Guidance and Other Information of Special Interest to MCM Stakeholders

FDA develops guidance to provide its policy perspectives and recommendations on a wide variety of topics. The guidance documents below may be of special interest to existing or prospective medical countermeasure (MCM) sponsors, and other stakeholders, including state, tribal, local and territorial public health preparedness personnel. 

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Current COVID-related guidances may be found using Guidance Document Search. For additional MCM-related guidance and industry announcements from previous years, please visit our archives, available by date.

On this page:

General Guidance

Indication-Specific Guidance (Drugs)

Vaccines, Gene Therapies, and Cell Therapies (Biologics)

Diagnostics, Medical Devices and Personal Protective Equipment (PPE)

Pediatric Guidance of Interest to MCM Stakeholders

Other Information of Interest (press releases, notices, etc.)

General Guidance

• Draft guidance: Risk Management Plans to Mitigate the Potential for Drug Shortages (May 2022) Also see: FDA Urges Drug Manufacturers to Develop Risk Management Plans to Promote a Stronger, Resilient Drug Supply Chain
• Availability of Regulatory Management Plans (Dec. 2019)
• Material Threat Medical Countermeasure Priority Review Vouchers - Draft Guidance for Industry (Jan. 2018)
• Emergency Use Authorization of Medical Products and Related Authorities - Guidance for Industry and Other Stakeholders (Jan. 2017)
• Product Development Under the Animal Rule - Final Guidance for Industry (Oct. 2015)

Additional select MCM-related guidance/information (General Guidance)

• May 2023: FDA is taking additional steps to support the use of decentralized clinical trials (DCTs) for drugs, biologics and devices, where some or all the trial-related activities occur at locations other than traditional clinical trial sites. The agency released a new draft guidance that provides recommendations for sponsors, investigators and other stakeholders regarding the implementation of DCTs to advance medical product development and research. Examples of decentralized elements include obtaining laboratory tests at a local facility rather than a research medical center or conducting a clinical follow-up visit in the trial participant’s home using telemedicine. Submit comments on the draft guidance by August 1, 2023. 

• April 2023: FDA announced the availability of a draft guidance to help prevent and mitigate drug shortages: Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients (API) Under Section 506C of the FD&C Act. The purpose of this guidance is to assist applicants and manufacturers in providing FDA timely, informative notifications about changes in the production of certain finished drugs and biological products as well as certain APIs that may, in turn, help the agency in its efforts to prevent and mitigate drug shortages. While some supply disruptions and product shortages cannot be predicted or prevented, early communication and detailed notifications to FDA from manufacturers play a significant role in decreasing the incidence, impact, and duration of supply disruptions and product shortages. These notifications allow the agency to evaluate the situation and determine an appropriate course of action. Submit comments by June 5, 2023.

• October 2020: Product developers may want to review this new web page, Developing and Manufacturing Drugs Including Biologics.

• December 2019: Draft guidance - Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products - To provide guidance to applicants planning to file new drug applications (NDAs), biologics license applications (BLAs), or applications for supplemental indications on the evidence to be provided to demonstrate effectiveness. 

• December 2019: FDA published a new web page, Availability of Regulatory Management Plans. The Pandemic and All-Hazards Preparedness Reauthorization Act of 2013 (PAHPRA), as amended by the Pandemic and All-Hazards Preparedness and Advancing Innovation Act of 2019 (PAHPAIA), furthered FDA’s mission of fostering the development and availability of medical countermeasures (MCMs) by codifying the development of Regulatory Management Plans (RMPs) for certain high-priority MCMs. Under section 565(f) of the Federal Food, Drug, and Cosmetic (FD&C) Act, sponsors or applicants of eligible MCMs can request an RMP, setting forth a formal process for obtaining scientific feedback and agency interactions regarding the development and regulatory review of eligible MCMs. This page provides information about RMPs for MCM sponsors or applicants. Also see: MCM-Related Counterterrorism Legislation

• September 2019: Draft guidance - Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products - Provides guidance to sponsors and applicants on interacting with the FDA on complex innovative trial design (CID) proposals for drugs or biological products. FDA is issuing this guidance to satisfy, in part, a mandate under section 3021 of the 21st Century Cures Act (Cures Act). In accordance with the Cures Act mandate, this guidance discusses the use of novel trial designs in the development and regulatory review of drugs and biological products, how sponsors may obtain feedback on technical issues related to modeling and simulation, and the types of quantitative and qualitative information that should be submitted for review. Comment by December 23, 2019.

