Final enactment of the Food and Drug Administration Amendments Act (FDAAA) took longer than expected and forced the legislators
to compromise on many specific issues. Congress managed to approve it in time to reauthorize the Prescription Drug User Fee
program (PDUFA IV) before that expired on September 30, 2007, but the last-minute rush could have long-term ramifications
for agency operations as well as company R&D decisions.
Overall, manufacturers applauded Congress' enactment of FDAAA. In addition to continuing user fee support for FDA approval
of drugs, biologics, and medical devices, the final bill curbed the scope of new penalties for fraud and noncompliance and
provided FDA with leeway to weigh the need for special distribution safeguards for riskier products. At the same time, FDA
gained more authority to control drug marketing and labeling, to require postapproval studies, to establish active surveillance
systems, and to make clinical trial operations and results more transparent.
NEXT YEAR FOR FOLLOW-ON BIOLOGICS
One of the final decisions was to put off establishing a legal pathway for FDA to approve follow-on versions of biotech therapies
(FOBs), also called biosimilars. The Senate Health Committee approved bipartisan legislation in June that authorizes biosimilars, but the House did not address
the issue. Despite Senate efforts to include FOB legislation in FDAAA, Congressional leaders conceded in early September that
time was running out for such action. Rep. Henry Waxman (D-CA) told the Generic Pharmaceutical Association (GPhA) that the
chances of adding generics biologics legislation to FDAAA were "extremely slim." And Rep. Frank Pallone (D-NJ), who chairs
the House Energy & Commerce Health subcommittee, said that the House would not just "take the Senate bill" but would hold
hearings on FOBs later in the year. Although some Congressional leaders talked of pushing through a bill this year, most are
now looking to 2008 or beyond. Democrats may decide to wait until they take over the White House to really tackle the issue.
FOB advocates recognize that gaining House and Senate consensus on biosimilar legislation will take time because of considerable
disagreement over what kind of exclusivity to grant FOBs. The Senate bill provides protection for 12 years, a compromise designed
to ensure continued R&D investment by innovators. This is less than the 14 years demanded by the Biotechnology Industry Organization
(BIO), but much more than generics makers consider reasonable. Waxman told GPhA that he wants to see hard data supporting
any exclusivity period, and that the brand industry "can't just make up these numbers." A related issue is whether innovators
could extend exclusivity beyond the 12 years by reformulating products. Another challenge is interchangeability. Innovators
maintain that "similar" does not mean "the same" and that unlike conventional generic drugs, a biosimilar should not have
the same generic name as the innovator, as is the case for conventional generic drugs.
Meanwhile, the biogenerics bandwagon is picking up steam. The European Union recently approved new biosimilar versions of
erythropoietin, (see BioPharm International, October 2007) and other biosimilar applications are pending. Generics makers point to analytical advances that make characterizing
of FOBs more precise, but are willing to accept FDA case-by-case review of FOB applications. Developing biosimilars is "a
moral issue," said Hospira CEO Chris Begley at the GPhA meeting, because it will allow patients to access low cost, high-quality,
life-saving treatments. Hospira hopes to build on its expertise in producing generic injectables to become a biosimilars leader;
a number of generics makers and small biotech companies are eyeing similar opportunities. FOB enthusiasts predict significant
savings for patients even though biosimilars are likely to cost only 25% less than innovators.
NEW CHANGES AND CHALLENGES
The industry is mulling over hundreds of pages of the FDAAA's fine print. The bill contains at least 200 specific provisions,
noted FDA commissioner Andrew von Eschenbach in discussing next steps for the agency. Delays in finalizing the legislation
and late additions to the user fee program also put off the calculation of actual user fees for the fiscal year that began
October 1, 2007. The final bill provides $225 million in additional safety funding over five years, which boosts drug fees
by another $25 million for 2008.