FDA may allow unapproved use of medical products during emergencies

In the event of emergencies, such as pandemic threats or attacks involving chemical, biological, radiological, and nuclear (CBRN) agents, the FDA may authorize emergency use of medical products that are unapproved, or of approved products for an unapproved use. The FDA may take action to employ “medical countermeasures” (MCMs) after a relevant U.S. agency has declared an emergency or threat justifying such use.

Emergency Use Authorizations

The FDA’s emergency use authorization (EUA) power is distinct from the power to use a medical product under an investigational application. Before an EUA may be issued, the HHS Secretary must make an EUA declaration based on one of the following:

  • the Secretary of Homeland Security’s determination that there is a domestic emergency, or potential for emergency, involving a risk of attack with a CBRN agent;
  • the Secretary of Defense’s determination that there is a military emergency, or potential for emergency, involving a risk of attack on U.S. military forces involving CBRN agents;
  • the Secretary of HHS’ determination that  there is a public health emergency, or potential for emergency, that would affect national security or the health and security of U.S. citizens living abroad, involving a CBRN agent or related disease; or
  • the Secretary of Homeland Security’s identification of a material threat sufficient to affect national security of the health of US citizens living abroad.

After the EUA declaration is issued, the FDA Commissioner is expected to confer, to the extent feasible and appropriate, with the Assistant Secretary for Preparedness and Response, the Director of the National Institutes of Health, and the Director of the Centers for Disease Control and Prevention, before authorizing emergency use of a product.

EUA Criteria

The CBRN agents in the EUA declaration must be capable of causing serious or life-threatening harm. Medical products eligible to be considered for an EUA require a lower level of evidence than the typical effectiveness standard that the FDA uses when approving products, and are those with the possibility of effectiveness in preventing, diagnosing, or treating serious or life-threatening conditions. The FDA may also make available products that might mitigate a disease or condition caused by an FDA product (including emergency use products) used due to the CBRN agent.

The product must be one that has benefits known to outweigh known and potential risks, as determined by assessing the totality of scientific evidence available. Additionally, there must be no adequate, approved, and available alternative to the product in an EUA. This criterion is satisfied if the potential alternative product lacks sufficient supplies, is contraindicated for special populations, lacks an approved dosage form for special populations, or may not withstand the CBRN agent.

EUA Requests

Although most EUA requests will likely be submitted by government agencies, industry sponsors are eligible to submit such a request. In the event that a sponsor wishes to do so, the FDA recommends the inclusion of an organized summary of scientific evidence supporting a product’s safety and effectiveness, as well as approved alternatives. Other relevant information includes a description of the product and its intended use, a description of the product’s FDA approval status, the need for the product, and information about the adequacy and availability of approved alternative products.

FDA tells manufacturers what it means to be an accessory

The FDA encourages manufacturers of medical device accessories to use the de novo classification process under Section 513(f)(2) of the Federal Food, Drug, and Cosmetic Act (FDC Act). In a new guidance document, the FDA explains the definition of accessory for FDC Act purposes, the regulation of such devices, and the process by which manufacturers can obtain a risk-based classification of a new accessory type.

Background

Under Section 201(h) of the FDC Act, the definition of the term “device” includes “accessories.” Thus, all accessories to articles meeting the definition of device, are, themselves, devices. The classification of device accessories has historically taken on one of two methods: (1) shared classification with a “parent” device or (2) by issuance of a unique classification for the accessory. In the second circumstance, an accessory obtains a unique classification because the FDA determines that a classification regulation for an accessory should be separate from that of the corresponding parent device—a designation typically reserved for accessory types that may be used with multiple parent devices or that have unique standalone functions. However, the FDA recognized that some accessories have a lower risk profile than that of their parent device and, therefore, warrant a lower classification. Section 513(b) of the FDC Act was amended by the 21st Century Cures Act (P.L. 114-255) to reflect that thinking with a category of classification known as de novo classification.

Accessories

After the FDA determines that an article is an accessory, the agency determines whether the article is intended for use with one or more parent devices and then asks whether the article is intended to support, supplement, and/or augment the performance of one or more parent devices. The guidance explains that an article does not become an accessory simply by virtue of the fact that it is used in conjunction with another device. For example, the FDA would not consider a mobile phone to be an accessory merely because it is used as a general platform for applications that include mobile medical applications that are themselves medical devices.

