The GiveAway Act

The 21st Century Cures Act should be called the 21st Century GiveAway Act.

The Act may not even deliver on its main attraction– the funding of new research– but will instantly definitely give a regulatory boost to a host of special interests, including drug and device makers. As Michael Hiltzik writes in the LA Times, “the 21st Century Cures Act is a huge deregulatory giveaway to the pharmaceutical and medical device industry, papered over by new funding for those research initiatives. The punchline is that the regulatory rollback is real, but the funding may not be—it’s subject over the next decade to annual appropriations by Congress which might never come.”

Derek Lowe, an industry scientist, points out that the Act is based on a fundamental misconception: “I really don’t think that there’s this huge backlog of wonderful therapies piling up behind a big FDA roadblock.”

The bill gives billions to precision medicine and cancer moonshots, but reduces support for less glamorous but far more effective public health measures. As Julia Belluz notes in Vox, a significant portion of the increased funding for research is going to be paid by “cutting $3.5 billion from public health efforts like immunizations and tobacco prevention.”

Among many other slippery slopes, the Act will expand use of real world evidence to make regulatory decisions. As Belluz notes, “Unlike clinical trials, these types of data aren’t very rigorous — they’re just observations about things that are already happening, not experiments with placebo controls. Drugmakers could conceivably find any correlation they want in the data and present them as proof their products work. (The researchers I spoke to said this opens the door for data dredging, and there’s really no way to know whether a drug is safe or effective based on that kind of research.)”

Another repulsive aspect of the act is that it will provide comfort to the sleazy purveyors of untested and unproven stem cell therapies.

Devices will also get a big break, according to Trudy Lieberman in Health News Review: “Some medical devices would get an easier pathway to market, one that is already relatively easy. Overall devices are held to a lower standard than drugs, but the bill gives device makers even more wiggle room by calling for the FDA to ensure that the design of clinical trials for devices designated as ‘breakthroughs’ is as ‘efficient and flexible as practicable when scientifically appropriate’.” As I’ve written recently, the real problem now with cardiac devices is that they are already being approved and used in clinical practice despite a lack of evidence demonstrating benefit. This is one slippery slope that could easily end up as a disastrous mudslide.

Lieberman also writes that the Act supports expanded use of “patient experience data to inform its regulatory decisions,” which means we can expect to see more repeats of the eteplirsen/Sarepta fiasco. It should be no surprise that the Act has received strong support from industry-supported patient groups, she notes.

Summary Level Review

Let me conclude with one seemingly obscure detail that is unlikely to gain a lot of attention but is really quite frightening. The Act will allow the FDA to rely upon “qualified data summaries” to support supplemental indications for a drug. In other words, companies could submit their own own reviews for new indication approvals. Currently FDA reviewers have full access to raw data and they perform their own rigorous analysis of that data. Anyone who has observed the FDA approval process knows that these independent reviews often turn up important issues that might otherwise go unnoticed but that could later spell disaster.

People should not take comfort because the Act specifies that summary data can only be used for supplemental indications of already approved drugs. Remember, many companies first seek approval on the narrowest possible indication. Then, with their foot in the door, they seek to expand the indication over time using data from clinical trials. These broader indications are much more likely to entail difficult clinical decisions. Think, for instance, of PCSK9 inhibitors. Most people support their use in the rare cases of genuine familial hypercholesterolemia. Before these drugs are unleashed on a larger population nearly everyone agrees that we need to see data from the outcomes trials. The idea that this indication would not undergo FDA’s intensive independent review is terrifying.

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