Prescription Drug User Fee Act (PDUFA V)
In 1992, Congress authorized the Food & Drug Administration to charge a fee to pharmaceutical companies upon submission of an application to market a new drug in a statute titled the Prescription Drug User Fee Act (PDUFA). Revenue raised from the fee program must be used to expedite the drug development process and the review of human drug applications. This expansion in FDA’s capacity to review new products has led to a dramatic decrease in the time required to review applications for new drugs while maintaining or improving standards for the safety and efficacy of new drugs. This means that new drugs approved by FDA are now available in the marketplace sooner than was possible before passage of PDUFA, with the time required for review having been cut in half in most cases.
Since 1992, Congress has reauthorized PDUFA three times, and the Act will require a fourth reauthorization in 2012 as PDUFA V. Without reauthorization, FDA will be unable to fulfill its mission. Previous reauthorizations have increased the fees to keep up with inflation, expanded the program to include biologic products, and, importantly for the rare disease community, exempted new drug applications for orphan products from all application fees. This is an important milestone that NORD helped to accomplish to further incentivize the development of orphan products. Learn more about PDUFA.
On July 7, 2011, the United States House of Representatives Health Subcommittee of the Committee on Energy & Commerce held its first hearing on the Reauthorization of PDUFA. Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research, US FDA, testified before the subcommittee during the hearing. Her written testimony contains several comments on the development of drugs for rare diseases, and may be found here.
During the January 11, 2011 stakeholders meeting between FDA and patient advocate groups, information about the Rare Disease Program proposal was discussed. NORD strongly advocates that the proposal be included as part of PDUFA. The proposal would direct FDA to allocate resources toward the development of specific guidelines for the review of Orphan Products. This proposal will lead to a more efficient FDA where rare diseases are concerned and provide a more comprehensive process for innovators to develop new products that treat rare diseases. Minutes from the meeting can be found here.
FDA recently released a draft document summarizing its position to date on the PDUFA reauthorization. NORD responded with a statement of support for the content related to rare diseases and orphan products on page 23 of the document. Read the FDA draft and, in particular, the section on rare diseases on page 23.
On February 1, 2012, NORD Vice President for Public Policy Diane Dorman was one of eight witnesses, and the only patient representative, invited to present testimony on the PDUFA V reauthorization. Read the statement, or view the video from the hearings (Diane begins reading her statement at 3:07:35).
On April 30, 2013, NORD signed-on to the following letter. Read the letter.
