Medical Countermeasure Monitoring and Assessment

Electronic health records are an important part of post-dispensing medical countermeasure monitoring and assessment

Background | FDA Guidance & Regulatory Information | Contact | FDA MCM Monitoring & Assessment Projects | Medical Product Assessment in General | Additional Resources

Background

There is a critical need for the U.S. Government (USG) to build and maintain a national capability to monitor and assess medical countermeasures (MCMs) after they are dispensed or administered in response to a chemical, biological, radiological, or nuclear threat or an emerging infectious disease.  In collaboration with the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE), the FDA has been a key partner in developing such a capability.

Historically, research as part of a public health emergency response has been limited. However, that is changing. Experiences responding to public health emergencies highlight the need for a coordinated plan to leverage every opportunity to learn more about an MCM’s performance.  This information is vital to protecting public health, particularly in the case of MCMs that may have limited human efficacy data prior to their use.  The emergency may present the only opportunity for clinical assessment.

The resources below highlight relevant FDA guidance and regulatory information, specific FDA projects related to monitoring and assessment of MCMs, some of the ongoing FDA work related to medical product assessment in general, and key efforts relevant to MCMs that are ongoing outside of the FDA.

FDA Guidance & Regulatory Information

FDA guidance and general FDA regulatory information to provide important context on the need for MCM monitoring and assessment:

• Guidances and other information of special interest to MCM stakeholders (including the January 2017 guidance on Emergency Use Authorization of Medical Products and Related Authorities; October 2015 guidance on Product Development Under the Animal Rule (PDF, 574 KB); and the June 2016 guidance on Expanded Access to Investigational Drugs for Treatment Use (PDF, 180 KB))

• Animal Rule Information - In developing MCMs for potential bioterror threats, human challenge studies (exposing people to the threat agent) are often not ethical or feasible. In these cases, FDA may grant approval based on well-controlled animal studies, when the results of those studies establish that the drug or biologic product is reasonably likely to produce clinical benefit in humans. The product sponsor must still demonstrate the product’s safety in humans.

• FDA Postmarketing Requirements and Commitments - Postmarketing requirements and commitments refer to studies and clinical trials that sponsors conduct after approval to gather additional information about a product's safety, efficacy, or optimal use.

FDA Contact

FDA Medical Countermeasures Initiative (MCMi)
AskMCMi@fda.hhs.gov

NOTE: Non-FDA links below are included for reference, and do not necessarily reflect FDA's positions or opinions. Consistent with FDA website policies and disclaimer, reference in any referred (linked) website to commercial products, services, manufacturers, or companies does not constitute an endorsement by the U.S. government, the Department of Health and Human Services, or FDA.

FDA Information about Past and Current MCM Monitoring and Assessment Projects

• FDA Sentinel System’s Coronavirus (COVID-19) Activities
  The FDA’s Sentinel System is engaged in numerous activities to protect and promote public health during the COVID-19 pandemic, including an ongoing project led by the (former) FDA Office of Counterterrorism and Emerging Threats (OCET) to examine outcomes among hospitalized patients with influenza-like illness and acute respiratory disease that is being expanded to also capture data on hospitalized patients diagnosed with COVID-19.

• Using a Handheld Device for Patient Data Collection: A Pilot for Medical Countermeasures Surveillance
  This project demonstrated the feasibility of using a handheld device to collect patient information and accurately link individuals to their health record.

• Adverse Events Monitoring and Analysis Pilot Program (completed MCMi Regulatory Science project)
  This project assessed the feasibility of rapidly detecting, verifying, and assessing adverse events to conduct risk-benefit analyses during emergency situations and communicate related risk.

• Influenza antiviral drug use 2010-2015
  This study involved a modular program-based, one-time assessment of incident use of two influenza antiviral drugs (Oseltamivir and Zanamivir) during the five previous influenza seasons (2010-2015).

