National Preparedness: HHS Is Monitoring the Progress of Its Medical Countermeasure Efforts but Has Not Provided Previously Recommended Spending Estimates
Released: 2013-12-27T00:00:00

Letter

Background

Since 2004, Congress has authorized over $8 billion for medical countermeasure procurement. The Project BioShield Act of 2004 authorized the appropriation of $5.6 billion from fiscal year 2004 through fiscal year 2013 for the Project BioShield Special Reserve Fund, and funds totaling this amount were appropriated. The act facilitated the creation of a government countermeasure market by authorizing the government to commit to making the Special Reserve Fund available to purchase certain medical countermeasures, including those countermeasures that may not be FDA-approved, cleared, or licensed. In 2013, PAHPRA authorized an additional $2.8 billion to be available from fiscal year 2014 through fiscal year 2018 for these activities,funding has not yet been appropriated for these years. In addition to the but Special Reserve Fund, Congress has also made funding available through annual and supplemental appropriations to respond to influenza pandemics, including developing vaccines and other drugs.

Federal Roles and Responsibilities Related to Medical Countermeasures

HHS is the primary federal department responsible for public health emergency planning and response, including medical countermeasure development, procurement, and distribution. HHS also coordinates with other federal departments, such as DHS, through PHEMCE.

Within HHS, several offices and agencies have specific responsibilities for public health preparedness and response.

HHS’s ASPR leads PHEMCE and the federal medical and public health response to public health emergencies, including strategic planning, medical countermeasure prioritization, and support for developing, procuring, and planning for the effective use of medical countermeasures.

Within ASPR, BARDA—established by the Pandemic and All-Hazards Preparedness Act of 2006—oversees and supports advanced development and procurement of some medical countermeasures into the SNS.

NIH conducts and funds basic and applied research and early development needed to develop new or enhanced medical countermeasures and related medical tools for CBRN and infectious disease threats.

CDC maintains the SNS, including purchasing commercially available products as necessary, and supports state and local public health departments’ efforts to detect and respond to public health emergencies, including providing guidance and recommendations for the mass dispensing and use of medical countermeasures from the SNS.

FDA assesses the safety and effectiveness of medical countermeasures; regulates their development; approves, clears, or licenses them; and conducts postmarket surveillance as part of its overall role to assess the safety and effectiveness of medical products. FDA also provides technical assistance to help ensure that product development meets FDA’s regulatory requirements and provides technical support for the development of regulatory science tools. FDA may authorize the emergency use of medical products that have not yet been approved, cleared, or licensed or were approved, cleared, or licensed only for other uses.

DHS develops material threat assessments (MTA), in coordination with HHS, to assess the threat posed by given CBRN agents or classes of agents and the potential number of human exposures in plausible, high- consequence scenarios. DHS uses the MTAs to determine which CBRN agents pose a material threat sufficient to affect national security and to provide HHS with a basis for determining needed countermeasures for those agents. DHS also develops terrorism risk assessments (TRA) to assess the relative risks posed by CBRN agents based on variable threats, vulnerabilities, and consequences.

HHS’s PHEMCE is responsible for establishing civilian medical countermeasure priorities for CBRN and emerging infectious disease threats, including influenza; coordinating federal efforts to research, develop, and procure medical countermeasures to enhance preparedness and response for public health threats; and developing policies, plans, and guidance for the use of countermeasure products in a public health emergency. PHEMCE is composed of officials from ASPR, including BARDA; CDC; FDA; NIH; and other federal departments, including the Departments of Agriculture, Defense, Homeland Security, and Veterans Affairs. HHS and PHEMCE establish federal medical countermeasure development and procurement priorities through a multistep process. This process includes assessing the threat posed by CBRN agents and the potential consequences they pose to public health, determining medical countermeasure requirements—the type of countermeasure (vaccines, drugs, or medical devices such as diagnostics), the amount needed, and characteristics of the countermeasures (such as formulations, dosing, and packaging)—for these agents, evaluating public health response capability, and developing and procuring countermeasures against these CBRN agents. (See fig. 1.)

PHEMCE Strategy, Implementation Plan, and Priorities

The 2012 PHEMCE Strategy lays out the four PHEMCE strategic goals and their underlying objectives for building HHS’s countermeasure capabilities to respond to a public health emergency. The 2012 PHEMCE Implementation Plan updates the 2007 implementation plan and describes the activities that HHS and its interagency partners plan to conduct to achieve the four strategic goals and their associated objectives, the medical countermeasures HHS wants to develop and procure, and the capabilities HHS wants to build to support countermeasure development and procurement. The plan also includes 72 items that HHS selected as key priorities for fulfilling PHEMCE’s strategic goals within the next 5 years, which the agency placed into three categories. For the purposes of this report we refer to the items in these categories as “priority activities,” “priority threat-based approaches,” and “priority capabilities.”