• April 2019: FDA has posted a Compliance Program for the Inspection of Nonclinical Laboratories Conducting Animal Rule-Specific Studies (CP 7348.007) on its Bioresearch Monitoring Program (BIMO) Compliance Programs web page. This compliance program provides instructions for the inspection of nonclinical laboratories conducting the Animal Rule-specific studies (i.e., the natural history studies that define the animal model in which the efficacy of an investigational drug or biological product will be tested, the adequate and well-controlled animal efficacy studies intended to provide the primary evidence of effectiveness to support marketing approval of the product, and the pharmacokinetic and/or pharmacodynamic studies in animals used to select a dose and regimen in humans). Inspections of these studies are conducted to verify, to the extent practicable, the quality and integrity of the data contained in the final reports of the Animal Rule-specific studies submitted to FDA.

• February 2019: Electronic data standards for Animal Rule studies - notice of upcoming comment periods and webinar - FDA has been working with the Critical Path Institute and the Clinical Data Interchange Standards Consortium (CDISC) to develop electronic data standards for the natural history and efficacy studies conducted in animals that support Animal Rule applications. The draft Standard for Exchange Nonclinical Data (SEND) Implementation Guide for Animal Rule studies (SENDIG-AR) will be available for public review and comment on the CDISC website by February 25, 2019, with the comment period closing on April 29, 2019. A free webinar providing an overview of the SENDIG-AR is scheduled for March 5, 2019, 11:00 a.m. – 12:30 p.m. ET. A webinar recording is available.

• August 2018: FDA adds four tropical diseases to priority review voucher program to encourage drug development in areas of unmet need - FDA announced the addition of Lassa fever, chikungunya virus disease, rabies, and cryptococcal meningitis to the list of tropical diseases. Applicants who submit applications for drug or biological products to prevent or treat these diseases may qualify for a tropical disease priority review voucher (PRV). A tropical disease PRV can be used to obtain priority review of a subsequent drug application that does not itself qualify for priority review. (Federal Register notice)

• April 2018: Guidance for industry - Special Protocol Assessment (PDF, 182 KB) - This guidance provides information about the procedures and general policies adopted by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research for special protocol assessment (SPA). Several protocols are eligible for SPA, including animal efficacy protocols for studies intended to provide primary evidence of effectiveness required for approval or for licensure for products developed under the Animal Rule. This guidance finalizes the draft guidance of the same name issued May 4, 2016, and replaces the guidance of the same name issued May 17, 2002. (Federal Register notice) Also see: FDA In Brief: FDA advances policies to bring greater predictability and certainty to the drug development process

• January 2018: Draft guidance - Material Threat Medical Countermeasure Priority Review Vouchers (PDF, 174 KB) - The 21st Century Cures Act established a new priority review voucher (PRV) program to encourage development of material threat medical countermeasures (MCMs). This draft guidance explains to internal and external stakeholders how FDA intends to implement its material threat MCM PRV program. Comment by March 20, 2018. (Federal Register notice) Also see: FDA In Brief: FDA takes steps to spur development of medical countermeasures needed to protect, prepare for emerging threats to public health and national security and 21st Century Cures Act: MCM-Related Cures Provisions

• January  2017: Guidance - Emergency Use Authorization of Medical Products and Related Authorities - This guidance finalizes the draft guidance, Emergency Use Authorization of Medical Products and Related Authorities (April 2016) and replaces the following two guidance documents: Emergency Use Authorization of Medical Products (July 2007) and Emergency Use Authorization Questions and Answers (April 2009). (Federal Register notice)

• October 2015: Final Guidance - Product Development Under the Animal Rule (PDF, 563 KB) 

• February 2012: Postmarket Adverse Event Reporting for Medical Products and Dietary Supplements During an Influenza Pandemic (PDF, 218 KB) - FDA response to comments on this guidance (January 6, 2015)

• March 2011: Planning for the Effects of High Absenteeism to Ensure Availability of Medically Necessary Drug Products

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Indication-Specific Guidance (Drugs)

• Guidance, compliance and regulatory information for drugs (from CDER)

• Meetings, Conferences & Workshops (Drugs)

• April 19, 2023: FDA announced availability of a draft guidance for industry, Acute Radiation Syndrome: Developing Drugs for Prevention and Treatment. The purpose of this draft guidance is to provide information and recommendations to assist sponsors and other interested parties in the development of drugs to prevent or treat acute radiation syndrome (ARS) caused by exposure to ionizing radiation from accidental or deliberate events. Generally, drugs developed for such indications will require approval under the regulations commonly referred to as the Animal Rule. Submit comments by July 19, 2023.

• March 2, 2023: FDA issued a draft guidance, Potency Assay Considerations for Monoclonal Antibodies and Other Therapeutic Proteins Targeting Viral Pathogens. This guidance provides drug manufacturers with recommendations for developing and implementing potency assays to ensure each lot of monoclonal antibodies (mAbs) or other therapeutic proteins is produced consistently with the potency necessary to achieve efficacy and that potency is maintained over the shelf life of the product. In January 2021, FDA published a guidance specific to potency considerations for mAbs and therapeutic proteins targeting SARS-CoV-2 (the virus causing COVID-19). This draft guidance expands the scope of the January 2021 guidance to include all mAbs and other therapeutic proteins targeting viruses, not just mAbs targeting SARS-CoV-2. Submit comments by May 1, 2023. 

