Genentech's $94,000-a-Year Drug Tarceva: Why Faster FDA Approvals Could Backfire

Published: Feb 08, 2017

Genentech's $94,000-a-Year Drug Tarceva: Why Faster FDA Approvals Could Backfire February 7, 2017
By Mark Terry, BioSpace.com Breaking News Staff

One of the first things President Trump did upon winning the election was change his website, greatagain.gov. On that site, his plans were updated to reflect intended policies for various things, including healthcare and bipharma. Of the six points for healthcare, one was, “Reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products.”

As yet, President Trump hasn’t selected anyone to head the U.S. Food and Drug Administration, although one being vetted is Jim O’Neill, a former Health and Human Services Official. Although not a physician or scientist (he holds both a BA and MA in Humanities), from 2005 to 2007, he served as Associate Deputy Secretary/Senior Advisor to the Deputy Secretary of the Department of Health and Human Services, then as Principal Associate Deputy Secretary from 2007 to 2008. He is currently managing director of Peter Thiel’s Mithril Capital.

O’Neill has given talks as recently as 2014 where he said that the FDA should approve drugs based only on safety, not efficacy.

A recent FDA report cited 22 specific cases of drugs that passed Phase II clinical trials, but crashed and burned in Phase III, underlining why O’Neill’s arguments could be dangerous.

The Los Angeles Times presented another case study today why this approach to drug regulation can be problematic.

In 2004, the FDA approved Genentech ’s Tarceva for lung cancer. At that time, there weren’t a lot of options for lung cancer, and although the drug wasn’t blowing anyone away with how great it was, and it had some unpleasant side effects, such as intense rashes, it was at the time better than nothing. The drug cost about $94,000 for a year of treatment.

About the same time Tarceva was approved, scientists identified a gene mutation associated with epidermal growth factor receptor (EGFR) that is blocked by Tarceva. In short, they had discovered a way to test patients to determine if Tarceva would actually work for them—because the drug didn’t work for about 90 percent of the patients receiving it.

The Los Angeles Times writes, “Yet for years, Genentech and OSI downplayed the importance of testing for the mutation or levels of the protein and avoided using those tests to narrow the pool of patients. Instead the companies’ early trials—and promotion of the drug—were aimed at all patients with non-small cell lung cancer whether they had the mutation or not.”

In June 2016, Genentech sent a letter to physicians saying they should stop prescribing Tarceva to most patients with lung cancer. This turned over a 2010 FDA decision that significantly expanded use of the drug—despite warnings from experts that there was not much evidence the drug worked.

“Clinical trials answer specific questions about the safety and efficacy of a medicine, but also lead to new hypothesis-generating information,” Genentech said in a statement. “It is not uncommon for a medicine’s approved indications to change over time.”

True enough. But again, it does underline part of the problem with Trump’s statements regarding streamlining the approval process, as well as O’Neill’s. In an August 2014 speech, O’Neill said, “Let’s prove efficacy after they’ve been legalized.”

Setting aside the fact that taxpayers and insurers would likely be paying millions and potentially billions for drugs that potentially do nothing, as the recent FDA study suggested, Phase II safety trials are regularly overturned with larger Phase III data. Or as The Los Angeles Times writes, “That would mean more patients would become part of a continuing experiment and be prescribed medicines their doctors know little about.”

Although O’Neill and other critics of the FDA’s timelines seem to focus on how long it can take to get a drug approved, The Los Angeles Times article suggests that it’s a double-edged sword—it takes a long time to prove that an approved drug is ineffective, dangerous and should be taken off the market. Some of it is simply that new science comes along that gives insight into the drug’s activity. Some of it is just the slow grinding of the regulatory mill and possibly pharmaceutical companies not rushing to release data that might kill their golden goose.

In the case of Tarceva and Genentech, the drug was originally approved in patients who had failed chemotherapy and had few other options. In 2009, Genentech and OSI revisited the FDA in an attempt to get its usage expanded. New data showed the drug increased survival by a month. They wanted it prescribed as “a maintenance therapy” for patients earlier in the diseases and who had responded to chemotherapy. The FDA committee voted against the approval, 12 to 1. The FDA went ahead and approved the drug for expanded use anyway.

In 2011, Tarceva’s worldwide sales jumped to $1.5 billion. But now, eight years later, physicians are being told not to prescribe the drug unless they specifically have the gene mutation that makes it effective.

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