Health & Fitness

Cancer drugs and the FDA: Safety vs. saving lives

Dr. Leonard Kalman, deputy medical director and chief clinical officer of Miami Cancer Institute at Baptist Health South Florida, stands inside an MRI suite for breast center treatment. The walls are made of copper. Kalman was giving a tour of the new facility on Feb. 10, 2106.
Dr. Leonard Kalman, deputy medical director and chief clinical officer of Miami Cancer Institute at Baptist Health South Florida, stands inside an MRI suite for breast center treatment. The walls are made of copper. Kalman was giving a tour of the new facility on Feb. 10, 2106.

One of America's leading cancer experts says many lives could be saved if patients got the best available treatment — and he places considerable blame on federal regulators for being slow to approve desperately needed drugs.

“There are incredibly promising therapies out there,” writes Dr. Vincent T. DeVita Jr., former director of the National Cancer Institute.

“If used to their fullest potential on all patients, I believe we could cure an additional 100,000 patients a year,” he says in his new book, The Death of Cancer, which was written mostly in Deerfield Beach, where he spends the winters.

The book — being heatedly discussed among oncologists locally and nationally — comes as the Obama administration launches a “moonshot” to find cures for cancer, the second-leading cause of death in America, after a decade in which funds for research of the disease have been in decline, when adjusted for inflation.

DeVita blames the Food and Drug Administration as a major cause for patients not getting the best possible care.

“There is an urgency in the cancer field not present in other fields,” he wrote in the book, co-authored with his daughter, Elizabeth DeVita-Raeburn, a science writer.“And if a drug works to some degree, [patients] want and need access to it long before the creaky approval process allows. The FDA refuses to acknowledge this.”

Many patient advocates agree with DeVita, who points to the growing “right to try” movement. In the past two years, more than 20 states — including Florida — have passed bills demanding that terminally ill cancer patients have easier access to yet-to-be-approved cancer drugs.

Still, top local researchers at the Miami Cancer Institute and the Sylvester Comprehensive Cancer Center said their experiences with the FDA have been generally positive.

Dr. Leonard Kalman, deputy medical director and chief clinical officer of the Miami Cancer Institute, which is part of Baptist Health South Florida, said that in recent years, “there have been more rapid delivery of drugs to market, both because “the FDA has heard some of the criticism,” and because some of the drugs now being presented for approval are much better drugs.

There’s at least one local FDA critic, however: Miami urologist Dr. George Suarez. In 2003, he first sought approval for the use of high intensity focused ultrasound for treating prostate cancer — a method that had already been approved in Europe and Japan because it showed good results with considerably less danger of impotence.

Waiting for the FDA to act, Suarez spent a dozen years doing HIFU treatments for several thousand men in the Caribbean, Mexico and Canada. When the Herald wrote about him in 2004, he noted that 80 of his first 100 patients were physicians. His personal investment in HIFU technology will be covered in an article on prostate cancer in The Herald's Men's Health section, to be published on Sunday.

In a recent interview, Suarez said he kept telling the FDA to look at the data on his outcomes, but the FDA insisted on trials comparing HIFU to other treatments. Once, he said, the FDA asked him to compare HIFU to a treatment virtually guaranteed to cause impotence. He found only one person willing to accept the alternative care.

In October 2015, after countless meetings, the FDA finally approved HIFU and Suarez can now treat patients in the United States.

“We’ve been very busy,” Suarez said. He continues to have offices in the Caribbean, but American patients prefer Miami, where he treats them at an outpatient surgical center on Sunset Drive. “It’s been a very rewarding time.”


Suarez may be an exception. In a recent interview, DeVita, 81, speaking from his Broward home, said most doctors are afraid to offend the FDA because they need regulators’ approval for their research. “They can’t hurt me,” DeVita said, because he no longer seeks FDA approvals.

One of his main complaints: The FDA often approves a drug only for a specific stage of a specific cancer, not allowing researchers the kind of leeway that DeVita had in the 1970s when he and colleagues experimented to find a blend of drugs for chemotherapy that led to a cure for Hodgkin’s lymphoma.

