Biosimilar User Fees: Public Meeting Kicks Off Negotiations for BsUFA II

The US Food and Drug Administration (FDA) began negotiations on the second biosimilar user fee program on Friday with a public meeting highlighting some of the roadblocks the agency has seen so far and what remains to be done to help make the US biosimilar market more robust.

The current legislative authority for the Biosimilar User Fee Act (BsUFA) expires in September 2017, so FDA has to work with a number of stakeholders (industry, patients, academia, etc.) to devise a new program that provides FDA with the revenue it needs to hire more staff, ensure the timely review of biosimilar applications, and improve its performance goals on reviewing applications and meeting with sponsors.

Since the US biosimilar market is still in its infancy (FDA has only approved one so far, which is less than what the agency had predicted), the agency uses a lot of the revenue from the current user fee program to engage with industry early in the development process.

And as Leah Christl, associate director for therapeutic biologics at FDA’s Center for Drug Evaluation and Research (CDER), noted on Friday, with 59 biosimilar programs currently in some stage of development, FDA has so far underestimated the extent of the market.

But FDA’s cautious approach so far seems to be intentional. Janet Woodcock, director of CDER, explained to Senators in September that FDA is still working to ensure the scientific framework is "bulletproof" as it works with sponsors to develop the US biosimilar market.

Progress on BsUFA I

At the public meeting on Friday, David Gaugh, VP of the Generic Pharmaceutical Association (GPhA), offered an in-depth review of FDA’s work so far under BsUFA, noting that policy efforts and supporting sponsors’ development programs has consumed the greatest amount of FDA’s resources.

As far as how well FDA has met its performance goals, the agency clearly has work to do.

“To date, 8 BLAs [biologics license applications] have been announced as submitted to FDA and the action date for five of these submissions has passed,” Gaugh said. “We only know the review outcome and timing for two of these five. And for the two, FDA has completed their review within the 10-month goal – Sandoz’s filgrastim with an approval and Pfizer’s epoetin alfa with a complete response letter. Of the remaining three, we do not have public information concerning their status.”

In addition, FDA has issued four final and eight draft guidance documents on biosimilars, plus a proposed rule on nonproprietary naming, though the agency has yet to issue guidance (despite saying it would at the outset of 2015) on interchangeability, labeling and statistical considerations for demonstrating analytical similarity.

Gaugh also called on FDA to prepare guidance to address life-cycle management or post-approval requirements for biosimilars.

On the sponsor meetings front, however, Gaugh noted that interactions under BsUFA have been constructive and can lead to extensive feedback to improve companies’ dossiers.

And as far as the 10-month reviews for biosimilars, which is two months less than what other sponsors expect for new drug applications, GPhA says the timing is “justified” because of these early interactions between FDA and industry.

Expectations for BsUFA II

Although the majority of speakers on Friday discussed policy issues (which is what FDA has said should not be included in the negotiations), the overall sentiments from nearly all of the speakers remain the same: How can FDA ensure the timely review of biosimilars, protect the US from any unexpected immunogenicity issues or other tracking problems and ensure the market begins to take off as it has in the EU?

Kay Holcombe, SVP for science policy at industry group BIO, said that it would be helpful for FDA to offer more information on what BsUFA resources have been used in the development of policies.  

And with little visibility into the amount of time it actually takes FDA to review biosimilars because the market has yet to mature, Holcombe said it may be difficult to determine “whether the current [BsUFA] structure is appropriate for long-term sustainability.”

Regardless, what Woodcock and other speakers made clear: FDA needs to use at least some of these biosimilar user fees to hire and retain scientists that can work on biosimilars, particularly as they are highly sought-after by industry, which offers more competitive salaries.

The docket for public comment on BsUFA is open until 20 January 2016.

BsUFA

BIOSIMILAR BIOLOGICAL PRODUCT AUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FISCAL YEARS 2013 THROUGH 2017