Experts concerned about lack of clinical trials for biosimilar drugs

Medical experts on Monday warned that the absence of mandatory clinical trials for biosimilar drugs could compromise their safety and effectiveness.

The Democrats' new healthcare reform law outlined a pathway for the Food and Drug Administration (FDA) to approve next-generation biopharmaceuticals modeled on original breakthrough drugs. But the provision is vague, the critics say, and leaves FDA with a great deal of leeway to okay those drugs — including the flexibility to decide whether clinical trials are necessary at all.

That provision, sponsored by Rep. Anna Eshoo (D-Calif.), was designed to get biosimilars — and the cost savings that accompany them — to the marketplace more quickly by allowing FDA to base its approval, in part, on the safety and efficacy record of the original breakthrough drug.

But Craig Kessler, professor of medicine and pathology at Georgetown University, said Monday that the difference in manufacturing processes between companies can alter the drugs "in ways that technology can't detect" — something of particular concern when doctors prescribe biosimilars for off-label uses, he warned.

"If you don't have clinical trials to take a look at all of these other off-label uses," Kessler said during a Capitol Hill discussion hosted by the Congressional Health Care Caucus, "then you don't really know what the equivalency in dosing is going to be like, and what the safety — the long-term safety — [issues] will be."

Kessler wasn't alone. 

Merrill Matthews, a drug expert with the conservative Institute for Policy Innovation, cautioned that the law's "lack of clarity on safety issues" leaves the United States behind Europe when it comes to ensuring the well-being of patients prescribed biosimilars.

"The country needs a clear pathway," Merrill said. "But we need to ensure that the safety and efficacy are taken care of."

Meanwhile, Rep. Michael Burgess (R-Texas), an Ob/Gyn who founded the Congressional Health Care Caucus, used Monday's forum to slam the Democratic leaders of the Energy and Commerce Committee for not holding any hearings on implementation of the health reform law. 

"We have yet to have a single oversight hearing," Burgess said. "HHS has missed some deadlines and accelerated some others. … Congress is failing in its oversight obligations."

Eshoo's office did not immediately respond to requests for comment Monday.