Safe and Effective

An FDA advisory panel recently approved atypical antipsychotics for use for children, clearing the way for these drugs to be approved formally by the FDA in the near future. These drugs were determined to be "safe" and "effective" for the treatment of pediatric schizophrenia, bipolar disorder and related other mental conditions. It is pronouncements such as this, I'm sad to say, which broaden the chip on my shoulder for modern medicine. The FDA voted on whether to approve antipsychotics based on reviews of the scientific data, which means the published clinical research. Using words such as "safe" and "effective" are extremely misleading, as much to clinicians as to patients, families and the general public, who may have an interest in the health and well being of our children. As one trained in child psychiatry, I can unequivocally state that these drugs are not entirely safe and not entirely effective, not for children, and not for adults.

Years of experience with these drugs, and a massive NIH funded trial, known as the CATIE trial, which was completed about 2 years ago, have shown that these drugs have high drop out rates due to intolerable side effects or lack of efficacy, and that all of the atypical apsychotics appear to be not significantly safer or more effective than the "typical" or "first generation" antipsychotics.
Indeed, when reading the article further (published in the July issue of Psychiatric News), the author qualifies his original report by stating that the committee found that "the results of the clinical trials met the established criteria for short-term use...despite the known risks."
Meeting criteria for safety and efficacy, as it turns out, is very different from being safe and effective. Safe implies to me that there are no serious risks, or even quasi-serious risks. It implies trustworthiness. Effective implies a predictable effect -- reliability and consistency. It means if you take it, it will work. Any other definition dilutes the meaning of the word.

Yet the CATIE study demonstrated that at least for adults there were many problems with serious adverse effects, including metabolic syndrome, which includes obesity, diabetes, hypercholesterolemia and hyperlipidemia, neurologic effects, including Parkinsonian effects, dystonic reactions, akathisia and permanent neurologic damage in the form of tardive dsykinesia, and various other problems, including cardiac arrhythmia and neuroleptic malignant syndrome, the latter a rare but potentially life threatening reaction to neuroleptic medicines. The drop out rate was so high in the CATIE study that the effectiveness had to be measured in days on the medicine until relapse occurred. In other words, during the study, almost all the patients relapsed-- it was just a matter of how long it took.

The medicines are described as safe and effective for "short-term" treatment of bipolar disorder and schizophrenia, both of which are considered to be chronic illnesses. How useful is it to have Insulin available for the "short-term" treatment of diabetes? Does the committee really think that these drugs are going to be used only for the short term? How can we not avoid concluding from what has been written that children are going to be exposed to the dangerous long term effects of these medications?

I am not happy about anyone -- children or adults -- being exposed to the risks of hazardous treatments, especially when safer treatments may be available, such as homeopathy. What gets my goat however, is less the use of these risk-prone and at best partially effective treatments, than the misrepresentation of these drugs by the FDA, by physicians, by drug companies and their representatives, and by the advertising industry. They are called safe when they are not safe (they merely have met safety criteria, which seem to be consistent with something less than mass extermination of all the research subjects, but is certainly far worse than is likely to be believed). They are considered effective when their effectiveness is often partial, temporary, or absent. True, these drugs can work wonders for some individuals, who inexplicably appear to be insensitive to the side effects, and sensitive to the positive effects, but these numbers are in actuality a small minority.

Are the people promoting these drugs evil or deluded or what? I believe that those responsible for misrepresenting unsafe and only partially effective drugs to the public mean well. They are guilty of over-valuing drugs because of their sincere wish to make available to the public tools of potential use for improving health. To some extent they may be guilty of allowing themselves to be influenced by the pharmaceutical industry, which clearly has a pecuniary as well as eleemosynary interest in seeing these drugs succeed. And these drugs do have some potential value. Unfortunately, by focussing excessively on the potential value and minimizing the potential harm, the FDA, the pharmacuetical industry and the medical establish continue to place those of us who are willing to take their pronouncements at face value at risk for serious health problems. These drugs cost a lot of money and the harm they cause is very expensive too, so this is not a mistake to be taken lightly. Ultimately, it is a betrayal of the trust we have placed in government and in physicians to carefully protect the welfare of the public, and to first do no harm.

We will continue to see more and more drugs and medical devices removed by the FDA or given black box warnings as the overly optimistic and ultimately disingenuous ballyhoo around these medical interventions gives way to a more frightening and sober realism. What a shame for those who have to endure such harm. What a shame to allow it to happen in the first place.