• Streamlining Countermeasure Data Collection During Public Health Emergencies
  In collaboration with the Biomedical Advanced Research and Development Authority, the FDA awarded Discovery, the Critical Care Research Network, a contract to help speed access to and evaluation of MCMs during public health emergencies.

• FDA Real Time Application for Portable Interactive Devices (RAPID)
  The FDA’s Real-Time Application for Portable Interactive Devices (RAPID) is a real-time bidirectional communication and analytic system that is customizable to respond to emergency and general FDA requirements for safety and patient outcome surveillance.

• Use of an FDA Real-time Mobile Communication Platform System during Medical Countermeasure Events: RAPID
  May 31, 2017 presentation at the 2017 FDA Science Forum by Henry "Skip" Francis, MD, Director for Data Mining and Informatics Evaluation and Research, CDER (recording at 53:20 – 1:11:00)

• Building a National Capability to Monitor and Assess MCM Use in Response to Public Health Emergencies
  June 6, 2017: Presentation by the FDA's RADM Carmen T. Maher on medical countermeasure monitoring and assessment (slides from keynote at the NAS workshop listed immediately above).

• Building a National Capability to Monitor and Assess Medical Countermeasure Use During a Public Health Emergency: Going Beyond the Last Mile: Proceedings of a Workshop
  October 31, 2017: Proceedings of the June 2017 NASEM workshop to further discussion on the need for a more robust national capability for monitoring and assessing MCMs during public health emergencies.

• Descriptive Analyses in the Sentinel System for the FDA Office of Counterterrorism and Emerging Threats (OCET)
  July 18, 2018 (posted): OCET-sponsored project to explore how the Sentinel System may inform study protocols for MCM safety and effectiveness, and to provide a baseline for comparison during a public health emergency.

• Public Health Emergencies: Unpacking Medical Countermeasures Management for Preparedness and Response
  September 7, 2018: Article in the American Journal of Public Health.

• Examining the Ability to Conduct Influenza Antiviral Effectiveness Studies in Sentinel by Improving Confounding Control
  March 21, 2019 (posted): OCS/OCET-sponsored project to explore how the Sentinel System may inform study protocols for medical countermeasure safety and effectiveness and to provide a valuable baseline for comparison during a public health emergency.

FDA Information regarding Medical Product Assessment in General

Public information from the FDA about monitoring and assessment of medical products for safety and effectiveness

• FDA Sentinel Initiative
  The FDA’s Sentinel System is an active surveillance system that uses routine querying tools and pre-existing electronic healthcare data from multiple sources to monitor the use and safety of regulated medical products.

• CBER Biologics Effectiveness and Safety (BEST) System
  Launched in October 2017 to expand and enhance CBER access to new and better data sources, methods, tools, expertise and infrastructure to conduct surveillance and epidemiologic studies. BEST is part of the Sentinel initiative and it promotes CBER's Office of Biostatistics and Epidemiology's (OBE) mission to assure the safety and effectiveness of biologic products including vaccines, blood and blood products, tissues and advanced therapeutics.

• CDER Conversation: The FDA'€™s Sentinel Initiative (November 2017)
  Robert Ball, MD, MPH, deputy director of CDER’s Office of Surveillance and Epidemiology, discusses Sentinel’s progress and future.

• Sentinel Post-licensure Rapid Immunization Safety Monitoring (PRISM) Program (archived FDA Voice blog post)
  PRISM, a component of FDA’s Sentinel initiative, is an active national vaccine safety surveillance system used to evaluate for vaccine-adverse events.

• National Evaluation System for health Technology (NEST)
  The FDA is building the foundation for a National Evaluation System for health Technology (NEST) to more efficiently generate better evidence for medical device evaluation and regulatory decision-making.

• Medical Device Epidemiology Network Initiative (MDEpiNet)
  The MDEpiNet initiative is a collaborative program through which the FDA’s Center for Devices and Radiological Health and external partners share information and resources to enhance our understanding of the safety and effectiveness of medical devices.

