Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Initiative is one step in agency’s road to developing the final abbreviated approval pathway for biosimilars.

More than a year after launching a dialogue with industry regarding biosimilars, FDA is holding a morning-long public meeting today. The proposed approval pathway and fees drug developers must pay for the five fiscal years starting October 1, 2012, will be discussed. The agency is soliciting public comment through January 6, 2012

Those comments are expected to shape a final FDA recommendation on biosimilar user fees, which the agency plans to send to Congress by January 15, 2012. On December 7, the agency published “Biosimilar Biological Product Authorization Performance Goals and Procedures, Fiscal Years 2013 through 2017.”

The agency noted, “The proposed biosimilars user fee program for FYs 2013 through 2017 addresses many of the top priorities identified by public and industry stakeholders and the most important challenges identified within FDA.”

Companies aren’t expected to disagree too much with the framework, which was developed by FDA, representatives from several biopharma companies, the Biotechnology Industry Organization (BIO), and the Pharmaceutical Research and Manufacturers of America (PhRMA).

“PhRMA supports the development of a robust user fee program for biosimilar products to provide FDA with the resources needed to review biosimilars without diverting resources from the review of innovative medicines,” Matthew Bennett, an svp with the organization, told GEN.

The user fee program is expected to aid FDA in developing the final abbreviated approval pathway for biosimilars, which was required under the Biologics Price Competition and Innovation Act (BPCIA) of 2009. BPCIA was tucked into page 686 of the Patient Protection and Affordable Care Act enacted last year by President Obama. Janet Woodcock, M.D., director of FDA’s Center for Drug Evaluation and Research co-authored a paper published this August in The New England Journal of Medicine that provided some clues on the overall approval pathway.

User fee funding would supplement dedicated nonuser fee funding to ensure sufficient resources for the agency’s biosimilars review program. In each fiscal year, in order to spend biosimilar user fees, FDA would be required to have available and allocate at least $20 million, adjusted for inflation, in nonuser fee money for biosimilar review activities. In FY 2011, FDA received an appropriation of $1.8 million plus the use of four full-time staffers for biosimilar product review. Hence, the agency is proposing a phased approach to implement performance goals for biosimilars, commensurate with the availability of additional funds to increase staffing and program review capacity.

“We recognize FDA having adequate dedicated and independent resources to devote to meeting with biosimilar sponsors and timely review of those applications will help ensure successful implementation,” Andrew Emmett, BIO’s managing director of science and regulatory affairs, explained.

User Fee Proposals

FDA envisions creating four categories of user fees. The first would be biosimilar product development (BPD) fees, starting when sponsors submit an IND or meet with FDA. The initial fee would be 10% of the fee established for a drug application under PDUFA each year from FY 2013 through 2017. The agency would collect only one initial BPD fee per product, regardless of the number of proposed indications.

Sponsors would also pay FDA an annual biosimilar product development fee—10% of the PDUFA fee—until they either submit a marketing application or end participation in the BPD program for the product.

Second, sponsors that submit marketing applications would pay fees equal to those established for drug applications under PDUFA minus the cumulative amount of BPD fees. Under PDUFA, 2012 fees for drug products go up as high as $1.84 million.

“By providing FDA with these resources, they would be able to meet with sponsors, provide clear and established guidelines for regulatory action, and as a result that should reduce the barriers to market entry even more than what would be represented through a modest fee like this,” Emmett said. Since established biopharma companies are more likely to produce biosimilars than early-stage companies, “I wouldn’t anticipate that $180,000 would be a significant barrier to market,” Emmett added.

The final two fee categories are establishment fees and product fees, both of which would be equal to those paid by drug developers under PDUFA for any fiscal year. “FDA anticipates a modest level of funding from these sources initially because only biosimilar biological products that are approved for marketing would be subject to these fees,” the agency said.

FDA’s Promises

In return for collecting user fees, FDA agreed to a series of goals for its review of applications for new and resubmitted biosimilar products. The agency committed to reviewing and acting upon 70% of original and resubmitted biosimilar submissions within 10 months of receipt in FY 2013. That percentage is expected to climb to 80% in FY 2015, 85% in FY 2016, and 90% in FY 2017.

For original and resubmitted supplements to applications, FDA committed itself to reviewing and acting upon 90% of original supplements with clinical data within 10 months of receipt date. That timeframe drops to six months for resubmitted supplements with clinical data and for original manufacturing supplements.

For original biosimilar applications and supplements with clinical data, FDA said it will inform applicants of “substantive” issues identified during initial filing reviews within 74 calendar days of receipt. If the review turns up no substantive review issues, FDA will notify applicants.

“The draft FDA performance goals are consistent with Congressional intent to create a unique user fee program to meet the needs of biosimilar product applicants and to provide FDA with the means necessary to build, essentially from scratch, its capacity for science-based review of biosimilar applications,” Bennett said.

Developing a Biosimilar

FDA has also set performance goals and procedures for meetings related to biosimilar development programs. Following a Biosimilar Initial Advisory Meeting, the agency anticipates holding:

  • “Type 1” meetings with sponsors trying to revive stalled drug development programs;
  • “Type 2” meetings to discuss specific issues such as proposed study design or endpoints or questions where FDA will provide what it calls “targeted advice;”
  • “Type 3” in-depth data review and advice meetings; and
  • “Type 4” meetings to discuss the format and content of a biosimilar application or supplement.

“These meetings relate to sponsors’ biosimilar development programs and are not mandatory,” Mahoney explained. “Sponsors choose the combination of meetings matching their specific development needs.”

The additional pre-application meetings should help address industry’s longstanding lament, as with the traditional drug development process: FDA keeps companies in the dark for long periods of time, only to respond with demands for additional data or studies much further down the sponsor’s development road. That sort of red tape would be unwelcome where biosimilars are concerned as well.

It is hoped that steps to define user-generated funding for the biosimilar approval program and development goals for developers will also aid FDA in fulfilling the promise made in BPCIA of creating a biosimilars pathway that balances innovation and consumer interests.

Alex Philippidis is senior news editor at Genetic Engineering & Biotechnology News.

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