Dr. Stephen Nimer, Sylvester’s director, noted that in the past several years, FDA approval of cancer drugs has been soaring at an “incredible” pace. “And these are very good drugs.”

The FDA is “damned if you do and damned if you don't,” said Nimer at Sylvester, which is part of UHealth — the University of Miami Health System. Desperate patients complain about FDA delays while critics object that quick approvals mean the FDA is putting patients at risk — or kowtowing to drug manufacturers.

Both Nimer and Dr. Miguel Villalona-Calero, deputy director and chief scientific officer of the Miami Cancer Institute, said the FDA these days is showing considerable flexibility. “The FDA is very happy to work with you” to design trials to explore the value of cancer drugs, Nimer said.

Villalona-Calero said the FDA is “trying to adapt to new times.” He has been able to “get some exemptions” to treat patients outside of approved drug regimes, and said that drugs showing promise in early trials are now getting accelerated paths to approval. Of the FDA: “They are trying to adapt to new times.”

Still, a continuing issue is that while great new drugs are becoming available, Kalman points out that some of them cost $8,000 to $10,000 a month, and insurers generally won’t pay for them unless they are used in exactly the way the FDA has approved them to be used.


In one case, Villalona-Calero said, a cancer drug approved by the FDA for use only after chemotherapy is now being made available at Miami Cancer Institute for those who haven’t had chemo by enrolling them in a clinical trial, with the expensive drug being paid for by the manufacturer. “It’s worked extremely well,” said Villalona-Calero. The manufacturer makes the drug available in hopes it can later expand its marketing.

Villalona-Calero said “some paperwork” was involved, but “it wasn’t difficult.”

That didn’t impress DeVita, who said that such paperwork can delay or preclude many patients from getting needed treatment. He has seen “some improvements” by the FDA, but “it’s still a very tedious process.”

Several reviewers of DeVita’s book noted that in early January, after it was published, The New York Times reported that the FDA’s approval of cancer drugs has speeded up since 2012, when the wife of FDA oncology chief Dr. Richard Pazdur was diagnosed with ovarian cancer.

Before, the Times noted, Pazdur had been viewed “by many cancer patient advocates as a slow, obstructionist bureaucrat.” Pazdur acknowledged to The Times: “I have a greater sense of urgency these days.” He added that approvals became easier because “the drugs simply got better.”

The FDA responded for this article with an email from spokeswoman Sarah Peddicord: “Increasing access to new cancer therapies is a priority for the agency. In 2015, the FDA approved 14 new molecular entities (NMEs) for cancer drugs,” many of them on accelerated schedules. . . . Our mission at the FDA is to ensure that drugs marketed in this country are safe and effective, and we strive to approve these medications as quickly as possible.”

DeVita wants the FDA to do a lot more. Another of his major complaints: The FDA may approve drug A and drug B for chemotherapy, but using A and B in combination requires a separate study before approval.

DeVita, now a Yale professor, said he’s “horrified” by this requirement. In his pioneering work with Hodgkin’s and many other diseases, the researchers found that the key was to use several chemo drugs in combination, often after clinical experimentation unencumbered by the regulatory process.

Said Villalona-Calero, at the Miami Cancer Institute: “I agree with Dr. DeVita. Once the drug is approved, we should be able to combine them.”

All the cancer doctors interviewed feel encouraged about the Obama administration’s $1 billion “moonshot” to cure cancer — although they’re not sure how much it will mean.

“For the last 10 years, the field has been starved for resources,” DeVita said.

Nimer at Sylvester welcomes the discussion, but “most doctors aren’t going to refer to cures for cancer,” since researchers know cancer is really many diseases with myriad treatment options, and no silver bullet is going to permanently fix all these diseases. In such a context, $1 billion doesn’t go far.

DeVita said that when he referred to 100,000 more people who he believed could be cured each year, he was citing American Cancer Society reports that include all of those who don’t now get proper treatment.

One example: Nimer pointed to a recent New York Times report that less than half of patients with ovarian cancer are getting the recommended care.

Still, Nimer added: “The one message to take away is this: “We’re making incredible progress against cancer.”