AAOS Now

Published 7/1/2016
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Judi Buckalew, BSN, MPH, CAE

2017 MDUFA Reauthorization Meetings Get Early Start

Anticipating delays typically experienced during presidential elections, Congress and the U.S. Food and Drug Administration (FDA) are starting the 2017 Medical Device User Fee Amendments (MDUFA) Reauthorization negotiations nearly 2 years ahead of schedule.

In September 2015, FDA held its first public MDUFA IV reauthorization meeting, which focused on introductory commentary and FDA statements expressing commitment to the goal of patient access to high-quality, safe, and effective medical devices. Subsequent meetings held monthly from October 2015 through January 2016 addressed the following:

  • FDA's responses to industry data requests
  • results of the analysis conducted on presubmissions for investigational device exemption (IDE) submissions
  • Center for Devices and Radiological Health (CDRH) information systems for premarket reviews
  • proposals related to addition of review staff, use of "real world evidence," and shifts in compliance and surveillance activities

FDA used these meetings to announce findings that IDEs with presubmissions now have a greater likelihood of first cycle approval. It also outlined implementation of CDRH's Plan of Action and discussed other infrastructure and workload initiatives.

Background
Two branches of FDA are responsible for the review of medical devices: CDRH and the Center for Biologics Evaluation and Research (CBER). Most medical devices are reviewed by CDRH, which requires the submission of either a premarket notification (510[k]) or premarket approval (PMA) for medical device review. CDRH also reviews IDE submissions.

To receive industry user-fee funding, FDA negotiates performance goals that are outlined in each MDUFA reauthorization. For example, in 2013, FDA promised to hire 200 full-time workers to reduce device review application times. It also agreed to have an outside firm evaluate the premarket approval process and agency progress in meeting performance goals.

Industry funding for new drug reviews was established 20 years ago, with passage of the Prescription Drug User Fee Act (PDUFA). User-fee laws were first enacted in response to mounting pharmaceutical industry frustration with the time FDA was taking to complete the new drug application review process. FDA blamed the delays on insufficient funding to hire the staff needed to review drug applications in a timely manner.

Because Congress was unwilling to provide increased funding, the FDA, Congress, and the drug industry negotiated an agreement that industry would provide user fees to supplement Congressional funding in exchange for a commitment from FDA to shorten the time required for new drug reviews.

Industry user fees were made contingent on a base level of appropriations funding so that they would supplement, rather than replace, federal funding of FDA. The original law also required Congress to reauthorize the program every 5 years, setting the stage for ongoing and predictable fighting over each reauthorization.

The FDA user-fee program was expanded in 2002, with passage of MDUFA. As with PDUFA, MDUFA must be reauthorized every 5 years and industry fees are tied to Congressional funding for FDA medical device reviews. MDUFA was reauthorized in 2007 (FDA Amendments Act) and in 2012 (FDA Safety and Innovation Act).

On July 9, 2012, President Obama signed the "Food and Drug Safety and Innovation Act" (FDA-SIA). This legislation reauthorized PDUFA for the fifth time and MDUFA for the third time. Current MDUFA III authority expires in September 2017. In addition, the FDA-SIA created two entirely new user fee programs—the Generic Drug User Fee Amendments (GDUFA) and the Biosimilars User Fee Amendments (BsUFA).

User fees are a vital part of FDA's operations because Congressional funding of the agency has not kept pace with the demands of additional responsibilities and ever more complex drugs and devices. User fees account for 44 percent of the agency's budget and pay for more than 4,700 full-time employees. They have been the singular predictable source of funds for an agency that regulates 25 cents out of every dollar spent in the United States. User fees will provide $595 million to the FDA for fiscal years 2013 through 2017.

AAOS Advocacy
The American Association of Orthopaedic Surgeons (AAOS) has been actively engaged in all MDUFA activities since the creation of the program. Most notably, the AAOS office of government relations used the 2012 MDUFA III negotiations to secure changes to the custom device exemption program. Custom devices deviate from those under an approved PMA or 510(k) clearance and are manufactured in response to a specific request from a physician to meet the unique needs of an individual patient.

The AAOS amendment broadened the custom device exemption. Previously, only one unique device for a specific patient was allowed. The AAOS provisions amended the existing custom device exemption regulations to allow up to five devices per patient. It also introduced new aspects and procedures, such as the following:

  • the potential for multiple units of a device type (not to exceed five units per year) qualifying for the custom device exemption
  • annual reporting requirements by the manufacturer to FDA about custom devices
  • inclusion of devices that are for a patient's unique physiologic or pathologic needs as well as for the unique practice needs of a physician, surgeon, or dentist

The AAOS is presently involved in the MDUFA IV negotiations. On Aug. 12, 2015, AAOS submitted comments to the FDA in partnership with 10 orthopaedic specialty societies in anticipation of the MDUFA IV Reauthorization public meetings. AAOS noted that "the overall performance of the medical device user fee program under MDUFA III has been highly effective…but issues still exist and more could be done to minimize the impact of reviewer assignments on variability of the review process."

AAOS pointed out that problems in accessing the custom device exemption, humanitarian device exemption, and compassionate use pathways are ongoing. Clinicians have reported confusion among administrators and institutional review boards regarding the regulations and review requirements.

AAOS urged FDA to thoughtfully consider the infrastructure required to transition the emphasis on device regulation from a premarket to a postmarket model, pointing out the burden on clinicians to capture postmarket data. MDUFA Comments, August 2015 (PDF).

Judi Buckalew, BSN, MPH, CAE, is the senior manager, regulatory and government relations, in the AAOS office of government relations. She can be reached at buckalew@aaos.org

Bottom Line

  • User fees paid by industry help support FDA's review programs of prescription and generic drugs, medical devices, and biosimilars.
  • Reauthorization negotiations present an opportunity for AAOS to effect change, such as the recent expansion in the definition of custom devices.
  • Congressional funding has not kept pace with additional requirements placed on FDA or greater demands for review of more complex drugs and devices, increasing the importance of the user-fee program.

Partnering with other orthopaedic organizations
AAOS submitted comments to the FDA in partnership with the following orthopaedic specialty societies in anticipation of the MDUFA IV Reauthorization public meetings:

  • American Association for Hand Surgery
  • American Orthopaedic Foot and Ankle Society
  • American Orthopaedic Society for Sports Medicine
  • American Society for Surgery of the Hand
  • Arthroscopy Association of North America
  • Musculoskeletal Tumor Society
  • Orthopaedic Research Society
  • Orthopaedic Trauma Association
  • Pediatric Orthopaedic Society of North America
  • Scoliosis Research Society