Mark Leahey05.01.07
Beyond MDUFMA: Other Issues Under Consideration by Congress
Mark Leahey
Pediatrics Legislation
With the passage of The Medical Devices Technical Corrections Act of 2004 in April 2004, Congress required the Secretary of Health and Human Services to submit a “report on the barriers to the availability of devices intended for the treatment or diagnosis of diseases and conditions that affect children.” What followed was a series of public meetings and a public comment period to identify areas of need and develop solutions.
One issue that arose from these meetings and the comment period was that many stakeholders felt the prohibition from generating profits for the Humanitarian Device Exemption (HDE) served as a significant disincentive to develop products for the pediatric population. (An HDE is an application that is similar to a premarket approval application but exempt from the effectiveness requirements of sections 514 and 515 of the act. FDA approval of an HDE authorizes the company to market a Humanitarian Use Device [HUD].) As a result, Sen. Chris Dodd (D-CT) introduced The Pediatric Medical Device Safety and Improvement Act on March 8 this year, eliminating the profit prohibition for HDEs that are specifically designed to meet a pediatric need.
Another issue stakeholders highlighted to promote the development of pediatric devices was to increase the HUD threshold. The FDA currently defines an HUD as a “medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in fewer than 4,000 individuals in the United States per year.” Increasing the HUD threshold to 8,000 or 10,000 affected patients per year would provide a greater incentive for companies to develop these products and ultimately improve the quality of care for the pediatric population. However, the current version of the pediatrics bill does not increase the HUD threshold.
Other provisions of the Pediatric Medical Device Safety and Improvement Act include requiring the National Institutes for Health (NIH) to designate a contact point or office to help innovators and physicians access existing funding for pediatric medical device development and direct the NIH, FDA and Agency for Healthcare Research and Quality to submit a plan for pediatric medical device research that identifies gaps and proposes a research agenda for addressing those gaps. The bill also would establish demonstration grants for non-profit consortia to promote pediatric device development; members would include coordinating among inventors, manufacturers and physicians.
While the industry generally supports the bill, it is working to improve one additional provision. The proposed legislation would expand Section 522 of the Federal Food, Drug and Cosmetic Act to permit the FDA to require, as a condition of clearance or approval, post-market studies for any Class II or Class III medical device if, in addition to the existing criteria, it is expected to have significant use in pediatric populations. From the industry’s perspective, the need for this provision is unclear given the FDA’s current authority to require post-market studies of a device to provide reasonable assurance of safety and effectiveness. Moreover, in the absence of reliable data on the need for pediatric devices and the affected pediatric populations, the agency would have no basis on which to determine whether a device is expected to have “significant” pediatric use.
Trial Registry, Results Database
In addition to the possibility of the pediatrics bill being attached to the reauthorization of MDUFMA and/or PDUFA, speculation exists that the Enhancing Drug Safety and Innovation Act would be tied to this vehicle as well. The bill, introduced by Sens. Edward Kennedy (D-MA) and Mike Enzi (R-WY), would require pharmaceutical manufacturers and the FDA to engage in better safety planning before a drug is approved for release while improving the agency’s response to risks identified after a drug is on the market.
In the device realm, these post-market issues have been addressed by the Centers for Devices and Radiological Health (CDRH) in its Medical Device Postmarket Transformation Initiative launched last November. The initiative designates four areas for improvement: (1) create a culture of collaboration among all parties within CDRH; (2) develop world class data systems to enhance the CDRH’s analysis and tracking capabilities; (3) enhance risk/benefit communication efforts by engaging clinical practitioners and professional communities; and (4) collaborate on enforcement strategies and outcomes among the CDRH, the Office of Regulatory Affairs and the Office of Chief Counsel. Given the significant efforts by the CDRH in the area of device safety, many on the Hill understand that additional post-market regulations for devices are unwarranted.
However, even with the significant steps taken by the CDRH, there has been talk that the Hill may try to include language that would require a clinical trial registry and results database for medical devices. Some believe that the registries help patients learn about innovative treatments earlier and assist the industry for enrollment purposes. While this analysis may be true for drugs, it does not hold for devices. Enrollment in device trials is driven by the premier physicians in a particular field—not an Internet registry. This is a direct result of physician’s role in a medical procedure versus the administration of pills and monitoring the patient’s response with drug therapies.
In developing a results database for devices, the industry has communicated the importance of developing a system that focuses on pivotal trials for approved Class III products. Attempts to include feasibility or marketing studies would not provide the patient with useful scientific information. In addition, given the policy goal of ensuring that patients have the most up-to-date information on devices in the marketplace, requiring the posting of trials for non-approved products would be too broad and not serve the intended objective. Finally, the industry strongly believes that any public posting of pivotal trial data for approved Class III products must not include any information that would compromise the intellectual property of a company. Doing so would significantly undermine future investment in this industry.
Timing
While these are busy times in Washington, the reauthorizations of both MDUFMA and PDUFA before the Sept. 30 deadline are priorities for Congress and the affected industries. Therefore, we are likely to see a strong push for passage before the Memorial Day recess. Time will tell if that push will be successful and what additional bills members of Congress will try to include in the “must pass” vehicle. As of now, all indications are that the two aforementioned items will be part of the discussion.