The 33 priority activities reflect activities that support PHEMCE’s overall mission and include pursuits such as developing systems to track countermeasure activities across all PHEMCE partners, enhancing national laboratory capabilities, and developing guidance documents and information for the public on using medical countermeasures in an emergency. (See table 1 for examples of PHEMCE priority activities by strategic goal.)

In addition to the 33 priority activities, the 25 items identified as priorities for threat-based approaches are intended to directly address threats such as anthrax or smallpox. These priorities include pursuits such as publishing updated clinical guidance for anthrax countermeasures; developing and qualifying with FDA animal models to test the safety and efficacy of medical countermeasures for certain biological, radiological, and nuclear threats; and developing new plans for the distribution and dispensing of pandemic influenza antivirals.

The remaining 14 items identified as priority capabilities reflect what HHS refers to as crosscutting capabilities. The priority capabilities are a mix of programs or technological applications that may, for example, support the development of countermeasures for a range of existing CBRN threats or for any new threats that may emerge in the future, or build infrastructure to provide countermeasure developers assistance with advanced development and manufacturing services. The priority capabilities include such pursuits as initiating a research program to fill gaps in knowledge in the area of patient decontamination in a chemical incident and establishing a network of facilities to support the filling and finishing of vaccines and other countermeasures.

In addition to the 72 items HHS selected as key priorities for fulfilling PHEMCE’s strategic goals, the implementation plan also identifies the medical countermeasures that constitute HHS’s priorities for development and procurement to fulfill strategic goal 1, which we refer to as “priority countermeasures” for the purposes of this report. (See table 2.) Many of the threat-specific countermeasures for which PHEMCE set procurement priorities in 2007 continue to be priorities for development and procurement in the 2012 plan, such as anthrax vaccine, smallpox antivirals, chemical agent antidotes, and diagnostic devices for radiological and nuclear agents. The 2012 plan also includes pandemic influenza countermeasures and nonpharmaceutical countermeasures, such as ventilators, as priorities, whereas the 2007 plan focused on CBRN medical countermeasures only.

HHS Has Established Timelines and Milestones for Key PHEMCE Priorities but Has Not Yet Provided Previously Recommended Anticipated Spending Estimates for Priority Countermeasures

HHS has established timelines and milestones for the 72 priority activities, threat-based approaches, and capabilities identified in the 2012 PHEMCE Implementation Plan as key to fulfilling PHEMCE’s strategic goals. However, while HHS has developed spending estimates for its priority medical countermeasures for internal planning purposes, it has not made these estimates publicly available, as we previously recommended in 2011.

HHS Has Established Timelines and Milestones for Key PHEMCE Priorities

HHS has established timelines and milestones for the 72 items it selected as key priorities for fulfilling PHEMCE’s strategic goals. Leading practices for program management call for establishing time frames and milestones as part of a plan to ensure that organizations achieve intended results.In the implementation plan, HHS has assigned each of the 33 priority activities, the 25 priority threat-based approaches, and the 14 priority capabilities to one of three time frames for completion—near-term (fiscal years 2012 through 2014), midterm (fiscal years 2015 through 2017), and long-term (fiscal year 2018 and beyond). In addition, HHS has placed PHEMCE’s priority countermeasures into these time frames. All but 2 of the 33 priority activities, and all of the priority threat-based approaches and capabilities, are slated for completion in either the near term or the midterm.

HHS has also identified deliverables and milestones for some of the priority activities, threat-based approaches, and capabilities, and assigned them more specific timelines. For 21 of the 33 priority activities, 10 of the 25 priority threat-based approaches, and 8 of the 14 priority capabilities, HHS and the PHEMCE agency or office responsible for carrying out the activity have identified specific deliverables intended to complete them. PHEMCE partners have tied each deliverable to a specific milestone or set of milestones, which delineate the steps necessary to complete the deliverable. In addition, the deliverables and milestones may have more specific timelines, such as an actual month or year of expected completion within the broader multiyear near- or midterm time frame. Examples of deliverables, milestones, and more specific timelines for PHEMCE priorities include the following:

For the priority activity that states that ASPR is to lead PHEMCE in developing or updating medical countermeasure requirements for certain CBRN threats by the end of fiscal year 2014, ASPR has identified the requirements for each specific threat—such as requirements for countermeasures for mustard gas and other blister agents—as the individual deliverables for this activity. The blister agents requirement deliverable has four associated milestones that reflect the various activities of a PHEMCE working group to develop the requirements and the levels of PHEMCE and HHS approval needed, culminating in the approval by the ASPR Assistant Secretary by September 2013.

