Letters to The Trump Administration: Advice for The FDA

Continuing my Series of Open Letters to Specific People in The Incoming Trump Administration

As a Child and Adolescent psychiatrist, I've often been in the position of having to have conversations nobody wants to have. I've worked in emergency rooms and inpatient psychiatric settings. These are places where conversations are had not because they are voluntary, but, rather, because they are necessary.

Many years back, I had the pleasure of being a keynote speaker for the American Academy of Emergency Psychiatry. Physicians are in a very peculiar role in emergency psychiatric settings. We are often in positions in which we have tremendous power and control over others. There are laws in place that limit what we can do—for example, in an emergency, my signature would allow someone to be legally restrained and injected with medicines, even if it was against that person's will. The caveat, of course, is that our ability to use force upon someone else is premised on acting in their best interest. Simply put, I don't get to shoot someone up with meds because I want it or because it's convenient for me; it has to be good for them.

It's a fundamentally different role than that of judges and others empowered by society to use force or coercion to control others. Their charge? It does not require the actions taken to be in the interest of the person they are acting upon.

Psychiatrists, more than any other type of physician, are empowered to use extraordinary methods— that the people affected might object to—but only do so in the interest of that individual. Other fields of medicine don't allow for much-forced healing. You can't leave a psychiatric unit against medical advice if you're involuntarily committed. Still, you can only be involuntarily committed if it's in your interest, according to physicians, including a psychiatrist, and usually a judge as a last stop. You can't sue a judge for putting you in prison. You can sue a Psychiatrist for putting you in restraints, forcing you to take a medication, or even not discussing the suitable risks and benefits of a treatment with you in the process of prescribing it.

We have to act in the interest of others. So, too, with advice. When you're providing therapy, as I do in my role as a psychiatrist, the things you’re saying are for the benefit of the other person. They're not to gratify ourselves. Patients pay us, and that's our side of the deal, but the guidance and change we catalyze in the lives of others? We act in the other person's interest, not our own.

So these letters, the ones I'm writing to the incoming administration, have a particular frame. That frame is in keeping with my role as a physician; I'm writing them to be in the interest of the people I’m addressing them to. I want this advice to be helpful to people who have the health of all of us as part of their purview. No matter what you think of the incoming administration—and many people have feelings about that—the success of the administrators in charge of the healthcare and regulatory apparatus must succeed, both for themselves and the good of all of us. I've already written letters to RFK advocating for the appointment of Dr. Drew to the director of SAMSHA and Dr. Oz, recommending a focus on payment models for medical devices.

Today, I am writing to the incoming director of the FDA and providing some relatively limited advice for what would be successful for him. Dr. Martin Makary is the administration's pick for the new FDA director.

The following letter is to him…

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Dear Dr. Makery,

Welcome to the helm of the government's second most crucial regulatory agency in healthcare. From my perspective, the first is now under the aegis of your colleague, Dr. Oz.

This is obvious to you, but I will include the information in the spirit of an open letter so the audience can follow along. The FDA has not always existed and hasn't always regulated the effectiveness of medicines. The FDA began regulating the effectiveness of medications in 1962 with the passage of the Kefauver-Harris Amendment to the Federal Food, Drug, and Cosmetic Act. This was a reaction to horrors related to the drug thalidomide, and the public outcry led to the passage of the above bill; however, as physicians, and particularly as interventionists—you as a surgeon and myself as an interventional psychiatrist—we know that many things done in the service of healing are not under the purview of the FDA. The recent kerfuffle around MDMA is an excellent example of this going badly. The agency was asked to review a “drug plus therapy combination,” which isn't what the FDA does. As a brief aside, we should admit to ourselves:

Most things that are therapeutic aren't therapy. Much of therapy isn't therapeutic.

At the FDA, that goes for food and drugs, but it also includes medical devices. The FDA has a limited resources problem. This can be ameliorated by the fact that the industry pays to interact with your agency.

I would make one recommendation first: currently, one of the ways the FDA is funded is through the 513 (g) process.

