The 21st Century Cures Act, passed by Congress and signed into law by President Barack Obama last December, speeds up the approval process for drugs and medical devices, in part by loosening the standards by which the Food and Drug Administration (FDA) evaluates those products’ effectiveness and safety.

President Trump may go even further. In a meeting with pharmaceutical executives after his inauguration, he promised to slash regulations and “streamline” the FDA to help companies get their drugs and devices to market more quickly.

He also said he had a “fantastic person” whom he would soon be putting in charge of the FDA. The name most frequently cited as being that person is Jim O’Neill, a former official at the Health and Human Services Department during the George W. Bush administration who is currently managing director of Mithril Capital Management, which was co-funded by Silicon Valley billionaire and Trump supporter Peter Thiel.

As the New York Times has reported, O’Neill “has argued that companies should not have to prove that their drugs work in clinical trials before selling them to consumers.”

“At an anti-aging conference in 2014,” writes Times reporter Katie Thomas, “Mr. O’Neill advocated something he called ‘progressive’ approval, in which drugs that were proved safe, but not yet proven effective, could be allowed on the market. ‘Let people start using them, at their own risk,’ Mr. O’Neill said. ‘Let’s prove efficacy after they’ve been legalized.’”

A cautionary tale

The danger of loosening regulations — essentially lowering the evidence bar — to speed drugs and devices to market can be seen in the troubling tale of the lung cancer drug Tarceva, which is the focus of a recent article in the Los Angeles Times.

As biotech reporter Melody Petersen explains, the FDA approved Tarceva in 2004 for the treatment of severely ill lung cancer patients who have not been helped by chemotherapy and have few options.

But that was a very small market. So Genentech and OSI Pharmaceuticals, the two companies marketing Tarceva at the time, asked the FDA to expand the drug’s approval to less ill lung cancer patients who have been helped by chemotherapy. In doing so, they downplayed their own research that showed that only a small percentage of lung-cancer patients — those with a particular gene mutation — benefited from the drug.

One Tarceva executive told investors that expanding the drug’s patient base to all lung cancer patients could add $500 million a year to the drug’s sales.

Two patient-advocacy groups, both partially funded by Genentech and OSI, helped lobby the FDA to make the drug available to more patients. The companies also flew in individual lung cancer patients to plead their case.

Those efforts had the desired effect. Despite warnings from a panel of expert advisers that there was not enough scientific evidence that Tarceva actually worked, the FDA approved the expanded use of the drug in 2010.

The FDA employee who signed the approval letter just happened to be a former Genentech senior scientist, Petersen reports.

Rise — and fall

Sales of the $94,000-a-year drug climbed rapidly after the expanded FDA approval, reaching $1.5 billion globally in 2011. But concerns about the drug’s safety and effectiveness also rose.

“The FDA’s decision resulted in more harm than the hundreds of millions of dollars wasted on a drug that was ineffective for 90% or more of patients,” writes Petersen. “Over the years, thousands of patients were prescribed a drug that had no chance of helping them but could frequently cause terrible rashes covering their faces and bodies, according to prescription data and adverse event reports. Some patients with the most aggressively growing cancers died before trying other treatments that may have lengthened their lives.” 

It took a whistle-blower to finally expose the company’s misrepresentation of Tarceva’s effectiveness. Writes Petersen:

Brian Shields, a former Genentech sales rep, said he complained in a 2010 meeting of the Tarceva marketing team that the company was promoting the drug to patients it could not help. His boss dismissed his concerns, he said, telling him he “was not a team player.” 

Shields later filed a lawsuit as a whistle-blower under the federal False Claims Act, which the government joined. The suit claimed Genentech and OSI had illegally sold the drug, including by sending doctors and nurses to attend all-expense-paid retreats at resorts where they were “groomed” to tell others about how Tarceva could extend survival in “a broad range of patients.”

The companies also recruited patients whose cancers had responded to the drug, and then trained and paid them to talk to others in what it called the “Patient Ambassador Program,” his lawsuit said.

The companies discouraged doctors from testing patients for the mutation, the lawsuit said, because they knew it would reduce sales.

“This is a failure of the system,” Shields said in an interview. “An FDA approval doesn’t necessarily mean that a drug works.”

Last June, the U.S. Justice Department announced that Genentech and Astellas Pharma (the company that acquired OSI in 2010) would pay $67 million to settle the lawsuit. Shields received about $10 million of that settlement.

That month, the companies also sent letters to doctors saying that Tarceva should no longer be prescribed to lung cancer patients without the gene mutation.

And in October, the FDA removed its approval of Tarceva — except in cases of patients who have the mutation.

FMI:  You can read Petersen’s article on the Los Angeles Times website.