For the priority threat-based approach of qualifying animal models for biological threats, the deliverable is FDA qualification of the animal model, and the three milestones are the development of animal models for anthrax, plague, and tularemia in fiscal year 2015.

For the priority capability of initiating funding for the development of diagnostic systems for biological and chemical threat agents, and systems to identify and characterize unknown threats, the deliverable is NIH’s awarding of funds to eligible applicants; the set of milestones for this deliverable are obtaining NIH approval to publish a solicitation for proposals for development of the diagnostics, publishing the solicitation in July 2013, and making awards in fiscal year 2014. NIH also plans to award additional funds in fiscal year 2015 for the development of multiplex diagnostic platforms for multiple threats.

For the priority countermeasures, HHS officials told us that the department includes specific milestones in the contracts it awards to developers; these milestones reflect the expected course for research and development, such as holding and completing clinical trials to test the efficacy of a countermeasure or submitting inventory and storage plans, and have associated completion dates.

For the remaining 12 priority activities, 15 priority threat-based approaches, and 6 priority capabilities, HHS has not established specific deliverables with milestones and timelines other than the overall completion of the priority within the specified near- or midterm time frame. HHS officials told us that some activities do not have specific timelines because HHS considers them to be ongoing activities that PHEMCE conducts regularly. For example, at least every 18 months, ASPR conducts formal reviews across participating PHEMCE agencies of medical countermeasure portfolios for specific threats in order to monitor progress in developing and procuring medical countermeasures for those threats, identify remaining gaps and challenges to developing and procuring countermeasures, and develop potential solutions. For activities in the implementation plan that are slated for completion in the long term, HHS officials said that they intend to develop more specific timelines as the near- and midterm activities are completed.

ASPR tracks the progress of participating PHEMCE partners in implementing the priority activities, threat-based approaches, and capabilities by holding monthly meetings to collect information on progress. According to HHS officials, during these monthly meetings, PHEMCE participants discuss their progress in completing deliverables, potential barriers to completion, and any options to help mitigate these barriers. ASPR officials told us they rely on the PHEMCE partner responsible for the activity to have adequate project management controls in place to determine the amount of progress that the partner agency has made. If an agency anticipates delays in or barriers to completing and meeting certain milestones, ASPR officials may assist in identifying additional support within PHEMCE partner agencies or within other federal agencies. For example, HHS officials told us that for one priority activity’s deliverable—developing requirements for anthrax antitoxins—CDC and FDA officials differed in their professional opinions on guidance for clinicians to administer the drug. PHEMCE senior management worked with the agencies to develop consensus wording for the guidance document to complete that deliverable. ASPR officials told us that they enter information collected in the meetings into a spreadsheet that contains descriptions of the PHEMCE priority activities, threat-based approaches, and capabilities; their associated deliverables, milestones, and timelines; and information on current progress, barriers to completion, and mitigation options. ASPR follows up with PHEMCE partners after the meetings to obtain any additional information, if necessary. ASPR distributes the finalized spreadsheet to PHEMCE partners about 1 week in advance of the next monthly meeting for them to use as reference for that meeting. ASPR officials told us they developed the tracking spreadsheet in response to the recommendation in our 2011 report that HHS develop a written strategy to monitor the implementation of recommendations from HHS’s 2010 PHEMCE review and incorporated the PHEMCE priorities into the spreadsheet when HHS updated the implementation plan.

At the completion of our review, PHEMCE was halfway through its near- term period of fiscal year 2012 through fiscal year 2014. As of September 2013 (the most recent information available):

PHEMCE partners reported completing five deliverables for the 21 priority activities. For example, for the priority activity that specifies that HHS, DHS, and other federal partners are to formalize roles, responsibilities, policies, and procedures for conducting the next generation of MTAs and TRAs, HHS and DHS completed one of two deliverables by developing and cosigning a strategic implementation plan to conduct MTAs.

PHEMCE partners reported completing three deliverables for the 10 priority threat-based approaches. For example, for one of the threat- based approaches, PHEMCE partners report completing the sole deliverable of developing guidance that establishes the order in which different groups of affected individuals would receive anthrax vaccination in a public health emergency. The completion of the three deliverables resulted in the completion of three priority threat-based approaches.

