In this CDER Conversation, Dr. Sarah Yim, M.D., director of the Center for Drug Evaluation and Research (CDER) Office of Therapeutic Biologics and Biosimilars discusses BsUFA.

BsUFA allows FDA to collect user fees, which create a reliable funding source that helps ensure the timely review of biosimilar and interchangeable biological products.

Can you explain BsUFA and the reauthorization process?

Certainly. BsUFA stands for the Biosimilar User Fee Act and was originally enacted in 2012 for a period of five years. This year marks our second reauthorization – BsUFA III.

In short, BsUFA is a law that authorizes FDA to collect fees from companies developing biosimilar and interchangeable biological products.

But much more than that, BsUFA provides additional revenues so that FDA can hire more staff, improve systems, and establish a better-managed biosimilar biological product review process to make important therapies available to patients sooner without compromising review quality or FDA’s high standards for safety, efficacy, and quality. As part of FDA’s agreement with industry during each reauthorization, the agency agrees to certain performance goals. These goals apply to the process for the review of new biosimilar biological product applications, resubmissions of original applications, and new and resubmitted supplements to approved applications.

Currently, FDA is operating under the first reauthorization of BsUFA - BsUFA II - for fiscal years 2018 through 2022. The upcoming reauthorization will allow FDA to continue to assess and collect fees for biosimilar biological products from October 2022 through September 2027. FDA dedicates these fees to continue to perform and make improvements related to the biosimilar and interchangeable product development and approval process.

How do biosimilars, and more specifically BsUFA benefit public health?

Biosimilars are biological products and are safe and effective medications for treating many illnesses such as chronic skin and bowel diseases, arthritis, kidney conditions, and cancer. Biosimilars can provide important public health benefits and have the potential to offer life-saving or life-altering treatments at reduced cost to patients. BsUFA facilitates the development and approval of safe and effective biosimilar products for the American public.

As part of FDA’s review process, biosimilar products, undergo a rigorous evaluation to ensure the safety and efficacy of these products. FDA closely regulates the manufacturing of biological products, including biosimilar and interchangeable products. Biosimilars, like all biological products, must be manufactured in accordance with Current Good Manufacturing Practice requirements, which cover methods, facilities and controls for the manufacturing, processing, packaging, or holding of a drug product. This helps to prevent manufacturing mistakes or unacceptable impurities, and to ensure product quality.

Related, we’ve heard of the Purple Book. How does this tie into BsUFA and your other work with biosimilars?

Yes, great question. Before I get into the Purple Book, I want to take a minute to talk a little about the FDA’s Biosimilars Action Plan, or BAP. The BAP summarizes the actions FDA has taken and outlines actions that we will take to advance FDA policies on biosimilar products to help make the development process more efficient and facilitate greater competition in the biologics marketplace. The BAP includes four main goals and several key deliverables. One of the deliverables in the BAP is to develop an enhanced Purple Book that includes more information about approved biological products and will provide a modernized, interactive user experience. Our BsUFA II commitments also include goals related to the Purple Book to publish information about newly approved or withdrawn Biologics License Applications or BLAs and about reference product exclusivity determinations.

Addressing these goals and commitments, earlier this year, FDA released The Purple Book: Database of Licensed Biological Products, which is a searchable, online database that contains information about FDA-licensed (approved) biological products, including biosimilar and interchangeable biological products, in addition to all FDA-licensed allergenic, cellular and gene therapy, hematologic, and vaccine products. The new database provides users with a public-facing database that includes more robust data about each product entry. The updated database includes information about product presentations, strength, and dosage form, in addition to other searchable and sortable data fields.

Ultimately, the Purple Book can help patients find information about a biological product they are currently taking or may be prescribed, and to view options for FDA-approved biosimilar and interchangeable products, once available. Prescribers and pharmacists can use it to see all associated products for a biological product they prescribe, dispense, or are considering prescribing for a patient, and, once we have approved interchangeable products, the Purple Book will provide information related to the substitution of those products at the pharmacy.

Good to know. Back to BsUFA. How would you say BsUFA II has set the stage for BsUFA III?

With BsUFA II we focused on a number of key areas including:

• Continued improvements to review processes and procedures, meeting management goals, and processes for the review of biosimilar biological products.
• Implementing an application review model for original 351(k) BLAs to promote the efficiency and effectiveness of the first cycle review process and minimize the number of review cycles necessary for approval.
• BsUFA II offers drug sponsors clearer pathways for biosimilar biological product development, improved predictability and consistency across drug review processes, and Americans increased access to safe, effective, and high quality biosimilar and interchangeable biological products.

The additional experience we’ve had with biosimilar product development and review within the agreed-upon BsUFA II processes will have identified areas or processes which we can refine further, or gaps that may need to be addressed, which may require a change or shift in resources that we will have the opportunity to address during the reauthorization process. So from a starting point of the processes, commitments, and timelines described in the BsUFA II commitment, a first question is what should be continued and what should be discontinued or changed (and if the latter, how should it be changed). Then a second question is, what are we missing that would be important to enable the biosimilar and interchangeable biologic product development program to succeed in meeting its intended public health objectives of increasing the availability of high quality, safe, and effective biologic treatment options and increasing patient access to these important therapies.

What can we expect to see with the BsUFA III reauthorization?

The current legislative authority for BsUFA expires in September 2022. There is an upcoming public meeting November 19, 2020 on the reauthorization of BsUFA for fiscal years 2023 through 2027 that marks the beginning of the negotiation and reauthorization process.

The purpose of this virtual public meeting is to provide interested stakeholders an opportunity to offer their views on the program’s reauthorization as FDA considers which, if any, features to propose, update, or discontinue in the next BsUFA. The BsUFA III meeting agenda will include presentations by FDA staff and panels representing multiple stakeholder groups, as well as time allotted for public comment and discussion. Public comments will also be accepted through December 19, 2020 by submission to docket No. FDA-2015-N-3326.

Registrants will receive instructions to access the live webcast of the virtual meeting. The meeting’s agenda, webcast information, additional materials, and any other updates will be posted to FDA’s website as they become available.

There will likely be proposals regarding potential enhancements or efficiencies for the biosimilar product development and review process, additional proposals toward maximizing scientific and regulatory clarity, and other enhancements such as a possible regulatory science program to provide additional information that could enable more efficiency in safety or efficacy assessment - such as data from real-world experience, pharmacodynamic biomarkers, and immunogenicity response. FDA is looking forward to the opportunity to discuss these and other proposals that will continue to make the biosimilars program successful and to enable increased treatment options for patients.