Amid the partisan gridlock of the past few years, one of the few things Democrats and Republicans have agreed on is the need to reform FDA! Accordingly, Congress has just passed, and the President has indicated he will sign, the 21st Century Cures Act (Cures Act) (H.R. 34). The numbers were lopsidedly in favor of the bill (392-26 in the House and 94-5 in the Senate). A copy of the final version that is expected to be signed by the President is available here.
The Cures Act has a host of provisions intended to improve how the FDA reviews and approves drugs and medical devices. The following is a quick summary of the device related provisions:
Section 3051. Breakthrough Devices. This section requires FDA to establish a program to provide priority review and management attention for devices that provide “more effective treatment or diagnosis of life threatening or irreversibly debilitating human disease or conditions” and that represent “breakthrough” technologies for which no cleared or approved alternatives exist, that offer significant advantages over existing alternatives, or “the availability of which is in the best interest of patients.”
This section would allow a sponsor to request priority review designation from FDA at any time prior to the device submission. FDA would be required to respond within 60 days of receiving the request for priority review designation.
This program will no doubt build on FDA’s already-established Expedited Access Pathway (EAP). Unlike the EAP program, the legislation opens priority review to device types subject to 510(k) clearance and not just PMA approval or de novo requests.
Section 3052. Humanitarian Device Exemption. FDA’s existing HDE program allows approval of devices addressing rare diseases or conditions with relaxed effectiveness data (a showing of “probable benefit” rather than “effectiveness”). The existing HDE program is limited to disease or conditions affecting up to 4,000 persons annually. This provision doubles the ceiling to 8,000.
Section 3053. Recognition of Standards. This provision allows any party to obtain a ruling from FDA within 60 days on recognition of a standard, and requires FDA to make the ruling public in a manner deemed appropriate by FDA. It also has some other modest improvements, such as periodic training of FDA employees in the use of standards in premarket review.
Section 3054. Certain Class I and Class II Devices. FDA will now be required in early 2017 and once every 5 years to review class I and class II devices and consider whether they may be declared 510(k) exempt with reasonable assurance of safety and effectiveness. Instead of amending the classification regulations, which can take a long time, FDA will accomplish the exemption by publishing a list of newly exempted device types. The class II exemptions will require a proposed list, public comment, followed by a final list. Both the class I and class II lists will be deemed to amend the pertinent classification regulations. This provision is an excellent house cleaning measure that will now allow FDA to more easily prune the list of device types that require 510(k) clearance.
Section 3055. Classification Panels. This section would require FDA to ensure that any classification panel has “adequate expertise” to assess “the disease or condition which the device is intended to cure, treat, mitigate, prevent, or diagnose,” and “the technology of the device.” FDA must also provide an opportunity for the company whose product is being reviewed to provide recommendations on the expertise needed among the voting members of the panel.
“Adequate expertise” is defined to include “two or more voting members, with a specialty or other expertise clinically relevant to the device under review” and “at least one voting member who is knowledgeable about the technology of the device.” FDA must provide an annual opportunity for patients, representatives of patients, and sponsors of medical device submissions to provide recommendations for individuals with appropriate expertise to fill voting member positions on classification panels.
This provision will hopefully fix past problems with panels that do not have the right expertise.
Section 3056. Institutional Review Board Flexibility. This provision allows for a single IRB to oversee a multicenter device trial (rather than an IRB at each center). This additional flexibility should make IRB oversight more efficient.
Section 3057. CLIA Waiver Improvements. This provision takes aim at a section of FDA’s 2008 guidance on Clinical Laboratory Improvement Amendment (CLIA) waivers for in vitro diagnostic devices. FDA has a year to revise the 2008 guidance to allow demonstration of accuracy through comparable performance between a waiver user and a moderately complex laboratory user. This approach would replace the requirement to demonstrate accuracy based upon a gold standard. This provision will make it easier for some IVD tests to be exempted from routine inspections and most CLIA requirements.
Section 3058. Least Burdensome Device Review. This section is intended to address the device industry’s long-held position that CDRH does not comply with the statutory requirement to review only the data necessary to make a determination of substantial equivalence or device effectiveness, and to determine the “least burdensome means” of establishing substantial equivalence or effectiveness.
Section 3058 would amend section 513 of the FD&C Act by requiring each reviewer at CDRH to receive training “regarding the meaning and implementation of the least burdensome requirements” and would require FDA to assess implementation of the least burdensome requirements to ensure that they are “fully and consistently applied.”
