True drug breakthroughs are few and far between. In a good year, the FDA might approve a handful of new medications that are truly innovative. But teaching old drugs new tricks is becoming increasingly common.
Pharmaceutical companies have discovered a huge business in “off-label” prescribing. These are unofficial uses for approved medicines.
Once the FDA clears a drug for a specific purpose, doctors can prescribe it for all kinds of other problems. For example, Neurontin is an anticonvulsant developed to treat epilepsy. FDA has also given the drug a green light for relieving pain following shingles.
Neurontin is widely prescribed, however, for other medical conditions including persistent hiccups, migraines, restless legs, nerve pain (peripheral neuropathy) and bipolar disorder (manic depression). In fact, more prescriptions are written for these unofficial uses than for epilepsy.
Propranolol has been used for decades to treat high blood pressure and other heart problems. But some physicians prescribe it for stage fright or performance anxiety, even though it has never been approved for such purposes.
More recently Provigil was approved to treat daytime sleepiness due to narcolepsy. This uncommon sleep disorder causes people to fall asleep without warning in the middle of the day. Provigil is also being used by truck drivers or military personnel who simply want to stay alert and push their bodies beyond normal limits.
The antidepressant Desyrel (trazodone) is also used off label to treat insomnia, aggressive behavior, panic disorder, and withdrawal from cocaine or alcohol addiction. How well the drug works for such conditions remains somewhat controversial.
In fact, that is one of the biggest problems with off label prescribing. Frequently physicians learn about such applications through informal channels.
A colleague or friend may mention an anecdote or a small uncontrolled study.
The research behind off label uses is not always rigorous. Appropriate doses and potential side effects may differ from those found in the official drug label.
Doctors are allowed to prescribe medicines for any use they think will be helpful. Drug companies are not supposed to promote these unapproved uses. Nevertheless, there have been whispers of scandal. The maker of Neurontin, for example, has been accused of paying prominent physicians to talk up the off label uses of the anticonvulsant.
Sales of Neurontin soared. But the research that was eventually conducted didn’t support some unofficial practices. Two studies demonstrated that the drug was no better than placebo in treating bipolar disorder. In fact, some patients on Neurontin did worse.
Opinion leaders paid by drug companies can influence their medical colleagues to prescribe medicines for unproven purposes. But without objective research, it is impossible for physicians to know if off label uses of medications will live up to the claims and do more good than harm.
Until the FDA figures out how to harness the potential power of unapproved drug use, patients should ask their doctors to verify that there is good science behind the pills they are prescribing.