‘It’s not working’: An FDA insider’s view of where medical innovation falls short

At first, it sounded like a breakup.

“It’s not working, and it won’t work in the future,” said Dr. Janet Woodcock, the U.S. Food and Drug Administration’s top drug regulator.

She was speaking to an audience of about 150 researchers who are toiling to discover new medicines that will save lives. The room was silent.

“I don’t want to bum everyone out,” she said. “The science is fabulous, but that’s not enough.”

To be sure, Woodcock wasn’t looking to sever ties with the researchers, who work at universities, research institutes and pharmaceutical companies. But she did call for major reforms in the scientists’ relationships with regulators, doctors and one another. Without a wide-ranging overhaul, she said, their successes in the lab won’t make it to the patients who need them most.

“The goal isn’t just improving knowledge. The goal isn’t FDA approval. The goal is to improve human health,” she said Friday at the Breakthroughs in Medicine conference in Rancho Palos Verdes. (The Los Angeles Times is the presenting sponsor of the event.)

“Is the guy living under the bridge going to get a $2-million CAR-T cell curative therapy?” she asked, referring to a breakthrough cancer treatment. “If that guy can’t get the drug, then we’ve failed.”

Woodcock pointed to various causes for the disconnect between discoveries in the laboratory and medicines that reach the clinic. The current structure of the academic research discourages collaboration: Grant review processes, promotion criteria and even the concept of tenure ought to be reconsidered, she said.

In addition, she argued, the process of creating and commercializing a drug has been hindered by corporate secrecy. When the science is valuable and “not to be shared,” drug trials are fragmented and costly. Meanwhile, important clinical questions go unanswered when they don’t have obvious commercial gain, she said.

And above all other concerns was patient access. Other industries focus on making their products cheaper. But that’s not the case with the pharmaceutical industry, where high failure rates and expensive clinical trials have led to drugs far beyond the financial reach of those in need.

“I would never have thought, ever, in the ’90s, that if somebody came up with a cure for hepatitis C, that people wouldn’t be able to be treated. That was inconceivable,” she said. “But fast-forward to now, and that’s the reality we’re seeing.”

Some changes to drug development have disrupted the ecosystem in positive ways. As researchers aim to conquer rare diseases, for example, patients and advocacy groups have become more than just cheerleaders for innovation — they’ve become active participants in finding solutions.

But even that fervor has caused collateral damage, Woodcock said. Health burdens such as cardiovascular illness, addiction and antimicrobial resistance are now less attractive candidates for research, even though they affect far more people.

Woodcock urged researchers to leverage big data — such as electronic health records — to study those diseases with efficient, universal protocols. She also encouraged her audience to incorporate practicing doctors in the research process.

“You can’t just develop breakthroughs and then throw them over the wall to practitioners,” she said. “We need the whole system to evolve and change if we’re going to do what we set out to do: help every patient feel better and live longer.”