The bill is formerly called the 21st Century Cures Act and passed the US House of Representatives on July 10, 2015. Presently, the legislation is winding its way through the Senate.
Its supporters tout the bill as a patient’s best friend in that it will allow for speedy passage of new “breakthrough” drugs and medical devices. Some 250 organizations have expressed support for the bill and note that it will “advance the discovery and development of treatments, strengthen the patient voice in the regulatory environment, increase funding for the National Institutes of Health and Food and Drug Administration, and greatly improve our innovation ecosystem.”
That sounds nice even though it is nebulous in clearly articulating how this bill will actually help patients. What we do know is this-the bill is designed to streamline and quicken drug and medical device approval processes by submitting evidence of safety and efficacy based on sources other than clinical trials, including case histories which could mean the experiences of individual patients.
Even the head of the FDA has expressed caution concerning the legislation. On May 5, FDA Commissioner Robert Califf also said he has concerns that if the 21st Century Cures legislation “is not carefully crafted it could pose a significant risk” for the agency and patients because it could cause drugs and devices to be approved too quickly.
“Innovative therapies are not helpful to patients” if they don’t work or they cause harm, Califf said at the Food and Drug Law Institute’s Annual Conference.
Proponents of the bill, such as US Senator Lamar Alexander, continue to use the term “breakthrough” as if that smooths over the fact that the legislation weakens existing FDA regulations on drugs and medical devices.
The Senate legislation is now 19 separate bills. A reporter, or Alexander’s constituents for that matter, wouldn’t learn much from his committee’s news release that summarizes those bills in the most glowing terms. It described the “Advancing Breakthrough Medical Devices for Patients Act” as a bill “to help anyone whose health could be improved by a truly breakthrough device—it will focus more of Food and Drug Administration’s (FDA) attention and resources and put more senior people at FDA in charge of the review of these breakthrough devices so that they will move more efficiently through the review process and get to patients more quickly.”
Dr. Sanket Dhruva, a Yale cardiologist, unraveled what this actually means in a blog post for The Hill. The bill defines breakthroughs as devices that offer “significant advantages over existing approved or cleared alternatives” or are in the “best interests of patients.” Dhruva argued that those requirements are vague and lack meaningful benchmarks and that “once a device is designated as a breakthrough, the legislation erodes that device’s standards.”
In an interview Dhruva questioned whether a breakthrough pathway is even necessary. “I say ‘no.’ Let the evidence speak for itself. This is more a marketing sales pitch than something that’s truly going to benefit patients.” The FDA already has a process for designating new drugs as “breakthroughs,” and grants more than one-third of requests for the designation. Evidence is also coming in that neither doctors nor patients understand what a breakthrough really means, but that labeling something a breakthrough is powerful stuff and sells a lot of drugs.
Don’t get me wrong, cures are good as are breakthroughs. Yet, calling them such doesn’t make them so. Cures and breakthroughs come about after rigorous clinical trials and scientific evidence, not some Pollyanna public relations campaign designed to help the pharmaceutic and medical device industries sell more products.