Food and Drug Administration (FDA) Center for Devices and Radiological Health (CDRH) Initiatives – Public Notification of Emerging Signals for Medical Devices
CDRH is responsible for the regulation of products which meet the regulatory definition of a medical device. At the time a medical device reaches the market, it has a benefit-risk profile that health care providers, patients, and consumers use to make treatment decisions. However, not all information regarding benefits and risks for a given device may be known before the device reaches the market. New information about a device’s safety and/or effectiveness, including unanticipated adverse events, may become available once the device is more widely distributed and used under real-world conditions and in broader patient populations. Also, subsequent changes made to the device, its manufacturing process, or supply chain might lead to new safety problems. CDRH considers new information suggesting a new potentially causal association or a new aspect of a known association between a medical device and an adverse event to represent a “signal.” An “emerging signal” is a signal for which the Agency has conducted an initial evaluation and determined that the information has the potential to impact patient management decisions and/or the known benefit-risk profile of the device.
CDRH is currently putting in place, a process by which each emerging postmarket safety signal being evaluated by a cross-Center team, will be assessed for potential early public notification (e.g., before we have finished gathering and analyzing all of the relevant information). This includes having an established set of factors that would be used in making the decision, the timelines for making the decision and re-assessing the decision, as well as the processes and timing for updating such notifications. The Center plans to finalize a guidance document which speaks to this effort, and begin instituting the effort in the coming months.
Through this activity, CDRH is attempting to increase its public transparency by having a more consistent approach to sharing new device performance-related information that comes to light in the postmarket period, while that information is still under evaluation. Timely notification about those emerging signals is intended to provide health care providers, patients, and consumers with access to the most current information concerning the performance and potential benefits and risks of marketed medical devices so that they can make informed decisions about their treatment options. It may also reduce or limit the number exposed to the potential risk while the issue is being further evaluated, and promote enhanced vigilance on the part of clinicians, risk managers, patients and consumers. The awareness raised by a notification may also promote voluntary reporting of events to FDA. This in turn may provide the Agency with additional insight into the issue and assist in the formulation of potential risk mitigation strategies or recommendations.
FDA Medical Device Regulatory Guidance Webinars
CDRH issues approximately 40 draft/final guidances each year. These guidances are developed to help the medical device industry understand what to include in its medical device marketing applications. While the medical device industry is able to comment on the draft guidance, once it is issued in final they must now use that guidance to submit their applications. In 2014 FDA/CDRH began to offer public webinars for industry to discuss the contents of the guidance and answer any questions they had about the guidance. The webinar includes the Subject Matter Expert discussing the purpose, content, changes and clarification of the guidance, after the presentation there is a significant 2-way dialogue/question and answer session where the participants can ask specific clarifying questions. These are held 2 weeks after the issuance of guidance to allow stakeholders time to read the guidance and schedule time to participate. On average 1,000 sites participate in these webinars, with some reaching 2,000 participants. After the webinar the presentation, slides, and transcripts are posted to our webinar website for future viewing. Since 2014 CDRH has held 41 webinars, reaching at least 41,000 sites where between one and multiple stakeholders are participating. Informing and educating the medical device industry ensures better quality submissions and 2-way dialogue with our stakeholders helps us to produce better guidance in the future.
National Institutes of Health (NIH) Plans for Increasing Access to Digital Scientific Data
The NIH has a long history and continued commitment to ensure that, to the fullest extent possible, the results of federally-funded scientific research are made available to and are useful for the general public, industry, and the scientific community. The NIH has maintained the principle that “data sharing is essential for expedited translation of research results into knowledge, products, and procedures to improve human health.” Validation and progress in science are predicated on access to research results. The NIH has developed a number of policies to support this effort and has many activities underway to further promote sharing of data, such as the 2003 NIH Data Sharing Policy (http://grants.nih.gov/grants/guide/notice-files/NOT-OD-03-032.html), the 2014 NIH Genomic Data Sharing Policy (http://grants.nih.gov/grants/guide/notice-files/NOT-OD-14-124.html), and the 2015 NIH Intramural Human Data Sharing Policy.
On February 22, 2013, the White House Office of Science and Technology Policy (OSTP) released its memorandum entitled Increasing Access to the Results of Federally Funded Scientific Research https://obamawhitehouse.archives.gov/sites/default/files/microsites/ostp/ostp_public_access_memo_2013.pdf). In February 2015, in response to this memorandum, the NIH issued the NIH Plan for Increasing Access to Scientific Publications and Digital Scientific Data from NIH Funded Scientific Research (http://grants.nih.gov/grants/NIH-Public-Access-Plan.pdf). The goals of this directive are in keeping with the NIH’s ongoing and future commitments to facilitate data sharing, and the NIH Plan outlines mechanisms for expanding and strengthening access to data and publications from NIH-funded research. In the near future, and after public engagement, the NIH is planning to expand upon its 2003 Data Sharing Policy and develop a data management and sharing policy that will apply to all NIH-funded research, regardless of funding level.
Many of the NIH activities related to public access to digital scientific data are overseen by the NIH Scientific Data Council (SDC), an internal NIH committee comprised of senior NIH leaders and staff. The SDC was originally created in 2013 and constituted as part of a multifaceted expansion of NIH programs and governance related to data science. The SDC, in a trans-NIH manner, addresses the growing challenges and opportunities associated with ‘big data’ and data science in biomedical research, thereby improving data utilization by the research community in order to transform the impact of biomedical, clinical, and public health research.
