A Federal Drug Administration review process created 30 years ago called accelerated approval allows pharmaceutical companies to license promising treatments without proving they're effective, says KHN.
It's become a common path to market, accounting for 14 of the 50 approvals of novel drugs in 2021, compared with four of the 59 approvals in 2018, KHN says.
The accelerated approval usually is based on a “surrogate marker” of effectiveness — evidence of lower viral loads for HIV, for example, or shrinking tumors for cancer.
"Debate rages over the validity of some of these stand-ins, and some of the drugs,” says KHN.
“If you’ve got a game-changing drug that truly is going to make a difference, you don’t need surrogate markers to prove that. If it’s effective, patients will survive longer,” says Dr. Aaron Mitchell, an oncologist at Memorial Sloan Kettering Cancer Center. The shortened approval process, he says, is one reason “we are getting a lot of marginally effective, not clinically meaningful, more expensive drugs on the market.”
In 1992, the year the accelerated approval process was launched, Congress passed the Prescription Drug User Fee Act, a law committing the drug industry to pay so-called user fees to help fund the FDA’s drug approval process.
The fees have steadily increased in importance, accounting for $2.9 billion of the FDA's $6.5 billion 2022 budget, including two-thirds of the drug regulation budget, and the work of at least 40 percent of the FDA’s 18,000 employees, KHN says. In recent years, companies have paid between $2.5 million and $3 million to have each drug application reviewed.
In most cases, companies that get accelerated approval must submit additional data after the drug goes to market that proves it cures or successfully treats the disease, KHN says.
"Although FDA-regulated products account for about a fifth of every dollar spent by U.S. consumers, Congress has never shown appetite for dramatically increasing its budget,” says KHN.
So every five years the user fee renewals become must-pass legislation. And this is the year. The user fee accords — one for each brand-name, generic, and over-the-counter drug, as well as for animal drugs, biologics and medical devices — "are packed with new programs, tweaks to old ones, regulatory deadlines, and other items negotiated by the FDA and industry, with Congress tacking its priorities onto the authorizing bill.”
The final bill, which became part of stopgap spending legislation, stripped out language that would have made it harder for accelerated products to stay on the market if manufacturers failed to produce evidence of lasting value in a timely way, says KHN.