This section also requires the CDRH Ombudsman to conduct an audit of the required training with respect to implementing the least burdensome provisions, and to interview industry representatives to obtain their perspective regarding the application of the least burdensome provisions to “premarket review and decision making.”
This provision also adds section 515(c)(5) to the FD&C Act, stating that whenever FDA requests additional information from a PMA applicant, FDA “shall consider the least burdensome appropriate means necessary to demonstrate a reasonable assurance of device safety and effectiveness.” “Necessary” is defined to mean “the minimum required information that would support a determination by [FDA] that an application provides a reasonable assurance of the safety and effectiveness of the device.” FDA is also directed in PMA review to consider the “role of postmarket information in determining the least burdensome means of demonstrating a reasonable assurance of device safety and effectiveness.”
Section 3059. Cleaning Instructions and Validation Data Requirement. The recent crisis over disease transmission between patients due to inadequate cleaning of duodenoscopes led to this provision. FDA had already upped its game in this area as a result of the duodenoscope situation, and this provision solidifies it. Within 180 days of enactment of the Cures Act, FDA must publish (and revise when appropriate) a list of reusable device types where 510(k) submissions must include validated instructions for use and validation data for cleaning, disinfection and sterilization.
This provision also includes an unrelated requirement for FDA to finalize its draft August guidance (see our previous post here) on when a new 510(k) is required for modifications to a cleared device. This guidance, of course, is of critical importance to the device industry.
Section 3060. Clarifying Medical Software Regulation. There has been a great deal of uncertainty about the degree to which FDA can and should regulate stand alone medical software. This type of software is steadily increasing in importance and capability. This provision modifies the definition of a “device” to remove a number of categories of software from FDA’s jurisdiction. The categories of software removed from the device definition are:
- Software that provides administrative support of a healthcare facility. This is non-controversial and arguably was never subject to the device definition in the first place.
- Software for maintaining or encouraging a healthy lifestyle, and not related to the diagnosis, cure, mitigation, prevention, or treatment of a disease or condition. This is non-controversial and is consistent with FDA’s General Wellness and Mobile Medical Applications guidance documents.
- Software that serves as electronic patient records, provided (among other things) that such function is not intended to interpret or analyze patient data or images for the diagnosis, cure, mitigation, prevention, or treatment of a disease or condition. This codifies policy already implemented by FDA as a matter of enforcement discretion.
- Software for transferring, storing, converting formats, or displaying data/results and associated findings by a healthcare professional (e.g., medical device data systems (MDDS)), unless intended to interpret or analyze the data, results or findings. This codifies FDA’s exemption of MDDS technology from regulation.
- Software for: (i) displaying, analyzing, or printing medical information about a patient or other medical information (such as practice guidelines); (ii) supporting or providing recommendations to a healthcare professional (i.e., clinical decision support) about prevention, diagnosis or treatment of a disease or condition, AND (iii) enabling the health professional to independently review the basis for such recommendations rather than primarily rely on it when making diagnostic and treatment decisions. There is an exclusion for medical images, signals from in vitro diagnostic devices and signals or patterns from signal acquisition systems, i.e., they are not part of this carve out from the definition of a medical device.
The last carve out from the device definition is important. FDA has long regulated image analysis software and IVD technology and wanted to make sure they would continue to regulate these devices, which could arguably be said to provide clinical decision support. This provision appears to give FDA continued jurisdiction in these areas. But, importantly, this provision removes FDA from regulating evolving software that uses “big data” and advanced computing capability to provide sophisticated clinical decision support to health care professionals.
This provision has an escape hatch if FDA believes that software of the types described above in C, D, and E, “would be reasonably likely to have serious adverse health consequences.” To bring this software back under FDA regulation, the agency must issue a final order in the Federal Register providing its rationale based upon the potential for and severity of patient harm if the software does not perform as intended, the extent to which the software is intended to support the judgment of a healthcare professional, whether a healthcare professional has a reasonable opportunity to review the basis of the information or treatment recommendation provided, and the intended user and use environment.
Finally, this section also provides that FDA shall regulate accessories based upon their intended use and not that of the parent device with which they are intended to be used. This comports with FDA’s 2015 draft guidance on accessories. FDA historically had taken the position accessories take on the classification of their parent devices. This position has not worked very well when it comes to recent software products.
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