Notice Number: NOT-FD-11-001

Key Dates
Release Date: August 19, 2011
Response Date:  September 30, 2011

Issued by
U.S. Food and Drug Administration

Purpose

Replace text below as appropriate. To facilitate conversion to HTML, text formatting in this document is limited.  To apply formatting, highlight the text and apply the style (described below).

In March 2011, the U.S. Food and Drug Administration (FDA) sponsored a workshop—held by the Institute of Medicine (IOM)—to identify opportunities to advance regulatory science for developing and evaluating medical countermeasures for chemical, biological, nuclear, radiological (CBRN) and naturally occurring emerging infectious disease (EID) threats–such as pandemic influenzaⁱ. The purpose of this request for information (RFI) is to build on the information elicited during the IOM workshop and garner information that will inform FDA investments in its medical countermeasures regulatory science program by identifying medical countermeasure regulatory science needs, enhancing FDA’s understanding of existing regulatory science capabilities, and identifying opportunities for collaboration.

Background

In August 2010, FDA launched its Medical Countermeasures Initiative (MCMi) in response to the U.S. Department of Health and Human Services’ comprehensive review of the Public Health Emergency Medical Countermeasures Enterprise (Enterprise)ⁱⁱ. The Enterprise Review lays out a vision for a flexible, nimble system to produce medical countermeasures rapidly in the face of any attack or threat, known or unknown and recommends—among other things—a robust 21st-Century medical countermeasures regulatory science program led by FDA to help achieve this vision.

FDA has organized its MCMi around a three pillar action plan based on the expected outcomes and elements of the 21st-Century regulatory science initiative as envisioned in the Enterprise Review:

• Pillar I: Enhance the Medical Countermeasure Regulatory Review Process and Capacity
  
• Pillar II: Advance Regulatory Science for Medical Countermeasure Development and Evaluation
  
• Pillar III: Modernize the Legal, Regulatory, and Policy Framework for Effective Public Health Response

Mission of the FDA Medical Countermeasures Initiative

The MCMi is designed to bolster the US government’s efforts to increase the availability of and access to safe, effective medical countermeasures—including drugs, vaccines, medical devices  (including diagnostic tests)—against CBRN and EID threats. The overall mission of the MCMi is to promote the development of medical countermeasures by enhancing FDA’s regulatory processes and fostering the establishment of clear regulatory pathways for medical countermeasures, and to facilitate the timely access to available medical countermeasures by establishing effective regulatory policies and mechanisms.

General Program Needs and Scope of Program

Regulatory science refers to the broad range of activities needed to develop and apply new methods, tools and standards to facilitate development of safe, effective, and high quality medical products, including products suitable for at-risk populations (e.g., children, pregnant women, and people with chronic health conditions). The goal of FDA’s MCMi regulatory science program (Pillar II) is to facilitate, simplify, and speed the development and evaluation of medical countermeasures by establishing medical countermeasure regulatory science programs that address complex scientific issues that impede medical countermeasure development.  For example, research aimed to incorporate innovative methodologies that lead to improved potency and sterility assays or may lead to improved data available for regulatory review, allowing for better informed benefit-risk, science-based regulatory decision-makingⁱⁱⁱ. Topic areas of special interest for medical countermeasure regulatory science include: (1) animal model development and qualification, including those for radiation injury protection, (2) identification and qualification  of  biomarkers for safety and efficacy, (3) immune responses including identification of correlates of protection, (4) methods to assess product quality and related product release assays, (5) risk communication to improve public health outcomes, (7) validation of next-generation in vitro diagnostics platforms, (9) assessing performance of emergency medical equipment, and (10) real-time tracking and evaluation of MCM safety and efficacy during public health emergencies.