• January 19, 2023: FDA announced availability of a draft guidance for industry: Mpox: Development of Drugs and Biological Products. FDA is issuing this guidance to support sponsors in their development of drugs and biological products for mpox. This guidance provides nonclinical, virology, and clinical considerations for mpox drug and biological product development programs, with a focus on recommendations to support initiation of clinical trials. Preventive vaccines are not addressed in this guidance. Submit comments by March 21, 2023. 

Select MCM-related guidance/information (Drugs)

• September 29, 2021: FDA worked with the Clinical Data Interchange Standards Consortium (CDISC) to develop electronic data standards for the natural history and efficacy studies conducted in animals that support Animal Rule applications. The Standard for Exchange of Nonclinical Data (SEND) Implementation Guide-Animal Rule v1.0 (SENDIG-AR v1.0) was published by CDISC on September 17, 2019, and FDA’s support for these data standards began on March 15, 2020. SEND data sets will be required in Animal Rule submissions to the Center for Drug Evaluation and Research (CDER) for studies initiated after either March 15, 2022, or March 15, 2023, depending on the type of regulatory submission. To learn more about the requirements for data standards for Animal Rule submissions to CDER, see FDA’s Animal Rule Information web page. 

• December 2019: Draft guidance - Qualification Process for Drug Development Tools Guidance for Industry and FDA Staff - Provides FDA’s current thinking regarding the qualification process for drug development tools (DDTs) for a specific use, as defined in the 21st Century Cures Act. Qualification is a voluntary process that permits use of a DDT for its context of use— the defined boundaries within which the available data justifies use of the DDT(s)—across multiple drug development programs. DDTs are methods, materials, or measures that have the potential to facilitate drug development, including, for example, an animal model used for efficacy testing of medical countermeasures under the Animal Rule. 

• November 2019: Final guidance - Smallpox (Variola Virus) Infection: Developing Drugs for Treatment or Prevention - designed to assist drug manufacturers designing studies to appropriately establish the safety and efficacy of drugs to treat or prevent smallpox infection Also see: FDA In Brief: FDA issues final guidance for development of smallpox treatments as part of critical preparedness efforts

• April 2019: FDA published a final guidance for government public health and emergency response stakeholders: Extending Expiration Dates of Doxycycline Tablets and Capsules in Strategic Stockpiles. This document provides guidance to government stakeholders on testing to extend the shelf life (i.e., expiration date) under the FD&C Act of stockpiled doxycycline tablets and capsules for public health emergency preparedness and response purposes for an anthrax emergency. This finalizes the draft guidance published on April 25, 2017. (Federal Register notice) More on Expiration Dating Extension

• June 2018: In response to public feedback, FDA updated the format of the Antibacterial Susceptibility Test Interpretive Criteria webpages for clarity. The new format includes the pathogens that FDA recognizes as part of a standard and any exceptions, additions, or additional FDA-identified susceptibility test interpretive criteria for certain drug-bacteria combinations not included in a recognized standard. Also see: FDA-Recognized Antimicrobial Susceptibility Test Interpretive Criteria

• June 2018: Draft guidance for industry: Limited Population Pathway for Antibacterial and Antifungal Drugs (PDF, 128 KB) - The LPAD pathway is intended to encourage the development of certain antibacterial and antifungal drugs to help address the critical public health and patient care concern that has resulted from the current decline in antibacterial drug research and development as serious antibacterial and antifungal drug-resistant infections increase.  FDA is committed to using the tools at its disposal, including the LPAD pathway, to help encourage the development of safe and effective drug products that address unmet needs of patients with serious bacterial and fungal infections. (Federal Register notice) -  Also see: Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s efforts to foster discovery and development of new tools to fight antimicrobial-resistant infections

• May 2018: Guidance - Anthrax; Developing Drugs for Prophylaxis of Inhalational Anthrax (PDF, 116 KB) - The purpose of this guidance is to assist sponsors in the development of new drugs for the prophylaxis of inhalational anthrax. This guidance finalizes the draft guidance of the same name issued on February 16, 2016. (Federal Register notice) - Also see FDA In Brief: As part of a longstanding program encouraging the development of medical countermeasures; new FDA policy promotes innovation to thwart inhalational anthrax 

• April 2018: Draft guidance: Pregnant Women: Scientific and Ethical Considerations for Inclusion in Clinical Trials (PDF, 90KB) - This draft guidance discusses the ethical and scientific issues when considering the inclusion of pregnant women in clinical trials of drugs and biological products. This draft guidance is intended to advance scientific research in pregnant women, and discusses issues that should be considered within the framework of human subject protection regulations. (Federal Register notice)

