After a panel of experts echoed his concerns this week, a top House Energy and Commerce Republican stepped up his charge that Democratic leadership on the committee is ignoring the fact that FDA may be struggling with safety concerns surrounding the healthcare reform law provisions that created the accelerated biosimilars approval pathway.

A July 19 panel of medical experts sponsored by Rep. Michael Burgess (R-TX) cautioned that FDA's exercise of its health reform law option to forgo clinical trials in approving biosimilar drugs could lead to safety and efficacy concerns. Panel members suggested that FDA may not approve some products because it doesn’t know whether to waive clinical trials requirements.

Congress created the biosimilars pathway to get relatively inexpensive biopharmaceuticals to consumers faster by letting FDA use an original drug's safety and effectiveness history to approve a follow-on biosimilar. It was touted as one of reform’s cost-saving provisions because payers, such as CMS, would buy fewer higher-cost innovator products. But the experts assembled by Burgess' Congressional Health Care Caucus cautioned that such a focus on speed – including an option to forgo clinical trials – leaves FDA far too much discretion to approve biosimilars.

This overt flexibility gives Burgess pause and requires some public discussion over the approval process, he told FDA Week directly after the panel ended. Specifically, Burgess wants the Democrats who run the Energy and Commerce Committee to summon FDA Commissioner Margaret Hamburg or Principal Deputy Commissioner Joshua Sharfstein to discuss the safety implications of a lack of clinical trials for biosimilars.

“We need the opportunity to get Dr. Hamburg or Dr. Sharfstein in to talk to them about what their vision is on this,” Burgess told FDA Week. “It's important to know what the regulatory regimen is going forward. Is it going to be that on some things, the safety and efficacy are pretty well worked out and we're going to proceed, or is it something that's really going to require something like a new drug application?”

FDA is going to be faced with that choice as events play out, Burgess said, “but it would be great to be able to talk to them on the record in a committee hearing.” Though the committee did recently bring in the Federal Trade Commission to brief committee members on its role, this misses Burgess' focus: “I'm not worried about the trade aspects. I'm concerned about safety.”
The agency is not to blame for this lack of formal conversation, said Burgess, who pointed the finger at Energy and Commerce Chair Henry Waxman (D-CA) and health subcommittee chair Frank Pallone (D-NJ).

“It's why they had the FTC in, so they could say, OK, we checked the box on this issue,” Burgess said. “The committee has not shown the appropriate level of curiosity for a major change in policy. If I were cynical, I would say they weren't really interested in the first place,” instead focusing more on the length of time of exclusivity and the economics of biosimilars instead of their safety aspects.

Neither Waxman nor Pallone responded for comment by press time.

During the panel discussion, Georgetown University professor of medicine and pathology Craig Kessler said that without clinical trials, off-label prescriptions for biosimilars become increasingly dangerous, creating a lack of trust in the process. “The issue is when FDA approves any biosmilar, the patients accept the imprimatur with confidence,” Kessler said.

After the panel, Kessler told FDA Week that there are several products “sitting, waiting to be approved” but “FDA hasn't decided what it wants to do yet.”

It is notable that FDA hasn't approved these drugs yet despite the intense pressure from lawmakers on the agency on the need to lower health care costs. But the health concerns force FDA to need “the congressional support and the mandate and the funds to be able to initiate the post-marketing surveillance work that's required.”

Georgetown’s Kessler said FDA does have some responsibility to “be a little more vocal about their concerns” about their new biosimilars role. One solution would be to follow the strict process created by the European Medicines Agency, he added (see FDA Week, Aug. 26, 2009).

“I think the FDA is in a very difficult situation because they didn't get this mandate until the bill was signed,” Kessler said. “I think the anxiety in the public would be dispelled if the FDA said we're going to mandate clinical trials in a way similar to what [Europe] has, and let's harmonize the worldwide approval of drugs.”