Biosimilar Takeaways From Recent Energy And Commerce Hearing
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
Last week, I watched the livestream of the Energy and Commerce Committee’s hearing on the FDA’s Generic Drug (GDUFA) and Biosimilar User Fee (BsUFA) programs. These programs, which are both in their second iterations, authorize the FDA to collect fees from manufacturers to fund timely reviews and approvals of new generics and biosimilars.
This hearing featured witnesses from the heavy-hitter individuals and organizations in the biosimilar and generics space, including the FDA’s Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), and representatives from the Biosimilars Forum, the Biosimilars Council, BIO, and the newly renamed Association for Accessible Medicines (formerly GPhA).
Topics of discussion broached by members of the committee ranged from (sometimes heated) commentary on surging U.S. drug prices, to concerns about the hiring freeze and the “draconian” executive order requiring the elimination of two federal regulations for each new regulation to be enacted. But the majority of the hearing focused on the importance of the user fees' roles in accelerating the review and approval of generic and biosimilar drugs. Both GDUFA II and BsUFA II need to be reauthorized by Congress by the end of September to remain in effect.
While the overarching argument was that Congress should reauthorize these user fee programs, I came away with a few more takeaways worth noting for the biosimilar community.
Generics Were The Star Of The Show
I walked away from this hearing surprised by the amount of time spent discussing GDUFA specifics, rather than BsUFA. This isn’t to say biosimilars weren’t brought up or given the spotlight at times. But I’d argue small molecule generics and GDUFA garnered the most attention — no doubt because of the market’s 20-plus-year tenure and the agency’s highly publicized generics approval lag.
As the committee’s chairman, Texas Congressman Michael Burgess, shared, following the authorization of GDUFA in 2012, median review times for generic drug applications rose to 48 months. Only 9 percent of generic drugs were approved in the first review cycle. (This is compared to new drugs, which often receive an affirmative nod after six months and are primarily approved upon the first review cycle.)
Though quicker turnaround in the generics space is critical, I was disappointed to see so little time spent discussing the still-fledgling biosimilars market. The same committee held a hearing in January 2016 focused solely on the implementation of the Biologics Price Competition and Innovation Act (BPCIA). At that hearing, the individuals within this committee asked some insightful and important questions about the formation of the biosimilar market. I went into this most recent hearing hoping to hear some of the same. After all, the FDA’s slow release of key guidances and the small number of biosimilar approvals have both been concerns to industry and legislators alike.
Why BsUFA II Is Encouraging To The Biosimilar Industry
One of the biggest themes throughout this meeting was timeliness. A majority of questions circled around how the FDA plans to decrease review times and increase first-cycle generic approvals. But this goal was also emphasized on the biosimilar side of things, thanks to Bruce Leicher, VP and general counsel of Momenta and current president of the Biosimilars Council.
Going into BsUFA II negotiations last year, the industry called for more staff to encourage quicker reviews and a more reliable meeting schedule, and more transparency on how user fees were being spent by the agency. As Leicher highlighted, the new BsUFA II agreements promise the release of new guidances, more meeting opportunities, resource planning, and new hires.
Texas Congressman Gene Green homed in on the importance of the new meeting opportunities in particular. Ultimately, these improvements aim to enhance communication with the agency to ensure sponsors are receiving necessary development information early. This way, they are able to make any corrections prior to filing for approval. In particular, BsUFA II adopted the Prescription Drug User Fee Act’s (PDUFA) program review model. This program establishes specific communication timelines and opportunities to talk through any complicated issues in development that could keep the application from being approved.
Another important fact to note is the agency’s move from a 10-month to a 12-month review period. Leicher emphasized that this change was made to help sponsors get their biosimilars approved in the first cycle. “The agency learned in PDUFA that additional time was important to enable communication,” Leicher said. “This time helps the sponsor respond to information requests and communications within the established timeframe and finish the application the first time around. Otherwise, the sponsor could be stuck waiting an additional six months beyond the original 10-month review period.”
REMS, Hiring Freeze Pose Biggest BsUFA II Challenges
Inevitably, however, there are some notable barriers in place that could make it challenging for the FDA to maintain its BsUFA goals. The hiring freeze, which was brought up at multiple points during the hearing, is a primary concern for the FDA and some of the representatives in attendance.
It’s no secret that hiring has challenged the agency for the past five to six years. Woodcock emphasized the need for additional resources to carry out its reviews in a timely manner. “The additional interactions with the industry to help get submissions in shape the first time is a labor-intensive activity,” she stated. The hiring freeze, however, will likely cause issues in recruiting additional staff to review biosimilars.
As California Congresswoman Anna Eshoo described, “If you don’t have the staff, forget the timely review of these applications or the quick arrival of these products on the market.” As such, she urged advocates of the majority party to relay the serious impact these hiring freezes could have on approval of more cost-effective drugs.
Risk Evaluation and Mitigation Strategies (REMS) programs were another topic garnering considerable attention during this hearing. Comparator sourcing has become a notable challenge in the biosimilar space, and is an ongoing issue for small molecule generics. Certain levels of these REMS programs (not all) can restrict general access because the innovator drugs may have high-risk profiles. As such, innovators have used REMS programs to directly or indirectly delay or keep generics makers from receiving the comparators for determining bioequivalence to the reference product.
Unfortunately, these instances are not easily stopped by the FDA. The agency is only capable of determining that the generic company’s protocol for testing the innovator is acceptable. FDA officials then write a letter to the innovator company emphasizing the safety of this protocol and highlighting the inappropriate use of REMS. The agency is unable to compel the reference company to give up batches of the drug to the generic or biosimilars company.
As Leicher argues, however, challenges with comparator sourcing extend beyond REMS. Securing an adequate amount of the comparator is a much bigger problem in the biosimilar space. While small molecule generics need a small quantity of the reference drug to run analytical testing, biosimilar development requires a large quantity and numerous batches of the innovator in order to run analytical tests and blinded clinical trials. In many situations, when a company approaches a wholesaler to purchase the innovator, it’s being told it’s not permitted to receive the innovator because it’s doing biosimilar testing.
“This is why we strongly support the Fair Access for Safe and Timely [FAST] Generics Act or the Creating and Restoring Equal Access to Equivalent Samples [CREATES] Act as a solution to make that practice unlawful,” said Leicher. “It should be a condition of approval that products are made available to licensed, FDA-regulated companies that are creating biosimilars.”