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