How to reboot the FDA

In the United States, there’s just one way to sell a medical drug or device: Get it approved by the Food and Drug Administration, a century-old institution with thousands of reviewers who rigorously check evidence to ensure drugs and devices are effective and safe. That sounds like a good thing, but a growing number of people and institutions — including the Defense Department — aren’t so sure. They argue that there are too many drugs and products bound up in regulatory review that could save lives, if only the government’s health care agency wasn’t so hidebound and slow.

That argument swayed some congressional Republicans, who last month attempted to go around the FDA and give the Defense Department authority to approve its own medical products. The move was beaten back in a joint assault from congressional health staff and the FDA itself.

While the Pentagon’s insurgency has ended, calls for alternatives to FDA approval have not. The agency has long been targeted by critics who blame its review process for drugs that are too expensive and too slow to reach market — arguing that if we really want more and cheaper drugs in the future, we must reform or even scale back the FDA’s role. That’s far from a unanimous view; much of the medical community deeply trusts the FDA and its stamp of approval, and the nation’s powerful drug industry has a vested interest in preserving the current system. But a number of ideas, both radical and nuanced, have been proposed for how the process could work differently. Here’s a look at five proposals that could create new paths to drug approvals or transform the way FDA operates.

Option 1: Skip FDA. That’s the preference of the Right-to-Try movement, which has argued that the agency should get out of the way and let terminally ill patients decide how much risk they’re willing to take on unproven treatments. “We don’t necessarily see it as an alternative to the FDA,” said Christina Sandefur of the Goldwater Institute, which has championed the initiative. “Right to Try is an alternative pathway for patients who need immediate access and can’t get it any other way.”

Under Right-to-Try legislation — which is on the books in more than three dozen states, has passed the Senate and currently is in front of the House of Representatives — terminally ill patients can work with doctors and insurers to get access to in-development treatments, if the drug company grants necessary permissions.

“We have an entity that has essentially substituted its own judgment for the judgment of the patient,” Sandefur said. “I would like to see an FDA that operates more like our FDA did when it was first founded: It just ensured that patients had information, and that it was accurate.”

The flip side: Despite the widespread adoption of Right-to-Try laws, there’s little evidence that companies are willing to take the risk. Sandefur puts some blame on the FDA itself, saying drug companies fear that if patients try an experimental therapy and have an adverse reaction it could jeopardize their chance of getting the treatment approved. And the FDA already accommodates many terminally ill patients’ requests for access through its own experimental drug pathways, which critics of Right to Try say renders the Goldwater-backed movement unnecessary.

Physicians and researchers also warn that without FDA safety reviews, patients aren’t equipped to judge what’s an appropriate risk. “We as Americans have tried that. And it was terrible,” said Holly Fernandez Lynch, a professor at the University of Pennsylvania who has studied tragedies from unregulated drugs, like the deaths of more than 100 Americans from a single product’s mass poisoning in 1937. “And that’s why we have the FDA that we have.”

Option 2: Let the market decide. Conservative economists like Tomas Philipson, who sits on the White House Council of Economic Advisers, have argued that FDA should approve drugs and devices using limited early data and then revise its decisions later. For instance, drug developers could get a temporary license to sell a fixed amount of a drug — companies with more compelling early evidence could sell more and companies with a riskier product would be allowed to sell less. The companies would apply for expanded approvals as more data are collected.

“Partial approvals make sense to me,” said Darius Lakdawalla, a USC health economist who has argued for the idea alongside Philipson. In Lakdawalla’s vision, FDA would deny only “egregiously unsafe drugs,” and health insurers and doctors could then make decisions about what they would pay to cover or choose to prescribe.

“I don’t think FDA’s role should be to decide on risks and benefits in difficult cases,” added Lakdawalla, who thinks market power, not government oversight, should be playing a larger role in health care. “Making a single value determination for the entire country seems impossible from an economist’s perspective. Imagine if the U.S. government could determine which car Americans could drive, and you could only choose six.”

Read more of this Politico article by Dan Diamond by clicking here.

Photo: Taylor Callery for Politico