• January 2018: FDA released draft guidance on its policies and procedures related to the designation of a qualified infectious disease product (QIDP) under the Generating Antibiotic Incentives Now (GAIN) Act: QIDP Designation Questions and Answers (PDF, 390 KB). (Federal Register notice)

• December 2017: FDA announced the establishment of the Susceptibility Test Interpretive Criteria website, which will help to efficiently update susceptibility test interpretive criteria for antimicrobial drugs  when necessary for public health, and may allow for more efficient development and evaluation of antimicrobial susceptibility test (AST) devices. (Federal Register notice) - Also see FDA launches new tool for sharing information that allows doctors to better manage antibiotic use; improve patient care; Treating Infections – FDA is Forging a More Efficient Path to Help Healthcare Providers Treat their Patients; and 21st Century Cures Act

• September 2017: Guidance - Advancement of Emerging Technology Applications for Pharmaceutical Innovation and Modernization (PDF, 71 KB) - provides recommendations to companies that are interested in participating in the FDA’s Emerging Technology Program (Federal Register notice) - also see FDA issues guidance to help advance novel technology to improve the reliability and safety and help lower the cost of pharmaceutical manufacturing

• August 2017: Guidance - Antibacterial Therapies for Patients With an Unmet Medical Need for the Treatment of Serious Bacterial Diseases (PDF, 149 KB) - This guidance finalizes the draft guidance of the same name issued July 2, 2013. (Federal Register notice)

• April 2011: Influenza: Developing Drugs for Treatment and/or Prophylaxis (PDF, 417 KB)

• November 2007: Smallpox (Variola) Infection: Developing Drugs for Treatment or Prevention (PDF, 242 KB) (Draft)

• March 2006: Internal Radioactive Contamination - Development of Decorporation Agents (PDF, 177 KB)

• August 2004: Calcium DTPA and Zinc DTPA Drug Products – Submitting a New Drug Application Guidance for Industry (PDF, 168 KB)

• January 2003: Prussian Blue Drug Products — Submitting a New Drug Application (PDF, 159 KB)

• December 2002: KI in Radiation Emergencies - Questions and Answers (PDF, 161 KB)

• December 2001: Potassium Iodide as a Thyroid Blocking Agent in Radiation Emergencies (PDF, 40 KB) 

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Vaccines, Gene Therapies, and Cell Therapies (Biologics) 

• Vaccine EUA Questions and Answers for Stakeholders

• Guidance, compliance and regulatory information for biologics (from CBER)

• Workshops, Meetings & Conferences (Biologics)

• Biosimilars Guidances

Select MCM-related guidance/information (Biologics)

• December 2019: Final guidance - Considerations for the Development of Dried Plasma Products Intended for Transfusion. This guidance provides recommendations for the development of safe and effective dried plasma products intended for transfusion; it finalizes the guidance of the same title dated October 2018.

• February 2019: Important Information for Human Cell, Tissue, and Cellular and Tissue-Based Product (HCT/P) Establishments Regarding Zika Virus Transmission Risk in the World - The Centers for Disease Control and Prevention (CDC) has changed information on its Blood and Tissue Safety webpage used to communicate epidemiological information about Zika virus (ZIKV) to the blood and tissue collection community. The webpage includes a world map of areas with risk of Zika for other countries and territories outside of U.S. states. A new process has been developed to indicate risk for these areas that assigns one of four categories. FDA considers countries and territories outside the U.S. states categorized as “Red” (current outbreak) or “Purple” (any prior or current reports of mosquito-borne Zika transmission) as areas with increased risk of ZIKV transmission. Also see: Guidance for Industry: Donor Screening Recommendations to Reduce the Risk of Transmission of Zika Virus by Human Cells, Tissues, and Cellular and Tissue-Based Products (PDF, 86 KB, updated May 2018)

• December 2018: Draft guidance - Bacterial Risk Control Strategies for Blood Collection Establishments and Transfusion Services to Enhance the Safety and Availability of Platelets for Transfusion  - Provides recommendations for additional measures to help control the risk of bacterial contamination of room temperature stored platelets intended for transfusion. (Federal Register notice) Also see: FDA In Brief: FDA promotes the development and adoption of innovations that can ensure the continued safety of the U.S. blood supply

• January 2018: FDA issues new rule to improve efficiency, effectiveness of oversight over biologics manufacturing - FDA has issued a direct final rule that amends the general biologics regulations to remove outdated requirements and help eliminate inconsistencies and duplicative processes – specifically, how frequently the FDA is inspecting certain facilities and the actual duties of the inspector.