De novo classification

Under Section 513(f)(2) of the FDC Act, the FDA may classify an accessory of a new type under the de novo classification process. Such a classification request is a request for risk- and regulatory control-based classification of a new type of accessory. To fall into the classification of “new category type,” the accessory under consideration should not be previously classified or the subject of any approved premarket approvals (PMAs) or cleared 510(k)s for that accessory type. The de novo classification is intended as a pathway to Class I and Class II device classification for accessories for which general controls or general and special controls provide a reasonable assurance of safety and effectiveness, despite the lack of legally marketed predicate device.

Submission and classification

A manufacturer of a medical device accessory, who submits a de novo classification request, must include a description of the device and detailed information regarding the reasons for the recommended classification. The FDA is obligated to make a classification determination for the device, by written order, within 120 days of the request. If the submitter satisfies the regulatory criteria (i.e. presents an accessory for which general controls or general and special controls provide a reasonable assurance of safety and effectiveness) the FDA will grant the de novo request, classifying the new accessory (and new accessory type) as Class I or Class II. The FDA will then publish an announcement in the Federal Register of the new classification and the general and special controls necessary to assure safety and effectiveness for the device type.

The Empire State woos pharma, biotech industries

The 21st Century Cures Act (Cures Act) was passed by the House on November 30, 2016 and the Senate on December 7, 2016. The President signed it into law on December 13, 2016.  The Cures Act contains three primary titles that makes good on the promise of its name through FDA reforms by accelerating drug and device development and delivery. The Cures Act also creates new administrative positions related to mental health and substance abuse and provides state funding to combat opioid addiction. The President applauded Congress’ approval of the bill, commenting, “I think it indicates the power of this issue and how deeply it touches every family across America.”

In a similar vein, New York Governor Andrew Cuomo and New York City Mayor Bill de Blasio recently unveiled two initiatives that would commit $1.15 billion in funding and tax incentives for education, business development, and job creation in the life sciences sector. Of the total amount,  New York City will be investing $500 million in biotech and life sciences over the next decade via a program called LifeSci NYC, the largest piece is composed of $300 million in tax credits that will be made available to companies building lab space in the city, in order to defray the high costs of construction in the city. The state’s contributions include $250 million in tax incentives for new and existing life science companies, $200 million in state capital grants to support investment in wet-lab and innovation space, and $100 million in investment capital for early stage life science initiatives with an additional match of at least $100 million for operating support from private sector partnerships.

Citing the lack of affordable and appropriate lab space as a barrier to industry, especially in the New York City real estate market, the state and city initiatives will provide more than 3.2 million square feet of innovation space and 1,100 acres of developable land available tax-free at 45 colleges and universities statewide. The availability of grants, land and space would offer an incentive for life science industry to access labs, infrastructure, and other equipment for product development.

 

FDA: No lab-developed test final guidance in the near future

The FDA will not finalize its 2014 draft guidance on “FDA Notification and Medical Device Reporting of Laboratory Developed Tests” any time soon due to the changing political administration, according to a statement the agency provided to GenomeWeb. Intended to balance “patient protection with continued access and innovation,” the draft guidance is unpopular in the laboratory and pathologist communities, which view lab-developed tests (LDTs) as services that should be regulated under the Clinical Laboratory Improvement Amendments (CLIA) of 1988, rather than medical devices within the FDA’s regulatory purview. The FDA’s hesitation stems, in part, from the uncertainty of the new political landscape, including the views of the as-of-yet unnamed incoming HHS Secretary.

The FDA has taken the position that LDTs are subject to its enforcement discretion pursuant to the Medical Device Amendments because they are intended for clinical use and designed, manufactured, and used within one laboratory (see FDA seeks notification of lab developed tests, October 3, 2014). According to GenomeWeb, the FDA believes that gaps in the CLIA-authorized system present a public health risk. However, some senators from the Republican party—which will control Congress—believe the agency is “too plodding to keep up with innovation in the molecular diagnostics space.”

The agency continues to emphasize the importance of its role in regulating LDTs and is considering issuing best practices, rather than a final guidance. Senator Lamar Alexander (R-Tenn) and Representative Fred Upton (R-Mich) applauded the FDA’s decision not to finalize the guidance in the near future, while the American Clinical Laboratory Association (ACLA) called it “a victory for diagnostic innovation and most importantly, patients.”