False cures in the Cures Act

Congress Just Quietly Handed Drug Companies a Dangerous Victory

By Adam Gaffney
New Republic, December 14, 2016

Last week, the Senate followed the House in passing the 21st Century Cures Act. Though this bill has been lauded by liberals for providing much-needed funds for medical research, its real impact will be elsewhere. Whereas drug approval traditionally required the demonstration of real clinical benefit in a randomized clinical trial, under the Act drug firms will increasingly be able to rely on flimsier forms of evidence for approval of their therapies (incremental steps in this direction, it is worth noting, have already occurred). The Act, by reconfiguring the drug regulatory process, lowers the standards for drug approval — a blessing for drug makers, but an ill omen for public health.

In the Senate, a grand total of five senators — including Bernie Sanders and Elizabeth Warren — voted against it. The media, meanwhile, has for the most part done a poor job dissecting its actual contents. As a result, few now realize how detrimental the act is likely to be for drug safety, or appreciate the mix of conservative ideology and pharmaceutical industry greed underlying the longstanding campaign that brought it to fruition.

The 21st Century Cures Act allows “the potential use of real world evidence” to support the approval of a novel indication for an approved drug and “to help to support or satisfy postapproval study requirements.” That sounds rather reasonable: who could possibly oppose the use of “real world evidence”? The problem, however, is that “real world evidence” actually means here “really bad evidence.” The law defines “real world evidence” as “data… derived from sources other than randomized clinical trials,” which is to say uncontrolled observational data.

This is perhaps the bill’s most dangerous clause Randomized clinical trials are — in the vast majority of cases — the only way to know if a drug actually works, as observational studies can be notoriously unreliable in determining the efficacy of drugs.

Randomized clinical trials are not perfect, especially when they are conducted by pharmaceutical companies with a vested interest in distorting trial methodology (or hiding trials results when they don’t go as planned). But they are the best we have when it comes to assessing drug efficacy. With this new provision, however, randomized clinical trials can — in some instances — be skirted altogether, leaving both doctors and patients forever in the dark as to a drug’s actual therapeutic utility for a particular indication. Their profitability, in contrast, will be rather more clear.

Adam Gaffney is an Instructor in Medicine at Harvard Medical School and a board member-elect of Physicians for a National Health Program.

More on this topic by Trudy Lieberman:

And winners and losers by Sheila Kaplan:



By Don McCanne, M.D.

We did not have to wait for the incoming, anti-government administration to take over. Yesterday President Obama signed the 21st Century Cures Act. It will reduce government oversight of drugs - allowing on the market products that may not be effective and might even be dangerous.

Yes, there were some good things in the legislation, but some of the appropriations were deferred until future years - under control of a Congress that has already demonstrated that it is quite willing to refuse to appropriate funds for existing programs. This could be an empty promise.

The politics is depressing. As the concerns of Sen. Warren and Sen. Sanders were beginning to gain coverage in the media, the Congressional leadership called in Joe Biden to give an emotional appeal on behalf of his cancer moonshot that was included in this legislation. And to top it off, one section of the legislation was renamed after his son, Beau Biden, who tragically died in his prime of brain cancer. How could a member of Congress be so heartless as to vote against this legislation?

In reality, those members of Congress voted for countless deaths and suffering that will occur from defective drugs and devices. That is truly heartless. They also voted for an open conduit of money flowing to the pharmaceutical and medical device industries - our money, whether through taxes, insurance premiums, or direct consumer purchases. Heartless greed, pure and simple.

Wait until the billionaires are in charge of our government, not they they don’t already own it. We likely haven’t seen anything yet.