More …Paid Advice from FDA

The 513 (g) process came to be from a citizen's petition—my colleague Fred Ma, M.D., another surgeon, first asked for it! It is a formal mechanism through which medical device manufacturers can request the FDA's classification and regulatory requirements for a device. It originated from Section 513(g) of the Federal Food, Drug, and Cosmetic Act, enacted to provide clarity on device classifications (Class I, II, or III) and applicable regulatory controls.

Industry pays the FDA to get advice. Currently, the 513 (g) process for medical devices is completely backed up. Predictably, this is because there are no metrics for the FDA to meet regarding timelines for reviewing 513 (g) submissions. I recommend setting standards inside the FDA regarding the timeframe for the review of these submissions. It will allow your agency to get more money, it will encourage industry to pay you for the advice before the submissions, and this will lead to less uncompensated work reviewing submitted applications. Think of it as a study hall, but you get paid extra to proctor it. It will improve the quality of submissions to the FDA and teach medical device manufacturers, many of whom are from outside traditional biosciences, to submit better applications to your agency, which speeds up everything later.

More of Approval like these…

I think we have had excellent predicates recently, and I would encourage you to look at medical devices like the Huma app, with its extremely broad Indication for Use, as a model for expediting the review of novel technologies that are very similar in terms of risk to prior modalities.

Similarly, I think the agency did excellent work with the 510 K clearance of transcranial magnetic stimulation (TMS) as a first-line adjuvant treatment in adolescence, based on post-marketing data. However, TMS still needs to be appropriately covered, with medically unlikely edits on the CMS side restricting the best deployments of the technology, such as accelerated TMS.

What to Change at FDA, According to Me

The more FDA can incorporate post-marketing data into approval requirements, the better. It shunts funding away from too expensive clinical trials that capture only a tiny subset of the population. It moves therapeutic development towards broader population health-level surveillance as essential.

Currently, clinical trials cost a lot, and drugs make a phenomenal amount of money. This is because CMS has abdicated any responsibility to negotiate the prices of those drugs.

Payment is a problem for Congress and Dr. Oz to fix, not for FDA. However, the more we can drive down the cost of getting a therapeutic approved while increasing requirements for cost-effectiveness, the better. It shouldn't be that FDA determined safety and efficacy in the smallest subset of people necessary to get a therapeutic over the approved is the end., and then never again is a therapeutic required to prove it does anything useful.

We have some excellent legislation proposed last year, including the Breakthrough Therapies Act, which I would strongly recommend reviewing and working with your colleagues in the administration to pass.

The focus should revolve around partnering with CMS to make sure that more FDA-approved treatments move seamlessly through the New Technology Add-On Payment (NTAP) program plus outpatient.

This requires a crucial expansion of the Emerging Technologies program, like the TCET program in the outpatient setting for all breakthroughs.

Breakthroughs + Payment

We can develop all the remarkable technologies we want, but if it is too expensive to obtain approval and those therapies never get paid for in the healthcare marketplace? Investors will not put money into them and will not get to the public. Pharmaceuticals will not be the best way to treat every problem. So let's let Industry pay the FDA for advice at market rates, and that will speed up the process of getting things approved, especially if the FDA and CMS work hand-in-hand to ensure approval and payment are the same thing.

If we don't get this part right, we're incentivizing capital to go flow into innovations that fail to get anyone healthier. One of the highlights of the Breakthrough Therapy Act was the four-year time clock because, in exchange for accelerating payment, they have four years to demonstrate cost-effectiveness, after which the payment gets yanked if it’s not good enough. This is a good idea. The more we tie meaningful reimbursement to better and more cost-effective outcomes, the better it is for the budget and the population's health.

Thank you for agreeing to take on this challenge. None of this will be easy, and all of it will matter. If all else fails, you can set up a phase 3 camp at Burning Man and make the FDA fun again.

Don't Forget…More Enforcement

P.S. Currently, the Bad Ad Program only applies to drugs, not devices. Change that. It must also apply to devices. There's going to be a lot of bad behavior in the industry. If the hammer doesn't come down, open up the floodgates for approval on bad behavior. Make sure you open up enforcement on fraud, waste, and predatory behavior so that people worry about going to jail realistically if they're going to pray on the vulnerable public with lies. More approval and more enforcement, please.