Unbiased Analysis of Today's Healthcare Issues

Off-label drug use is…good?!?!

Written By: Jason Shafrin - Jun• 29•06

I claim that ‘off label’ drug use is good.  But according to a recent Forbes article “the practice is potentially risky, since three-quarters of these off-label uses lack scientific support.”  The article (“Off-label…“) claims that this practice is very common and that one in five prescriptions for drugs are for an off-label use.and effective.  If efficacy is shown for one disease, the company must re-run some of their trials at great expense in order to prove that the drug is effective for other diseases.  Because of this, pharmaceutical companies prefer to rely on their marketing team to explain alternative uses to physicians instead of obtaining explicit FDA approval.  Waiting for additional FDA approval would mean that many patients will go without treatment while the drug application is stuck in the bureaucratic process.    

A better alternative would be for the FDA to only require a drug to be safe.  In the UK, safety–but not efficacy–is the current standard for prescription drug approval.  This way, off-label uses would be out in the open.  Private agencies–such as Underwriters Laboratories–could certify that the drug is effective and an open debate could be held about which drug is the best, instead of having these decisions made behind closed doors.

One drawback to requiring only safety is that physicians may begin to over-prescribe some medicines.  If the drug is safe, but ineffective, the provider may decide to recommend it to patients simply to earn more cash.  The principal-agent problem would be strong since the doctor would not fear significant adverse effects since the drug was already considered safe by the FDA.  Thus, medical costs and unnecessary care could increase.  

Overall, however, I believe requiring safety only and allowing the market, physicians, and patients–instead of bureaucrats–to decide which drugs are the most effective will lead to a better healthcare system

You can follow any responses to this entry through the RSS 2.0 feed. Responses are currently closed, but you can trackback from your own site.

No Comments

  1. Curious to know Healthcare Economist’s email address.

  2. Jerry Tsai says:

    With respect, and revealing that I have trained as a biostatistician and as someone who could benefit financially from drug approval work, I disagree. We already have prominent examples of how drugs not assessed for efficacy (or effectiveness) would fare in the market — the substances known as “complementary or alternative medicines”.

    Unfortunately, people in general are quite gullible and are generally willing to believe ridiculous health claims for just about anything, whether it is noni juice, St. John’s wort, etc. They spend their money on

    In an ideal society, people would be better educated as to the efficacy of substances they take. But as long as there are plenty of charlatans and snake-oil sellers out there, it is a public good to make sure that drugs are not simply approved for safety, but for efficacy as well.