FDA Reevaluates User Fees for Biosimilar Reviews

Tony Hagen

Tony Hagen is senior managing editor for The Center for Biosimilars®.

Even before the pandemic, the FDA was having trouble estimating its resource needs for biosimilar application reviews. An ongoing process may fix that.

Coronavirus disease 2019 (COVID-19) put a crimp in just about everything, including the FDA’s new drug and biosimilars review activities. In so doing, the pandemic helped make the case for an FDA project to upgrade its system for assessing workplace capacity and setting user fees required from manufacturers that make drug application submissions.

“Our staff is currently in a primarily telework environment,” the agency said in a recent statement that described the strain on its resources caused by COVID-19 and expressed uncertainty about the FDA’s ability to continue drug application reviews on schedule.

“At this time, the New Drug Program, the Generic Drug Program, and the Biologics and Biosimilars Programs are continuing to meet key review program user fee performance goals, approve applications, and communicate with applicants. With many staff working on COVID-19 activities, it is possible that we will not be able to sustain our current level of performance indefinitely,” the agency said.

Revising the User Fee Methodology

Just as COVID-19 hit, the agency had embarked on an effort to revise its methodology for determining resource and capacity needs for its human drug and biosimilar biologic review programs under the Prescription Drug User Fee Act (PDUFA) and Biosimilar User Fee Act (BsUFA).

A previous problem with these programs was that unanticipated increases in workload did not result in comparable increases in user fees collected from manufacturers who submitted review applications for new drug products, leaving the FDA with a funding shortfall.

“In other words, the agency would have more work while fee revenue remains fixed and would not be able to afford hiring the additional staff required to maintain review timeline performance,” the FDA wrote in an April 2020 statement explaining its decision to develop a new workload—user fee calculation methodology.

In 2003, a workload adjustment plan was introduced for the PDUFA program, and it allowed for adjustments based on long-term changes in the volume of regulatory submissions; however, it has been revised multiple times owing to the complexities of regulatory submissions, and studies conducted in the hopes of fine-tuning this methodology have been considered “suboptimal,” the agency said.

No such adjustment methodology was adopted for BsUFA, and in its current initiative, the FDA seeks to incorporate private sector analysis and feedback into what it believes is a more efficient plan.

In an analysis commissioned by the FDA, management consulting firm Booz Allen Hamilton has given the new workload capacity tool a thumbs-up, stating that it “comprehensively includes workload submission types in a manner that will likely forecast resource demands close to real-world figures” and is also scalable as FDA resource needs evolve or increase.

A considerable amount of money is collected from user fee programs at the FDA. For example, the base PDUFA target revenue for 2020 is slightly more than $1 billion, and the base target revenue for the BsUFA in 2020 is $40.9 million.

Change Recommendations

Booze Allen has also recommended the FDA consider creating prediction intervals (a range with upper and lower limits) for its resource demand forecasts to address future uncertainties in its estimates. The firm said the FDA should consider making its forecast models more interpretable to build public trust in its methods and suggested that it find a way, within the model, to account for work that is not directly related to drug application submissions, “such as postmarket safety and some subsets of policy and guidance development.”

The firm also recommended that the FDA make further refinements by evaluating the accuracy of user fee adjustments made in previous years, develop business scenarios to model workplace needs, and closely evaluate its ability to enlarge the agency workforce to accommodate needs.

During the month-long public comment period that closed in May, the FDA received just 1 comment, from the trade organization Pharmaceutical Research and Manufacturers of America (PhRMA). The group expressed agreement with the findings and recommendations from Booze Hamilton but called for reassurance that the resources requested by the FDA would be used for the purposes intended.

“If the [capacity planning adjustment] provides additional resources due to increases in workload for specific functions, PhRMA believes there should be an effective allocation of agency staff and resources to meet those needs,” PhRMA said in its comment. The group asked for clarification on how resource needs are determined and follow-up reports explaining how resources were allocated to those needs.

The group also requested that the FDA improve transparency on how much is being spent on overlapping needs by different departments within the agency that may be funded by multiple user fee programs. “The FDA should include a comprehensive and aggregate accounting of the cross-cutting activities and how each funding source contributes to the effort,” the group said.