Donald Trump

Trump and How to Speed Up FDA Regulatory Approvals

Slashing the restraints on the agency's slow and burdensome process.

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FDA

President Donald Trump described the Food and Drug Administration's drug approval process as "slow and burdensome" in his speech to a joint session of Congress on Tuesday. He observed that the FDA process "keeps too many advances… from reaching those in need." His solution is to "slash the restraints" at the FDA to speed new drugs to the bedsides of patients. To illustrate his criticisms Trump pointed to Megan Crowley in the gallery, a young woman who has survived Pompe disease which is caused by a genetic glitch that prevents her cells from properly processing glycogen. After she and her younger brother was diagnosed with the disease in 1998, her father John Crowley co-founded the biotech company Novazyme to develoop a treatment. Megan and her brother began taking the recombinate enzyme replacement therapy in 2003.

The enzyme treatment was tested on several dozen patients and submitted to the FDA in 2005. The agency took only 9 months to approve the drug in 2006. That may not seem particularly slow and burdensome, but a new Tufts University study reports more typically that getting a new drug from development through the FDA process takes more than a decade and costs about $2.6 billion. As it happens, Crowley is head of Amicus Therapeutics which has developed an enzyme replacement treatment for Fabry Disease. In November, the FDA rejected its initial submission for approval and ordered the company to conduct further clinical trials. Interestingly, the Amicus' new drug was approved by European Union drug regulators in May, 2016.

Another way to think about the development of the Pompe treatment is that it took three years for the drug to get FDA approval after Crowley had begun dosing his children with it. If they had had to wait an extra three years for FDA approval, Megan and her brother might well have succumbed to their illness.

As I have argued, the FDA should be modernized so that new treatments become available to patients once they have made it through the Phase II safety testing. Patients who choose the new treatments would essentially be enrolled in Phase III efficacy trials. This would drastically cut the time and the expense it takes to get new medicines to people.

I am not alone in urging this reform of the drug approval process. In a February 14, 2012 Wall Street Journal op-ed, former FDA Commissioner Andrew von Eschenbach argued that "after proof of concept and safety testing, the [new therapeutic] product could be approved for marketing with every eligible patient entered in a registry so the company and the FDA can establish efficacy through post-market studies." Elsewhere von Eschenbach pointed out this FDA reform would mean that new drugs could …

…come to market after promising early-stage research in targeted patients, with appropriate post-marketing studies required. Payers and patients would be the ultimate judge about the quality of the product, and companies could learn from the experience to develop superior products if needed.

Companies would still be liable for unforeseen side effects, but patients and doctors would be warned—through the drug's labeling—that the product had been approved based on promising but provisional research.

Gradually replacing or reducing dependence on Phase 3 trials with smaller, faster adaptive trials and post-market surveillance would have a positive impact on medical innovation and the U.S. economy….

To head the FDA, Trump is reportedly thinking of nominating venture capitalist Jim O'Neill who argued in 2014: "We should reform [the] FDA so there is approving drugs after their sponsors have demonstrated safety—and let people start using them, at their own risk. Let's prove efficacy after they've been legalized." Sounds good to me.

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11 responses to “Trump and How to Speed Up FDA Regulatory Approvals

  1. I don’t particularly like the idea that “Companies would still be liable for unforeseen side effects”, in that the whole point of early adopters is that there are plenty of unknown unknowns. If you sign up for an experimental drug, you should get up to date access to all progress reports as fast as they come out — unfiltered, raw, scare-you-to-death reports. Maybe make sure your doctor or other medical contact for the experimental drug gets them too, to explain them to you. But liability? Not unless they hid it from the early users.

  2. “Companies would still be liable for unforeseen side effects.”

    There is SOMETHING WRONG with that.

    1. SR&C & w: I understand your concerns – but the requirement would encourage companies to look really hard for side effects – thus revealing to patients “foreseen” side effects that would presumably protect the company from liability since they would have been disclosed to patients and physicians. Otherwise, companies might be tempted to not look for side effects that could reasonably have been “foreseen.”

      1. No offense, Ron, but I call a bit of bullshit or soft pedalling on this. The full statement, including “but patients and doctors would be warned — through the drug’s labeling — that the product had been approved” makes it more apparent that the idea is just to be seen moving the goal posts rather than actually making the drug, information, and/or treatment available. Still essentially involving the FDA in everything down to the labeling process in order to make sure doctors and patients don’t knowingly do something that they all consent to.

      2. Thanks for responding. Yeah, it’s a tricky problem. I buy insurance (I own a company) for things that will never happen but I am uninsured for many things that would. That’s just just the way it is. Risk managers are probably spot with widely used auto insurance and a big database, but go stray away from that and it’s all a joke.

      3. That’s a real problem, and probably only enhanced by letting in early adopters. The only solution I can see is back to the market (as usual) — full disclosure to the public (not just those who sign up) on all the testing so far, all the internal memos on all the testing avoided as being too early, etc.

        But of course, the usual problems come up. Without whistle blowers, it’s all too easy for private chats and hidden communication channels to hide nasty problems. On the other hand, this is a problem now too, so it wouldn’t be any worse than today.

      4. Just always list disability and/or death in the known side effects. We told you. You knew.

    2. Scarecrow beat to the punch and attempted to explain it.

      Bullshit. Let’s play 5 card stud where every card in my hand is wild.

  3. Hey, Ron, will you or anyone at Reason ever advocate actual health care *freedom*, where I can buy medicine/tests/health care from anyone who wants to sell them to me without having to pay a pound of flesh to the Medical Mafia and their government enabled rent seeking?

    You do realize that routine medical care is primarily an information technology, and would be getting geometrically cheaper like all medical technologies, and would probably be free on on cell phones by now but for government enable rent seeking?

    It really is pathetic that Reason will advocate for my freedom to purchase heroin, but not simvastatin.

    Yet another case where Reason’s “Free Markets” means “Corporatist Freedom to Make Profits”, not “Freedom for Peasants”.

  4. And can it be easy to get medicine that is approved and has been used for 2000 years ?!!! I.e. Opiates?!!

  5. Except a lot of drugs pass phase II and then toxicity is discovered in phase III (where the enrolled patients increase). This would mean drugs would get to market marginally faster, but be far less safer (and virtually no promise of efficacy). This can have all sorts of unintended effects (side effects if you will) such as doctors being more hesitant to recommend newer medications (actually slowing their use in the market) slowing the replacement of older less effective medications, an increase in cost of drugs as they are being pulled from the market instead of phase III (meaning companies are advertising more drugs that get pulled and are held liable for drugs to the populace that would otherwise have dropped in phase III) etc. Essentially, this sounds like a good idea for people who don’t have a good sense of the process but to those who know the drug review process see this as a means of getting more snake oil to market.

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