It's Getting Scarier: Is There a Way Out?

How about the enforcing agencies like the Food and Drug Administration in the United States----do they put in sufficient effort for a long enough period of time when evaluating the safety and efficacy of new drugs? In the recent past, the FDA approved fourteen new drugs, which it was forced to recall later. One of the most glaring of these recalls was the withdrawal of Bayer's Baycol because it was thought likely to have been the cause of 31 deaths, probably due to rhabdomyolysis. Not too long ago, Pfizer was forced to give up on its extremely lucrative anti-diabetic product Rezulin. It's scary alright because it seems to be happening more and more frequently. Is this because these companies are trying out riskier products, are faking their studies, are rushing their products to the market too quickly, or just don't care as long as they can get by? My own experience doing drug research for safety and efficacy, which was carried out under the tightest of security conditions, does not support these contentions, but that was a long time ago. Is it really a whole new ball game now, as some critics insist, or are we simply getting more aware of the dangers involved while the new drugs are becoming more complex, more sophisticated, more difficult to evaluate over the relatively short time period allowed for side effects to appear, and certainly a lot more expensive to bring to market? Let's face it, when you're the one who's sick or dying, you're not too likely to quibble about a statistic of 31 people who may have died out of a total of perhaps 10 million that benefited. It's especially disheartening to learn that a medication, which is literally saving you from suffering or even dying, will no longer be available.

Scott Gottlieb, a monthly contributor to the AMNews, in his February 11, 2002 "Street Smarts" makes some astute observations about the likelihood that there may be a new trend developing. He notes that the FDA is taking longer to okay new drugs. Consequently, pharmaceutical companies realize that their profits will suffer. We all know that the tech market has been under siege recently but most of us have been unaware that the Nasdaq's Biotechnology Index was also down about 13% at that time, perhaps in large part as a result of those delays in getting to market. Gottlieb puts his finger right on the mark when he points to the fact that there are more new drugs designed to combat rarer diseases among more diversified populations. Rare side effects are bound to arise in the long run. How, in heaven's name, can a company afford to carry out the trials necessary in order to uncover all the potential remote risks? The result is that finding these risks must be left to the prescribing physician, but the FDA believes that it's just not working. As a result, new drug approvals are harder to come by and more approved drugs are being withdrawn.

Dr. Gottlieb goes one step further. "What can the FDA do----?" he asks, then answers, "New technology---pharmacogenomics-can mitigate their efforts." Suppose we take a peek at his reasoning. After all the miracles we read of every day about what can be done with DNA, heck, it seems all our scientists have to do, now that they've cracked the human genetic code, is to locate that little "snip", or single nucleotide polymorphism, which is out of it's proper sequence on the chain, and voila, they have the answer as to whether our pill popper will end up with a rash or rhabdomyolysis or what have you. Now isn't that simple? Well let's hope so because a few of those companies such as Genaissance, Variagenics, and Sequenom are spending a great deal of money and time looking for those little "snips". Unfortunately, however, the stock market doesn't always seem to be betting on them these days. You had to pay $30 for a stock of Genaissance a while back, but if you were a fortuneteller, and waited a bit, you could have picked up the same stock, shortly afterward, for around $4. Variagenics did even worse at that time, going from about the same $30 to about $3, and Sequenom really tanked starting at a fabulous $100 and sinking to $7.45. Shareholders for the latter two were somewhat put out and got busy suing. However, Dr. Gottlieb remained an optimist because he believed that in the end DNA research would win out. By storing DNA samples from patients who had bad reactions and comparing them to those who didn't, they should be able to create snip profiles for screening patients likely to get side effects. There's a good deal more to all this and the future should indeed be bright. Let's just hope that we'll still be around to take advantage of all these potentially miraculous advances. If not, well maybe we can at least live out our later years a bit more comfortably, in spite of having a serious reaction to some new medicine, by suing the "bejabbers" out of them.