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Regulatory Focus™ > News Articles > PDUFA VI: FDA Hears Stakeholder Comments in First Public Meeting

PDUFA VI: FDA Hears Stakeholder Comments in First Public Meeting

Posted 15 July 2015 | By Michael Mezher 

PDUFA VI: FDA Hears Stakeholder Comments in First Public Meeting

The US Food and Drug Administration (FDA) today heard from various groups representing patients, healthcare professionals and the regulated industry for their input on the iteration of the Prescription Drug User Fee Act (PDUFA).


In the 1980s, the US lagged behind Europe in drug approvals, and individual drug reviews often took years to complete. These lengthy approval times were a significant source of frustration not only for patients and drug companies, but for FDA itself.

The problem, FDA argued, was that the agency lacked adequate funding to hire the staff it needed to review drugs in a more timely manner. The solution FDA proposed was to collect one-time fees from the companies it regulates for each new drug approval, which would provide the agency with a substantial source of funding to boost its staff and reduce review times.

While drugmakers had previously opposed attempts by FDA to collect fees for reviews, they eventually came to support the fees, with the condition that FDA would adhere to tighter timetables for completing its new drug reviews: six months for priority applications and 12 months for non-priority applications.

In 1992, PDUFA was passed by Congress and signed into law with a five-year mandate. Since then, PDUFA has been reauthorized four times. PDUFA is unique compared to other legislation that requires fees from industry in that FDA and the regulated industry negotiate the amounts of the fees directly, rather than the fees being set by lawmakers.

Overall, PDUFA has been a major success. Now, most new drugs are approved in the US before other countries, and FDA is consistently faster than its peers in reviewing new drugs.

The current act, PDUFA V, is set to expire in 2017, and introduced new opportunities for communication between FDA and drug sponsors, improvements to the Risk Evaluation and Mitigation Strategies (REMS) plans introduced in PDUFA IV and greater inclusion of patients' perspectives in the drug review process.


Today's event was the first public meeting on the PDUFA reauthorization process.

The meeting began with opening remarks from FDA's Acting Commissioner Stephen Ostroff, who called PDUFA a great facilitator of healthcare innovation and touted the agency's performance in approving new drugs.

Afterward, various stakeholders, including the Pew Charitable Trusts, National Organization for Rare Disorders (NORD), The Pharmaceutical Research and Manufacturers of America (PhRMA), the Biotech Industry Organization (BIO) and the Alliance for Regenerative Medicine (ARM) were able to present their views on PDUFA.

Prior to the meeting, FDA asked the public to address several questions:

  • What is your assessment of the overall performance of PDUFA V thus far?
  • What current features of PDUFA should be reduced or discontinued to ensure the continued efficiency and effectiveness of the human drug review process?
  • What new features should FDA consider adding to the program to enhance the efficiency and effectiveness of the human drug review process?

Industry Perspectives

Speaking on behalf of the pharmaceutical industry, Sascha Haverfield, vice president for scientific and regulatory affairs at PhRMA, praised PDUFA for its role in improving FDA's performance over the past two decades. The fees gathered through PDUFA were instrumental in FDA's dramatic increase in drug review staff, from roughly 1,900 in 1989 to more than 3,700 as of last year.

Improvements aside, Haverfield cautioned that more must be done to "gain a deeper understanding of diseases and condition elements that are of the greatest burden to patients." Building on the progress made under PDUFA V, Haverfield says that "additional clarity is needed on how to translate patient preference information into tangible outcomes."

To do so, PhRMA says it will support policies in PDUFA VI that:

  • "Better integrate the patient perspective in drug development and regulatory decision-making;
  • Enhance the scientific expertise, processes, and tools FDA uses to regulate increasingly complex medical products and public health issues; and
  • Promote the long-term stability of the PDUFA program by improving its financial transparency, efficiency, and accountability and ensuring FDA can recruit, hire and retain a highly-skilled workforce to advance its public health mission."

Next, Kay Holcombe, senior vice president for science policy at BIO, urged FDA to build on the success of PDUFA V by facilitating earlier access to innovative medicines. Holcombe also stressed the importance of keeping FDA funded, pointing to the period of time in 2013 when part of FDA's funding was sequestered.

Speaking on behalf of ARM, Michael Werner called for specific measures to improve the development of regenerative medicines, including:

  • "Establishment of a Standards Coordinating Body for [regenerative medicines]."
  • "Creation of a Qualified Regenerative Medicine Product designation whose sponsor can receive assistance from FDA."
  • "Improved coordination and communication among FDA review centers."

PDUFA Meeting 15 July 2015

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