Statement by
Stewart Simonson, J.D.
Assistant Secretary
Office Of Public Health Emergency Preparedness
U.S. Department Of Health And Human Services

BioShield: HHS Biodefense Preparedness Efforts

Committee on Appropriations
Subcommittee on Homeland Security
United States Senate

April 28, 2005

Good morning, Mr. Chairman, Senator Byrd and Subcommittee members. I am Stewart Simonson, Assistant Secretary for Public Health Emergency Preparedness. I appreciate the opportunity to share with you information on our progress in implementing the Project BioShield Act of 2004, which was enacted some nine months ago. Biodefense is a top priority for the Bush Administration and having an appropriate armamentarium of medical countermeasures is a critical aspect of the response and recovery component of the President’s “21st Century Strategy for Biodefense.” The acquisition and ready availability of medical countermeasures, such as antibiotics, monoclonal and polyclonal antibodies against infectious threats, therapies for chemical and radiation-induced diseases, and vaccines to protect against exposure from biological agents will have a substantial impact on our preparedness and response capabilities.

Protecting Americans
The events of September and October 2001 made it very clear that terrorism—indeed bioterrorism—is a serious threat to our Nation and the world. The Bush Administration and Congress responded forcefully to this threat by seeking to strengthen our medical and public health capacities to protect our citizens from future attacks. The Bioterrorism Act of 2002 substantially increased funding authorization for the Centers for Disease Control and Prevention’s Strategic National Stockpile. To encourage the development of new medical countermeasures against biological, chemical, or radiological agents and to speed their delivery and use in the time of an attack, President Bush, in his 2003 State of the Union address proposed and Congress subsequently enacted the Project BioShield Act of 2004. The Special Reserve Fund, pre-appropriated with $5.6 billion was created to assure developers of medical countermeasures that funds would be available to purchase critical products for use to protect our citizens.

The Strategic National Stockpile Today
The wake-up call that we received in the fall of 2001 brought clarity to the gaps in our medical countermeasure armamentarium and we immediately sought to address them. Although there is much work still to be done, we have made significant progress in building our Strategic National Stockpile from that time to what we have on-hand today. For example, our smallpox vaccine stockpile has grown from 90,000 ready-to-use doses in 2001 to enough vaccine to protect every man, woman, and child in America. Major strides have been made in building our medical countermeasure reserve against anthrax, plague, and tularemia. We are now able to protect and treat millions of Americans in the event of an attack with one of these agents. We have taken the botulism antitoxin program started by the Department of Defense in the early 1990s to completion and we are now building our antitoxin stockpile further. We have also built our stockpile of countermeasures to address the effects of radiation exposure with products such as Prussian Blue and diethylenetriaminepentaacetate, or DTPA. These countermeasures act to block uptake or remove radioactive elements such as cesium, thallium, or americium from the body after they are ingested or inhaled. Potassium iodide, a drug that can protect the thyroid from the harmful effects of radioactive iodine, is also in the Stockpile.

The Strategic Approach to Addressing Medical Countermeasure Gaps
The initial focus of our efforts to protect the nation was aimed largely at those threats that could do the greatest harm to the greatest number of our citizens, namely, smallpox and anthrax. A sense of urgency has pervaded our efforts and has defined new ways of doing business. Our new national security environment demanded accelerated product development timelines and new paradigms of interactions between industry and government with risk-sharing and enhanced intra-governmental collaboration. Using a robust interagency process, that mined intra- and extra-governmental expertise, requirements for medical countermeasures were identified, and options elaborated for addressing immediate and long-term needs. These experts continue to help us define the most expeditious way to traverse the critical pathway to develop and acquire usable countermeasures for the Strategic National Stockpile.

Application of the strategic approach: Anthrax.
Although not transmissible from person-to-person, an attack involving the aerosol dissemination of anthrax spores, particularly in an urban setting, was considered by public health experts to have the potential for catastrophic effects similar to smallpox... The potential for large-scale population exposure following aerosol release of anthrax spores, the threat demonstrated by the anthrax letters, and our knowledge that anthrax had been weaponized by state-actors, highlighted the nature of the treat. The Secretary of the Department of Homeland Security determined that anthrax posed a material threat to the Nation. And, because untreated inhalation anthrax is usually fatal, the Secretary of HHS identified anthrax as a significant threat to public health.

The approach to protect citizens against this threat demanded immediate, intermediate and long-term strategies and requirements. First, the existing stockpile of antibiotics in the Strategic National Stockpile was increased. Second, there was a need for a licensed vaccine to be used not only for pre-exposure protection for laboratory and other workers at known risk for anthrax, but for use along with antibiotics after an exposure to potentially decrease the currently recommended 60-day course of antibiotic therapy. Anthrax spores are stable in the environment and would have a profound impact if released in an urban population. Availability of a vaccine is a critical requirement for repopulation and restoration of the functionality of any exposed area.

The limitations inherent in the currently available anthrax vaccine were articulated in a 2002 Institute of Medicine report, “Anthrax Vaccine: Is It Safe? Does it Work?” The report stated, “...a new vaccine, developed according to more modern principles of vaccinology, is urgently needed.” An assessment of developing technologies was undertaken by HHS experts in the fall of 2001 and the decision was made that there was a sufficient scientific foundation, including a detailed understanding of the pathogenesis of anthrax and how anthrax vaccines provide protective immunity, to support the aggressive development of a next generation vaccine consisting of recombinant protective antigen (rPA). This research, spanning more than a decade from its inception in the early 1990s, was conducted in large part by the United States Army Medical Research Institute of Infectious Diseases at Fort Detrick, Maryland.

