Dr. Steven-Huy Han, a UCLA liver specialist, has prescribed Ocaliva to a handful of patients, although he’s not sure it helps.
As advertised, the drug is lowering levels of an enzyme called alkaline phosphatase in their blood, and that should be a sign of healing for their autoimmune disease, called primary biliary cholangitis. But “no one knows for sure,” Han said, whether less enzyme means they won’t get liver cancer or cirrhosis in the long run.
“I have no idea if the drug will make them better,” he said. “It could take 10, 20, or 30 years to know.”
Ocaliva came to market through an FDA review process created 30 years ago called accelerated approval, which allows pharmaceutical companies to license promising treatments without proving they are effective. It has become a common path to market — accounting for 14 of the 50 approvals of novel drugs in 2021 compared with four among 59 in 2018, for example.
The FDA’s accelerated approval is usually 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.
“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,” said …