“Thousands of lives lost”? Why calls for faster drug approvals need more scrutiny

A Times photo of Dr. Pazdur at his wife Mary's funeral. The paper appears to have received unusual access to the agency for its reporting on this piece.

A Times photo of Dr. Pazdur of the FDA at his wife Mary’s funeral. The paper appears to have received unusually liberal access to the agency for its reporting on this piece.

Last week the New York Times published an interesting hybrid of a piece that I found in the Health section, although its header was actually “Politics.”  On its face, it is a sad human interest story of a cancer nurse who died of cancer, with the ironic twist that her husband is the long-standing head of the Food and Drug Administration’s (FDA’s) division on cancer drugs, Dr. Richard Pazdur.

I,  too, was saddened by the story, and could only begin to imagine the pain and frustration of the couple.

So why was the story labeled as “Politics”? Its headline gives a clue: “F.D.A. Regulator, Widowed by Cancer, Helps Speed Drug Approval.” Drugs are big business, and the pharmaceutical industry is one of the top spenders when it comes to lobbying politicians and contributing to their campaigns. According to the article, some cancer specialists speculate that Dr. Pazdur’s experience with his wife’s illness contributed to FDA’s faster approval of cancer drugs. Could a newfound “passion to fight the disease” by one man in a large bureaucracy speed up what is supposed to be a scientific approval process? As a doctor who used to work at the FDA, I found that most of the professionals at the agency were dedicated to evaluating drugs as expeditiously and thoroughly as possible. Indeed, the FDA approves most drugs faster than its counterparts in Europe and Canada, with most drugs approved in six to 10 months.

To his credit, Dr. Pazdur cited a number of factors driving the faster approval of cancer drugs over the past few years. While they included his wife’s illness, he also noted considerations such as advances in genetic research and the development of better drugs.

The FDA is just one cog in the wheel of drug approvals, and many believe that wheel can be greased through political influence from other key players, including pharmaceutical companies and Congress. A particularly contentious political battle involving the FDA, in the form of the 21st Century Cures Act, was fought in the House of Representatives last summer and is now under consideration by the Senate. The bill pushes for faster drug approvals, though sacrifices efficacy and safety standards. It will increase drug costs for consumers, while likely lowering development costs for drug companies. Much of the bill in fact favors the interests of the drug and device industries

It’s somewhat unusual for an agency as tight-lipped as the FDA to provide the kind of access that it appears the Times received for its piece. And so I can’t help but wonder if this is part of a media strategy designed to advance the overall message that “speed is good” in drug approvals. Judging from the comments submitted to the Times, that is the message that many people took home from the article. But the “speed is good” message should also be placed in this context: drug companies directly fund approximately 65% of the FDA’s drug approval costs through user fees. For whom is speed better – patients or pharma?

While some consider faster drug approvals a positive for patients, the author of the article, seasoned health journalist Gardiner Harris, quoted two prominent voices who cautioned about approving drugs that haven’t yet been adequately assessed for their efficacy and safety: Fran Visco, President of the National Breast Cancer Coalition, and Dr. Sidney Wolfe of the consumer advocacy group Public Citizen.

I decided to blog about this article because of a particularly bothersome phrase regarding drug approvals: “where every month’s delay can mean thousands of lives lost . . .”

I tried to think of a drug for which this might be true in the last several years. “Thousands of lives lost.” Every month. Some of the drugs approved for melanoma represent significant advances, but it’s still a fairly uncommon condition.

The article makes reference to Opdivo, a drug already approved for skin cancer that Dr. Pazdur and his colleagues approved very quickly for lung cancer. In a blog post last month for HealthNewsReview.org, Dr. Michael Wilkes put its benefits and risks into perspective: patients lived an average of 90 days longer compared to standard treatment, although many experienced side effects so severe that some stopped the drug, and at a cost of $150,000 for the initial treatment, then $14,000 a month (with not all insurance companies covering the cost).

The article also duly notes that Ms. Pazdur received experimental cancer treatments—drugs that had not yet been approved by the FDA–through two different mechanisms. One through the expanded access “compassionate use” process and another through a clinical trial. These both illustrate that patients can gain access to drugs prior to approval, which in my view further undermines the bothersome statement above with regard to thousands of lives being lost. Unfortunately, neither treatment helped her, and she experienced dreadful side effects with one.

While advances are indeed being made in drugs to treat cancer, of the 54 cancer drugs approved by the FDA over the past decade, 74% had no proof they extended life and few had proof they improved quality of life, as reported last year by John Fauber and Elbert Chu. Improvements in treatments are often incremental, and understandably, the risk-benefit calculus may be different and/or more uncertain when it comes to approving drugs for life-threatening cancers. But, a number of researchers have said, “Most approved cancer drugs provide little gain for patients.” They describe the low standards used for the clinical trials for many cancer drugs, resulting in an overall median survival gain of only 2.1 months for the 71 drugs approved between 2002 to 2014 for solid tumors.

The financial costs of these drugs can be tremendous, as can their impact on quality of life. For example, bevacizumab (Avastin), which was withdrawn from the market for the treatment of breast cancer because it neither increased survival nor improved quality of life, included side effects such as bleeding, heart attack, heart failure, and perforations of the nose, stomach and intestines.

High costs and lack of access to good health care are much more likely to kill people than the FDA’s deliberate review process. Many people, despite having insurance, put off going to the doctor or filling prescriptions because of costs, as deductibles and co-payments continue to rise, and a significant number of claims are denied by insurance companies.

We can have all the drugs in the world, but if we wait too long to have that pain or that lump diagnosed, or if we can’t afford the care that doctors recommend, what’s the benefit of speedy approval?

Susan Molchan, MD, is a psychiatrist in the Washington, DC area and one of our regular editorial contributors.

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Comments (3)

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Dr. Peter Capell

January 7, 2016 at 5:08 pm

Well done- thoughtful and on target!

Leslie Taylor

January 7, 2016 at 7:36 pm

Dr. Molchan, thanks for continuing to emphasize the benefits of prevention, taking care of oneself, early diagnosis, as well as discussing the need for cost (both financial and physical)/benefits to be addressed, especially with respect to cancer drugs.

Marc Beishon

January 9, 2016 at 5:02 pm

For me the key quote in the piece is:

“Instead of having physicians assess drug toxicities, which they do a terrible job of, we’re going to start asking patients to assess them,” Dr. Pazdur said. “It’s a huge issue.”