BsUFA II: What Biosimilar Makers Need From FDA
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
As more biopharma companies enter the biosimilar space, the FDA is facing greater pressure to keep up with the impending biosimilar boom. In the past few months, the industry has seen the release of the FDA biosimilar labeling draft guidance and the FDA approval of the second biosimilar in the U.S. However, behind the scenes, biosimilar makers, trade organizations, and the FDA have been hard at work on the Biosimilar User Fee Act (BsUFA II) reauthorization negotiations to ensure streamlined and productive review processes in the future.
BsUFA I was launched in 2012. This Act enabled the FDA to collect fees from biosimilar companies to aid in the assessment of development programs and applications for approval. Kay Holcombe, SVP of science policy for BIO, describes how this reauthorization process has opened a dialogue between the industry and FDA on “how both parties would like to see the biosimilar program moving forward in the next five years.”
BIO is one of several trade organizations, including The Biosimilars Forum, The Biosimilars Council, and PhRMA, representing member companies in the negotiations. BIO’s negotiation process began roughly six months ago. After learning about members’ experiences working with the FDA, BIO created a list of ideas on how the program could be improved. This list was then shared with other trade organizations to determine additional goals for the reauthorization. As Holcombe says, “Our goal is to present a unified view of how to best move the industry forward.”
How Has BsUFA Changed Since 2012?
The primary goal of the FDA when instituting BsUFA four years ago was to bring alternative, more cost-effective therapies to market. Because of Europe’s 10-year lead over the U.S. with biosimilars, the FDA has been able to observe the pricing of biosimilars versus originators and the overall reduction of healthcare spending abroad. As such, FDA’s goal for BsUFA was “to bring that sort of competition into the marketplace in the U.S.,” Holcombe explains. This still remains the overall goal of the BsUFA today.
However, as the U.S. market matures, so do the needs of companies making biosimilars. The foremost goal of these reauthorization negotiations is to ensure the FDA receives adequate funding through the user fees. The FDA has observed increased interest in biosimilar development from the industry. Indeed, in recent weeks, the agency accepted Samsung Bioepis’ infliximab biosimilar application for review. This application has now joined a stack of several other biosimilar applications awaiting a final decision. But in 2012, there was not the same rush to bring biosimilars to market as there is today. As Holcombe describes, the biosimilar industry presented some unique challenges for the creation of a user fee program. “The BsUFA program was set up differently than other user fee programs,” she says. “Because it was a brand new pathway, companies were going to have a lot of questions. The industry didn’t know what information the FDA was going to be looking for and how receptive the agency would be to the information.”
Similarly, the FDA had its own questions about how many applications it was likely to receive and how long it would take to review them. “This was a brand new concept in the U.S. — everything was based on assumptions,” says Holcombe. “This was not like the beginning of the Prescription Drug User Fee program (PDUFA).” When launching PDUFA, the FDA understood what a drug application included, how long it would take to review, who needed to review it, and how many applications would be submitted in a year. But with biosimilars, the agency was left to make assumptions. Now, there are upwards of 60 products in development. As Holcombe acknowledges, “The data are still few, but there are a lot more products in development than there were.” This leads to the question: how far off were the agency’s assumptions?
“The Crucial Question”
When BsUFA was launched, the FDA established a meeting schedule. Companies would pay the FDA to have meetings over the course of their development programs. This was in the form of an annual fee rather than a pay-per-meeting. (It was presumed if a company had one meeting with the FDA, it would continue to do so throughout its development program.) When the product was ready for reviewed, the company would pay another fee, which was the same as what would be paid under the PDUFA. (Biosimilar companies that had paid for meetings were allowed to subtract this total from their application fee.)
Now that four years have passed and the agency and biosimilar companies have become more familiar with the process, the industry is left questioning this structure. There is more knowledge over how many companies are entering or planning to tackle biosimilar development. As such, Holcombe poses one of the industry’s biggest questions: “Do we know enough where we can say, ‘We don’t need to pay the meeting fees anymore. We can just set this up like any other user fee program where the company pays a certain amount for the application and the products.’” Before a decision could be made on this, however, the industry would need to determine how these fees would be collected, and more importantly, if the agency currently knows enough to change the whole structure.
“The crucial question being posed throughout these negotiations is whether this is still the right structure to have. Should we keep it this way for the next five years and then revisit it in the next reauthorization negotiations?” Holcombe asks. “That is the crucial question.” Revamping this structure too soon could alter the course of drug development. “We don’t want to set up a system that’s going to be a disincentive for people to develop these products,” she says.
Why The Hiring Issue Poses Problems For Biosimilars
It’s no secret that the FDA’s past hiring record has proven challenging for the growth of the biosimilar market. Following the reauthorization of BsUFA II, BIO and the other negotiators are pushing for user fees to be put toward hiring the appropriate number of employees. Having the appropriate number of staff members is especially important for biosimilar approval because the review must be carried out in the same division that reviewed the reference product. “You don’t want to have a situation where the review division is overwhelmed because they’re trying to get through all the new drug applications at the same time as biosimilar applications are rolling in. Biosimilar applications may require the same amount of time to review and thought as the novel biologics up for approval,” Holcombe explains. As such, hiring has been a prime topic of discussion in BsUFA II reauthorization negotiations.
To ensure that inadequate staffing doesn’t stunt biosimilar market growth, BIO has been urging the FDA to set internal goals. This will require the agency to determine how many people, and what kind of people, it will need to be hiring this year, next year, and in the future. Ultimately, the FDA will need to have the same mindset as a company in order to predict its hiring. A business would be in big trouble if the CEO were to wake up one day and find the company short by 500 employees. “You have to be able to do capacity planning,” argues Holcombe. “You have to be able to foresee that a few years down the road you will need 50 new pharmacologists.” In order to accomplish this, the FDA will need to perform the appropriate capacity planning exercises and then establish methods for finding and recruiting new employees.
Will BsUFA II Be Radically Different?
While these reauthorizations have yet to be resolved, Holcombe does not expect this process to “turn the Act upside down.” There’s always the chance for mistakes when establishing a new program. But the reauthorization process exists to help the industry and agency make course corrections along the way. “We’ve certainly learned a lot about how things have worked and how things are evolving,” offers Holcombe. “So far, this has been a very productive process. We have gone in as a united industry. We’ve been upfront with the FDA over what we can do and what we expect, and the agency tells us what it can realistically achieve,” she says. “It’s a serious back-and-forth where everyone’s goal is to succeed, and no one wants to fail.”