Uncertain Future for Waxman Follow-on Biologics Bill

    By Kevin E. Noonan --

If behavior can inform belief, it may be getting less and less likely that H.R. 1427, the "Promoting Innovation and Access to Life-Saving Medicine Act," has any reasonable chance of passing in this Congress.  The bill, introduced on March 11, 2009 by House Committee on Energy and Commerce Chairman Henry Waxman (D-CA; at right), has as one of its key provisions a data exclusivity period of only 5 years (see "Waxman Introduces Follow-on Biologics Bill"), a term most in the biotechnology community believe is too short to support innovation in the sector.  Indeed, Vaughn Kailian of MPM Capital LLC, and a seasoned veteran in the biotechnology sector, opined that Congressman Waxman's five year data exclusivity proposal was "dead in the water" at a Super Session on the challenges to biotechnology held at the BIO 2009 Convention in Atlanta last month (see "Docs at BIO: 'Perfect Storm' Super Session").

Another participant at that session, Dr. Scott Gottlieb, a senior fellow at the American Enterprise Institute, said that there will be legislation "in the near future" but that this had not advanced in Congress because Rep. Waxman could not move his own bill out of (his own) committee.  Indeed, there is a competing bill, H.R. 1548 ("The Pathway to Biosimilars Act"), introduced on March 17, 2009 by Ms. Eschoo, Mr. Inslee, and Mr. Barton that specifies 12 years of data exclusivity with provisions for an additional 2 years for a "medically-significant new indication" and 6 months for pediatric studies (see "Second Follow-on Biologics Bill Is Introduced in House").  This bill has more than 65 co-sponsors, and it is widely believed that Congressman Waxman's bill has not advanced out of committee because he does not have the votes for its passage.

Perhaps as a tacit acknowledgment of the political realities, or maybe simply as an attempt to ensure rapid implementation of whatever bill is ultimately passed, Congressman Waxman sent a letter to the President on Monday regarding how the Food and Drug Administration could approve follow-on biologics under its current regulatory scheme.  The tenor of his remarks suggests (as reported by modernhealthcare.com) that Mr. Waxman may be trying to have the FDA develop a pathway for biosimilar approval under existing law.

In his letter, the Congressman "urge[s] the Administration to consider what steps can be taken under existing authority to prepare and even begin to use a pathway for generic biologics" (emphasis added).  Since it is widely accepted that FDA has no such authority under existing law, the emphasized portion of Mr. Waxman's remarks is curious at best.

The rationale, of course, is cost; the letter prefaces this suggestion that the Obama administration "begin to use" a "pathway to generic biologics" that is non-existent under current FDA authority by reminding the President that:

Biotech drugs, while often life-saving, are the fastest growing and most expensive components of the nation's prescription drug costs.  Many of them cost tens and thousands of dollars a year and impose an unsustainable burden on patients, employers, and the federal and state governments.

Thus, "it is important for FDA to begin implementing the program as soon as possible" and "[t]he speed of FDA's action will determine how quickly safe and effective generic biologics become available to patients."  Because, after all, "[g]eneric biologics are a significant way to control costs," according to the Congressman.

It is certainly the case that there are enormous economic and political pressures to reduce healthcare costs over the near term, as the Baby Boom generation hits their geriatric years and the burdens on all payors (public and private) can be expected to intensify.  So it is understandable that politicians of all stripes (as Mr. Waxman notes, his (and Congresswoman Eschoo's) bill are "bipartisan") would be inclined to address (or appear to address) these issues.  But suggestions that the government use "existing [FDA] authority" to cobble together a follow-on biologics pathway ignore some very real impediments to providing biosimilars that deserve approval.  Issues such as safety and efficacy, interchangeability, and even the definition of a biosimilar are not accounted for under existing U.S. law.  For example, what constitutes a "biosimilar" can depend on whether differences in primary amino acid sequence, post-translational modifications, level of impurities, the mechanism of action, and the mode of administration are or can be accommodated.  The issue of interchangeability, ensuring that a patient on the innovator biologic can be "switched" to the generic and (if need be) back again is another undefined area of great interest to the public, since the bureaucratic consideration, "increased risk of an adverse event" translates to real patients suffering real consequences if the risks are not minimized (if not eliminated).  And, of course, in view of the recent Supreme Court decision in Wyeth v. Levine, FDA approval even under well-established guidelines is not necessarily sufficient to shield a biosimilar company from liability should a "generic biologic" fail in any of these critical criteria for safety.

Even more chilling, Congressman Waxman's letter can be interpreted to mean that the Congressman is willing to cut corners and avoid the political process to impose a biosimilars regime.  The biosimilars debate is an important one, balancing multiple competing interests and, with any luck, arriving at regulations that will reduce healthcare costs while protecting the incentives to invest that are vital to continued innovation.  These are just the kinds of questions that should be debated fully, fairly and openly.  Let's hope Congressman Waxman thinks so too.