Washington Editor

WASHINGTON - Any new law for approving follow-on biologics should give the FDA flexibility in determining the extent of clinical trial requirements, Deputy Commissioner and Chief Medical Officer Janet Woodcock testified on Wednesday. That caveat is needed to allow for future scientific advances that eventually might trump current necessities, she told a House panel.

Her comments came at a hearing of the health subcommittee of House Energy and Commerce panel, convened to provide information on crafting legislation to create a follow-on biologics approval pathway. And the lawmakers heard plenty from both sides of the fence, with Woodcock's testimony the centerpiece of safety concerns.

Acknowledging the agency's present belief that human immunogenicity trials will be needed "at a minimum" to assure the safety of follow-ons, and "probably human pharmacokinetic trials" as well, she nonetheless advised subcommittee members to consider "that the science will be advanced over time." Routine lab and animal studies always will be needed, Woodcock added, but she cautioned that imposing clinical trial requirements now for studies that could prove unnecessary down the road isn't ethical for patients.

"It's going to depend on the situation," Woodcock said, conceding that "in some cases, very extensive safety testing in humans may not be necessary." She said the FDA wants to work with lawmakers to craft a follow-on approval pathway under the Public Health Service Act, under which most biologicals are approved.

Language in a bill called the "Access to Life Saving Medicine Act of 2007," or H.R. 1038, seems to favor Woodcock's sentiment for variable clinical trial regulations, and its primary sponsor, Rep. Henry Waxman (D-Calif.), underscored the point.

"I would trust the FDA to make that decision, not members of Congress," he said in a comment that echoes generic industry advocates who are fighting against setting standards too high for such companies to even consider entering the follow-on business.

In contrast, a competing bill called the "Patient Protection and Innovative Biologic Medicines Act," or H.R. 1956, mandates clinical studies for follow-ons. That language, put forth by Rep. Jay Inslee (D-Wash.), is in line with pioneer drugmakers who were represented at the hearing by David Schenkein, Genentech Inc.'s vice president of clinical hematology and oncology. Speaking on behalf of the Biotechnology Industry Organization (BIO), he said "there will always be a need" for clinical testing of follow-ons to ensure their safety.

However, it's worth keeping in mind that such wishes don't jibe with Woodcock's remarks on behalf of the FDA. At the same time, she noted that while some "very, very simple" follow-ons will be able to be characterized within this decade, obviating the need for clinical trials, most won't be able to be characterized for more than a decade. "That is my prediction," Woodcock said, "but I don't have a crystal ball."

Schenkein also raised another seminal difference between the two bills: data exclusivity. Pioneer companies believe such protection is essential to preserve incentives for innovation, as do many patient advocates. The Inslee bill includes a provision for 14 years of data exclusivity and the potential for one more, while the Waxman bill provides none.

Failing to address data exclusivity could "strangle" innovation, Schenkein said, particularly in the "critical but costly research" to develop early stage, adjuvant cancer treatments because of the time needed to make such discoveries.

In the other camp, Bruce Downey, CEO of Barr Pharmaceuticals Inc., said that five years of data exclusivity would sit well with him, a shift away from the absence of such language in the Waxman bill. But he expressed outright opposition to the 14 years proposed in the Inslee bill.

This back and forth sets the stage for some sort of compromise between the competing pieces of legislation, and industry observers believe a bill that seeks a middle ground is forthcoming. As Rep. Jim Matheson (D-Utah) said, "there's a reasonable solution" between the lines drawn by the Waxman and Inslee bills.

In the context of continued congressional examination of growing health care costs these days, the follow-on issue seems to have undeniable traction. And even if legislative language to establish a follow-on approval pathway doesn't get attached to efforts to renew the Prescription Drug User Fee Act, as some have speculated in recent weeks, there still are signs pointing toward the possibility that a resolution could be reached in the relative near term. Rep. John Dingell (D-Mich.), chairman of the House Energy and Commerce Committee, said he would like to do that in the current Congress, taking into account patient safety and innovation.

"This will be a priority for our subcommittee," said Chairman Frank Pallone (D-N.J.), noting that Congress needs to pass a follow-on approval pathway.

"The time to move forward with an abbreviated pathway is now," Waxman said.