• December 2017: Draft guidance - Chemistry, Manufacturing, and Controls (CMC) Changes to an Approved Application: Certain Biological Products (PDF, 252 KB) - The draft guidance is intended to assist applicants and manufacturers of certain licensed biological products in determining which reporting category is appropriate for a change in CMC information to an approved biologics license application (BLA).  The updated guidance applies to certain biological products licensed under the Public Health Service Act, including in vitro diagnostics licensed under BLAs. (Federal Register notice)

• January 2017: Guidance - Recommendations for Assessment of Blood Donor Eligibility, Donor Deferral and Blood Product Management in Response to Ebola Virus (PDF, 99 KB) - notifies blood establishments that FDA has determined Ebola virus to be a transfusion-transmitted infection (TTI) and provides blood establishments that collect blood and blood components for transfusion or further manufacture, including Source Plasma, with FDA recommendations for assessing blood donor eligibility, donor deferral, and blood product management in the event that an outbreak of Ebola virus disease (EVD) with widespread transmission is declared in at least one country. The guidance document applies to Ebola virus (species Zaire ebolavirus). The recommendations apply to routine collection of blood and blood components for transfusion or further manufacture, including Source Plasma. The guidance announced in this notice finalizes the draft guidance of the same title dated December 2015. (Federal Register notice)

• May 2015: Final Rule - Requirements for Blood and Blood Components Intended for Transfusion or for Further Manufacturing Use - To better assure the safety of the nation's blood supply and to help protect donor health, FDA is revising the requirements for blood establishments to test donors for infectious disease, and to determine that donors are eligible to donate and that donations are suitable for transfusion or further manufacture.

• February 2010: Characterization and Qualification of Cell Substrates and other Biological Starting Materials Used in the Production of Viral Vaccines for the Prevention and Treatment of Infectious Diseases 

• September 2007: Manufacturing Biological Intermediates and Biological Drug Substances Using Spore-Forming Microorganisms (PDF, 184 KB)

• November 2007: Considerations for Plasmid DNA Vaccines for Infectious Disease Indications (PDF, 88 KB)

• May 2007: Clinical Data Needed to Support the Licensure of Pandemic Influenza Vaccines (PDF, 188 KB)

• February 2006: Considerations for Developmental Toxicity Studies for Preventive and Therapeutic Vaccines for Infectious Disease Indications (PDF, 50 KB)

• October 2001: Recommendations for Assessment of Donor Suitability and Blood and Blood Product Safety in Cases of Possible Exposure to Anthrax (PDF, 37 KB)

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Diagnostics, Medical Devices and Personal Protective Equipment (PPE)

• Device advice: comprehensive regulatory assistance (from CDRH), including How to Market Your Device

• Medical device webinars and stakeholder calls

• Workshops & Conferences (Medical Devices)

• Expedited Access Pathway Program - a voluntary program for certain medical devices that demonstrate the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions that are subject to premarket approval applications (PMA) or are eligible for de novo requests​​​​

Select MCM-related guidance/information (Devices)

• March 2023: FDA issued a guidance for immediate implementation: Cybersecurity in Medical Devices: Refuse to Accept Policy for Cyber Devices Under Section 524B of the FD&C Act. FDA generally intends not to issue “refuse to accept” (RTA) decisions for premarket submissions for cyber devices that are submitted before October 1, 2023, based solely on information required by section 524B of the FD&C Act. Instead, the FDA will work collaboratively with sponsors of such premarket submissions as part of the interactive and/or deficiency review process.

• November 2020: Final guidance - Regulatory Considerations for Microneedling Products - to provide industry with clarity about how to determine whether a microneedling product meets the definition of a medical device (section 201(h) of the Federal Food, Drug, and Cosmetic Act)

• March 2020: FDA's Center for Devices and Radiological Health (CDRH) has posted FAQs on Diagnostic Testing for SARS-CoV-2 

• February 2020: Policy for Diagnostics Testing in Laboratories Certified to Perform High Complexity Testing under CLIA prior to Emergency Use Authorization for Coronavirus Disease-2019 during the Public Health Emergency – As part of FDA’s ongoing and aggressive commitment to address the coronavirus outbreak, the agency issued a new policy for certain laboratories seeking to develop diagnostic tests for coronavirus in order to achieve more rapid testing capacity in the U.S. Also see: Coronavirus (COVID-19) Update: FDA Issues New Policy to Help Expedite Availability of Diagnostics

• March 2019: FDA finalizes requirements to help foster access to safe and effective tests to detect anthrax-causing bacteria - FDA issued a final rule classifying in vitro diagnostic devices for the detection of Bacillus bacteria into class II (special controls) and continuing to require a premarket notification (510(k)) for these devices. This final rule and the accompanying special controls guideline will help assure manufacturers continue to use appropriate practices for these devices and provide consistent information on testing criteria and performance evaluations for bringing safe and effective new Bacillus bacteria detection devices to market. Bacillus bacteria detection devices are prescription devices that provide a preliminary identification of Bacillus anthracis and other Bacillus species to help diagnose cases of anthrax and other diseases caused by Bacillus bacteria. These devices were previously unclassified preamendments devices that were legally marketed prior to May 28, 1976, when the Medical Device Amendments to the Federal Food, Drug, and Cosmetic Act were signed into law.