• FDA Adverse Event Reporting System (FAERS)
  FAERS is a database designed to support the FDA's post-marketing safety surveillance program for drug and therapeutic biologic products that contains information on adverse event and medication error reports submitted to the FDA.

• Vaccine Adverse Event Reporting System (VAERS)
  VAERS is a national vaccine safety surveillance program co-sponsored by the FDA and CDC to detect possible signals of adverse events associated with vaccines.

• Innovation in Medical Evidence Development and Surveillance (IMEDS) Program
  The IMEDS program, offered by the Reagan-Udall Foundation for the FDA, serves to advance the science and tools necessary to support post-market evidence generation on regulated products.

• Health Informatics at FDA
  Health informatics initiatives at the FDA combine the disciplines of information science, computer science, medicine, data science, and management science to evaluate and enable access to life-sustaining and life-saving products.

• Leveraging Real-World Data and Shared Clinical Trial Data to Inform Regulatory Decision-Making (Yale University-Mayo Clinic)
  The FDA has awarded Yale and Mayo Clinic a grant to establish a Center of Excellence in Regulatory Science and Innovation related to leveraging real-world data and shared clinical trial data to inform regulatory decision-making. 

• Use of Electronic Health Record Data in Clinical Investigations - Guidance for Industry (PDF, 327 KB)
  July 2018 - to assist sponsors, clinical investigators, contract research organizations, institutional review boards, and others on the use of electronic health record data in FDA- regulated clinical investigations.

Additional Resources

Resources from other sources, including other government agencies, relevant to monitoring and assessment of MCMs

• PHEMCE Strategy and Implementation Plan
  The PHEMCE annually assesses and updates a PHEMCE Strategy and Implementation Plan to ensure that PHEMCE priorities reflect current scientific progress and fiscal capabilities and to ensure optimal allocation of resources to address high-priority threats.

• BARDA core services, including CSN
  The BARDA Clinical Studies Network (CSN) is a network of clinical research organizations established to design and conduct clinical studies needed to develop MCMs.

• NIH Health Care Systems Research Collaboratory
  The NIH Collaboratory aims to strengthen the national capacity to implement cost-effective large-scale research studies that engage healthcare delivery organizations as research partners.

• NIH Disaster Research Response Program (DR2)
  The NIH Disaster Research Response Program (DR2) is the national framework for research on the medical and public health aspects of disasters and public health emergencies.

• HHS ASPR Technical Resources, Assistance Center, and Information Exchange (TRACIE)
  ASPR TRACIE was created to meet the information and technical assistance needs of regional ASPR staff, healthcare coalitions, healthcare entities, healthcare providers, emergency managers, public health practitioners, and other relevant stakeholders.

• CDC Vaccine Safety Datalink (VSD)
  The VSD is a collaborative project between CDC's Immunization Safety Office and nine health care organizations to monitor safety of vaccines and conduct studies about rare and serious adverse events following immunization.

• CDC Division of Health Informatics and Surveillance (DHIS)
  CDC's DHIS manages two public health surveillance programs that have cross-cutting utility for multiple CDC programs and public health jurisdictions.

• NAS Real-World Evidence Generation and Evaluation of Therapeutics A Workshop
  October 2016 public workshop hosted by NAS that examined opportunities and challenges for incorporating real-world evidence into evaluation of medical products.

• NAS Standing Committee on Medical and Public Health Research During Large-Scale Emergency Events
  Standing committee hosted by NAS that provides a venue for discussion of issues related to short- and long-term strategic planning

• Discovery, the Critical Care Research Network, University of Southern California
  Fosters collaborative research to promote the advancement of science in the field to improve outcomes for critically ill and injured patients.

• PCORnet, the National Patient-Centered Clinical Research Network
  PCORnet is designed to make it faster, easier, and less costly to conduct clinical research than is now possible by harnessing the power of large amounts of health data and patient partnerships.

• Observational Health Data Sciences and Informatics (OHDSI)
  The OHDSI program is a multi-stakeholder, interdisciplinary collaborative to bring out the value of health data through large-scale analytics.