Big Pharma is trying to ram two sweeping overhauls through Congress, the “21st Century Cures Act” and the “Reciprocity Ensures Streamlined Use of Lifesaving Treatments Act.” (The latter is so obnoxiously named that it is typically referred to as the Cruz-Lee proposal.) Both of these bills include good provisions, like increasing funding for the National Institutes of Health and allowing the Food and Drug Administration more flexibility to review drug approvals in other countries. However, the bills also include truly terrible provisions, like instructing the FDA to abandon the use of clinical trials and giving Congress the power to approve drugs on its own.
In favor of the bills, there’s economist Alex Tabarrok, who argues there’s an “invisible graveyard” of patients whose lives could have been saved by drugs that were not yet approved. In opposition, there’s medical school professor Dr. Aaron Kesselheim, who argues there’s no evidence the FDA has blocked innovation that actually helps patients.
Tabarrok calls that “laughable” and “magical thinking,” apparently unlike his “invisible graveyard.” He lays down a challenge: “We live in a world of tradeoffs. Let’s debate the tradeoffs.”
Let’s start with some basic facts:
- The FDA approves the majority of drug applications filed, and most of them are already through “expedited” programs that are meant to be the exception, not the rule.
- Only 10% of new medicines have been shown to be superior to medicines that were already available.
- More than 85% of cancer drugs approved on the basis of “surrogate markers” — one of the key changes proposed by the 21st Century Cures Act — failed to improve overall survival. (For more about the problem, see this article.)
- Medical device manufacturers, who already have their own version of “21st Century Cures” that allows them to sell medical devices before their safety and effectiveness are proven, routinely game the system by not doing the post-approval studies they promised. Only 13% of postmarket studies are completed within 5 years after FDA approval.
It would be nice if we had a crystal ball that allowed us to know which new drugs were actually going to make a difference in patients’ lives. But we don’t have that. For all the hubbub about “precision medicine” and the like, there really is no good way to tell if a drug will be effective, much less what side effects it may create, until we do clinical trials with the “primary endpoint” being an actual improvement in patients’ lives.
As David Gorski argued, while criticizing the way the 21st Century Cures Act would water-down the proof of efficacy needed for FDA approval,
A homeopath would love this provision, and, I’m sure, so would drug companies. Why bother with the time, bother, and expense of those pesky clinical trials to get your drug approved for additional indications, when you can rely on clinical experiences based on therapeutic use, uncontrolled observational studies, or registries instead? If I were the CEO of a pharmaceutical company, I’d love it. Indeed, the one thing this provision most definitely does not do is to speed effective treatments to patients. Rather, it smacks of being a payoff to pharmaceutical companies.
For some reason, this is a controversial statement, but corporations exist to make money. If we’re discussing the “tradeoffs” of our pharmaceutical regulatory system, we have to start by acknowledging two key facts. First, drug companies routinely produce “new” drugs that don’t do any better than drugs already on the market. Second, when it comes to “life-saving” treatments, drug companies typically produce drugs that don’t help at all.
As I mentioned a month ago, when a drug is shown to be useless after being on the market, there’s usually nothing anyone can do about it. The patients who were cheated – and sometimes given a useless medication in lieu of a useful one, thereby leaving them worse off – typically can’t sue. The health insurers and health plans that were ripped off typically can’t sue either. The drug company just takes the money and runs, funneling it back into development of new worthless drugs. A robust FDA is the only thing we have to keep drug companies from making billions of dollars by selling us ineffective pills with horrible side effects.
The only “magical thinking” I see in this debate comes from Tabarrok, who simply assumes that every drug a pharmaceutical company wants to sell is going to help patients, when all the evidence is to the contrary. In the pharmaceutical industry, “snake oil” isn’t the exception, it’s the rule.