FDA Wraps Up Responses to 2003 & 2004 Petitions on “Biosimilars”; Denies Outstanding Requests

By Kurt R. Karst –      

Just days after FDA announced the release of three draft guidances on biosimilar product development (see our previous post here), and on the same day FDA officially noticed their availability in the Federal Register (here, here, and here) (as well as a proposed collection on § 351(k) biosimilar applications), FDA denied the outstanding requests in two citizen petitions submitted to the Agency in April 2003 (Docket No. FDA-2003-P-0003) and April 2004 (Docket No. FDA-2004-P-0214) by the Biotechnology Industry Organization (“BIO”) and Genentech, Inc. (“Genentech”), respectively.  Both petitions were submitted to FDA well before the March 2010 enactment of the Biologics Price Competition and Innovation Act of 2009 (“BPCIA”), which amended the Public Health Service Act (“PHS Act”) to, among other things, create an approval pathway for biosimilar and interchangeable versions of reference products (§ 351(k) biosimilar applications).  As such, the petitions primarily pertain to the submission and approval of 505(b)(2) applications under the FDC Act for “biotechnology-derived products” (i.e., therapeutic drug or biological products developed by biotechnology methods) or “follow-on therapeutic proteins” (i.e., polypeptide therapeutic products, including recombinant versions of therapeutic protein products, not submitted to FDA with a full complement of clinical data).

FDA had previously addressed most of the issues raised in the petitions in the Agency’s October 2003 and May 2006 petition responses (here and here); however, two outstanding requests remained:

(1) that FDA decline to publish a draft guidance regarding the “similarity” or “sameness” of a “biotechnology-derived product” or “therapeutic protein” that relies on confidential or trade secret information; and

(2) that FDA provide advance notice and a pre-deprivation hearing if FDA does decide to issue such a guidance using trade secret or confidential information.

Genentech broadly requested in its petition that FDA “refrain from publishing a draft guidance document setting forth standards for the ‘similarity’ or ‘sameness’ of biotechnology-derived products. . . that relies, directly or indirectly, on Genentech’s trade secret and confidential commercial data.”  FDA says in its 2012 petition response that Genentech “suggests that any guidance document FDA creates on this subject impermissibly will be infused with protected confidential and trade secret information.”  Any such argument must fail, however, says FDA, because the Agency’s “mandate is to protect the public health in the areas within its purview – including biotechnology-derived products – and it may, among other things, create guidance to industry regarding the laws it administers.”  With respect to the draft biosimilars guidances issued on February 9, 2012, FDA comments that they “do not violate any protections available to applicants by relying, either ‘directly or indirectly,’ on Genentech’s or any other entity’s confidential information. . . [,] do not disclose any trade secret or confidential information, and [that] FDA did not impermissibly use or rely upon any such information in developing them.”

BIO more specifically requested in its 2003 citizen petition that FDA “refrain from preparing, publishing, circulating, or issuing any new guidance for industry, whether in draft or final form, concerning follow-on applications for therapeutic proteins, particularly human growth hormone or insulin, under [FDC Act § 505(b)(2)] which was intended simply to codify FDA’s earlier ‘paper NDA’ policy.”  Insofar as BIO’s request is limited to guidace involving 505(b)(2) applications for therapeutic proteins, FDA comments in the Agency’s February 2012 response that FDA has general authority to issue guidance and notes that “if FDA deems such an exercise of its authority appropriate or necessary, the Agency may prepare, publish, circulate, or issue new draft or final guidance for industry concerning applications for therapeutic proteins (or any other subset of products) eligible for approval under [FDC Act § 505(b)(2)].”  Insofar as BIO’s petition more generally requests that FDA refrain from issuing guidance on class-specific biological products, FDA points to the text of the BPCIA, which permits the Agency to issue “specific” guidance “with respect to the licensure of a biological product under [PHS Act § 351(k)].”  Perhaps signaling future guidance, FDA comments that while the Agency “thus far has not published draft guidance on the subject of product class biological products, the [BPCIA] clearly contemplates that the Secretary may issue this type of guidance.”

Genentech specifically requested in its citizen petition that FDA provide advance notice and a pre-deprivation hearing if the Agency decides to issue guidance on biotechnology-derived product/therapeutic protein similarity/sameness issues using trade secret or confidential information.  In FDA’s February 2012 petition response, the Agency declined to address Genentech’s Constitutional and trade secret law arguments, and instead reasserted that that Agency “acts within its authority when it issues guidance documents in its areas of scientific expertise for purposes of executing its mandate as an agency.”  Moreover, says FDA, the draft biosimilars guidances “do not implicate, disclose, or rely upon Genentech’s (or any other company’s) confidential commercial or trade secret information.”