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Biosimilar User Fee Agreement Reached, But Will Require Appropriations Boost


Executive Summary

Tentative agreement closely resembles FDA's original proposal.


FDA would have to receive about 11 times more in dedicated federal funding for biosimilar application reviews than fiscal year 2011 in order to collect user fees, if the tentative agreement awaiting stakeholder ratification is implemented.

The agency and industry reached what was termed a "tentative agreement, pending ratifier approval," on statutory language to create the user fee program and proposed performance goals at an Aug. 26 meeting, according to minutes released Sept. 21.

The agreement includes an appropriations trigger that would require at least $20 million a year in "non-user fee funds," i.e. federal funding, to be devoted to biosimilar product reviews. That figure also would be adjusted for inflation, according to the minutes. Without the federal money, FDA would not be allowed to collect or spend biosimilar user fees.

A similar trigger is included in the Prescription Drug User Fee Act legislation to ensure that user fees supplement, rather than supplant, appropriations.

Assuming the agreement is ratified and the program launches in FY 2013, Congress would be forced to devote significantly more money to the biosimilar program. In FY 2011, only $1.8 million was allocated. Still, FDA has stated it could accept biosimilar applications and had been talking to several sponsors ("FDA “Busy” With Biosimilars; Sponsors Already In Pre-IND Meetings" — "The Pink Sheet," May 16, 2011).

The new user fee program also would be separate from the others, namely PDUFA, according to the minutes.

The Generic Pharmaceutical Association and a Momenta Pharmaceuticals representative argued at previous meetings that Congress intended the program to remain part of PDUFA throughout the first five-year program cycle. The Biotechnology Industry Organization and Pharmaceutical Research and Manufacturers of America disagreed, saying the intent was for a separate program.

The issue lingered through a negotiating session in early August, but appeared to be resolved by Aug. 8 ("Biosimilar Product Development Meetings With FDA To Be Flexible Under User Fee Program" — "The Pink Sheet" DAILY, Sep. 15, 2011).

Ratification Questions?

GPhA has ratified the agreement and BIO's board of directors is considering it, according to officials at the two groups. PhRMA refused to comment about the negotiations.

Ratification could become a thorny issue because of the nature of the negotiations. FDA allowed any trade group or company with a stake in the biosimilar market to sit at the negotiating table because the sector is fragmented and still emerging in the U.S.

A relatively small group of stakeholders emerged during the negotiations, including BIO, PhRMA and GPhA. Representatives from 10 pharmaceutical companies also attended the Aug. 17 and Aug. 26 meetings.

FDA did not respond to questions Sept. 22 about whether the companies that participated in the negotiations will be required to ratify the agreement. After it is ratified, the agreement will go to the Department of Health and Human Services and White House for clearance, then to Capitol Hill.

Ratification efforts also appear to have started about a month later than FDA hoped. The agency had wanted to finish the talks by the end of July ("Biosimilar User Fee Negotiations Under Way; Timetable Compressed" — "The Pink Sheet," Jul. 4, 2011).

The biosimilar user fee agreement likely will be included in an omnibus user fee bill that also will include reauthorizations for PDUFA and the Medical Device User Fee and Modernization Act, as well as the new generic drug user fee program.

FDA and industry completed PDUFA negotiations in late March, but added another provision in mid-May. Generic user fee negotiations finished in early September ("ANDA User Fee Negotiations Finished; Backlog To Be Mostly Cleared By Year Five" — "The Pink Sheet," Sep. 12, 2011).

Fee Schedule Largely Resembles Original Agency Proposal

The program would include an application fee, product fee and establishment fee, according to minutes of an Aug. 17 negotiating session. It also would include the new product development fee to be collected annually as long as an application is in active development. FDA proposed collecting the fee at that time of the IND filing in order to support pre-marketing application scientific activities at the agency.

FDA proposed setting the application, product and establishment fees equal to the corresponding PDUFA amounts each fiscal year. The product development fee would be 10% of the PDUFA application fee in a given fiscal year, according to the Aug. 17 minutes.

FDA set the cost of an application requiring clinical data at $1.842 million for FY 2012, an increase of 19% from the previous year ("NDA, BLA Submissions Expected To Fall Again in FY 2011" — "The Pink Sheet" DAILY, Aug. 1, 2011).

The biosimilar user fee proposal largely fits with the agency's original idea for the program. Biosimilar applicants were meant to be charged the same as BLA sponsors, with some of the money collected before the marketing application is filed ("Biosimilar User Fees Would Be Same As BLAs, Only Earlier" — "The Pink Sheet" DAILY, May 9, 2011).

GPhA had argued biosimilar fees should be 50% to 65% of the full BLA fee, in part to reflect that biosimilar data packages are expected to be less extensive ("Biosimilar Product Development Fee Should Be Temporary, Trade Groups Say" — "The Pink Sheet," Jun. 20, 2011).

FDA also intends to discount product development fee payments from the marketing application charge. A reactivation fee, set at twice the product development fee, also was included in the proposal for applications that had fallen out of active development after IND filing, but had been restarted, according to the Aug. 17 minutes.

Reactivation fees also would be subtracted from a marketing application charge.

Performance goals also were set for the program, but not specified in either set of meeting minutes. In addition to review, major dispute resolution, clinical hold and meeting management goals, both sides also agreed to a special protocol assessment goal and created a new formal meeting to address stalled biosimilar development programs.

Both sides already had decided meetings would not be required at specific times or for specific purposes during the IND phase to give sponsors more flexibility.

- Derrick Gingery (d.gingery@elsevier.com)