A group of Senators led by Sen. Lamar Alexander (R-TN), the Chairman of the Senate Committee on Health, Education, Labor, and Pensions, sent a letter last month to Dr. Stephen Ostroff, the Acting Commissioner of the U.S. Food and Drug Administration, "to express concern and raise questions over how the Food and Drug Administration (FDA) is implementing critical elements of the biosimilar product pathway established by the Biologics Price Competition and Innovation Act [BPCIA]."  In particular, the Senators were concerned about the FDA's failure to resolve "unsettled questions about the biosimilar approval pathway," including the failure to provide policies on nonproprietary naming of biosimilar products, interchangeability, and production of patent information.

Noting that members of Congress had sought guidance on these issues in 2013 and 2014, the letter points out that "[d]espite this repeated urging, FDA did not make public its policy on the review and approval process before moving forward with the first approval of a biosimilar product" (see "The First Biosimilar Application Has Been Approved -- But What About the Patent Issues?").  The Senators suggest that "FDA's failure to resolve fundamental science and policy questions prior to approving a biosimilar for the first time last month raises a number of serious concerns."  Among those concerns is the FDA's use of a "placeholder" nonproprietary name for the biosimilar it just approved.  The letter states that "[i]t is unclear to us what it means for a nonproprietary name to be a 'placeholder,'" and asks "what authority FDA has to make such a designation."  The Senators also express "concern[] that hospitals, consumers, patients, doctors, and others may be confused by a name that appears temporary or not fully approved."

While noting that the FDA recently released the first final guidance on the biosimilar pathway, the Senators also point out that "much of the guidance FDA has provided on the biosimilar pathway remains in draft form."  And as for the three finalized guidance documents, the Senators note that "FDA did not finalize these documents until after reviewing and approving the first biosimilar application."  The letter continues:

FDA's failure to issue complete and final guidance before beginning to review and approve biosimilar applications raises significant questions.  In particular, it is not clear to what extent FDA staff has been following draft guidance when reviewing biosimilar applications.  And, if FDA staff has not been following draft guidance when reviewing applications, it is not clear what agency policies, if any, have been governing the process.

The letter closes by listing a series of questions and requesting that the FDA provide answers to those questions by May 22.  Among the questions posed by the Senators are the following:

• What is a "placeholder" nonproprietary name, and what is FDA's legal authority to issue and/or to change such a name?

• What guidelines have FDA staff members been following in reviewing biosimilar applications?

• What guidance documents regarding biosimilar or interchangeable products does FDA currently intend to publish, and on what schedule?

• Why has FDA declined to provide guidance regarding whether the information exchange provisions of Section 351(l) of the PHSA are mandatory?

In addition to providing several questions for the FDA to answer, the Senators "urge FDA to prioritize the publication of final guidance on the issues identified [in the letter], and to improve the transparency of its biosimilar review and approval process going forward."

The text of the letter was made available by the FDANews (see "GOP Senators Request FDA Clarification of Biosimilars Policy").