	Background

Several federal departments and agencies have responsibilities for  assessing the threat of CBRN agents and determining requirements and  priorities for developing and acquiring medical countermeasures for these  agents, as part of their mission and, in some cases, as specifically required  by law. PHEMCE is a federal interagency  decision-making body responsible for providing recommendations to the  Secretary of HHS on (1) prioritized requirements for CBRN medical  countermeasures, (2) coordination of medical countermeasure  development and acquisition activities to address the requirements, and  (3) strategies for distributing medical countermeasures held in the U.S.  Strategic National Stockpile (SNS), the national repository of medications,  medical supplies, and equipment for use in a public health emergency. In addition, PHEMCE  includes officials from DHS, the Department of Defense (DOD), the  Department of Veterans Affairs, the Department of Agriculture, and the  Executive Office of the President. Within HHS, ASPR is responsible for leading federal government efforts to  research, develop, evaluate, and acquire public health emergency medical  countermeasures to prevent, treat, or mitigate the potential health effects  from exposure to CBRN agents. HHS, Through PHEMCE, Uses a Four-Step Process to Determine Acquisition Priorities for Medical Countermeasures and Oversees Their Development

HHS coordinates and leads federal efforts to determine CBRN medical  countermeasure priorities and develop and acquire CBRN medical  countermeasures, primarily through PHEMCE. HHS’s medical  countermeasure acquisition strategy is based on a four-step process: (1)  identify and assess the threat of CBRN agents, (2) assess medical and  public health consequences of attacks with these agents,   (3) establish medical countermeasure requirements, and (4) identify and  prioritize near-, mid-, and long-term development and acquisition  programs. Because desired CBRN medical countermeasures may not be  immediately available for acquisition, HHS oversees and supports the  various stages of research and development of these countermeasures,  also under PHEMCE. 1.) In the third step, PHEMCE uses the consequence modeling results to  determine requirements for needed medical countermeasures, including  the needed quantity and the desired characteristics, such as how they  would be used and stored. In this stage, potential medical  countermeasures are further evaluated in animal studies to demonstrate  safety and effectiveness for preventing, diagnosing, or treating disease in  humans. Challenges to Development and Acquisition of Medical Countermeasures Include High Failure Rates in Research and Difficulties Meeting Regulatory Requirements

The federal government faces a variety of challenges in developing and  acquiring medical countermeasures, such as the high failure rate in  research and development and difficulties meeting regulatory  requirements. One scientific challenge is that, as with other medical  products, the failure rate for development of certain CBRN medical  countermeasures can be high, depending on the stage of scientific  research and development. HHS estimates that the failure rate for  development and licensure of most drugs, vaccines, and diagnostic devices  in the early development stage can be more than 80 percent, with an  increasing probability of success as the product moves further through  development. Given the high risk of failure in research, as well as a lack of a commercial  market for most CBRN countermeasures, attracting companies  experienced in meeting the complex requirements necessary to develop a  new product is also challenging. As a result, FDA officials told us that they have to provide more regulatory  and scientific guidance to these companies than they might provide to  larger pharmaceutical companies, which generally have more experience  with bringing products through the regulatory process. There are also several challenges related to the regulatory processes for  evaluating the development of promising medical countermeasures. For  example, researchers face challenges proving the effectiveness of  potential countermeasures because they cannot ethically or feasibly test  the effectiveness of countermeasures on humans due to the dangers posed  by CBRN agents. Determining appropriate doses of CBRN countermeasures for children,  who may be more vulnerable to the adverse effects of a CBRN agent, also  involves regulatory challenges. Finally, CDC faces the logistical challenge of ongoing replenishment of  expiring medical countermeasures in the SNS.
Why GAO Did This Study

The anthrax attacks of 2001 and a radiation leak after the recent natural disaster in Japan highlighted concerns that the United States is vulnerable to threats from chemical, biological, radiological, and nuclear (CBRN) agents, which can cause widespread illness and death. Medical countermeasures--such as drugs, vaccines, and diagnostic devices--can prevent or treat the health effects of exposure, but few are currently available for many of these CBRN agents. GAO was asked to testify on the Department of Health and Human Services' (HHS) CBRN medical countermeasure development and acquisition activities. This statement focuses on (1) how HHS determines needed CBRN medical countermeasures and priorities for development and acquisition and (2) selected challenges to medical countermeasure development and acquisition. This statement of preliminary findings is based on ongoing work. To do this work, GAO examined relevant laws and presidential directives, analyzed federal agency documents and reports from advisory boards and expert groups, and interviewed officials from HHS and the Department of Homeland Security (DHS) about the processes for developing and acquiring CBRN medical countermeasures and the challenges related to those efforts. GAO shared the information in this statement with HHS. HHS provided technical comments, which GAO incorporated as appropriate.

What GAO Found

HHS coordinates and leads federal efforts to determine CBRN medical countermeasure priorities and develop and acquire CBRN medical countermeasures, primarily through an interagency body that includes other federal agencies with related responsibilities, such as DHS and the Department of Defense. HHS's medical countermeasure acquisition strategy is based on a four-step process: (1) identify and assess the threat of CBRN agents, (2) assess medical and public health consequences of attacks with these agents, (3) establish medical countermeasure requirements, and (4) identify and prioritize near-, mid-, and long-term development and acquisition. Through these processes, HHS determines which countermeasures to buy for specific CBRN agents, including the desired characteristics of these countermeasures--such as how many doses a vaccine requires to confer immunity--the needed quantity of certain medical countermeasures, and the acquisition priorities. While a few CBRN countermeasures can be immediately acquired, most have not yet been developed. Therefore, HHS and the interagency body support and oversee several stages of research and development to try to obtain usable countermeasures. These include basic cellular and biological research to understand the effects of these agents on humans; applied research to validate approaches, such as testing the effectiveness of treatment in animals; early development to assess the safety of potential countermeasures; and advanced development, in which the products are more fully evaluated for safety and effectiveness, including their formulation and manufacturing processes. The federal government faces a variety of challenges in developing and acquiring medical countermeasures, such as the high failure rate in research and development and difficulties meeting regulatory requirements. For example, the failure rate for development and licensure of most drugs, vaccines, and diagnostic devices can be more than 80 percent, depending on the stage of scientific research and development. Given this risk, as well as a lack of a commercial market for most medical countermeasures, attracting large, experienced pharmaceutical firms to research and develop them is challenging. Smaller biotechnology companies are more likely to be developing medical countermeasures, but HHS must provide more guidance to these less experienced small companies than might be typical with larger companies. In addition, several challenges exist related to regulatory processes for evaluating promising medical countermeasures. These challenges include (1) proving a countermeasure's effectiveness using animals as proxies for humans, because humans cannot ethically be used in studies involving CBRN agents; (2) determining appropriate doses of countermeasures for children, who may be more vulnerable to exposure to CBRN agents; and (3) evaluating the safety and effectiveness of medical countermeasures for use in a public health emergency if they have not yet been approved or licensed. Finally, HHS faces the logistical challenge of ongoing replenishment of expiring medical countermeasures in the U.S. Strategic National Stockpile, the national repository of medications, medical supplies, and equipment for public health emergencies.