• December 2018: Guidance - Breakthrough Devices Program - Describes policies that FDA intends to use to implement the new Breakthrough Devices Program, established by the 21st Century Cures Act. The Breakthrough Devices Program supersedes and combines elements from FDA's Expedited Access Pathway (EAP), which was intended to facilitate the development and expedite review of certain devices that demonstrate the potential to address unmet medical needs, as well as the Priority Review Program, which implemented statutory criteria for granting priority review to premarket approval applications (PMAs) and applied those criteria to other types of premarket submissions for medical devices. (Federal Register notice) FDA will host a webinar on January 17, 2019, to help clarify the Breakthrough Devices Program final guidance for manufacturers. Also see: Statement from FDA Commissioner Scott Gottlieb, M.D., and Jeff Shuren, M.D., Director of the Center for Devices and Radiological Health, on new steps to promote innovations in medical devices that help advance patient safety, and Breakthrough Devices Program

• November 2018: FDA published two draft guidance documents that aim to help manufacturers of in vitro diagnostic (IVD) devices apply for and receive Clinical Laboratory Improvement Amendments (CLIA) waivers: Select Updates for Recommendations for CLIA Waiver Applications for Manufacturers of IVD Devices (Federal Register notice) and Recommendations for Dual 510(k) and CLIA Waiver by Application Studies (Federal Register notice). Both draft guidances were originally published on November 29, 2017 and have been updated based on feedback received during the open comment period. Both guidances are being re-issued as draft guidances to allow additional comments. CDRH will host a webinar for industry on these draft guidances on January 9, 2019. Also see: Clinical Laboratory Improvement Amendments (CLIA)

• October 2018: Draft guidance - Content of Premarket Submissions for Management of Cybersecurity in Medical Devices - Provides FDA’s updated recommendations for the device design, labeling, and documentation to be included in premarket submissions for devices with cybersecurity risks. This draft replaces the previous guidance finalized in 2014. (Federal Register notice) Also see: FDA In Brief: FDA proposes updated cybersecurity recommendations to help ensure device manufacturers are adequately addressing evolving cybersecurity threats

• May 2018: A Federal Register notice announces the FDA’s exemption of certain N95 filtering facepiece respirators and surgical N95 respirators (herein collectively referred to as “N95s”) from premarket notification [510(k)] requirements. The final order and NIOSH Memorandum of Understanding (MOU) streamline the regulation of N95s to help manufacturers easily identify, understand, and work to meet marketing requirements, and help ensure the availability of safe and effective medical products, particularly during times of increased demand, such as a public health emergency. Also see: FDA exempts certain N95 respirators from premarket notification requirements (PDF, 212 KB), and NIOSH and FDA Collaboration Streamlines Regulatory Oversight for N95 Filtering Facepiece Respirators

• December 2017: Draft guidance - Investigational In Vitro Diagnostics (IVDs) Used in Clinical Investigations of Therapeutic Products (PDF, 731 KB) - This draft guidance is intended to assist sponsors of clinical investigations of therapeutic products that also include investigational IVDs and Institutional Review Boards (IRBs) that review such investigations in complying with the Investigational Device Exemption (IDE) regulation. (Federal Register notice) Also see Statement from FDA Commissioner Scott Gottlieb, MD, on new FDA efforts to support more efficient development of targeted therapies

• December 2017: Guidance: Technical Considerations for Additive Manufactured Medical Devices guidance (PDF, 619 KB) - FDA is the first in the world to provide a comprehensive technical framework to advise manufacturers creating medical products on 3D printers. On January 10, 2018, FDA will hold a webinar for industry to review this guidance and answer questions. Also see: Statement by FDA Commissioner Scott Gottlieb, MD, on FDA ushering in new era of 3D printing of medical products, and 3D Printing of Medical Devices

• October 2017: Draft guidance - Breakthrough Devices Program - This program is intended to help patients have more timely access to devices and breakthrough technologies that provide for more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases, for which no approved or cleared treatment exists or that offer significant advantages over existing approved or cleared alternatives. This guidance describes the policies that the agency intends to use to implement the program. (Federal Register notice)

• October 2017: FDA has qualified the first tool under the medical device development tools (MDDT) program, a voluntary process intended to reduce regulatory burden for medical device developers and FDA reviewers through the qualification of tools that can aid in the development and evaluation of medical devices. Also see: Medical Device Development Tools: Helping to Speed Medical Device Evaluation and Approval

• September 2017: Draft guidance - Regulatory Considerations for Microneedling Devices (PDF, 441 KB) - this draft guidance is being issued to assist industry in understanding when a microneedling product is a device as defined in the FD&C Act (Federal Register notice)

• September 2017: Guidance - Evaluation and Reporting of Age, Race, and Ethnicity Data in Medical Device Clinical Studies (PDF, 1.1 MB) - the primary intent of these recommendations is to improve the quality, consistency, and transparency of data regarding the performance of medical devices within specific age, race, and ethnic groups (Federal Register notice) - FDA will discuss this final guidance document and respond to questions during a webinar on October 31, 2017, 12:00 - 1:30 p.m. ET.

