Stakeholder Meeting on MDUFA III Reauthorization

July 27, 2011, 1:40 – 4:00 PM

HHS Humphrey Building, Washington, DC

Room 325A


To review status of MDUFA III negotiations.


Malcolm Bertoni Office of the Commissioner (OC)
Ashley Boam Center for Devices and Radiological Health (CDRH )
Nathan Brown Office of Chief Counsel (OCC)
Kate Cook Center for Biologics Evaluation and Research (CBER)
Natalia Comella CDRH
Susan Cummins CDRH
Sharon Davis CDRH
Cindy Garris CDRH
Elizabeth Hillebrenner CDRH
Toby Lowe CDRH
Michelle McMurry-Heath CDRH
Barbara Myklebust CDRH
Thinh Nguyen OC
Arleen Pinkos CDRH
Ruth Watson Office of Legislation (OL)
Nicole Wolanski CDRH
Barbara Zimmerman CDRH
Cynthia Bens Alliance for Aging Research
Paul Brown National Research Center for Women & Families
Eric Gascho National Health Council
Suzanne Henry Consumers Union
Jay Hunter Parkinson’s Action Network
Jeanie Kennedy American Academy of Orthopedic Surgeons
Jenny Liljeberg American Society of Cataract and Refractive Surgery
Heidi Moline
Meredith McGreary Women Heart
Martha Nolan Society for Women’s Health Research
Kate Ryan National Women's Health Network
Lisa Swirsky Consumers Union
Additional Registered Stakeholders
Mellanie True Hills

Meeting Start Time: 1:40 pm

Update on Negotiations

FDA provided an update of FDA’s discussions with Industry, including details regarding the July 15, 2011 meeting with Industry. FDA noted that the Agency had also met with Industry again on July 26, 2011. FDA answered questions from stakeholders regarding these discussions up to the July 15 meeting. In response to FDA’s plan for mitigating program uncertainties, stakeholders asked for clarification regarding the types of underlying assumptions potentially implicated by the resolution of program uncertainties in the Fall. FDA explained that projected device review workload, in terms of anticipated numbers of pre-market submissions, is a key assumption that affects both the staffing levels needed to accomplish performance goals, as well as the calculations regarding user fee amounts for different submission types. Additional assumptions regarding amount of data required per submission and complexity are more difficult to judge but could also affect the amount of effort required to complete each review. Stakeholders also asked about the length of a potential reauthorization agreement. FDA reiterated its position that a five year reauthorization is needed, consistent with the statute; stakeholders indicated their support for this position.

Response to Stakeholder Letter

FDA acknowledged a letter received from the Patient, Consumer, and Public Health Coalition and thanked these stakeholders for their feedback. A representative from that coalition provided a summary of the letter for the group. The letter reflects their response to all negotiation meeting minutes available at the time of writing, through June 1, 2011. The group is concerned with CDRH’s ability to meet statutory responsibilities without adequate funding. The group also commented on proposals they would like to see addressed through funding under MDUFA: Unique Device Identifier (UDI), MedWatch/Maude database improvements, and the CDRH labeling initiative. The group indicated their support for FDA proposals for increased review staff, training, specific expertise development, teams across the Center for backup, facilitating postmarket data into premarket decision making, improving the guidance document development process, refuse to accept checklists, and improving U.S. clinical trial infrastructure. In particular, the Coalition strongly supports using postmarket data to inform updating labeling, reclassification of devices, and resolution of safety problems through changes in design.

FDA addressed specific questions from the Coalition. In response to the Coalition’s request for additional information regarding FDA’s proposal for clinical trial registries, FDA explained that the infrastructure provided by development and support for registries of medical device utilization and associated outcomes will provide desired information to address gaps in knowledge about how devices are used in the clinical setting and how patients receiving those devices fare compared to those observed in premarket trials. These registries will be useful in addressing questions that are currently managed on an individual device basis in post-approval studies imposed as a condition of approval for class III devices.  For example, external registries that are available may be linked to implant (or other utilization) data in order to gain data for longer term endpoints.   In addition, registries may be used for active or enhanced surveillance mandated as conditions of approval.  A formal post-approval study may be nested or embedded within an existing registry, eliminating the need to create new study infrastructure, and allowing collection of in-depth data on a small subset of people while collecting a shorter set of elements on a broad swath of the patient population.  Finally, when a post-approval study occurs prior to final establishment of an external registry, then the patients may be rolled into the registry for continued follow-up for longer-term outcomes.