Key major milestones and anticipated completion dates:
- January 2015: The NIH Genomic Data Sharing Policy became effective and is now being implemented across all NIH Institutes and Centers. This policy applies to all NIH-funded research that generates large-scale human or non-human genomic data, regardless of the funding level, and use of these data for subsequent research.
- October 2015: The NIH Intramural Human Data Sharing Policy became effective and is now being implemented across all NIH Institutes and Centers with the purpose of responsible sharing of all human data and secondary research with human data generated in the NIH Intramural Research Program (IRP).
- Spring-Summer 2016: The NIH completed the analysis of nearly 900 public comments on the Notice of Proposed Rulemaking on Clinical Trials Registration and Results Submission and is working with the FDA on the consideration of the public comments and development of the Final Rule. In parallel, work is also proceeding on the development of the final NIH policy on clinical trial information dissemination. Both the rule and policy are expected to be released in the summer of 2016.
- 2014 – Present: Many other NIH programmatic initiatives are also advancing implementation elements discussed in the NIH Plan for Increasing Access to Scientific Publications and Digital Scientific Data from NIH Funded Scientific Research (Public Access Plan). In particular, through the NIH Big Data to Knowledge Initiative (BD2K), awards have been made for the development of:
- A Commons as a shared virtual space in which data and associated analytics can be utilized.
- A data catalog in the form of a data discovery index, called bioCADDIE (biomedical and health care data discovery index ecosystem) to facilitate the FAIR principles of Finding, Accessing, Interoperating, and Reusing data.
- 2016 - 2017: The Scientific Data Council’s Sustainability Working Group will develop and implement economic, technical, and administrative approaches to enhance long-term support of biomedical data resources.
- Fall 2016: In order to inform the development of a future NIH data management and sharing policy that would encompass the different types of data generated by the vast amount of research supported by the NIH, the NIH will first establish priorities for data sharing. The priority setting process will involve obtaining stakeholder input on a number of topics, such as data sharing strategy (e.g., what data should be shared; which data types have the greatest value for secondary analysis; costs and value of sharing different types of data; long-term preservation and sustainability of sharing data; metric for assessing biomedical repositories; and standards for citation of data.
- 2016-2019: The NIH plans to develop, to seek stakeholder input on, and to release and implement a new data management and sharing policy that will apply to all NIH-funded research, regardless of funding level.
NIH Accelerating Medicines Partnership (AMP) Type 2 Diabetes Knowledge Portal
The AMP Type 2 Diabetes (T2D) Knowledge Portal is aimed at advancing type 2 diabetes research and treatment, and includes data from over 100,000 genetic samples obtained from clinical consortia supported by the NIH and the Foundation for the NIH. Encouraging greater collaboration between and participation of the scientific community and all interested in diabetes and genetic research, this online library allows open-access searching of human genetic and clinical information on type 2 diabetes. Individual data remains confidential. Truly a source for Big Data, the portal includes information from a growing list of major international networks, collected from decades of research.
By using human genetic samples, the portal provides a way to identify the most promising therapeutic targets for diabetes from troves of potentially relevant human data. The innovative curation and the scale of data enable researchers to translate differences in an individual’s genome into an understanding of how those differences affect a person’s risk of developing type 2 diabetes. The portal also enables advances in Precision Medicine, where investigators can examine genetics and disease in relation to differences in race, ethnicity, and locality.
The knowledge portal makes genetic and clinical information searchable in myriad ways to help researchers identify and describe the effects of genes on disease, test biological hypotheses, and conduct many other analyses. The portal is publicly searchable and can be used as a tool to learn about genetics and health. In 2016, the portal enhanced its accessibility to the public, allowing anyone to query detailed data from the portal. The portal is also available in Spanish.
By making available the fruits of government-funded research, the portal increases transparency of how those research dollars have been spent and encourages users to amplify the government’s investment by finding new uses for that data in scientific inquiry.
Administrative Law Judges
The Office of Medicare Hearings and Appeals has a number of activities focused on Administrative Law Judges (ALJ) and their work to address open government. These include:
Appeal Status Information System (AASIS): Individuals appealing unfavorable decisions regarding the coverage or payment of Medicare claims and other related matters need timely acknowledgement of the receipt of their appeals and definitive contact information should they need to make an inquiry regarding the status of their appeals. To meet this need, OMHA implemented AAISIS, a web-hosted solution that allows for verification of receipt, up-dates regarding the status of any appeals, and contact information for ALJ staff assigned to adjudicate the appeal.
Electronic Case adjudication and Processing Environment (ESCAPE), Appellant Public Portal (APP): a public facing portal, that will serve as a full two-way communication tool and will allow authorized parities to file requests for hearing, submit additional evidence, check appeal status, and view the appeal case file contents online.
Adjudication manual: Provides staff with a single resource for policy and procedural guidance that can be updated on a rolling basis. The manual sets forth a standardized business process aimed at increasing consistency for appellants and provides a framework to move from a paper-based to a fully electronic adjudication process which will further increase access and transparency to the administrative appeals process through features such as online access to real-time case status information and the administrative record.
Data Transparency Initiative: The agency is developing reports, to share additional workload and appeal disposition information with the public and appellant community on its website and update this information on a regular basis in future years.