And, indeed, the bill’s supporters come from both sides of the political aisle. Haley Barbour, the former Republican governor of Mississippi, for example, has said the bill “offers new hope for patients” while Democratic Sen. Patty Murray of Washington has predicted the bill will “advance biomedical innovation for patients and families.”
Editorials and commentaries in support of the legislation have appeared in such disparate media outlets as the Huffington Post and Fox News. The editorial board of the Minneapolis Star Tribune has called it “worthy legislation” that will “spur the next generation of medical treatments.”
‘Profound safety consequences’
At first glance, any legislation that increases the budget of the National Institutes of Health — a budget that has stagnated in recent years — would seem like a good thing. But, as health reporter Trudy Lieberman points out in an article published Tuesday on the Minnesota-based HealthNewsReview website, other provisions in the 21st Century Cures Act threaten to weaken the standards used by the Food and Drug Administration (FDA) to approve new drugs and medical devices.
And that, as Lieberman points out, “could have profound safety consequences for the public.”
She explains why:
Companies that market “breakthrough” medical devices would be able to submit evidence of safety and efficacy based on sources other than clinical trials, including case histories — the experiences of individuals. In other words, they would not be required to conduct a clinical trial, randomized or not, but instead could submit documentation showing that Joe Blow Patient’s health had improved by having some device implanted in his chest. The reasoning, as [Dr. Rita Redberg, a cardiologist from the University of California San Francisco Medical Center] writes [in a New York Times commentary], is that “the need outweighs the risk.”
The bottom line is that “anecdotal evidence, rather than the scientific studies, could be used to approve drugs and devices,” Redberg says. She believes the legislation may shift the burden of evidence to clinical studies conducted only after a device is on the market, and there are plenty of problems doing that. Such studies are often delayed for years after a device gets the go-ahead, and many are never completed.
The act would also let the FDA rely on data that was submitted previously for a different purpose to expedite the development of certain drugs. Furthermore, it would require the FDA to use a third party to review the safety and efficacy of medical devices when manufacturers make small changes, as they often do. Currently the agency does the review. Will allowing third parties chosen by the manufacturer from an approved list to handle this process open the door to new conflicts of interest?
In an interview Redberg told me, “This will result in a lot of dangerous drugs and devices on the market with no evidence. Allowing case studies as the standard is crazy. It lowers the data standard to non-existent.”
Safety trials: Not worth the money?
As Lieberman points out, the 21st Century Cures Act “did not pop out of the blue”:
The Manhattan Institute, a free market think tank, has been promoting its Project FDA for the last several years in an effort, it says, “to modernize the FDA’s policies and procedures.” (Such think tanks often provide the intellectual ammunition for corporations in their battles with Congress.) The Institute believes the agency “can become a bridge for innovation rather than a barrier to it,” and its work has helped educate members of Congress. The think tank’s work paid off in late January when the Bipartisan Policy Center, an influential think tank founded in 2007 by former members of Congress, announced a one-year effort for a new initiative to modernize the FDA. The Center also released a document, “Innovation for Healthier Americans,” co-authored by Senator [Lamar] Alexander [R-Tenn.], which presented a blueprint for “meaningful reform” of drug development and marketing.
One section about opportunities for improving clinical trials is worth noting. Manhattan Institute health expert Avik Roy argued the “enormous cost and risk of Phase III trials create incentives for researchers and investors to avoid work on medications for the chronic conditions and illnesses that pose the greatest threat to Americans.” Not searching for these medicines, Roy warned, harms health and drives up the cost of care. In other words, the cost of a trial to determine safety and efficacy is not worth the money drug and device makers have to spend. So the solution, presumably, is to do away with the requirement for such trials. This reasoning no doubt has found its way into the 21st Century Cures Act.
A lesson from the 1930s
Lieberman concludes her article with a bit of history.
“In 1933,” she writes, “Arthur Kallet, a founder of Consumers Union, and F. J. Schlink wrote a book, ‘100,000,000 Guinea Pigs,’ which detailed the dangers of patent medicines as well as food and cosmetics, all of which fell under the FDA’s jurisdiction. They argued that corporations often knowingly sell products that may not do what they purport to do or have dangerous side effects and that the FDA had been captured by businesses it regulated.”
“Will further FDA modernization move us back to the time of 100,000,000 guinea pigs?” asks Lieberman. “Are Kallet and Schlink’s conclusions relevant today?”
“We may soon find out,” she says.