PHEMCE partners reported completing two deliverables for the eight priority capabilities. For example, for one of the priority capabilities, PHEMCE partners have reported completing the sole deliverable that specifies that BARDA will initiate a research program to address knowledge gaps in chemical decontamination of exposed individuals by awarding a contract to a university to gather data and develop decontamination procedures. The completion of the two deliverables resulted in the completion of two priority capabilities.

HHS Has Not Provided Previously Recommended Spending Estimates for Priority Countermeasures

HHS has not provided publicly available spending estimates for research, development, or procurement for the countermeasures it identified as priorities in the 2012 implementation plan. We previously recommended that HHS provide more specific information on anticipated countermeasure spending when it updated its 2007 plan. Additionally, PAHPRA directs HHS to include anticipated funding allocations for each countermeasure priority in the PHEMCE strategy and implementation plan. The implementation plan contains information on the source of the funds for research, development, and procurement, such as the Special Reserve Fund. However, the plan does not include any estimates of how much of these funds HHS may spend to develop or procure specific priority countermeasures. HHS officials told us that while PHEMCE has developed spending estimates for internal planning, they are hesitant to provide these estimates to manufacturers because they do not want to create the expectation that the estimates would reflect any final contract amounts. In addition, anticipated spending estimates for future years may be unreliable because, according to HHS officials, the Special Reserve Fund will be appropriated annually after fiscal year 2014, as opposed to the fiscal year 2004 appropriation, which appropriated funds for a 10-year period. Additionally, officials stated that because HHS published the PHEMCE Implementation Plan prior to the passage of PAHPRA, the department did not include any spending estimates in the plan because it was unaware that PAHPRA would include that requirement. HHS officials said that they plan to include estimates in the next iteration of the plan, which they anticipate publishing in September 2014, based on the time frames laid out in PAHPRA. However, the nature and format of the spending estimates that would be included in the plan had not been determined. As we stated in our previous recommendation, information on anticipated spending would allow HHS’s industry partners to suitably target research and development to fulfill PHEMCE’s countermeasure priorities, especially in tighter budget climates. While HHS officials expressed concerns regarding sharing internal spending estimates and the short-term nature of annual appropriations, these concerns could be addressed by agency communications with manufacturers when providing the spending estimates to make clear that spending estimates may not reflect final contract amounts, which depend on enacted appropriations levels, among other factors.

Concluding Observations

Developing and procuring medical countermeasures is a complex process that requires engagement across the federal government and with countermeasure developers in private industry. HHS has strengthened PHEMCE planning and oversight and has made progress in developing and procuring some medical countermeasures. However, given its almost 10-year efforts and the continuing lack of available countermeasures to fulfill PHEMCE’s many priorities, HHS would benefit from sharing information on its anticipated spending estimates with industry, to assist countermeasure developers with long-term business planning. PAHPRA’s requirement for HHS to include spending estimates for each medical countermeasure priority in future PHEMCE implementation plans is consistent with our 2011 recommendation. HHS’s plans to include more specific spending estimates in future plan updates could help implement both this requirement and our 2011 recommendation, provided the department makes meaningful estimates of spending for countermeasure research, development, and procurement available to industry. These estimates—or ranges of estimates—will provide HHS’s industry partners with more transparency on anticipated returns on investment in the face of competing priorities for developing other drugs with a commercial market. We believe the value of making this information available outweighs HHS’s concerns, especially those related to uncertainty over future appropriations; anticipated countermeasure spending would provide industry with the information it needs to determine whether and how to suitably target their research and development programs in tight budget climates.

Agency Comments

We provided a draft of this report to HHS, and its comments are reprinted in appendix II. In its comments, HHS acknowledged the effort we have taken to document HHS’s tracking processes for the activities in the 2012 PHEMCE Implementation Plan. HHS commented that the 72 activities we focused on in this review—which were described in the implementation plan as key to HHS’s efforts in the near and midterm—were a subset of 255 near- and midterm activities delineated in the implementation plan and that these 72 items were meant to be an illustrative but not comprehensive list of priorities. Further, HHS stated that it considered all 255 near- and midterm activities as priorities. HHS provided information on its efforts to track its progress on the remainder of these items that we did not discuss in the report and to establish deliverables and interim milestones for the activities slated for the midterm (fiscal years 2015 through 2017) as that period approaches. Finally, HHS provided information on its efforts to quantify its resource needs and provide more transparent anticipated spending information for its medical countermeasure development efforts while maintaining the integrity of the federal contracting process. HHS stated that it is working to find a compromise solution that will provide this transparency in light of statutory requirements and GAO’s 2011 recommendation. HHS also provided technical comments, which we incorporated as appropriate.

