September 13, 2004

Off With Their Labels!

TCS: Tech Central Station - Pill, Anyone?

A blog entry in the defense of off-label prescription of cognitive enhancer drugs.

This might very well be the next big medical battlefield. On one hand, off-label prescriptions enable exploration, customization to patient needs and new treatment for disorders. On the other hand there is room for abuse, prescriptions based on bad or weak science or pharma advertising. And that is just the normal debate about off-label usage (a quick google suggests that there are several anti-off-label campaigns out there).

Given that governments everywhere are running scared of escalating health-care costs it is not inconceivable that a crackdown on off-label use might occur simply for economic reasons. In a finite budget, any new option that will be requested by patients or doctors will be a new cost, and either force an enlargement of the budget or competition with earlier options. In the ears of many health-care politicians technological advancement is bad, no matter the good effects on patients. Just look at how the pharmaceutical industry is being increasingly seen as evil and manipulative at the same time as health budgets get more stretched. Coincidence?

Add to this the bio-conservatives, who strongly seek to limit medical enhancement (be they human nature defenders like Fukuyama or anti-modernists), and it seems plausible that policymakers might be tempted to add further restrictions on medical freedom. It is good for the budget, saves the human soul and might prevent some maltreatments. No problems at all.

The only thing lost is the flexibility. And the ability of the medical profession to learn things in a bottom up manner. In a world where only approved treatments can be done, only those who can afford to get their treatments approved will develop new ones. And given the rising costs of testing and bureaucracy, that means an even greater concentration in big pharma. Limiting off-label prescriptions will in the long run slow medical advances and limit the quality of life of many patients.

While it is not clear how many patients who are hurt yearly by bad off-label usage, it seems likely that they are far fewer than those who are helped. Limiting off-label usage by X% means many more lost quality-years from the helped group than gained years from the damaged group.

A better approach is of course to improve detection of side-effects, to enable better informed consent from the patients and better reporting of the results. This is a matter of information technology and medical practice rather than regulation. By stimulating these areas many of the basic problems with off-label usage can be dealt with. Better information sharing and transmission makes experience cheaper. In fact, it would even help put many of the fears of the dehumanizing effects of medical enhancement to the test.

What we need is a dynamist approach to off-label usage of medicine.

Posted by Anders at September 13, 2004 10:52 PM

Good points. Just allow me a moment to air one of my complaints about the debate over off-label prescribing. In this debate, the word "approved" generally is used as shorthand for "approved by the US Food and Drug Administration for a particular purpose." When someone says "the drug is not approved for that use," what they mean is "the US FDA has not explicitly approved that drug for that use."

The implication is that, somehow, the US FDA is the final authority on the matter. They are not. Additionally, the implication is that the lack of such approval means something. All it means is that the manufacturer of the product has not spent the time and money to prove to the satisfaction of the FDA that the product is safe and effective for the purpose in question.

Once a drug has gone generic, it no longer makes sense for a company to make that investment. Therefore, if a new use is found for an old drug, it is unlikely that anyone is going to spend the money to get approval. The lack of that approval cannot be taken to imply that the drug in not safe or effective for the new purpose.

No one gave the US FDA the authority to have the last word on the appropriateness of a particular drug for a particular patient. In fact, it was a well-considered policy decision for the FDA not to have that authority. The authority resides in the collaborative effort of physician and patient.

Posted by: Joseph j7uy5 at September 14, 2004 07:50 PM

I agree that flexibility in pharma development and application is desireable, but aren't you minimizing the possibility for both abuse and mis-application by incompetence?

The labelling and communication of efficacy and side-effect profile is a complicated business, possibly too complicated to be left to government regulation, but it certainly isn't a simple problem. And increasing the amount of 'nonstandard' uses and/or new uses and new drugs means that the problem becomes more complicated.

Certainly there is the potential for great assymetrical gains, but how can we ensure they remain greater than the losses? Saying that it's an information technology problem doesn't mean that it's trivial or possible.

Do you have a pet theory for some public database of drug use, profile, and collabrative reporting?

Posted by: Justin Corwin at September 15, 2004 11:09 PM

I am seeing significant strides in the area of what is called "mass customization", which is moving from online ordering of custom tailored clothing to nutriceuticals, customized in powder form to your personal mix, based on your lifestyle and/or an analysis of your DNA. Prices for DNA/lifestyle profiling are running a few hundred dollars now.

What I forsee next is neurochemical profiling via either DNA, MRI, lifestyle, spinal tap, or a combination of these, which will result in a neurological profile to provide custom mixed cognitive enhancement drugs, available as pill, powder, patch, or inhaler.

Posted by: Mike Lorrey at September 23, 2004 02:13 AM