Washington Editor
WASHINGTON - After percolating for months, the biogeneric debate spilled out on Capitol Hill Thursday at a hearing of the Senate Health, Education, Labor and Pensions Committee, and its overall tone seemed to favor pioneer drugmakers.
Chairman Edward Kennedy (D-Mass.) said the hearing would "help to provide the information the committee needs" to determine if a proposal to allow follow-on biologics should be included in a coming bill on drug safety and the Prescription Drug User Fee Act, though he offered no specifics for precise timing on legislative action. However, he indicated that a bill on follow-ons should be "led by the science," and added that "protecting patient safety is essential" and "innovation must be valued."
Sens. Charles Schumer (D-N.Y.) and Hillary Rodham Clinton (D-N.Y.), along with Rep. Henry Waxman (D-Calif.), have introduced legislation that would give the FDA discretion to determine whether there are clinically meaningful differences between follow-ons and original biologicals, as well as prudence on mandating clinical studies on a case-by-case basis.
The measure aims to provide the agency with "power and flexibility to make science-based decisions," Clinton told reporters after the hearing.
But critics say that the bill, as currently written, sacrifices safety standards for speed and savings, and also poorly accounts for pioneer drugmakers' intellectual property. So in addition to questions on timing, it's also not yet clear how strictly a final bill would adhere to the "Access to Life Saving Medicine Act of 2007," or H.R. 1038. In fact, speculation has arisen that competing legislation could be brought forward that addresses concerns about protecting patient safety and patent rights.
Aside from such gazing into the crystal ball, though, opponents of the bill were generally content with the hearing's outcome.
"We were pleased that folks recognized that these are not interchangeable generics," said Amit Sachdev, the executive vice president of the Biotechnology Industry Organization's health section, "and that science matters."
In addition, he applauded the committee's willingness to examine the debate beyond potential cost savings and also consider complexities around scientific, regulatory and patent questions. Given that range of unresolved issues, Sachdev cautioned against hastily advancing the bill in lockstep with pending user fee legislation. Importantly, he said he walked away with "a clear sense that there's bipartisan support for looking at something other than" the Schumer-Clinton-Waxman proposal. "It seems like [final legislative language] is not going to be around the current draft of the bill that was introduced," he told BioWorld Today, "and that's positive."
Innovator companies and their allies have long angled for a slow, conservative approach for allowing follow-on biologics on the market through a pathway that mandates some degree of clinical trials and other testing for all applications. BIO consistently has called for clinical studies of all follow-ons, a point echoed by the committee's ranking member, Mike Enzi (R-Wyo.), who likened the difference between biopharmaceuticals and chemically synthesized drugs to that between skyscrapers and three-bedroom houses. "One girder out of place could cause the entire structure to fall," he said.
That's the point made by pioneer drugmakers: Changes in manufacturing, no matter how minor, impact the final product's immunogenicity profile and other characteristics. Therefore, there will "always be a need" for clinical studies of follow-ons, testified Johnson & Johnson's Jay Siegel. "To ensure safety and efficacy, testing in humans will be needed."
European regulators have adopted guidelines that require clinical testing for all follow-ons, sometimes to a large degree, depending on the complexity of the original biopharmaceutical. In addition, the Europeans have labeled such products biosimilars, an acknowledgement that they are not exact copies.
The "primary objective" of that framework is "to apply equal standards," as opposed to establishing interchangeability and substitutability, testified Nicolas Rossignol, the administrator of the European Commission's pharmaceuticals unit, via satellite from Brussels, Belgium. "There is no one-size-fits-all approach."
In addition, that system provides a period of market exclusivity for pioneer products, prohibiting follow-on applications until after an 11-year period of data exclusivity expires, which innovator companies say is essential. The Schumer-Clinton-Waxman proposal makes no such provision.
Sen. Orrin Hatch (R-Utah), who has predicted that some form of follow-on legislation would pass this year, backed the European method. He said that system should be given "every consideration" in crafting a final bill, adding that he doesn't "quite agree" with existing language in the proposed bill.
In contrast, generic drugmakers favor the flexibility the legislation would give the FDA in determining when an abbreviated application process for follow-ons is applicable, relying on pioneer products' safety and efficacy data for reference in such cases. They believe the measure does not shortchange purity and potency, claiming that manufacturing discrepancies are inherent in the production of all biopharmaceuticals.
"Product is not the process," testified Sandoz GmbH's Ajaz Hussain. "Batch to batch variation is inevitable for all biologic drugs."
The Generic Pharmaceutical Association (GPhA) said that because pharmacokinetic studies are used in conjunction with advanced analytical tools to detect major brand product differences in post-approval manufacturing and product changes, relying on clinical studies to determine treatment outcomes would prove more cumbersome and less precise. Avoiding clinical work would save time and capital, allowing biogeneric makers to price their products measurably below original biopharmaceuticals, according to some estimates. That's music to the ears of some insurers, pharmacy benefit managers, consumer groups and large employers that have recently thrown their weight behind the bill.
Representatives of those groups, along with generic industry executives, have been in Washington all week to discuss the issue with congressional members. To counter that advocacy, BIO bought advertising in political publications read by lawmakers and their staff members. The ad, written to highlight the "twists and turns" of follow-ons, calls "thoughtful" deliberation "the best prescription for this complex issue."
Catalysts appear in motion for something to take shape. In addition to Hatch's prediction, made at a recent GPhA meeting, Mark McClellan, the former head of the FDA and Centers for Medicare & Medicaid Services, told the same audience that follow-on legislation could indeed pass this year. Whether or not it happens in the same time frame as renewing user fees in the next few months remains to be seen, not to mention an apparent lack of movement in the House of Representatives.
After the hearing, members of Kennedy's staff declined to comment on how quickly the issue would move forward. Clinton also demurred, though she conceded that "this is the beginning of the process."