Dangerous $30,000-Per-Dose-Drug Approved in Record Time
Meanwhile, in March 2011, the FDA's Center for Biologics Evaluation and Research approved a drug called Yervoy™ (ipilimumab) to treat advanced melanoma, based on data that the drug extended survival by about four months.
Yet it has potentially life-threatening side effects that are so severe that the FDA required manufacturer Bristol-Myers Squibb to place a boxed warning on its prescribing information and documentation.
The side effects relate to immune-mediated adverse reactions due to T-cell activation and proliferation, including enterocolitis, hepatitis, dermatitis, neuropathy, and endocrinopathy.
Still, Yervoy™ was approved in record time under priority review in just seven months. "I'm a huge fan of anything that can help patients," said Gulfo. "But the data are that, while there were some great responders, overall ipilimumab (Yervoy™) added just four months of life. Four months of the least productive, lowest quality, and most expensive life." Yervoy™ costs about $120,000 for four doses. That equates to $30,000 dollars a month of life riddled with horrific side effects, though use of Yervoy™ in earlier-stage melanoma patients might eventually prove to be more cost effective
"Detect melanoma at the earliest, most curable stage," noted Gulfo, "and you'll have forty years of high quality life! One of the massive ironies here is there is a tremendous disconnect between the FDA's laudable goals and what is happening as they stymie innovation that could save lives," Gulfo said. "It's almost as if the FDA didn't get the memo."
Bureaucratic Resistance Exports Innovation Overseas
Gulfo blames the delay on what he calls "innovation inertia," fostered in part by the FDA's long history of turnover and unstable leadership. In the last nine years, the beleaguered agency has had four commissioners, dotted by excessive periods of no leadership in between posts. "Yes, continuity is a big problem at the FDA," said Gulfo, "and it is worse on the device side." In February, FDA's Center for Devices and Radiologic Health (CDRH) Director Dr. Jeffrey Shuren testified before the Subcommittee on Health of the Committee on Energy and Commerce that high rates of turnover were affecting the center's ability to efficiently approve product applications.4 The result is stymied innovation and unnecessary delays in the availability of life-saving products for the public.
Just weeks before, FDA and CRDH launched the "Innovation Pathway" program, "a priority review program for new, breakthrough medical devices designed to encourage cutting-edge technologies among medical device manufacturers," according to an FDA press release.5 The program is meant to "accelerate the development and regulatory evaluation of innovative medical devices."
"So here you have another irony," said Gulfo. "We have just what the innovative program is stated to be looking for and it is right in front of them."
While the FDA appears to want to fuel innovation, the reality is that it is more comfortable approving a toxic, late-stage cancer drug—a familiar model in drug development—than approving a safe, non-invasive diagnostic device because it is novel. "Yervoy™ is a pretty common story in cancer drug approval," said Gulfo. "Drug developed for late-stage disease shows some reasonable benefit. In fact, that is every story in cancer drug approval. But we are a breakthrough."
It is this apparent aversion to progress and bureaucratic nitpicking that is causing patient groups, industry groups, and investment groups to sound the alarms that FDA's policies and procedures are burdensome, unclear, and threatening medical innovation itself. "Venture capitalists are openly saying they won't invest in companies if trials are done in the United States because they don't want their returns to be based on an FDA decision," said Gulfo. "So innovation is leaving America. Not even the big companies are doing their innovative work here."
Earlier this year, a report by investment house PriceWaterhouseCoopers showed that medical technology innovation, long centered in the United States, is moving offshore and that US consumers could eventually be the last to have access to innovative medical technology.6
A second survey of more than 350 medical device development experts sponsored by the Institute for Health Technology Studies at Northwestern University found that two-thirds of small medical device and diagnostic firms look to Europe for their first regulatory clearances.7
Not surprisingly, MelaFind® has already been approved for use in the European Union. (See Sidebar.)
"We are a test case. Everybody is watching this. We hit every endpoint and we hit every part of the innovation program. If FDA doesn't approve this—we say innovation is leaving, investment is drying up—it's all gone," said Gulfo. "If this doesn't get approved, it's the death knell. This case is much bigger than just MelaFind®. I believe a whole industry is at stake."
One American dies of melanoma every hour, yet it is virtually 100% curable if detected in time. Owing to flawed methodology, dermatologists may be missing up to 30% of curable melanomas.
An application was filed in June 2009 for MelaFind®, a completely safe, advanced optical diagnostic device that can help diagnose early melanoma with 98% accuracy. Citing concerns over "safety," the FDA is still reviewing the application for this lifesaving technology, while fast-tracking approval for ipilimumab (Yervoy™), a $30,000-per-dose, largely ineffective melanoma drug with potentially lethal side effects.
MelaFind® was approved by the European Union in September 2011, while the technology languishes here in the US thanks to the FDA. Numerous reports show that FDA bureaucracy and bias are sending other lifesaving medical innovations like MelaFind® overseas and out of reach for most Americans.
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