We are sending copies of this report to the Secretary of Health and Human Services. In addition, the report is available at no charge on the GAO website at http://www.gao.gov.

If you or your staffs have any questions about this report, please contact me at (202) 512-7114 or dsouzav@gao.gov. Contact points for our Offices of Congressional Relations and Public Affairs may be found on the last page of this report. GAO staff who made key contributions to this report are listed in appendix III.

Appendix I: HHS Spending on Advanced Research, Development, and Procurement of Medical Countermeasures

The Department of Health and Human Services (HHS) spent approximately $3.6 billion in advanced research, development, and procurement of chemical, biological, radiological, and nuclear (CBRN) and pandemic influenza medical countermeasures from fiscal year 2010 through fiscal year 2013. Of this amount, HHS spent 30 percent for countermeasures against influenza, 20 percent for smallpox countermeasures, and 19 percent for anthrax countermeasures. (See fig. 2.) The spending on influenza countermeasures reflects, in part, HHS’s response to the 2009 H1N1 influenza pandemic using annual and supplemental funds appropriated for that response.

Of HHS’s total medical countermeasure spending of $3.6 billion, from fiscal year 2010 through fiscal year 2013, HHS spent almost $2.1 billion on contracts dedicated to advanced research and development, of which HHS’s Biomedical Advanced Research and Development Authority (BARDA) spent nearly $700 million (almost 34 percent) for influenza antivirals, diagnostics, and vaccines. (See table 3.) Of the remaining $1.5 billion, HHS spent nearly $403 million on contracts dedicated to the procurement of pandemic influenza antivirals and vaccines. (See table 4.) BARDA also spent almost $1.2 billion on contracts dedicated to both advanced research and development and procurement of CBRN medical countermeasures. (See table 5.) In addition to the contracts that have already been awarded, HHS issues annual announcements for additional funding opportunities in the areas of advanced research and development of CBRN medical countermeasures; advanced development of medical countermeasures for pandemic influenza; and innovative science and technology platforms for medical countermeasure development. The announcements state anticipated funding for the overall program. For example, the announcement for CBRN countermeasure advanced research and development states that anticipated funding for the overall effort—not per award—ranges from an estimated $2 million to an estimated $415 million, subject to congressional appropriations, and does not reflect a contractual obligation for funding.

Appendix II: Comments from the Department of Health and Human Services

Appendix III: GAO Contact and Staff Acknowledgments

GAO Contact

Staff Acknowledgments

In addition to the contact named above, Karen Doran, Assistant Director; Shana R. Deitch; Carolyn Feis Korman; Tracey King; and Roseanne Price made significant contributions to this report.

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National Preparedness: Improvements Needed for Measuring Awardee Performance in Meeting Medical and Public Health Preparedness Goals. GAO-13-278. Washington, D.C.: March 22, 2013.

High-Containment Laboratories: Assessment of the Nation’s Need Is Missing. GAO-13-466R. Washington, D.C.: February 25, 2013.

National Preparedness: Countermeasures for Thermal Burns. GAO-12-304R. Washington, D.C.: February 22, 2012.

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National Preparedness: Improvements Needed for Acquiring Medical Countermeasures to Threats from Terrorism and Other Sources. GAO-12-121. Washington, D.C.: October 26, 2011.

Influenza Pandemic: Lessons from the H1N1 Pandemic Should Be Incorporated into Future Planning. GAO-11-632. Washington, D.C.: June 27, 2011.

Influenza Vaccine: Federal Investments in Alternative Technologies and Challenges to Development and Licensure. GAO-11-435. Washington, D.C.: June 27, 2011.

National Preparedness: DHS and HHS Can Further Strengthen Coordination for Chemical, Biological, Radiological, and Nuclear Risk Assessments. GAO-11-606. Washington, D.C.: June 21, 2011.

Public Health Preparedness: Developing and Acquiring Medical Countermeasures Against Chemical, Biological, Radiological, and Nuclear Agents. GAO-11-567T. Washington, D.C.: April 13, 2011.

Combating Nuclear Terrorism: Actions Needed to Better Prepare to Recover from Possible Attacks Using Radiological or Nuclear Materials. GAO-10-204. Washington, D.C.: January 29, 2010.