FDA-Approved Targeted Drugs Are Ripe for Off-Label Marketing

The FDA at times gives its stamp of approval to drugs that only work in small subset of the patient population. For example, a few days ago, the FDA approved Pfizer’s lung cancer drug Xalkori, which was only proven effective in less than 7% of the lung cancer patient population. However, because the efficacy was tied to a recognizable and testable gene mutation, the FDA only approved the drug as a treatment for lung cancer patients with this specific mutation.

“There’s been a change of paradigm,” Pfizer scientist James Christensen told the Wall Street Journal. “The new school of thought is, ‘If you find the patients that the drug will work in, and if you see enough benefit, we will find a way to get this to market.’”

These “targeted drugs” or “niche drugs” are oftentimes accompanied by a hefty price tag. For example, in the case of Xalkori, a year’s supply will run over $115,000. Similar sticker shock is tied to Roche’s melanoma drug Zelboraf (vemurafenib), which is priced at $56,400 for 6 months of treatment.

While there is nothing nefarious about a drug company reaping the monetary benefits of the economic supply and demand curve, there is heightened potential for fraud with hyper-specific FDA drug labels. Specifically, a narrow FDA-approved label provides drug companies with the proverbial “camel’s nose in the tent.” In other words, once a drug has been blessed by the FDA for a subset of the patient population, wayward drug companies will, inevitably and illegally, expand their marketing efforts to capture the rest of the patient population. This scenario has multiple off-label case recoveries over the past decade.

For more information about qui tam law and pharmaceutical fraud, contact Nolan and Auerbach, P.A.