• August 2017: Guidance - FDA released the final guidance Qualification of Medical Device Development Tools - Guidance for Industry, Tool Developers, and Food and Drug Administration Staff (PDF, 174 KB). This guidance formalizes the Medical Device Development Tools (MDDT) program, which the FDA launched as a pilot in 2013 (Federal Register notice).  More about MDDT

• June 2017: Final rule - technical amendment: FDA is amending regulations to reflect changes recently enacted into law by the 21st Century Cures Act. Specifically, certain requirements related to humanitarian device exemptions (HDEs) and institutional review boards (IRBs) for devices have changed. This action is being taken to align the regulations with the FD&C Act as amended.

• May 2016: Draft Guidance - Infectious Disease Next Generation Sequencing Based Diagnostic Devices: Microbial Identification and Detection of Antimicrobial Resistance and Virulence Markers  - (Federal Register notice)

• April 2016: Final Guidance - Radiation Biodosimetry Medical Countermeasure Devices (PDF, 514 KB) (Federal Register notice)

• December 2015: Final Guidance - Premarket Notification Requirements Concerning Gowns Intended for Use in Health Care Settings (PDF, 319 KB) (Federal Register notice) - read more about medical gowns 

• August 2014: Highly Multiplexed Microbiological/Medical Countermeasure In Vitro Nucleic Acid Based Diagnostic Devices (PDF, 799 KB)

• April 2014: Types of Communication During the Review of Medical Device Submissions (PDF, 134 KB)

• March 2014: Premarket Assessment of Pediatric Medical Devices (PDF, 137 KB)

• November 2011: In Vitro Diagnostic Devices for Yersinia spp. Detection Class II Special Controls Guidance Document (PDF, 173 KB) (Draft)

• July 2011: Establishing the Performance Characteristics of In Vitro Diagnostic Devices for the Detection or the Detection and Differentiation of Influenza Viruses (PDF, 339 KB)

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Pediatric Guidances of Interest to MCM Stakeholders

Also see the Pediatric Medical Countermeasures guidance page from CDER, including considerations for bioterrorism and radiation emergencies.

• May 17, 2023: FDA issues two draft guidances for industry to support the approval of pediatric drug products - FDA issued two draft guidances for industry entitled, Pediatric Drug Development: Regulatory Considerations – Complying with PREA and Qualifying for Pediatric Exclusivity Under the BPCA and Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations. Both guidances, once finalized, will provide recommendations to support the assessment of pediatric drugs, biological products, and vaccines under the Pediatric Research Equity Act (PREA) and/or the Best Pharmaceuticals for Children Act (BPCA). The guidance documents revise and replace the draft guidance for industry How to Comply with the Pediatric Research Equity Act. Comments on both draft guidances are due by July 17, 2023. 

Select MCM-related guidance/information (Pediatrics)

• March 2019: Guidance - Pediatric Information Incorporated Into Human Prescription Drug and Biological Product Labeling - To assist applicants in determining the appropriate placement and content of pediatric information in human prescription drug and biological product labeling as described in the regulations for the content and format of labeling for human prescription drug and biological products. This guidance finalizes the draft guidance issued on February 28, 2013.

• April 2018: Guidance for industry:  E11(R1) Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population (PDF, 354 KB) - The guidance was prepared under the auspices of the International Council for Harmonisation (ICH), and is an addendum to the guidance published in 2000 entitled E11 Clinical Investigation of Medicinal Products in the Pediatric Population. This addendum complements and provides clarification and current regulatory perspective on topics in pediatric drug development. (Federal Register notice)

• June 2016: Final Guidance - Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices (PDF, 696 KB) - On August 8, 2016, FDA will host a webinar on this guidance, which provides a framework to consider extrapolating existing data to evaluate a device’s performance in pediatric patients in pre-market approval applications (PMAs), humanitarian device exemptions (HDEs) and de novo requests. It also facilitates continued efforts to address unmet medical device needs for pediatric patients.

• March 2016: Draft Guidance for Industry - Pediatric Study Plans: Content of and Process for Submitting Initial Pediatric Study Plans and Amended Initial Pediatric Study Plans (PDF, 408 KB) (Federal Register notice) 

• May 2015: Draft guidance - Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices (PDF, 1.1 MB)

• December 2014: Draft guidance - General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Products (PDF, 375 KB)

• November 2014: Draft guidance - Rare Pediatric Disease Priority Review Vouchers

• May 2014: Providing Information about Pediatric Uses of Medical Devices (PDF, 177 KB)

• March 2014: Premarket Assessment of Pediatric Medical Devices (PDF, 162 KB)

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Other Information of Interest (press releases, notices, etc.)

