shortly before doing so, we shared it with the FDA.

Per my employer's analysis, the trial showed that those competing products had severe performance problems in real clinical practice (that had gone undetected in previous trials of them that used contrived samples or cherry-picked non-representative patients). This had implications not only for clinical practice but also for the FDA's approval process, since the FDA had been evaluating new diagnostics of the sort that my employer had developed by requiring concordance tests between them and this class of competing products that - we now argued - frequently got wrong results. As a consequence, we argued, tests that were actually more accurate were at risk of being unfairly rejected, while this inferior class of diagnostics we were competing with would be easily permitted on the market.

(I should note here that the science is disputed and that many studies exist that reach opposing conclusions. Anyway, the meat of the story, at least to my mind, is what happened next...)

According to the various executives who've told me the story, upon receiving this data, FDA staff spoke to our CEO on the phone and demanded that we not publish the data, threatening, explicitly, that if it were published they would respond by revoking our existing EUA for our product, thus destroying our existing business. This was after we had already shared the data privately with the FDA, so this wasn't a discussion about what the FDA would hypothetically do if presented with this data; it was specifically about the consequences of publishing it for others to read. We assume that the threat was motivated by fear that data like ours would cast doubt on the FDA's previous decisions about which products to approve in our field.

(I should note here that I was not a direct witness to this threat, but I have a direct witness account of it in writing and believe it to be truthful - although I am not entirely sure. We don't have the threat itself in writing, since it was made over the phone. For what it's worth, the only person with experience working for the FDA who I've chatted to about this confirmed that the FDA likes to keep discussions with companies over the phone specifically to avoid a paper trail - so that part of our CEO's story checks out.)

Eventually we did in fact publish (presumably in defiance of the threat), but the company never spoke publicly about the FDA threatening us (presumably because we're afraid that doing so will trigger retaliation or cause us to receive less favourable treatment from the FDA in future). I have tried since then, unsuccessfully, to persuade the CEO to speak out - either to the press, or to some FDA HR or internal affairs department or to some other federal law enforcement agency that could take action against the FDA staff who made the threat. I have not succeeded.

The arguments for and against speaking out

I'm trying to figure out what to do next. The company's management is not keen to pick this fight and have tried to persuade me that it is a waste of time and energy for me to be thinking about. To me, it seems like shrewd action here could potentially save huge numbers of lives. To summarise our disagreements...

How many people do we expect to be seriously harmed if this behaviour is left unpunished?

My take: Potentially, vast numbers! If these FDA staff were willing to use this tactic against us, it's reasonable to expect they'll do it again whenever they see similar political/reputational/work-avoidance advantages to doing so. And such suppression of trial data has the potential to cause mass death; the most famous case I know of where non-publication of clinical trial data was blamed for subsequent deaths is the use of lorcainide on people who have had heart attacks, where initial trial data from 1980 that suggested it actually made them more likely to die was not published, and doctors in the US didn't realise and stop prescribing it until they'd killed 100000 people. And that was merely one case of a voluntary decision by researchers not to publish inconclusive data that they didn't trust the conclusions of. Repeated suppression by a regulator specifically of credible trial data that casts doubt on their past approvals could thus be expected to regularly have severe consequences like this.

Management's take: Thinking there are lots of lives at stake here is wild speculation. We don't know these kind of threats have ever been made to anyone other than us. We ultimately called the FDA's bluff and published our data despite the threats we received, so this tactic doesn't seem like it's even very effective at suppressing data. And the spectacular bad consequences of data on lorcainide not being published are clearly exceptional, which is exactly why lorcainide is famous; decisions not to publish trial data happen all the time, and obviously don't normally kill 100000 people. Nor would suppression by the FDA typically have such consequences, even if it were widespread.

Would the public and/or a regulatory or disciplinary body even see this behaviour as wrong and worthy of punishment?

My take: Of course! The FDA is meant to make approval decisions based on safety and efficacy. Here they explicitly threatened to make those decisions conditional on us burying trial data. That's contrary to the FDA's mission, a violation of the First Amendment, and almost certainly some sort of criminal corruption. Most people will see this the same way as me: clinical trial data should be published, and any story about it being deliberately buried deserves to be a scandal.

Management's take: Even if you convince people this really happened, nobody is going to care. It's not like the FDA reviewer was soliciting bribes; without some kind of direct personal gain for the FDA staff involved, nobody's going to see this as corrupt. Furthermore the FDA staff involved can simply argue that they didn't want the research published because they felt the methodology was flawed and the conclusions were wrong, and most people, including anyone who might take disciplinary action against them, will see that as perfectly reasonable.

(For what it's worth, the previously mentioned FDA insider I've spoken to about this was of the view that if it were proven this would be a scandal and should lead to the FDA firing the staff involved.)

Even if this would be seen as wrong, is it worth trying to take action without hard evidence?

My take: Of course! Just like with any crime or act of wrongdoing that you witness, you don't necessarily report it (to authorities, or to the press) with proof already in hand. You make the allegation, and leave it to the cops / HR people / journalists / etc. to investigate that allegation. Maybe other FDA staff who were present for the meeting but uncomfortable with the threat being made will snitch when asked about it. Maybe they emailed each other about it and there's an internal paper trail! An investigation has at least a fighting chance of finding proof, and that's enough to justify starting one.

Management's take: The only evidence we have is the word of our own staff. There are no recordings and no paper trail. Without hard evidence, nobody is even going to bother starting an investigation into this.

How much scope is there for speaking out to be counterproductive

My take: Not much. Even if an investigation fails to find any evidence, it should still instil fear in the guilty parties and deter them from making such threats in future - which is still a good outcome. And any public reporting about this threat, even without proof, may encourage other companies that have received similar threats, especially from the same individual FDA employees, to speak up.

Management's take: Lots. The FDA is a household name that people trust; we are not. The likely outcome of any attempt by us to take action on this is that it ends up in the press, the reporting makes us look like liars with a bad product, the FDA retaliates against us by refusing approvals on future products and thereby wrecks our company, and anyone else who has a similar experience to us in future and thinks of speaking out will look at the fate we suffered and think again.

My questions

With the above disagreements in mind, I'd like to seek some advice, especially from people with experience of the US medical industry, and most especially from anyone with direct experience of dealing with the FDA.

1. Is it normal for the FDA to try to control what trial data does and doesn't get published via threats of regulatory retaliation? Have you heard similar stories of this? Indeed, do you find the claim plausible, or think that our CEO must be lying to me for some reason?
2. Is fighting to get the FDA staff involved disciplined a worthwhile cause and potentially a way to save many lives, like I think it is? Or would I be wasting time and energy and putting my employer at risk with no real prospect of doing any good, as the company thinks? If you had to put an expected value in QALYS or lives saved on me or my employer pursuing this, what would it be?
3. If I were to leak what I know against the company's wishes, who should I leak to?
   1. The press? If so, which journalists or outlets?
   2. An internal team at the FDA? If so, who? There's an Office of Internal Affairs at the FDA; would they investigate an allegation like this?
   3. Some kind of federal law enforcement agency? If so, who? The GAO? The FBI? Who would be interested in this?
4. Are there any other people or organisations you'd suggest talking to for advice on this?
5. Besides "leak what I know to somebody with the ability to investigate further" and "do nothing", are there any other courses of action I'm failing to consider that could do some good?

And finally, putting it all together: what would you do in my place, and why?