FDA considers establishing a new ‘Office of Patient Affairs’

The FDA announced that it is establishing a public docket to solicit public input on ongoing efforts to enhance mechanisms for patient engagement at the agency. In addition, to achieve a more transparent, accessible, and robust experience for patient communities, the FDA is considering establishing a new Office of Patient Affairs.

On November 4, 2014, the FDA established a docket (FDA-2014-N-1698) for the public to submit information related to the FDA’s implementation of the Food and Drug Administration Safety and Innovation Act (FDASIA) (P.L. 112-144), Patient Participation in Medical Product Discussions under FDASIA section 1137.

Based on the comments received, the FDA identified objectives for its patient engagement activities. First, to develop a nuanced understanding of the patient experience of disease by: (1) gathering patient perspective on what is clinically meaningful; (2) assessing attitudes towards benefit-risk and tolerance of uncertainty; and (3) enhancing the science of eliciting and integrating patient input.

Second, to support patients and their advocates in understanding regulatory processes and navigating the FDA by: (1) communicating relevant FDA positions, procedures, and activities; (2) connecting patients and their advocates with the appropriate resources; and (3) resolving discrete challenges and needs.

To achieve these objectives, the FDA is considering establishing a central “Office of Patient Affairs.” The responsibilities of this central office would include:

  • offering a single, central entry point to the FDA for the patient community;
  • providing triage and navigation services for inbound inquiries from patient stakeholders;
  • hosting and maintaining robust data management systems that would incorporate and formalize knowledge shared with the FDA by patient stakeholders and the FDA’s relationships with patient communities; and
  • developing a scalable and forward-looking platform for communicating with patient stakeholders, particularly online channels.

The Office of Patient Affairs would be directly accountable to the medical product Centers. A regular evaluation of this central office and of FDA’s overall patient engagement efforts are also proposed.

 

 

Trump nominates Gottlieb for FDA Commissioner

President Trump intends to nominate Scott Gottlieb, M.D., a resident fellow at the conservative American Enterprise Institute (AEI), clinical assistant professor at New York University School of Medicine, and a member of the HHS Federal Health IT Policy Committee, to the post of FDA Commissioner. The White House announced the nomination, which brought varied reactions from opposite sides of the aisle and a general positive response from the pharmaceutical industry, via a tweet from Press Secretary Sean Spicer.

Gottlieb served as the Deputy FDA Commissioner from 2005 to 2007 and previously served as a senior official at CMS. He has testified before Congress on numerous occasions as an AEI felllow, most recently with respect to EpiPen® price increases and “How Regulatory Barriers Inhibit Pharmaceutical Competition.” Gottlieb noted that FDA regulatory policy has made developing less expensive copies of complex drugs after patent expiration difficult, discussed how the 340B program has put “upward pressure on drug prices,” while noting other change in drug insurance coverage structure, and described obstacles to competitive single source drug pricing.

Various sources report that Gottlieb has close ties to the pharmaceutical industry. Scientific American noted that Gottlieb believes in a quicker approval process for new drugs, but has focused on shortening waiting times for large, clinical trials rather than doing away with efficacy considerations. He commented on this, to an extent, in remarks he made at the 21st Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) in May 2016.

Gottlieb has also issued commentary about the Patient Protection and Affordable Care Ac (ACA) (P.L. 111-148). In May 2016, he testified before Congress that the law’s tiered marketplace approach has aided consumers with plan selection, but has forced insurers into narrow design corridors. His testimony regarding the ACA also included a suggestion that CMS move away from mandates and towards incentives to encourage individuals to enter into the insurance market (see Is there a better way than the ACA? Hearing asks experts, Health Law Daily, May 12, 2016). More recently, he coauthored a piece with another AEI fellow, opining that President Trump’s election provided, “a generational opportunity to pursue a new direction for American health care” and making suggestions about how a new health care system should operate. The authors suggested that the system should provide a path to catastrophic health insurance for all, accommodate individuals with pre-existing health conditions, allow access to health savings accounts, and deregulate the medical services market.

Senator Lamar Alexander (R-Tenn), Chairman of the Committee on Health, Education, Labor & Pensions, touted Gottlieb’s “impressive qualifications” in a released statement. His colleague, Ranking Member Patty Murray (D-Wash), expressed “initial concerns” about the nomination, including Gottlieb’s “work with multiple pharmaceutical companies, medical device companies, and investment firms.”

Bloodstream infection detection test approved for marketing

In a first of its kind, the FDA has approved the marketing of the PhenoTest BC Kit, which is the first test to identify organisms that cause bloodstream infections. The test kit, manufactured by Accelerate Diagnostics Inc., can provide information to health professionals about which antibiotics the organism is sensitive to and potentially reduce the amount of time to provide this information. As a result, antibiotic treatment recommendations can be formulated much quicker. Unlike traditional identification and antibiotic susceptibility tests that may take 24 to 48 hours after detection in a positive blood culture to provide test results, the PhenoTest BC Kit can identify bacteria or yeast from a positive blood culture in approximately 1.5 hours. The test can identify 14 different species of bacteria and two species of yeast that cause bloodstream infections, while also providing antibiotic sensitivity information on 18 selected antibiotics for a subset of the identified organisms as appropriate.

Bacterial or yeast blood infections can occur in individuals of all ages, but are particularly severe in infants, the elderly, and those with weakened immune systems. If not treated rapidly, such bloodstream infections can lead to severe complications, such as septic shock and death. The PhenoTest BC Kit can also provide treatment recommendations within 6.5 hours of organism detection in blood cultuers.

The FDA reviewed the data for the PhenoTest BC Kit through its de novo premarket review pathway, a regulatory pathway for devices of a new type with low-to-moderate-risk that are not substantially equivalent to an already legally marketed device and for which special controls can be developed, in addition to general controls, to provide a reasonable assurance of safety and effectiveness of the devices. Although some risks with the kit included false positive findings, the FDA’s decision was based on Accerlerate’s clinical study of 1,850 positive blood cultures.  In the study, bacteria or yeast in positive blood culture was identified correctly more than 95 percent of the time.

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.