HHS defined a three-stage development and acquisition strategy with open competition for awards at each stage. The early and advanced development programs were supported by the National Institutes of Health’s National Institute of Allergy and Infectious Diseases with contract awards in September 2002 and 2003, respectively. These were milestone-driven contracts with well-defined deliverables including the manufacture of clinical-grade vaccine and the conduct of Phase 1 and Phase 2 clinical trials. Large-scale manufacturing capacity would be required to support the civilian requirement for this medical countermeasure, which was defined through an interagency process to be the initial protection of up to 25 million persons. Senior officials throughout the United States government evaluated acquisition options to achieve this requirement and, in the fall of 2003, the decision was made to pursue the acquisition of rPA anthrax vaccine.

An evaluation of the status of the NIAID rPA anthrax vaccine development program suggested rPA vaccine could potentially become a licensed product within 8 years. In March 2004, the acquisition program for this vaccine, under the direction of my office, was launched using the Special Reserve Fund created in the FY 2004 Department of Homeland Security appropriations bill.

Utilizing a robust technical and business evaluation process, we reviewed multiple proposals and finally negotiated a contract with VaxGen of Brisbane, California, for 75 million doses of the vaccine, (anticipating a three-dose regimen). Using a milestone and deliverables approach utilized with the ACAM2000 smallpox vaccine development and acquisition program, and the rPA anthrax vaccine development related contracts at NIAID, the VaxGen contract lays out an ambitious program to include the delivery of the first 25 million usable vaccine doses to the Strategic National Stockpile within 2 years of contract award.

A unique and critical aspect of the rPA vaccine BioShield acquisition contract is the fact that no payment is made until a usable product is delivered to the Stockpile. While awaiting delivery of this new vaccine to the Stockpile my office will complete negotiations for 5 million doses of the currently licensed anthrax vaccine in the next few days to support immediate requirements. Delivery of the product to the Stockpile will begin very soon after the contract award and will have a direct impact on our preparedness.

Other Needed Countermeasures.
In an effort to fill other gaps in the Stockpile, we have made progress in contracting for products that will soon be delivered for use.

Potassium Iodide.
In March 2005 a contract was awarded under Project BioShield for a pediatric liquid formulation of potassium iodide, a drug that helps limit risk of damage to the thyroid, from radioactive iodine. This formulation is aimed at young children who cannot take pills and are at the highest risk of harmful effects from exposure to radioactive iodine. This acquisition will provide needed protection for at least 1.7 million children. Product delivery will begin next month.

Ongoing Project BioShield activities.
In addition to the Project BioShield acquisition contracts that have been awarded in the last nine months, there are several other important BioShield procurement-related activities underway. We are reviewing the responses for Requests for Proposals for anthrax therapies, and we are continuing to move forward on the acquisition of an antitoxin treatment for botulism. Furthermore, to signal our intent to acquire a next generation smallpox vaccine, we will be releasing a draft request for proposal for industry comment within the next two weeks. Finally, in anticipation of yet to be determined requirements, we actively monitor the state of the medical countermeasure pipeline-- both within and outside the government--- by evaluating USG research and development portfolios and engaging industry through the publication of Requests for Information (RFIs). For example, we have recently released three RFIs to assess the timeline to maturity of medical countermeasures to treat nerve agent exposure, acute radiation syndrome, and additional products that might be available to treat anthrax. These requests are a key tool for HHS to dialogue with industry partners and to inform the development of sound USG acquisition strategies.

Priority Setting Beyond Smallpox and Anthrax
The approach taken to rapidly expand our Nation’s response capacity to meet the medical and public health impact of either a smallpox or anthrax attack demonstrate our national resolve to address these threats. But, in many ways, anthrax and smallpox represent the “low hanging fruit” for medical countermeasure research, development and acquisition and was enabled by a substantial research base developed by USAMRIID and NIH. There was consensus that these were our highest priorities and we had countermeasures available or relatively far along in the development pipeline to permit acquisition. Given an almost endless list of potential threats with finite resources to address them, prioritization is essential to focus our efforts. We rely heavily upon our interagency partner, the Department of Homeland Security, to provide us with a prioritized list of threats along with material threat assessments that will provide reasonable estimates of population exposure. This information is critical for future strategic decision making regarding how best to focus our National efforts in countermeasure development and acquisition, including whether in the short-term, the so-called “one-bug, one-drug” approach should continue while simultaneously investing in more broad-spectrum prevention and treatment approaches for the longer term.

Challenges to Rapidly Expanding the Strategic National Stockpile
Although defining priorities and quantifying the size of the threat to the population are the key steps to focus our efforts, we must be mindful of the realities of the spectrum of efforts needed along the research and development pipeline to produce a useable medical countermeasure. The process of defining required specifications for a countermeasure often reveals few, if any, candidates in the pipeline. Basic research and early development efforts, even when robustly funded, often take years before a concept is mature enough for advanced development. When a product has reached the advanced development stage, Project BioShield Act of 2004 provides an important incentive for manufacturers to take the product the rest of the way through the pipeline. And, as I have outlined here today, in the 9 months since Project BioShield was enacted, the incentive has sped final development of several products for the Stockpile.

In closing, I must emphasize that the number of threat agents against which we could guard ourselves is endless and new and emerging threats introduced by nature will present continuing challenges. Although we cannot be prepared for every threat, we have the ability to create a strategic approach to identifying and combating the greatest threats. HHS and its agencies including NIH, CDC, and FDA, have a clear mandate from President Bush and Congress to lead the charge in this arena. We have already made important strides and will continue to work to address the obstacles identified. Mr. Chairman, I look forward to working with you and members of the Subcommittee to address the challenges of bioterrorism preparedness and its impact on public health.

I will be happy to answer any questions you may have.

Last Revised: June 6, 2006