Program Structure

The MCMi regulatory science program is focusing on high-priority projects that are of value for public health and national security based on Enterprise partner priorities. FDA is interested in hearing about potential projects that support the development and evaluation of specific medical countermeasure products, classes or groups of medical countermeasure products, or important medical countermeasure technologies. For more information on Enterprise partner medical countermeasure priorities see for example:

• HHS Public Health Emergency Medical Countermeasure Enterprise Implementation Plan for Chemical, Biological, Radiological and Nuclear Threats
  
• BARDA CBRN BAA- 11-100-SOL-00009 ^iv
  
• Report to the President on Reengineering the Influenza Vaccine Production Enterprise to Meet the Challenges of Pandemic Influenza

The MCMi regulatory science program comprises both intramural FDA and external, FDA-sponsored research components. FDA is strengthening and expanding its intramural medical countermeasures regulatory science program and plans to establish an extramural component to engage entities outside of FDA (e.g., other U.S. government agencies, academia, non-governmental organizations, and industry) in medical countermeasure regulatory science.

Information Requested

FDA is interested in obtaining stakeholder input into its medical countermeasure regulatory science program to help us assess and prioritize medical countermeasure regulatory science needs and gaps and further our understanding of existing regulatory science capabilities. Specifically, FDA is especially interested in the following:

• Medical Countermeasure Regulatory Science Needs

Please identify medical countermeasure regulatory science needs including:

• The specific medical countermeasure product, class or group of medical countermeasure products, or critical medical countermeasure technology that the regulatory science need applies to.
•  The medical countermeasure regulatory science topic area that the regulatory science need applies to (for example, animal model development and qualification including those for radiation injury protection; identification and qualification of biomarkers for safety and efficacy; immune responses including identification of correlates of protection; methods to assess product quality and related product release assays; risk communication to improve public health outcomes; validation of next-generation in vitro diagnostics platforms; assessing performance of emergency medical equipment; real-time tracking and evaluation of MCM safety and efficacy during public health emergencies; and any other relevant topics).
  
• How the regulatory science need identified relates to Enterprise partner medical countermeasure priorities as articulated by the Enterprise.
  
• The extent to which the identified regulatory science need is unmet and the significance of meeting this need with respect to furthering medical countermeasure development.

Potential Solutions to Medical Countermeasure Regulatory Science Needs

Please describe ideas and concepts that could lead to potential projects that would help advance regulatory science in areas of unmet needs, even if they involve radical changes to the current approaches and processes, including:

• An assessment of the potential for the project(s) to create significant value in improving the medical countermeasure development and evaluation pathway and regulatory decision-making process.
• The type and level of collaboration envisioned to complete the project(s) [e.g., independent investigator, multi-center, public-private-partnership, etc.].
• Concerns or limitations, if any, related to pursuing the project(s) (e.g., potential intellectual property issues). 

How to Submit a Response

Responses should be limited to a maximum of 5 pages and should be identified with NOT-FD-11-001. Responses are due by September 30, 2011.  Please submit your response to: [email protected].

This RFI is for information and planning purposes only and shall not be construed as a solicitation or as an obligation on the part of the Government. The Government does not intend to make any award on the basis of responses nor otherwise pay for the preparation of any information submitted or the Government's use of such information. Acknowledgement of receipt of responses will not be made, nor will respondents be notified of the Government's evaluation of the information received. However, should such a requirement materialize, no basis for claims against the Government shall arise as a result of a response to this request for information or the Government's use of such information as either part of our evaluation process or in developing specifications for any subsequent requirement. Information in responses will be held confidential to the extent permitted under applicable laws and regulations. Any proprietary, non-public information should be so marked.

All respondents are asked to indicate the type and size of your organization, e.g., Large Business, Small Business, Veteran-Owned Small Business, Service-Disabled Veteran-Owned Small Business, HUBZone Small Business, Small Disadvantaged Business, Women-Owned Business, 8(a), Historically Black College or University/Minority Institution (HBCU/MI), educational institution, profit/non-profit hospital, or other nonprofit organizations including those within the federal government.

Inquiries

Replace/delete all text below as appropriate for your notice
Please direct all inquiries to: [email protected]