• Animal Rule information and links

• Executive Order 13944 List of Essential Medicines, Medical Countermeasures, and Critical Inputs

• FAQs: What happens to EUAs when a public health emergency ends?

• Fast Track, Breakthrough Therapy, Accelerated Approval and Priority Review

• Designating an Orphan Product: Drugs and Biological Products

• Electronic regulatory submissions and review helpful links and presentations with updated info (CDER)

• Drug Supply Chain Security Act (DSCSA) information, including FAQs on product tracing requirements

• Questions and Answers on Current Good Manufacturing Practices, Good Guidance Practices, Level 2 Guidance - Records and Reports

• Study data standards resources, including Study Data Technical Conformance Guide v2.0 (December 17, 2014)

• For Industry: Using Social Media (draft guidance); slides from July 10, 2014 webinar (PDF, 157 KB) about this draft guidance; social media draft guidance webinar Q&As (PDF, 97 KB)

Select MCM-related guidance/information (Other)

• November 2018: FDA is posting links to computer code and a roadmap that will allow researchers and developers to customize and use the newly created MyStudies app. Patients can securely enroll and participate in large scale pragmatic clinical trials or registries involving multiple health care systems or data sources.  The agency expects that the MyStudies app will aid researchers and industry in collecting real world patient level data and that these data, when linked to existing electronic health data, will promote efficiencies in drug development and drug safety monitoring processes. The MyStudies app is also capable of supporting clinical trials that comply with FDA guidance and regulations regarding data authenticity, integrity, and confidentiality. Also see: FDA In Brief: FDA launches new digital tool to help capture real world data from patients to help inform regulatory decision-making

• April 2018: FDA is conducting a Model-Informed Drug Development (MIDD) Pilot Program to facilitate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources. MIDD approaches use a variety of quantitative methods to help balance the risks and benefits of drug products in development. When successfully applied, MIDD approaches can improve clinical trial efficiency, increase the probability of regulatory success, and optimize drug dosing/therapeutic individualization in the absence of dedicated trials. FDA will accept requests to participate in the program on a continuous basis beginning on April 13, 2018 through June 15, 2022. See the Federal Register notice for additional information.

• January 2018: FDA launched a new set of web pages that aims to provide a one-stop source for general information about Risk Evaluation and Mitigation Strategy (REMS) programs.

• October 2017: FDA Issues Final Guidance Clarifying FDA and EPA Jurisdiction over Mosquito-Related Products (archived) - The final Guidance for Industry #236 – Clarification of FDA and EPA Jurisdiction over Mosquito-Related Products (PDF, 85 KB) – clarifies that mosquito-related products intended to function as pesticides by preventing, destroying, repelling, or mitigating mosquitoes for population control purposes, and that are not intended to cure, mitigate, treat, or prevent a disease are not “drugs” under the Federal Food, Drug, & Cosmetic Act, and will be regulated by the EPA under the Federal Insecticide, Fungicide, and Rodenticide Act. The FDA will continue to have jurisdiction over mosquito-related products that are intended to prevent, treat, mitigate, or cure a disease (including by an intent to reduce the level, replication, or transmissibility of a pathogen in mosquitoes). (Federal Register notice) Also see Genetically Engineered Mosquitoes on the FDA Zika Response Updates page

• June 2014: Final rule - list of qualifying pathogens under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act (FDASIA). GAIN is intended to encourage development of new antibacterial and antifungal drugs for the treatment of serious or life-threatening infections, and provides incentives such as eligibility for designation as a fast-track product and an additional 5 years of exclusivity to be added to certain exclusivity periods.
   
  FDA has determined that the following pathogens comprise the list of “qualifying pathogens:” Acinetobacter species, Aspergillus species, Burkholderia cepacia complex, Campylobacter species, Candida species, Clostridium difficile, Coccidioides species, Cryptococcus species, Enterobacteriaceae (e.g., Klebsiella pneumoniae), Enterococcus species, Helicobacter pylori, Mycobacterium tuberculosis complex, Neisseria gonorrhoeae, N. meningitidis, Non-tuberculous mycobacteria species, Pseudomonas species, Staphylococcus aureus, Streptococcus agalactiae, S. pneumoniae, S. pyogenes, and Vibrio cholerae. For more information, view the Federal Register notice   Also see January 2018 draft guidance: QIDP Designation Questions and Answers (PDF, 390 KB). Comment by April 2, 2018. (Federal Register notice) and statement from Janet Woodcock, MD on examining ways to combat Antibiotic Resistance and Foster New Drug Development (June 14, 2016)

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Related Links

• Animal Rule Information
• 21st Century Cures Act: MCM-Related Cures Provisions
• Pandemic and All-Hazards Preparedness Reauthorization Act of 2013 (PAHPRA)
• FDA Basics for Industry: About Guidances
• Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review