In response to the Coalition’s question regarding the role the Medical Device Epidemiology Network (MDEpiNet) would play in the assessment of registries, FDA explained that the MDEpiNet will allow leveraging of the knowledge of a group of experts in both the infrastructure and methodology needed to advance our utilization of registries, as well as other data sources and structures.  Through international consortia opportunities, MDEpiNet is facilitating use of the vast amount of worldwide data available in the post-market setting.  Through data linkage, utilization of electronic healthcare records in the registries context, and other methodological advances, MDEpiNet will speed the collection of longer-term data for evaluation.  And, through the inventory of registries/databases available combined with the development of criteria for suitability of the available registries to address post-approval studies, MDEpiNet and the FDA will encourage utilization of the most appropriate data sources for postmarket questions. Stakeholders expressed concern with the idea of using registries in place of post-market studies. FDA explained that the intent is to advance available information to complement, not replace, post-market studies.

The Coalition asked if FDA’s proposal for annual meetings at the Division level would include all stakeholders, including patient groups. FDA replied that their initial intent was to hold such meetings with Industry stakeholders; however, FDA does believe it would be valuable to have industry, FDA, consumer/patient advocacy organizations, and academic institutions participate in yearly public meetings to provide feedback on product-specific issues. FDA stated they are willing to explore this possibility. Stakeholders indicated their appreciation and pointed out that patient and consumer groups bring different and unique information and expertise to bear when it comes to device-specific issues.

The Pediatric Medical Device Safety & Improvement Act of 2007

In response to a previous stakeholder request, CDRH’s Chief Pediatric Medical Officer Dr. Susan Cummins provided a presentation on the Pediatric Medical Device Safety & Improvement Act of 2007 (PMDSIA). The statute defines pediatric patients to include newborns up to their 22 nd birthday. This is different than the definition of pediatric patients used by CDER (i.e., up to age 16), but is consistent with definitions used by NIH and takes into consideration the brain and bone growth that takes place up until the 20s. The goal of PMDSIA is to foster pediatric device development through several means: financial incentives, simplifying pathways to development (i.e., extrapolating effectiveness from adults to children), development of infrastructure for innovation (i.e., requiring the development of the NIH Pediatric Medical Device Research Plan and establishing a non-profit Pediatric Device Consortia), tracking and annual reporting to Congress of PMA approved devices for pediatric patients, safety and profitability monitoring for pediatric Humanitarian Use Devices (HUDs), and GAO evaluation of its impact prior to reauthorization. Dr. Cummins explained that the Office of Orphan Product Development in the Office of the Commissioner is responsible for determining HUD status based on annual use in less than 4,000 Americans. This is the first step in the Humanitarian Device Exemption (HDE) approval pathway, which has a standard of approval based on safety and probable benefit rather than safety and effectiveness. Typically, devices approved through the HDE pathway are not permitted to earn a profit; however, the statute allows profit-earning for pediatric HUDs with requirements for adverse event and distribution reporting to the Office of Pediatric Therapeutics and review by a Pediatric Advisory Committee.

Dr. Cummins highlighted several PMDSIA successes to date. In January 2010, FDA approved an HDE for the first percutaneously delivered heart valve. This is the first pediatric HUD to be granted profit exemption and will be reviewed by a Pediatric Advisory Committee this Fall. Although FDA’s final decision is still pending, an Advisory Committee recently recommended approval of an HDE for a pediatric ventricular assist device. Additionally, over 90 pediatric device projects have been assisted by the Pediatric Device Consortia since October of 2009. The Center has also held multiple workshops to address pediatric device needs and has a project ongoing using computational modeling to look at defibrillation needs in pediatric patients.

Meeting End Time: 3:40 PM