The Tarceva Story: Why Faster FDA Approvals Could Backfire

Pulling an ineffective drug off the market can take years

In June 2016, Genentech sent doctors a letter saying they should stop prescribing the expensive blockbuster drug Tarceva (erlotinib) to most patients with lung cancer. A study had found that only a small number of patients with a certain gene mutation might be helped by the drug. According to an article in the Los Angeles Times, the story of Tarceva illustrates the danger of approving experimental medications before reliable scientific data show they are effective.

The FDA is now examining how to comply with the 21st Century Cures Act, a law Congress passed in December to speed the approval of drugs and medical devices. And critics of the FDA hope that President Trump will go even further. At a White House meeting with pharmaceutical executives this week, Trump promised them he was “going to streamline” FDA regulations.

One of Trump’s possible selections to head the FDA—Jim O’Neill, a Silicon Valley venture capitalist with no background in medicine or science—has raised the idea of approving drugs with no tests of their effectiveness.

“Let’s prove efficacy after they’ve been legalized,” he said in a 2014 speech, according to the Times.

In a written statement, the FDA defended its fast approval of Tarceva and other medications. The agency said lung cancer patients had few options when the drug was first approved in 2004.

Tarceva isn’t the only drug that may not work for most patients, the Times article contends. In a recent study, researchers found that of 36 cancer drugs approved by the FDA between 2008 and 2012, only five (14%) had been shown to actually extend survival by 2015.

Lead investigator Dr. Vinay Prasad, a professor at the Oregon Health and Science University, said the FDA’s lax approval standards, combined with the six-figure prices companies can charge for unproven or marginal medications, have encouraged the pharma industry to take marketing shortcuts rather than spend research dollars to find true breakthroughs.

Prasad pointed to a study that found the average gain in survival for the 71 cancer drugs approved from 2002 to 2014 was just 2.1 months.

“That’s not good enough for the people I see in my clinic,” he said. “As you lower the regulatory standards, you open the flood gates to more of these ineffective drugs.”

Some ineffective drugs have eventually been taken off the market, but the process can take years. For example, Mylotarg (gemtuzumab ozogamicin, Pfizer) was removed from pharmacy shelves in 2010—a decade after the FDA approved it for patients with acute myeloid leukemia.

Source: Los Angeles Times; February 7, 2017.