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For Patients, Does The FDA Play Fair?

They have a tough job, those government doctors, scientists, and bureaucrats who are charged with assessing the safety and effectiveness of proposed new medical products. As you know, they rely largely on studies presented by the applicants.

The Food and Drug Administration (FDA) has the power to not approve a new drug or product or even pull it off the market. Right now it is considering limiting or pulling GlaxoSmithKline’s (GSK) diabetes drug, Avandia, because of newly discovered data that it may have caused heart attack in some patients –- data mysteriously not shown in GSK’s own studies. If the drug is pulled it will cost GSK billions of dollars in lost revenue but, from the FDA’s point-of-view, it will be protecting the public. And, after all, there are safer diabetes drugs on the market as alternatives.

The FDA also is careful when considering approving new drugs. Sometimes applications come from big companies, GSK among them, and other times from small, biotech companies, that have devoted years and hundreds of millions of investors’ dollars to develop a useful, safe, effective new drug.

When it comes to the many forms of cancer, in this age of personalized medicine, increasingly the proposed new drug may be a “niche” product. It may help just a small number of people with a rare condition. And, with an aggressive cancer, it may not cure the cancer but only extend life by weeks or months. Plus, if a drug comes to market, it will be expensive. Biotech drugs are hard to develop and tricky to manufacture.

Over the past few years I have wondered whether the FDA was playing fair as it evaluated these niche drugs from smaller companies that have few resources to challenge or appeal action by the FDA. One such “little guy” is a Seattle company called CTI. A few months ago an FDA panel voted against approving a new drug from CTI called Pixantrone, a drug for an unmet need of treating patients with relapsed or aggressive Non-Hodgkin’s lymphoma. The panel, guided by Dr. Richard Pazdur, effectively the “Czar” of cancer drugs in America, said the drug’s study did not meet the agreed-upon endpoint, that it wasn’t effective.

A company spokesman told me they felt betrayed as they had had previous meetings with the FDA to agree upon a smaller sampling of patients than originally proposed and, with that smaller sample, the results were compelling in offering a life extending option to patients who otherwise had none. The company said, with the rare condition and short life span, it was impossible to get the larger sample originally discussed.

I am not a scientist nor a statistician. I can only fall back on what I witnessed with my own eyes and ears about three years ago when I attended another cancer drug advisory hearing shepherded by Dr. Pazdur. As I wrote then, patients got the short end of the stick. Another drug, a “bunt single” for CLL, my disease, went unapproved even though world experts advocated for it. The company went into a tailspin, researchers were laid off and disillusioned, and the type of patients who did well in the trial were denied the drug. My impression then was Dr. Pazdur had too much power and took pleasure in killing off a little guy rather than helping them reach the goaline in an ethical way. I felt patients lost and it sounds like that may have happened again with CTI’s Pixantrone.

Is there just smoke and no fire? Does Dr. Pazdur just have commercial entities expressing sour grapes and is style is only grating but not malicious or does he ruthlessly exercise power Congress never intended for him to have? I lean toward the latter and, in a NY Times article a few months ago, other people did too.

As for Pixantrone, they need to consider an appeal with Dr. Pazdur’s group –- obviously to be pursued as they watch over their shoulder every step of the way – or they might try to fund another trial. That costs hundreds of millions of dollars more and patients die while waiting for a new therapy.

My view is someone with a higher pay grade than Dr. Pazdur or a congressional committee should review his record and the transparency of the process. That would be the right thing for cancer patients who need every edge to beat –- or beat back –- their life threatening disease.

I welcome your comments and wish you and your family the best of health!


*This blog post was originally published at Andrew's Blog*

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